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International Journal of Technology Assessment in Health Care
Journal Prestige (SJR): 0.714
Citation Impact (citeScore): 1
Number of Followers: 17  
 
  Hybrid Journal Hybrid journal (It can contain Open Access articles)
ISSN (Print) 0266-4623 - ISSN (Online) 1471-6348
Published by Cambridge University Press Homepage  [352 journals]
  • Consolidated health economic evaluation reporting standards 2022 (CHEERS
           2022) statement: updated reporting guidance for health economic
           evaluations

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      Authors: Husereau; Don, Drummond, Michael, Augustovski, Federico, de Bekker-Grob, Esther, Briggs, Andrew H., Carswell, Chris, Caulley, Lisa, Chaiyakunapruk, Nathorn, Greenberg, Dan, Loder, Elizabeth, Mauskopf, Josephine, Mullins, C. Daniel, Petrou, Stavros, Pwu, Raoh-Fang, Staniszewska, Sophie
      First page: 13
      Abstract: Health economic evaluations are comparative analyses of alternative courses of action in terms of their costs and consequences. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) statement, published in 2013, was created to ensure health economic evaluations are identifiable, interpretable, and useful for decision making. It was intended as guidance to help authors report accurately which health interventions were being compared and in what context, how the evaluation was undertaken, what the findings were, and other details that may aid readers and reviewers in interpretation and use of the study. The new CHEERS 2022 statement replaces previous CHEERS reporting guidance. It reflects the need for guidance that can be more easily applied to all types of health economic evaluation, new methods and developments in the field, as well as the increased role of stakeholder involvement including patients and the public. It is also broadly applicable to any form of intervention intended to improve the health of individuals or the population, whether simple or complex, and without regard to context (such as health care, public health, education, social care, etc.). This summary article presents the new CHEERS 2022 28-item checklist and recommendations for each item. The CHEERS 2022 statement is primarily intended for researchers reporting economic evaluations for peer-reviewed journals, as well as the peer reviewers and editors assessing them for publication. However, we anticipate familiarity with reporting requirements will be useful for analysts when planning studies. It may also be useful for health technology assessment bodies seeking guidance on reporting, as there is an increasing emphasis on transparency in decision making.
      PubDate: 2022-01-11
      DOI: 10.1017/S0266462321001732
       
  • Building a model of health technology assessment cooperation: lessons
           learned from EUnetHTA joint action 3

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      Authors: Garrett; Zoe, Imaz-Iglesia, Iñaki, Willemsen, Anne
      First page: 14
      Abstract: ObjectiveThe European Network for Health Technology Assessment (EUnetHTA) was established in 2006. During its final project phase (joint action 3 [JA3]), it undertook an activity to define the scientific and technical principles of a model of health technology assessment (HTA) cooperation in Europe. This policy article presents the key learnings from JA3 partners about developing a model of HTA cooperation.MethodsThere were two phases to the activity: (i) A descriptive phase to describe the elements of HTA cooperation that were already in place in EUnetHTA JA3 and to identify which elements could be improved or were missing. (ii) An analytic phase synthesizing the data collected to identify learnings from the JA3 and to define the scientific and technical principles for a future model of HTA cooperation.ResultsLearnings for developing HTA cooperation were identified in regard to the framework used to support the cooperation, the HTA activities undertaken, the involvement of internal and external actors, managing decision making and the required human resources and support services needed to undertake HTA activities and to coordinate collaboration.ConclusionsThese learnings coming from the experiences of the EUnetHTA JA3 are useful to inform discussions on a European Union regulation for HTA cooperation as well as subsequent work to set up the structures that will be defined in the regulation. The findings also have broader applicability and are relevant to individuals, groups, and organizations setting up HTA programs or establishing their own international collaborations.
      PubDate: 2022-01-28
      DOI: 10.1017/S0266462321001719
       
  • Increasing the impact of budget impact analysis: incorporating uncertainty
           for decision-makers in small markets

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      Authors: Hofmeister; Mark, Clement, Fiona
      First page: 15
      Abstract: For decision-makers considering new medicines for reimbursement and public use, both value for money and affordability are important considerations. Whereas a cost-effectiveness model provides information about value for money, a budget impact assessment (BIA) is customized to a specific context and estimates the total investment needed; one part of affordability. Both analytic approaches have parameter uncertainty within them, yet comparatively little attention is given to parameter uncertainty in BIA. Currently, within BIA, uncertainty exploration is limited to point estimates for plausible scenarios, prompting the question: can a decision-maker be confident in point estimates' Within this paper, our intent is to revitalize the discussion of uncertainty in BIA. In the context of health technology assessments submitted to support reimbursement decision-making, we propose reliance on probabilistic sensitivity analysis conducted in the cost-effectiveness model. If assumptions made in a cost-effectiveness model are valid, probabilistic cost estimates from the model, with the same perspective adopted as the BIA, should also inform BIA. Mean and variance of population outcomes, given parameter uncertainty in model inputs, are estimable from model outputs. As sufficiently large random samples are drawn from a population, the distribution of sample means will follow an approximately normal distribution. Therefore, when drawing samples from the model to inform estimates of budget impact, the assumption of an approximately normal distribution for costs is reasonable. We propose that the variance in mean costs from the cost-effectiveness model also reflects the variance in budget impact estimates and should be used to estimate budget impact confidence intervals.
      PubDate: 2022-01-26
      DOI: 10.1017/S0266462321001707
       
  • Understanding innovation of health technology assessment methods: the
           IHTAM framework

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      Authors: Jiu; Li, Hogervorst, Milou A., Vreman, Rick A., Mantel-Teeuwisse, Aukje K., Goettsch, Wim G.
      First page: 16
      Abstract: Adequate methods are urgently needed to guarantee the good practice of health technology assessment (HTA) for technologies with novel properties. The aim of the study was to construct a conceptual framework to help understand the innovation of HTA methods (IHTAM). The construction of the IHTAM framework was based on two scoping reviews, one on the current practice of innovating methods, that is existing HTA frameworks, and one on theoretical foundations for innovating methods outside the HTA discipline. Both aimed to identify and synthesize concepts of innovation (i.e., innovation processes and roles of stakeholders in innovation). Using these concepts, the framework was developed in iterative brainstorming sessions and subsequent discussions with representatives from various stakeholder groups. The framework was constructed based on twenty documents on innovating HTA frameworks and fourteen guidelines from three scientific disciplines. It includes a generic innovation process consisting of three phases (“Identification,” “Development,” and “Implementation”) and nine subphases. In the framework, three roles that HTA stakeholders can play in innovation (“Developers,” “Practitioners,” and “Beneficiaries”) are defined, and a process on how the stakeholders innovate HTA methods is included. The IHTAM framework visualizes systematically which elements and stakeholders are important to the development and implementation of novel HTA methods. The framework could be used by all stakeholders involved in HTA innovation to learn how to engage dynamically and collaborate effectively throughout the innovation process. HTA stakeholders in practice have welcomed the framework, though additional testing of its applicability and acceptance is essential.
      PubDate: 2022-01-31
      DOI: 10.1017/S0266462322000010
       
  • Addressing uncertainty in relative effectiveness assessments by HTA
           organizations

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      Authors: Vreman; Rick A., Strigkos, George, Leufkens, Hubert G. M., Schünemann, Holger J., Mantel-Teeuwisse, Aukje K., Goettsch, Wim G.
      First page: 17
      Abstract: This study outlines the ways in which different health technology assessment (HTA) organizations deal with uncertainty in relative effectiveness assessments (REAs), using the GRADE framework as a common reference. Guidelines regarding REA and uncertainty assessment methods and three most recent HTA reports (as of April 2020) of seven HTA organizations in Germany, England and Wales, France, the Netherlands, Europe (EUnetHTA), the USA, and Canada were included. First, it was analyzed how each organization addressed uncertainty on the following levels of evidence: (i) individual studies, (ii) body of evidence for one outcome, (iii) body of evidence across all outcomes, and (iv) added net benefit. Second, the extent to which HTA organizations considered the eight domains of certainty of evidence defined by GRADE was assessed. For individual studies, checklists were the most common approach to express uncertainty (4/7 organizations). Uncertainty in the body of evidence for all outcomes and in added benefit was combined in a single conclusion by five organizations. All organizations reported on at least 4/5 downgrading domains of GRADE, while the three upgrading domains were reported less. The operationalization of the assessment of multiple domains was unclear due to vague or absent guidelines. HTA organizations consider most domains of the GRADE framework, but approaches to assess uncertainty within REAs on different levels of evidence differ substantially between organizations. More alignment and guidance on the best methods to deal with uncertainty within HTA could lead to more clarity for stakeholders and to more aligned reimbursement recommendations.
      PubDate: 2022-01-31
      DOI: 10.1017/S026646232100177X
       
  • Analysis of consumer comments into PBAC decision-making (2014–9)

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      Authors: Tjeuw; Emily, Wonder, Michael J.
      First page: 18
      Abstract: ObjectivesThe Pharmaceutical Benefits Advisory Committee (PBAC) is an independent expert body that recommends new technologies for listing on the Pharmaceutical Benefits Scheme. Its decision-making process is evidence-based and considers a technology's clinical effectiveness, safety, and cost-effectiveness compared with other technologies. Since 2014, the PBAC has formally taken into account input from those impacted by the technology via an online consumer comments portal and has also reported on received comments in the Public Summary Documents (PSDs). Comments are welcomed from those whose health the technology is trying to improve, as well as carers, clinicians, and organizations. Our objective was to analyze and review consumer comments in the PBAC's decision-making process.MethodsWe extracted information about consumer comments from the PBAC PSDs from 2014–9. We conducted simple descriptive analyses.ResultsOur findings reveal that two thirds of all submissions did not receive a single consumer comment. Of the remaining third, eight submissions (less than 1 percent) had a substantial number of consumer comments (>500). For these technologies, multiple submissions were required before a recommendation was issued. Submissions spanned multiple therapeutic areas, the therapeutic areas with the most consumer comments were genetic disease, pediatrics, and oncology.ConclusionsIn the light of our review, we have identified limitations to the current consumer comments process, and after an examination of the processes of other comparable health technology assessment agencies, we have identified a number of improvements that could be made to the PBAC's process to increase consumer engagement.
      PubDate: 2022-02-04
      DOI: 10.1017/S0266462321001744
       
  • Topic selection process in health technology assessment agencies around
           the world: a systematic review

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      Authors: Qiu; Yingpeng, Thokala, Praveen, Dixon, Simon, Marchand, Robert, Xiao, Yue
      First page: 19
      Abstract: ObjectiveThe purpose of this study was to systematically review the process for topic selection by health technology assessment (HTA) agencies around the world to provide the knowledge base for the improvement of topic selection frameworks in HTA agencies.MethodsA systematic search was conducted in PubMed and EMBASE to identify papers up to February 2019. Gray literature was identified by screening the Web sites of HTA agencies on the nonprofit member list of the International Network of Agencies for Health Technology Assessment (INAHTA). Data were extracted for each HTA agency and synthesized, with issues including general contextual information about each agency and the process of topic selection.ResultsOut of forty-nine nonprofit members of INAHTA, a total of seventeen HTA agencies with a framework for topic selection were identified from twenty-two included papers/documents. Multiple criteria were used for topic selection in all frameworks and agencies undertook multiple steps, which could include the specification of criteria for topic selection, identification of topics, short listing of potential topics, scoping of potential topics, scoring and ranking of potential topics, and deliberation and decision on final topics for HTA. Shortcomings were found in relation to methods of scoring and ranking as well as lack of monitoring and the evaluation of the process.ConclusionsOur study provides insights into the current practice of topic selection in HTA agencies. Multiple criteria decision analysis methodology appears highly relevant to these processes. A consensus approach for the development of methods of topic selection would be valuable for the HTA community.
      PubDate: 2022-02-07
      DOI: 10.1017/S0266462321001690
       
  • Synthesizing quantitative and qualitative information on multiple
           comparisons of health interventions to facilitate knowledge transfer: an
           example from an EUnetHTA multi-HTA

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      Authors: Formoso; Giulio, Jeroncic, Ana, Bonvicini, Laura, Djuric, Olivera, Erdos, Judit, Pezzarossi, Annamaria, Ballini, Luciana
      First page: 20
      Abstract: ObjectivePresenting an approach to synthesize quantitative and qualitative information from systematic reviews of multiple health interventions.MethodsWithin the context of an EUnetHTA multi-health technology assessment of twenty-three surgical techniques, we developed synthetic single tables, using color gradients and abbreviations, with information on which technologies had been compared, estimates of the size of differences for available comparisons, their clinical relevance, and certainty of the related evidence.ResultsThe proposed methodology provided, through a single depiction, information normally included in multiple figures/tables such as network plots, league tables, and summary of findings tables.ConclusionTransferring information on benefits, risks, and certainty of the available evidence on health interventions may be challenging, especially when assessing multiple treatments: more pieces of information need to be integrated in order to show an overall picture for each of the chosen outcomes, and usual reporting tools may be targeted to researchers more than to different kinds of decision makers. While more in-depth layers of information can always be added to satisfy needs of different audiences, the proposed tools could favor a quick interpretation of articulated scientific data by both decision makers and researchers.
      PubDate: 2022-02-09
      DOI: 10.1017/S0266462322000046
       
  • Eliciting uncertainty for complex parameters in model-based economic
           evaluations: quantifying a temporal change in the treatment effect

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      Authors: Jankovic; Dina, Payne, Katherine, Kanaan, Mona, Bojke, Laura
      First page: 21
      Abstract: BackgroundIn model-based economic evaluations, the effectiveness parameter is often informed by studies with a limited duration of follow-up, requiring extrapolation of the treatment effect over a longer time horizon. Extrapolation from short-term data alone may not adequately capture uncertainty in that extrapolation. This study aimed to use structured expert elicitation to quantify uncertainty associated with extrapolation of the treatment effect observed in a clinical trial.MethodsA structured expert elicitation exercise was conducted for an applied study of a podiatry intervention designed to reduce the rate of falls and fractures in the elderly. A bespoke web application was used to elicit experts’ beliefs about two outcomes (rate of falls and odds of fracture) as probability distributions (priors), for two treatment options (intervention and treatment as usual) at multiple time points. These priors were used to derive the temporal change in the treatment effect of the intervention, to extrapolate outcomes observed in a trial. The results were compared with extrapolation without experts’ priors.ResultsThe study recruited thirty-eight experts (geriatricians, general practitioners, physiotherapists, nurses, and academics) from England and Wales. The majority of experts (32/38) believed that the treatment effect would depreciate over time and expressed greater uncertainty than that extrapolated from a trial-based outcome alone. The between-expert variation in predicted outcomes was relatively small.ConclusionsThis study suggests that uncertainty in extrapolation can be informed using structured expert elicitation methods. Using structured elicitation to attach values to complex parameters requires key assumptions and simplifications to be considered.
      PubDate: 2022-02-18
      DOI: 10.1017/S0266462322000022
       
  • European Network for Health Technology Assessment’s Response to
           COVID-19: Rapid Collaborative Reviews on Diagnostic Tests and Rolling and
           Rapid Collaborative Reviews on Therapeutics

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      Authors: Ballini; Luciana, Wild, Claudia, Djuric, Olivera, Mayer-Ferbas, Julia, Willemsen, Anne, Huic, Mirjana
      First page: 22
      Abstract: ObjectivesIn spring 2020, The European network for Health Technology Assessment (EUnetHTA) decided to join forces to produce best evidence to inform health policy in the COVID-19 pandemic. The objective of this paper is to describe the process and output of the coordinated and collaborative activities of EUnetHTA.MethodsRelevant published and internal documents were retrieved for a descriptive analysis of EUnetHTA processes, methods, and outputs related to EUnetHTA’s response to the pandemic.ResultsProcess: In April 2020, a COVID-19 task force was set up and a survey collected pressing health policy questions across Europe. Two coordinating agencies for diagnostic tests and therapeutics were assigned. A process for prioritization and selection was set up for therapeutics, as well as explicit starting and stopping rules. Methodology: To increase a timely response, it was agreed that the rapid collaborative reviews (rapid CRs) would not require the consultation of manufacturers and the involvement of external experts, but would not differ in the methods and conduct of the systematic search, review, and synthesis of all available evidence, nor in the requirement for reviewing by EUnetHTA partners. Final reports: The joint effort resulted in the production of two rapid CRs on diagnostic tests, nineteen collaborative rolling reviews on therapeutics, three of which later moved to rapid CRs.ConclusionsDuring COVID-19 pandemic, the EUnetHTA partners proved capable of prompt collaboration, which allowed speeding up the production and release of high-quality EUnetHTA outputs, while the relationships with the other European institutions facilitated their quick dissemination.
      PubDate: 2022-03-08
      DOI: 10.1017/S0266462322000071
       
  • Faciliating More Efficient Negotiations for Innovative Therapies: A
           Value-Based Negotiation Framework

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      Authors: Whittal; Amanda, Jommi, Claudio, De Pouvourville, Gérard, Taylor, David, Annemans, Lieven, Schoonaert, Lies, Vermeersch, Sebastian, Hutchings, Adam, Patris, Julien
      First page: 23
      Abstract: ObjectivesAn increasing number of innovative therapies (e.g., gene- and cell-based treatments) have been developed in the past 20 years. Despite the significant clinical potential of these therapies, access delays may arise because of differing perspectives of manufacturers and payers regarding issues such as the value of the product, clinical and financial uncertainties, and sustainability.Managed entry agreements (MEAs) can enable access to treatments that would not be reimbursed by conventional methods because of such concerns. However, although MEA typologies exist, there is currently no structured process to come to agreements on MEAs, which can be difficult to decide upon and implement.To facilitate more structured MEA negotiations, we propose a conceptual “value-based negotiation framework” with corresponding application tools.MethodsThe framework was developed based on an iterative process of scientific literature review and expert input.ResultsThe framework aims to (i) systematically identify and prioritize manufacturer and payer concerns about a new treatment, and (ii) select a mutually acceptable combination of MEA terms that can best address priority concerns, with the lowest possible implementation burden.ConclusionsThe proposed framework will be tested in practice, and is a step toward supporting payers and manufacturers to engage in more structured, transparent negotiations to balance the needs of both sides, and enabling quicker, more transparent MEA negotiations and patient access to innovative products.
      PubDate: 2022-03-11
      DOI: 10.1017/S0266462322000095
       
  • Evidence-Informed Update of Argentina’s Health Benefit Package:
           Application of a Rapid Review Methodology

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      Authors: Alcaraz; Andrea, Alfie, Verónica, Gonzalez, Lucas, Virgilio, Sacha, Garcia-Marti, Sebastián, Augustovski, Federico, Pichon-Riviere, Andrés
      First page: 24
      Abstract: ObjectivesArgentina has a fragmented healthcare system with social security covering almost two thirds of the population. Its benefit package—called compulsory medical program (PMO; by its Spanish acronym Programa Médico Obligatorio)—has not been formally and widely updated since 2005. However, laws, clinical practice guidelines (CPGs), and a high-cost technology reimbursement fund complement it. Our objective was to comprehensively review such a PMO and propose an update considering the corresponding complementary sources.MethodsWe followed four steps: (i) identification of health technologies from the current PMO and complementary sources, (ii) prioritization, (iii) assessment through rapid health technology assessment (HTA), and (iv) appraisal and recommendations. We evaluated three value domains: quality of evidence, net benefit, and economics, which were summarized in a five-category recommendation traffic-light scale ranging from a strong recommendation in favor of inclusion to a strong recommendation for exclusion.ResultsEight hundred fifty technologies were identified; 164 of those, considered as high priority, were assessed through rapid HTAs. Those technologies mentioned in laws and CPGs were mostly outpatient essential medicines, whereas those from the reimbursement system were mostly high-cost drugs; of these 101 technologies, 50 percent were recommended to be kept in the PMO. The other 63 (identified by the Superintendence of Health Services, technology producers, and patients) were mostly medical procedures and high-cost drugs; only 25 percent of those resulted in a favorable recommendation.ConclusionsA methodology based on four clearly identified steps was used to carry out a comprehensive review of an outdated and fragmented benefit package. The use of rapid HTAs and a traffic-light recommendation framework facilitated the deliberative evidence-based update.
      PubDate: 2022-03-11
      DOI: 10.1017/S0266462322000034
       
  • Acceptability of Manufacturer-Proposed Utility Values for NICE Cancer
           Medicine Appraisals: Analysis of Manufacturers’ Information Sources

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      Authors: Takada; Shunsuke, Narukawa, Mamoru
      First page: 25
      Abstract: ObjectivesThe National Institute for Health and Care Excellence’s (NICE) method guide for technology appraisals (TAs) encourages medicine manufacturers to use the EuroQol 5 Dimensions (EQ-5D) in relevant clinical trials to obtain utility values; however, the EQ-5D may have low sensitivity when compared to disease-specific measures. This study investigated whether the NICE TA committee’s acceptance of manufacturer-proposed utility values is dependent on the manufacturers’ sources of the utility values.MethodsUsing publicly available data for 2011–2020, we identified 136 single TAs of cancer medicines, the health-related quality-of-life-measures used in relevant clinical trials, manufacturers’ sources of utility values, and the NICE TA committee’s acceptance of these values. Fisher’s exact tests were performed to compare the acceptability of different value sources and reasons for non-acceptance.ResultsThe number of appraisals for which the EQ-5D in the relevant clinical trials was the source of the manufacturer-proposed utility values increased continuously over time. The TA committee’s acceptance of values was not dependent on the information source. In cases where a submission for which the information source was the EQ-5D was rejected, the reason was generally related to inappropriate values for the UK population or inappropriate data adjustment, not data reliability.ConclusionsOur results demonstrated that according with the NICE’s method guide regarding utility values does not guarantee acceptance by the TA committee. Manufacturers must consider in advance possible differences between their clinical trials and clinical practice in the UK and refine plans for EQ-5D measurement in order to obtain convincing evidence.
      PubDate: 2022-03-17
      DOI: 10.1017/S0266462322000149
       
  • Developing and piloting a context-specified ethics framework for health
           technology assessment: the South African Values and Ethics for Universal
           Health Coverage approach

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      Authors: Krubiner; Carleigh B., Barsdorf, Nicola W., Goldstein, Susan J., Mosam, Atiya, Potgieter, Sunita, DiStefano, Michael J., Tugendhaft, Aviva, Merritt, Maria W., Li, Ryan, Chalkidou, Kalipso, Faden, Ruth R., Hofman, Karen J.
      First page: 26
      Abstract: ObjectivesWhile ethics has been identified as a core component of health technology assessment (HTA), there are few examples of practical, systematic inclusion of ethics analysis in HTA. Some attribute the scarcity of ethics analysis in HTA to debates about appropriate methodology and the need for ethics frameworks that are relevant to local social values. The “South African Values and Ethics for Universal Health Coverage” (SAVE-UHC) project models an approach that countries can use to develop HTA ethics frameworks that are specific to their national contexts.MethodsThe SAVE-UHC approach consisted of two phases. In Phase I, the research team convened and facilitated a national multistakeholder working group to develop a provisional ethics framework through a collaborative, engagement-driven process. In Phase II, the research team refined the model framework by piloting it through three simulated HTA appraisal committee meetings. Each simulated committee reviewed two case studies of sample health interventions: opioid substitution therapy and either a novel contraceptive implant or seasonal influenza immunization for children under five.ResultsThe methodology was fit-for-purpose, resulting in a context-specified ethics framework and producing relevant findings to inform application of the framework for the given HTA context.ConclusionsThe SAVE-UHC approach provides a model for developing, piloting, and refining an ethics framework for health priority-setting that is responsive to national social values. This approach also helps identify key facilitators and challenges for integrating ethics analysis into HTA processes.
      PubDate: 2022-03-08
      DOI: 10.1017/S0266462322000113
       
  • The transcatheter aortic valve implantation: an assessment of the
           generalizability of the economic evidences following a systematic review

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      Authors: Ruggeri; Matteo, Donatella, Mandolini, Federica, Campisi, Salvatore, Danese, Costanza, Santori, Marta, Chiappetta, Paolo, Roazzi, Marco, Marchetti
      First page: 27
      Abstract: ObjectivesScientific literature debates on the economic affordability of transcatheter aortic valve implantation (TAVI) in order to give a useful support to decision makers aiming at establishing a reimbursement scheme for TAVI. For this reason, it is important to assess the quality and the generalizability of the existing economic evidences.MethodsThe first step was to run a literature search according to a predefined population, intervention, comparator, and outcome on the cost and effectiveness of the TAVI procedure in comparison to medical therapy and traditional surgery. Second, a manual search was carried out on the Web sites of the main HTA agencies. Third, the checklist developed by Augustovski et al. was applied in order to assess the quality and the generalizability of the articles resulting from the selection process.ResultsOverall, 106 articles were obtained. Of these, sixty-five articles were excluded since the title was not consistent with the objective. Further selection took place after abstract and full-text reading. In the end, thirty-one documents were included for the review. According to the checklist, none of the articles was considered generalizable and only one was considered transferable which compares the TAVI procedure with Medical Management in inoperable patients.ConclusionsDespite the overall quality of the selected studies was considered good, there is still a lack of evidence on whether evidences generated in different contexts can be considered generalizable. Further research on resource consumption and preferences is needed in order to provide decision makers with more robust evidences.
      PubDate: 2022-03-24
      DOI: 10.1017/S0266462321001720
       
  • A Review of Overall Survival Extrapolations of Immune-Checkpoint
           Inhibitors Used in Health Technology Assessments by the French Health
           Authorities

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      Authors: Grumberg; Valentine, Roze, Stéphane, Chevalier, Julie, Borrill, John, Gaudin, Anne-Françoise, Branchoux, Sébastien
      First page: 28
      Abstract: ObjectivesExtrapolation is often required to inform cost-effectiveness (CE) evaluations of immune-checkpoint inhibitors (ICIs) since survival data from pivotal clinical trials are seldom complete. The objectives of this study were to evaluate the accuracy of estimates of long-term overall survival (OS) predicted in French CE assessment reports of ICIs, and to identify models presenting the best fit to the observed long-term survival data.MethodsA systematic review of French assessment reports of ICIs in the metastatic setting since inception until May 2020 was performed. A targeted literature review was conducted to collect associated extended follow-up of randomized controlled trials (RCTs) used in the CE assessment reports. Difference between projected and observed OS was calculated. A range of standard parametric and spline-based models were applied to the extended follow-up data from the RCT to determine the best-fitting survival models.ResultsOf the 121 CE assessment reports published, 11 reports met the inclusion criteria. OS was underestimated in 73 percent of the CE assessment reports. The mean relative difference between each source was −13 percent (median: −15 percent; IQR: −0.4 to 26 percent). Models providing the best fit were those that could reflect nonmonotonic hazards.ConclusionsBased on the available data at the time of submission, longer-term survival of ICIs was not fully captured by the extrapolation models used in CE assessments. Standard and flexible parametric models which can capture nonmonotonic hazard functions provided the best fit to the extended follow-up data. However, these models may still have performed poorly if fitted to survival data available at the time of submission to the French National Authority for Health.
      PubDate: 2022-03-25
      DOI: 10.1017/S0266462322000125
       
  • A Technical Review of the ISPOR Presentations Database Identified Issues
           in the Search Interface and Areas for Future Development

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      Authors: Cooper; Chris, Brown, Anna, Court, Rachel, Schauberger, Ute
      First page: 29
      Abstract: ObjectiveTo undertake a technical review of the search interface of the ISPOR Presentations Database. By technical review, we mean an evaluation of the technical aspects of the search interface and functionality, which a user must navigate to complete a search.MethodsA validated checklist (Bethel and Rogers, 2014, Health Info Libr J, 31, 43-53) was used to identify where the interface performed well, where the interface was adequate, where the interface performed poorly, where functionality available in core biomedical bibliographic databases does not exist in the ISPOR database, and to establish a list of any issues arising during the review. Two researchers independently undertook the technical review in October 2021.ResultsThe ISPOR database scored 35 of a possible 165 (27/111 essential criteria and 8/54 desirable criteria). Two issues arising were identified, both of which will cause searchers to miss potentially eligible abstracts: (i) that search terms, which include * or ' as truncation or wildcard symbols should not be capitalized (e.g., cost* not Cost*; organi'ation not Organi'ation) and (ii) that quotation marks should be straight sided in phrase searching (e.g., “cost analyses” not “cost analyses”).ConclusionsThe ISPOR database is a promising and free database to identify abstracts/posters presented at ISPOR. We summarize two key issues arising, and we set out proposed changes to the search interface, including: adding the ability to export abstracts to a bibliographic tool, exporting search strategies, adding a researcher account, and updating the help guide. All suggestions will further improve this helpful database.
      PubDate: 2022-03-08
      DOI: 10.1017/S0266462322000137
       
  • Early Dialogues for Pharmaceutical Products in European Network for Health
           Technology Assessment Joint Action 3: What Was Done and Where to Go in the
           Future

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      Authors: Galbraith; Margaret, Guilhaume, Chantal, Bélorgey, Chantal
      First page: 30
      Abstract: ObjectivesThe aim of this article is to describe the process, results, and experiences of European Network for Health Technology Assessment (EUnetHTA) Joint Action 3’s (JA3) Early Dialogue (ED) activities and to highlight opportunities for improving the processes.MethodsA descriptive analysis of the steps of the EUnetHTA ED process and evaluation of the data from the EDs conducted by EU health technology assessment (HTA) bodies, published guidelines, and documents, as well as internal statistics.ResultsIn JA3, an Early Dialogues Working Party (EDWP) was established, responsible for developing and improving processes and providing advice to pharmaceutical companies, supported by the ED Secretariat. From June 2017 to May 2021, 113 requests for pharmaceutical EDs were received and 38 conducted. The process was continuously optimized, and different approaches for involving patients were tested. Finally, a centralized procedure was chosen with the key documents produced by two responsible agencies and reviewed by the EDWP. Patient involvement was primarily done by interviewing a national patient representative to obtain general feedback on the disease and the planned study design.ConclusionsDuring JA3, EDs were established as an efficient, successful product. Pharmaceutical companies benefited not only from the positions of the individual agencies for the national HTA, but also from the recommendations that were common to all HTA authorities. In addition, regarding the European HTA Regulation, it will be important to conduct Joint Scientific Consultations with a view toward future Joint Clinical Assessments and to further develop processes aligned with the high demand for consultation.
      PubDate: 2022-03-24
      DOI: 10.1017/S0266462322000083
       
  • Should We Pay for Scientific Knowledge Spillovers' The
           Underappreciated Value of “Failed” R&D Efforts

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      Authors: Xie; Richard Z., Towse, Adrian, Garrison, Louis P.
      First page: 31
      Abstract: Recent experience with COVID-19 has reminded us of the importance of scientific progress in enabling pharmaceutical innovation. Developing novel therapies is a highly risky but rewarding process: it not only produces innovative drugs, but also valuable scientific knowledge that benefits the community of innovators. This paper examines whether the existing reward system for pharmaceutical research and development (R&D) leads to socially optimal levels of scientific knowledge generation and sharing, with a particular focus on the value of failures in the pharmaceutical R&D efforts. We first outline a conceptual approach based on the idea that pharmaceutical R&D efforts produce both medicines and scientific knowledge, and illustrate this with some examples of how failures may generate information beneficial to concurrent and subsequent R&D efforts. We then summarize the relatively small literature on failures in pharmaceutical R&D and their impact on R&D decision making. Lastly, we discuss several market-based and nonmarket-based policy approaches that can address potential shortcomings in the current reward system which may lead to suboptimal R&D and knowledge sharing.
      PubDate: 2022-03-17
      DOI: 10.1017/S0266462322000150
       
  • Key learnings from Institute for Clinical and Economic Review’s
           real-world evidence reassessment pilot

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      Authors: Jaksa; Ashley, Bloudek, Lisa, Carlson, Josh J., Shah, Kanya, Chen, Yilin, Patrick, Amanda R., McKenna, Avery, Campbell, Jon D.
      First page: 32
      Abstract: Health technology assessment (HTA) agencies are considering adopting a lifecycle approach to assessments to address uncertainties in the evidence base at launch and to revisit the clinical and economic value of therapies in a dynamic clinical landscape. For reassessments of therapies post launch, HTA agencies are looking to real-world evidence (RWE) to enhance the clinical and economic evidence base, though challenges and concerns in using RWE in decision-making exists. Stakeholders are embarking on demonstration projects to address the challenges and concerns and to further define when and how RWE can be used in HTA decision making. The Institute for Clinical and Economic Review piloted a 24-month observational RWE reassessment. Key learnings from this pilot include identifying the benefits and challenges with using RWE in reassessments and considerations on prioritizing and selecting topics relevant for RWE updates.
      PubDate: 2022-03-14
      DOI: 10.1017/S0266462322000162
       
  • Postlaunch evidence generation practices among health technology
           assessment bodies in Europe

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      Authors: Puñal-Riobóo; Janet, Varela-Lema, Leonor, Guilhaume, Chantal, Galbraith, Margaret, Bélorgey, Chantal, Faraldo, Maria José, Meillassoux, Amélie
      First page: 33
      Abstract: ObjectivesThe term Postlaunch Evidence Generation (PLEG) refers to evidence generated after the launch or licensing of a health technology. The aim of this paper is to provide an overview of the implementation of these practices in the European Union in order to explore cross-border cooperation opportunities.MethodsIn December 2019, a survey composed of nine closed-ended questions with multiple choice answers about the PLEG practices in each country was sent to all twenty-five dedicated work package (WP5B) partners of the European Network of Health Technology Assessment (EUnetHTA) Joint Action 3. In addition to the survey, the national practices were discussed during a face-to-face meeting with WP5B partners.ResultsTwelve Health TechnologyAssessment (HTA) bodies completed the survey. Of these, eleven reported procedures in place for official requests for PLEGs in their remit. In the large majority of cases, the requests are made at the time of the assessment/appraisal. Several agencies participate in the definition of the scope of the PLEG or review of its protocol. Data collection and analysis mainly lie with companies for pharmaceuticals, whereas it is more the responsibility of the HTA bodies for medical devices. Only one agency owns the data and is able to exchange them without asking permission.ConclusionsMost agencies recommend European collaboration on PLEG commence once the evidence gaps have been defined or during the production of the HTA report in the case of European joint assessment.
      PubDate: 2022-04-19
      DOI: 10.1017/S0266462322000174
       
  • European collaboration on health technology assessment: looking backward
           and forward

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      Authors: Ruether; Alric, Imaz-Iglesia, Iñaki, Bélorgey, Chantal, Lo Scalzo, Alessandra, Garrett, Zoe, Guardian, Marcus
      First page: 34
      Abstract: The establishment of health technology assessment (HTA) has been an important topic in Europe for many years. There have been a series of activities starting with first projects in 1994 leading to joint actions from the European Network of HTA (EUnetHTA) ending in 2021. This long interval of engagement with HTA structures, methodology, and processes by all member states led to a reliable basis for European collaboration in HTA. This article shows milestones and developments from EUR-ASSESS in 1994 through the progress of EUnetHTA and the accompanying EU-HTA-Network up to the recent elaboration of the EU-HTA-Regulation. With the EU-HTA-Regulation HTA collaboration is taken out of the trial phase of more than 15 years. Through the previous EU HTA collaboration, the appreciation and understanding of the differences and complexities behind the HTA processes in the EU healthcare systems have improved. It is now necessary to make the final steps toward a sustainable European Network for HTA.
      PubDate: 2022-04-19
      DOI: 10.1017/S026646232200006X
       
  • The development process of economic evaluation guidelines in low- and
           middle-income countries: a systematic review

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      Authors: Daccache; Caroline, Karam, Rita, Rizk, Rana, Evers, Silvia M. A. A., Hiligsmann, Mickaël
      First page: 35
      Abstract: ObjectivesTo review the development of economic evaluation guidelines (EEGs) in low- and middle-income countries (LMICs), with the goal of assisting those developing EEGs in LMICs.MethodsWe conducted a systematic search in MEDLINE (Ovid), PubMed, EconLit, Embase (Ovid), the Cochrane Library, and the gray literature until March 2021. We extracted data on the methods used in the EEG development process, the responsible party engaged, and the development team’s composition. We conducted a quality assessment, using the Appraisal of Guidelines for Research and Evaluation-Health Systems tool, and then carried out a relative comparison.ResultsFourteen EEGs and nine studies were identified. In ten countries, the Ministry of Health was responsible for handling the development process. The majority of LMICs who developed EEGs did not explicitly report the discipline of those involved in the process. The developers of EEGs followed four main steps: conducting a review on national guidelines, organizing workshops, and getting support from international experts or from organizations. One-third of the identified EEGs failed to engage multisectoral or multidisciplinary developers, and approximately 14 percent did not follow or report any recommended step.ConclusionsThis study identified a scarcity of published information related to the development process and the suboptimal quality of included studies. It provides relevant material to support international organizations and developers of guidelines in LMICs in developing EEGs that fit their national context. In addition, this paper recommends a transparent approach to the design of guidelines and to reporting on the methods for developing them.
      PubDate: 2022-04-22
      DOI: 10.1017/S0266462322000186
       
  • A systematic literature review of revealed preferences of decision-makers
           for recommendations of cancer drugs in health technology assessment

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      Authors: Wang; Yitong, Qiu, Tingting, Nikodem, Mateusz, Francois, Clément, Toumi, Mondher
      First page: 36
      Abstract: ObjectivesThis review intends to provide an overview of revealed preferences of decision-makers for recommendations of cancer drugs in health technology assessment (HTA) among the different agencies.MethodsA systematic literature search was performed in MEDLINE and EMBASE databases from inception to July 2020. The studies were eligible for inclusion if they conducted a quantitative analysis of HTA’s previous decisions for cancer drugs. The factors with p-values below the significance level of .05 were considered as the statistically significant factors for HTA decisions.ResultsA total of nine studies for six agencies in Australia, Belgium, France, South Korea, the UK, and Canada were eligible to be included. From the univariable analysis, improvements in clinical outcomes and cost-effectiveness were found as significant factors for the agencies in Belgium, South Korea, and Canada. From the multivariable analysis, cost-effectiveness was found as a positive factor for the agencies in the UK, South Korea, and Canada. Few factors related to characteristics of disease and technology were found to be significant among the included agencies.ConclusionsDespite the different drug reimbursement systems and the socioeconomic situations, cost-effectiveness and/or improvement on clinical outcomes seemed to be the most important factors for recommendations of cancer drugs among the agencies.
      PubDate: 2022-04-06
      DOI: 10.1017/S0266462322000216
       
  • Designing and Implementing Deliberative Processes for Health Technology
           Assessment: A Good Practices Report of a Joint HTAi/ISPOR Task Force

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      Authors: Oortwijn; Wija, Husereau, Don, Abelson, Julia, Barasa, Edwine, Bayani, Diana (Dana), Santos, Vania Canuto, Culyer, Anthony, Facey, Karen, Grainger, David, Kieslich, Katharina, Ollendorf, Daniel, Pichon-Riviere, Andrés, Sandman, Lars, Strammiello, Valentina, Teerawattananon, Yot
      First page: 37
      Abstract: ObjectivesDeliberative processes for health technology assessment (HTA) are intended to facilitate participatory decision making, using discussion and open dialogue between stakeholders. Increasing attention is being given to deliberative processes, but guidance is lacking for those who wish to design or use them. Health Technology Assessment International (HTAi) and ISPOR—The Professional Society for Health Economics and Outcomes Research initiated a joint Task Force to address this gap.MethodsThe joint Task Force consisted of fifteen members with different backgrounds, perspectives, and expertise relevant to the field. It developed guidance and a checklist for deliberative processes for HTA. The guidance builds upon the few, existing initiatives in the field, as well as input from the HTA community following an established consultation plan. In addition, the guidance was subject to two rounds of peer review.ResultsA deliberative process for HTA consists of procedures, activities, and events that support the informed and critical examination of an issue and the weighing of arguments and evidence to guide a subsequent decision. Guidance and an accompanying checklist are provided for (i) developing the governance and structure of an HTA program and (ii) informing how the various stages of an HTA process might be managed using deliberation.ConclusionsThe guidance and the checklist contain a series of questions, grouped by six phases of a model deliberative process. They are offered as practical tools for those wishing to establish or improve deliberative processes for HTA that are fit for local contexts. The tools can also be used for independent scrutiny of deliberative processes.
      PubDate: 2022-06-03
      DOI: 10.1017/S0266462322000198
       
  • A Review of Overall Survival Extrapolations of Immune-Checkpoint
           Inhibitors Used in Health Technology Assessments by the French Health
           Authorities – Erratum

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      Authors: Grumberg; Valentine, Roze, Stéphane, Chevalier, Julie, Borrill, John, Gaudin, Anne-Françoise, Branchoux, Sébastien
      First page: 38
      PubDate: 2022-05-17
      DOI: 10.1017/S0266462322000228
       
  • Is value portable' An examination of contextual and practical
           considerations that affect the transferability of value assessments
           between settings

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      Authors: Synnott; Patricia G., Lin, Pei-Jung, Hickson, Stacey S., Glaetzer, Christoph, Ollendorf, Daniel A.
      First page: 39
      Abstract: ObjectivesThe extent to which value assessments are uniquely deployed in any given geographic setting is variable. Increasingly, markets are seeking insights from external health technology assessments (HTAs) to assist with decisions surrounding the adoption of new technologies. We reviewed the environment, infrastructure, and practice of value assessment in six countries, with a focus on how these elements influence the transferability of value assessments between settings.MethodsWe reviewed the diverse settings in which six organizations conducting HTA operate, and explored how differences might affect the transferability of value assessment. We focused attention on Australia’s Pharmaceutical Benefits Advisory Committee, China’s National Center for Medicine and HTA, Germany’s Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Japan’s Center for Outcomes Research and Economic Evaluation for Health (Core 2 Health), the National Institute for Health and Care Excellence in England and Wales, and the Institute for Clinical and Economic Review in the United States.ResultsHTA is adopted to address unique objectives for a given health system and is tailored to support local standards and preferences. Some elements of a value assessment, such as evidence on clinical effectiveness, may be more transferable than others. It is challenging to appropriately adjust external assessments to the local context.ConclusionsContextual differences influence both the role and application of HTA. These differences limit the transferability of value assessments from one setting to another. De novo appraisals, customized to the local decision context, are the ideal approach to determinations about value.
      PubDate: 2022-04-21
      DOI: 10.1017/S026646232200023X
       
  • In Memoriam: H. David Banta

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      Authors: Oortwijn; Wija
      First page: 40
      PubDate: 2022-05-04
      DOI: 10.1017/S0266462322000253
       
  • EUnetHTA relative effectiveness assessments: efforts to increase
           usability, transparency and inclusiveness

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      Authors: Willemsen; Anne, Ettinger, Sabine, Helmink, Catharina, Erdos, Judit, Hviding, Krystyna, Ormstad, Sari Susanna
      First page: 41
      Abstract: ObjectivesThe objective of the European Network for Health Technology Assessment (EUnetHTA) Joint Action 3 (JA3) was to develop a sustainable European model for future collaboration on HTA, by reducing duplication in HTA production and increasing patient access to health technologies. Compared to the previous JA2, several procedural changes were made aiming to increase usability, transparency, and inclusiveness of relative effectiveness assessments (REAs). This article presents and highlights these changes, explains their rationale as well as their influence on HTA production.MethodsFeedback from REA teams and project managers was collected. At the end of JA3, all lessons learned were mapped, resulting in a set of recommendations for a future REA production process.ResultsIn JA3, forty-three EUnetHTA REAs have been produced. Efforts to increase the usability of the REAs were made by focussing on the needs of REA producers and users (HTA agencies) and by increasing stakeholder involvement. Huge steps were taken with regard to transparency, which was achieved through publication of guidances, templates, and up-to-date information on the EUnetHTA website. In an attempt to improve inclusiveness, (stakeholder) interaction and involvement as well as feedback procedures were enhanced and streamlined. The fine-tuned project management brought all aspects together and facilitated a consistent and reliable workflow.ConclusionsDespite that HTA agencies have different national requirements, the procedural changes made in JA3 proved to counteract some of these challenges. Nevertheless, it is of utmost importance that further perceived methodological differences are being resolved to ensure a strong base for future European collaboration on REA production.
      PubDate: 2022-05-26
      DOI: 10.1017/S0266462322000058
       
  • Transferability of a EUnetHTA relative effectiveness assessment to low-
           and middle-income countries setting

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      Authors: Padmasawitri; T. I. Armina, Fuady, Ahmad
      First page: 42
      Abstract: In 2020, European Network for Health Technology Assessment (EUnetHTA) published a relative effectiveness analysis (REA) of Pretomanid in combination with Bedaquiline and Linezolid for the treatment of extensively drug-resistant (XDR) or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB) (REA PTJA14). This REA may have a significant value for low- and middle-income countries (LMICs) outside Europe, particularly those with a high burden of drug-resistant TB. This commentary focuses on whether the REA PTJA14 can be transferred and to what extent a REA can be translated to LMICs context outside Europe. We found that the assessments on the clinical effectiveness and risks of bias reported in REA PTJA14 are useful for LMICs outside Europe. The highly standardized management of TB will support the applicability of the REA to LMICs outside of Europe. Transferring this REA can reduce workload and efficiently use limited resources to conduct health technology assessment (HTA). However, the transfer should consider several critical issues, including variations in health system delivery and clinical practice and setting-specific constraints. In the TB context, the differences in the current standard treatment for XDR or nonresponsive MDR TB, resources availability for drug-resistant TB management, and how healthcare is delivered in the countries can complicate the applicability of the REA PTJA14. Given that LMICs have limitations in doing HTA, it is now critical to develop standard guidelines for transferring REA or other HTA results from high-income countries or other LMICs to maximize the benefits of the REA for LMICs outside Europe.
      PubDate: 2022-04-28
      DOI: 10.1017/S0266462322000241
       
  • Facilitating More Efficient Negotiations for Innovative Therapies: A
           Value-Based Negotiation Framework – Corrigendum

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      Authors: Whittal; Amanda, Jommi, Claudio, De Pouvourville, Gérard, Taylor, David, Annemans, Lieven, Schoonaert, Lies, Vermeersch, Sebastian, Hutchings, Adam, Patris, Julien
      First page: 43
      PubDate: 2022-05-24
      DOI: 10.1017/S0266462322000290
       
  • Health Technology Assessment in Support of National Health Insurance in
           South Africa

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      Authors: Wilkinson; Maryke, Gray, Andrew Lofts, Wiseman, Roger, Kredo, Tamara, Cohen, Karen, Miot, Jacqui, Blecher, Mark, Ruff, Paul, Johnson, Yasmina, Poluta, Mladen, McGee, Shelley, Leong, Trudy D, Brand, Mark, Suleman, Fatima, Maramba, Esnath, Blockman, Marc, Jugathpal, Janine, Cleary, Susan, Nematswerani, Noluthando, Moodliar, Sarvashni, Parrish, Andy, Jamaloodien, Khadija K, Stander, Tienie, MacQuilkan, Kim, Crisp, Nicholas, Wilkinson, Thomas
      First page: 44
      Abstract: South Africa has embarked on major health policy reform to deliver universal health coverage through the establishment of National Health Insurance (NHI). The aim is to improve access, remove financial barriers to care, and enhance care quality. Health technology assessment (HTA) is explicitly identified in the proposed NHI legislation and will have a prominent role in informing decisions about adoption and access to health interventions and technologies. The specific arrangements and approach to HTA in support of this legislation are yet to be determined. Although there is currently no formal national HTA institution in South Africa, there are several processes in both the public and private healthcare sectors that use elements of HTA to varying extents to inform access and resource allocation decisions. Institutions performing HTAs or related activities in South Africa include the National and Provincial Departments of Health, National Treasury, National Health Laboratory Service, Council for Medical Schemes, medical scheme administrators, managed care organizations, academic or research institutions, clinical societies and associations, pharmaceutical and devices companies, private consultancies, and private sector hospital groups. Existing fragmented HTA processes should coordinate and conform to a standardized, fit-for-purpose process and structure that can usefully inform priority setting under NHI and for other decision makers. This transformation will require comprehensive and inclusive planning with dedicated funding and regulation, and provision of strong oversight mechanisms and leadership.
      PubDate: 2022-05-06
      DOI: 10.1017/S0266462322000265
       
  • A model-based study to estimate the health and economic impact of health
           technology assessment in Thailand

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      Authors: Kingkaew; Pritaporn, Budtarad, Nuttakarn, Khuntha, Sarayuth, Barlow, Euan, Morton, Alec, Isaranuwatchai, Wanrudee, Teerawattananon, Yot, Painter, Chris
      First page: 45
      Abstract: ObjectivesHealth technology assessment (HTA) plays a central role in the coverage and reimbursement decision-making process for public health expenditure in many countries, including Thailand. However, there have been few attempts to quantitatively understand the benefits of using HTA to inform resource allocation decisions. The objective of this research was to simulate the expected net monetary benefit (NMB) from using HTA-based decision criteria compared to a first-come, first-served (FCFS) approach using data from Thailand.MethodsA previously published simulation model was adapted to the Thai context which aimed to simulate the impact of using different decision-making criteria to adopt or reject health technologies for public reimbursement. Specifically, the simulation model provides a quantitative comparison between an HTA-based funding rule and a counterfactual (FCFS) funding rule to make decisions on which health technologies should be funded. The primary output of the model was the NMB of using HTA-based decision criteria compared to the counterfactual approach. The HTA-based decision rule in the model involved measuring incremental cost-effectiveness ratios against a cost-effectiveness threshold. The counterfactual decision rule was a FCFS (random) selection of health technologies.ResultsThe HTA-based decision rule was associated with a greater NMB compared to the counterfactual. In the investigated analyses, the NMB ranged from THB24,238 million (USD725 million) to THB759,328 million (USD22,719 million). HTA-based decisions led to fewer costs, superior health outcomes (more quality-adjusted life-years).ConclusionsThe results support the hypothesis that HTA can provide health and economic benefits by improving the efficiency of resource allocation decision making.
      PubDate: 2022-05-04
      DOI: 10.1017/S0266462322000277
       
  • Values, principles, strategies, and frameworks underlying patient and
           public involvement in health technology assessment and guideline
           development: a scoping review

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      Authors: Pinho-Gomes; Ana-Catarina, Stone, James, Shaw, Toni, Heath, Andrea, Cowl, Jane, Norburn, Laura, Thomas, Victoria, Scott, Sarah
      First page: 46
      Abstract: The importance of patient and public involvement (PPI) is recognized by agencies involved in health technology assessment (HTA) and guideline development. However, a comprehensive overview of the underlying PPI principles, values, strategies, and frameworks is lacking. This scoping review aimed to summarize the available evidence on principles, values, frameworks, and strategies underpinning PPI carried out by agencies involved in HTA and guideline development. A total of twelve records were included, of which four referred to guidelines and eight to HTA. Overall, this review demonstrated a lack of consistency in the definition and application of the concepts of values and principles to PPI in the context of guideline development and HTA. There was significant overlap between values and principles, with some broad themes emerging, such as representation, transparency, relevance, equity, fairness, and reconciling different types of knowledge. Frameworks were typically based on the stages of guideline development or HTA, despite heterogeneity in how stages were labeled and described. Strategies were also mapped to the stages of guideline development and HTA and varied substantially depending on the context and setting. Both strategies and frameworks demonstrated patients and the public can be involved, albeit to a variable extent, throughout the stages of guideline development and HTA. However, frameworks often failed to explicitly link the values and principles with the HTA and guideline development stages through actionable PPI strategies. Further research is warranted to better understand the values, principles, and frameworks underpinning PPI in guideline development and HTA.
      PubDate: 2022-06-03
      DOI: 10.1017/S0266462322000289
       
  • Assessing the value of healthcare innovations: a proposal for an
           integrated health technology assessment–responsible innovation in health
           approach in the “new normal”

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      Authors: Mukherjee; Kanchan, Walley, Tom
      First page: 47
      Abstract: There has been tremendous technological innovation in the healthcare sector, but it has also raised serious ethical and social concerns. The COVID-19 pandemic has only magnified these existing challenges. Hence, addressing these challenges becomes imperative in the “new normal.” In this context, this article uses a narrative synthesis approach to discuss the linkages of health technology, innovation, and policy to identify the challenges of this complex interaction by applying the principles of pragmatism and historicity to the existing literature. Moreover, the existing scientific mechanisms in the form of health technology assessment (HTA) and responsible innovation in health (RIH) are described to address these challenges. Using inductive epistemology, the linkages between HTA and RIH within a health innovation ecosystem framework are discussed for the future application of an integrated approach to address societal challenges. The proposed integrated approach of HTA and RIH is a work in progress and conceptualized as transdisciplinary, flexible, and adaptive, which is expected to facilitate future discussion, research, and policy action.
      PubDate: 2022-05-13
      DOI: 10.1017/S0266462322000319
       
  • Feasibility study to characterize price and reimbursement decision-making
           criteria for the inclusion of new drugs in the Spanish National Health
           System: the cefiderocol example

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      Authors: Calleja; Miguel Ángel, Badia, Xavier
      First page: 48
      Abstract: ObjectivesThe reimbursement of medicines by the Spanish National Health System (NHS) is based on a set of criteria included in the Royal Legislative Decree 1/2015 (RDL 1/2015). The Interministerial Committee on Pricing of Medicines and Healthcare Products (CIPM) is responsible for the final price and reimbursement (P&R) decision, including on its resolutions the criteria listed in the law by which the reimbursement of a drug is approved or denied. Nevertheless, the information behind its reasoning is not provided. The present study aims to characterize the P&R criteria of the RDL 1/2015 through criteria definitions from other countries to improve the P&R evaluation in Spain.ResultsA multidisciplinary experts panel with relevant experience in drug evaluation and decision making at national, regional, and local level in Spain was selected for this study. A literature review to characterize the criteria listed in the RDL 1/2015 was performed based on the most relevant and recognized Health Technology Assessment (HTA) agencies in Europe, UK, and Canada. Eventually, a feasibility study was performed to evaluate the novel drug cefiderocol using the characterized criteria, including a reflective discussion of the results.ConclusionsConsensus was reached among the multidisciplinary experts on the characterization of the criteria set by the law. The feasibility of their application to a new drug was exploratory, notwithstanding it showed the potential to improve the transparency as well as to offer a more structured rationale for the CIPM to evaluate the inclusion of new drugs in the Spanish NHS.
      PubDate: 2022-05-20
      DOI: 10.1017/S0266462322000332
       
  • Current Issues in Health Technology Assessment of Cancer Therapies: A
           Survey of Stakeholders and Opinion Leaders in Australia

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      Authors: Kim; Hansoo, Liew, Danny, Goodall, Stephen
      First page: 49
      Abstract: ObjectiveThe aim of this study was to find ways of bridging the gap in opinions concerning health technology assessment (HTA) in reimbursement submission between manufacturers and payers to avoid access delays for patients of vital medicines such as oncology drugs. This was done by investigating differences and similarities of opinion among key stakeholders in Australia.MethodsThe survey comprised of nine sections: background demographics, general statements on HTA, clinical claim, extrapolations, quality of life, costs and health resource utilization, agreements, decision making, and capability/capacity. Responses to each question were summarized using descriptive statistics and comparisons were made using chi-square statistics.ResultsThere were ninety-seven respondents in total, thirty-seven from the public sector (academia/government) and sixty from the private sector (industry/consultancies). Private and public sector respondents had similar views on clinical claims. They were divided when it came to extrapolation of survival data and costs and health resource utilization. However, they generally agreed that rebates are useful, outcomes-based agreements are difficult to implement, managed entry schemes are required when data are limited, and willingness to pay is higher in cancer compared to other therapeutic areas. They also agreed that training mostly takes place through on the job training and that guideline updates were a least favored opportunity for continued training.ConclusionsPrivate sector respondents favor methods that reduce the incremental cost-effectiveness ratio when compared to the public sector respondents. There still exist a number of challenges for HTA in oncology and many research opportunities as a result of this study.
      PubDate: 2022-06-15
      DOI: 10.1017/S0266462322000368
       
  • EUnetHTA’s contribution to the new legal framework for health technology
           assessment cooperation in Europe

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      Authors: Imaz-Iglesia; Iñaki, Wild, Claudia
      First page: 50
      Abstract: The history of the European cooperation on health technology assessment (HTA) has reached an important milestone with the adoption of the European HTA Regulation (HTA R) 2021/2282 in Dec 2021 (1). Its publication in the Official Journal of the European Union means a lot to those of us who wish to give HTA a stronger role in supporting health policy in favor of sustainable healthcare systems in Europe. The HTA R was prepared well by the European Commission with an impact assessment on policy options for enhanced EU cooperation on HTA (2) in 2017, followed by 3 years of negotiations with Member States. Now the ratified document stipulates that the European Cooperation on HTA will be based on a legal mandate and no longer stay a voluntary project-based initiative. It also means sustainability, since a permanent Secretariat at the European Commission will be set-up under the HTA R.
      PubDate: 2022-06-02
      DOI: 10.1017/S026646232200037X
       
  • “What’s Keeping Me Up at Night'” Reflections on the
           COVID-19 Pandemic in Asia

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      Authors: Mundy; Linda, Kearney, Brendon
      First page: 51
      Abstract: ObjectivesThe 2021 Health Technology Assessment International (HTAi) Asia Policy Forum (APF) aimed to explore the challenges and opportunities presented by the global COVID-19 pandemic for health systems and industry in the Asia region, to discuss how HTA changed during the pandemic, and what its role may be moving forward into a postpandemic era.MethodsDiscussions during the virtual 2021 APF, informed by a premeeting survey of HTA agencies and industry attendees from the region describing their experience during the pandemic, form the basis of this paper.ResultsDuring the pandemic, regulatory approval for COVID-related technologies was fast-tracked with fewer evidentiary requirements, and little or no HTA being conducted before these technologies were implemented in health systems in the region. “Living HTA” incorporating real-world evidence (RWE) as it was generated became part of the HTA landscape. In comparison, non-COVID technologies experienced regulatory approval and HTA delays. A major concern of APF members was future pandemic preparedness, and to ensure that lessons are learned from COVID-19. Governments need to continue to invest in innovation and allow early regulatory approvals with the increased use of RWE. Capacity building was identified as a key investment, including capacity in HTA, modeling, as well as local production of necessary supplies and equipment. Finally, collaboration at all levels of the health system was viewed as vital.ConclusionsPost-COVID, different forms of HTA must be embraced as part of the new HTA landscape in addition to increased intra- and intercountry collaboration across all sectors of the health system, including regulatory bodies.
      PubDate: 2022-06-15
      DOI: 10.1017/S0266462322000381
       
  • Integrating Empirical Analysis and Normative Inquiry in Health Technology
           Assessment: The Values in Doing Assessments of Health Technologies
           Approach

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      Authors: van der Wilt; Gert Jan, Bloemen, Bart, Grin, John, Gutierrez-Ibarluzea, Iñaki, Sampietro-Colom, Laura, Refolo, Pietro, Sacchini, Dario, Hofmann, Bjørn, Sandman, Lars, Oortwijn, Wija
      First page: 52
      Abstract: Health technology assessment (HTA) aims, through empirical analysis, to shed light on the value of health technologies (O’Rourke et al. [2020, International Journal of Technology Assessment in Health Care 36, 187–90]). HTA is, then, where facts and values meet. But how, where, and when do facts and values meet in HTA' Currently, HTA is usually portrayed as a sequential process, starting with empirical analysis (assessment), followed by a deliberation on the implications of the findings for a judgment of a health technology’s value (appraisal). In this paper, we will argue that in HTA, empirical analysis and normative inquiry are much more closely entwined. In fact, as we hope to show, normative commitments act as an indispensable guide for the collection and interpretation of empirical evidence. Drawing on policy sciences, we will suggest a concrete methodology that can help HTA practitioners to integrate empirical analysis and normative inquiry in a transparent way. The proposed methodology can be conceived as a concrete means for conducting a scoping exercise in HTA. Moreover, it offers a distinct way of giving stakeholders a structural and constructive role in HTA. This paper outlines the approach developed by the values in doing assessments of health technologies project, a project funded by the Erasmus+ program (contract number 2018-1-NL01-KA203-038960), which is the European Union’s program to support education, training, youth, and sport in Europe. The project has resulted in an E-learning course, an accompanying handbook, and a consensus statement, all freely available from the project’s website www.validatehta.eu.
      PubDate: 2022-06-23
      DOI: 10.1017/S0266462321001768
       
  • Health technology assessment: A matter of facts and values

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      Authors: van der Wilt; Gert Jan, Oortwijn, Wija
      First page: 53
      Abstract: The unique task and contribution of health technology assessment (HTA) is to help identify those health technologies and their uses that are most likely to preserve and restore a population’s health in a way that is consonant with its values, including, for example, equity and access to high-quality care (1). Such a task is challenging for at least two reasons. First, because of the vast and constantly evolving number and diversity of health technologies and their applications. Second, because of the usual wide variety of competing views within communities and their stakeholders regarding what strategies are likely to be conducive to the goal of preserving and restoring population health. Although perhaps tempting, it would be a grave mistake to hold that the controversies that result from such competing views can be resolved by taking recourse to the facts only. For such controversies are usually fueled by different notions of health and disease and different specifications of values such as equity and individual and collective responsibility for health. For this reason, they cannot be resolved in a satisfactory way without also addressing those normative issues and their interplay with empirical analysis.*
      PubDate: 2022-06-23
      DOI: 10.1017/S0266462322000101
       
  • Redefining Health Technology Assessment: A Comment on “The New
           Definition of Health Technology Assessment: A Milestone in International
           Collaboration”

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      Authors: Culyer; Anthony, Husereau, Don
      First page: 54
      Abstract: A new definition of health technology assessment (HTA), developed by an International Joint Task Group claims to be a “milestone,” “an historic achievement,” and “a cornerstone reference”—claims that we think to be unjustified. We too favor clear definitions, especially when confusion abounds. However, the Task Group seems to have developed a definition without the help of usual conventions regarding definitions and, in our view, through an ill-described process. A definition ought to differentiate the entity defined from other entities. This one fails to do so. It states traits that are true of HTA (e.g., that is interdisciplinary) but HTA is not alone in this. There are other concerns: examples of HTA’s use are embodied in the definition, precluding other uses; the adjectives used, although generally true of HTA, are not differentiating features; and attributing to HTA specific purposes, thereby excluding other purposes. We have sympathy for these purposes but cannot consider them HTA’s only purposes or even, its main purpose. A newcomer to HTA, on reading this definition, will have no idea of HTA’s true potential. These numerous failings, we feel, send all the wrong signals, and could ultimately weaken, rather than strengthen perceptions of HTA’s legitimacy and objectivity. The production of a good definition remains, therefore, a work in progress.
      PubDate: 2022-07-05
      DOI: 10.1017/S0266462321001756
       
  • Response to redefining health technology assessment: a comment on “the
           new definition of health technology assessment: a milestone in
           international collaboration”

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      Authors: O’Rourke; Brian, Oortwijn, Wija, Schuller, Tara
      First page: 55
      PubDate: 2022-07-05
      DOI: 10.1017/S0266462322000344
       
  • Tisagenlecleucel for relapsed/refractory acute lymphoblastic leukemia in
           the Irish healthcare setting: cost-effectiveness and value of information
           analysis

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      Authors: Carey; Niamh, Leahy, Joy, Trela-Larsen, Lea, McCullagh, Laura, Barry, Michael
      First page: 56
      Abstract: ObjectivesThis study evaluates the cost-effectiveness of tisagenlecleucel (a CAR T-cell therapy), versus blinatumomab, for the treatment of pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (R/R ALL) in the Irish healthcare setting. The value of conducting further research, to investigate the value of uncertainty associated with the decision problem, is assessed by means of expected value of perfect information (EVPI) and partial EVPI (EVPPI) analyses.MethodsA three-state partitioned survival model was developed. A short-term decision tree partitioned patients in the tisagenlecleucel arm according to infusion status. Survival was extrapolated to 60 months; general population mortality with a standardized mortality ratio was then applied. Estimated EVPI and EVPPI were scaled up to population according to the incidence of the decision.ResultsAt list prices, the incremental cost-effectiveness ratio was EUR 73,086 per quality-adjusted life year (QALY) (incremental costs EUR 156,928; incremental QALYs 2.15). The probability of cost-effectiveness, at the willingness-to-pay threshold of EUR 45,000 per QALY, was 16 percent. At this threshold, population EVPI was EUR 314,455; population EVPPI was below EUR 100,000 for each parameter category.ConclusionsTisagenlecleucel is not cost effective, versus blinatumomab, for the treatment of pediatric and young adult patients with R/R ALL in Ireland (at list prices). Further research to decrease decision (parameter) uncertainty, at the defined willingness-to-pay threshold, may not be of value. However, there is a high degree of uncertainty underpinning the analysis, which may not be captured by EVPI analysis.
      PubDate: 2022-07-11
      DOI: 10.1017/S0266462322000356
       
  • Challenges of Conducting Value Assessment for Comprehensive Genomic
           Profiling

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      Authors: Tarride; Jean-Eric, Gould, Toby, Thomas, David M.
      First page: 57
      Abstract: ObjectivesClinical practice is shifting toward an era of precision medicine. The use of comprehensive genomic profiling (CGP) in oncology has broad potential as a universal companion diagnostic for targeted therapies which may significantly improve health outcomes while using healthcare resources more efficiently. Given the nature of this technology, assessing the value of CGP presents unique challenges.MethodsThis paper draws on evidence from the academic and policy literature in oncology, as well as stakeholder interviews (health economists, payers, clinicians, and public policy officials) in countries using incremental cost-effectiveness ratios (ICER) as part of health technology assessment (HTA).ResultsThe degree to which CGP is subject to a value assessment varies significantly across healthcare systems. Current HTA processes focus on evaluating diagnostic testing through co-dependent assessment of diagnostic testing and associated therapeutic interventions. Diagnostic tests with multiple associated therapeutic interventions are rapidly evolving and poorly unsuited to current HTA approaches. Moreover, HTA approaches are limited in their ability to consider broader systemic benefits of the expanded diagnostic capabilities and enhanced opportunities for clinical trial participation offered by CGP.ConclusionsThe assessment of the overall value of CGP is limited by the current models of HTA. This paper suggests policy proposals for value assessment and funding reforms to help broaden patient access to CGP. These include investing in genomic testing infrastructure; decoupling the assessment of the value of CGP testing to identifying predetermined therapeutic interventions; tailoring evaluation methodology; and developing approaches to collecting evidence of clinical, healthcare system and societal benefit.
      PubDate: 2022-06-08
      DOI: 10.1017/S026646232200040X
       
  • Development, testing, and implementation of a new procedure to assess the
           clinical added benefit of pharmaceuticals

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      Authors: Dóczy; Veronika, Wernerné Sódar, Barbara, Hölgyesi, Áron, Merész, Gergő, Gaál, Péter
      First page: 58
      Abstract: ObjectivesThe reimbursement process for innovative health technologies in Hungary lacks any formalized assessment of clinical added benefit (CAB). The aim of this research is to present the development, retrospective testing, and implementation of a local assessment framework for determining the CAB of cancer treatments at the Department of Health Technology Assessment of the National Institute of Pharmacy and Nutrition in Hungary.MethodsThe assessment framework was drafted after screening existing methods and a retrospective comparison of local reimbursement dossiers to that of German and French methods. The Magnitude of Clinical Benefit Scale of the European Society for Medical Oncology was chosen to rate the extent of CAB in oncology, as part of a conclusion complemented by the assessment of endpoint relevance and the quality of evidence. Several rounds of retrospective assessments have been conducted involving all clinical assessors, iterated with semistructured discussions to consolidate divergence between assessors. External stakeholders were consulted to provide feedback on the framework.ResultsRetrospective assessments resulted in average more than 75 percent concordance between assessors on each element of the conclusion. Input from ten stakeholders was also incorporated; stakeholders were generally supportive, and they mostly commented on the concept, the elements of the framework, and its implementation.ConclusionsThe procedure is suitable for routine use in the decision-making process to describe the CAB of antineoplastic technologies in Hungary. Further extension of the framework is required to cover more disease areas for structured and comparable conclusions on CAB of innovative health technologies.
      PubDate: 2022-07-12
      DOI: 10.1017/S0266462322000411
       
  • Methods and criteria for the assessment of orphan drugs: a scoping review

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      Authors: Mohammadshahi; Marita, Olyaeemanesh, Alireza, Ehsani-Chimeh, Elham, Mobinizadeh, Mohammadreza, Fakoorfard, Zeinab, Akbari Sari, Ali, Aghighi, Mohammad
      First page: 59
      Abstract: ObjectivesThis study aimed to identify different criteria for priority setting of rare diseases to help policy makers in making evidence-informed decisions.MethodsA scoping review was conducted to comprehensively examine the existing various methods and criteria for prioritizing orphan drugs and rare diseases. We performed searching in Scopus, PubMed, Embase, and websites of health technology assessment (HTA) agencies, 2000–21, and data were extracted.ResultsFrom the 1,580 identified publications, eleven articles were included. Multicriteria decision analysis was the most frequent method (seven out of eleven studies) used for priority setting. The extracted criteria for priority setting of orphan products were analyzed based on six main categories as follows: health outcomes and clinical implications (six subsets which showed clinical implications), economic aspects (four subsets that indicated the economic effects of orphan drugs and rare diseases), disease and population characteristics (six subsets that included the characteristics of the rare diseases), therapeutic alternatives and uniqueness of orphan technologies (two subsets which discussed the alternatives and uniqueness of orphan technologies), evidence (three subsets which regarded the quality and availability of evidence), and other criteria (three subsets dealing with social and organizational criteria). Cost-effectiveness, budget impact, and disease severity were the most frequent criteria in the studies.ConclusionsBecause of the high price of orphan drugs and limitations of using HTA for reimbursement of them, it is critical to explore them by precise technical methods like multiple criteria decision making in priority setting.
      PubDate: 2022-06-22
      DOI: 10.1017/S0266462322000393
       
  • Assessing progression of health technology assessment implementation in
           Asia: a balanced scorecard for cross comparison of selected countries in
           Asia

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      Authors: Kumar; Ritu, Suharlim, Christian, Amaris Caruso, Ana, Gilmartin, Colin, Mehra, Meenakshi, Castro, Hector Eduardo
      First page: 60
      Abstract: ObjectiveTo provide an update on the use of health technology assessment (HTA) in Asia and lessons for countries seeking to advance HTA.MethodsBuild upon the research by Chootipongchaivat and the World Health Organization identifying eighteen “factors conducive to the development of HTA in Asia.” These factors were used to create a balanced scorecard to assess the progress of HTA, measuring progress against each factor in China, India, Indonesia, Malaysia, Philippines, South Korea, Taiwan, Thailand, and Vietnam. A scoring system was used wherein: 1, No progress; 2, milestone at early stages, ad hoc HTA use; 3, progress on milestone but limited impact; 4, significant progress but limited remit; and 5, significant progress on milestone, routine HTA informs decisions. Total scores indicated progress of HTA while milestone scores provided contextual insights within countries. Literature reviews and expert interviews were used to complete scorecards.ResultsSouth Korea and Thailand scored highest with seventy-three and seventy-one points, respectively, while Vietnam scored lowest at 28.5. Advanced HTA programs have independent HTA agencies with a broad remit, explicit process and methods, network of researchers, and routine use of HTA. Taiwan and Malaysia fall in a middle tier, with established HTA programs with limited remit. The final tier with China, India, Indonesia, Philippines, and Vietnam, emerging HTA processes.ConclusionsUniversal Health Coverage goals have catalyzed expansion of HTA. Political will, technical expertise, and sustained financing remain challenges for sustainable HTA programs. Legislation supporting HTA is helpful but political will is key. Recommendations for regional collaboration are provided.
      PubDate: 2022-07-21
      DOI: 10.1017/S0266462322000423
       
  • Adjustment of publication bias using a cumulative meta-analytic framework

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      Authors: Canestaro; W. J., Devine, E. B., Bansal, A., Sullivan, S. D., Carlson, J. J.
      First page: 61
      Abstract: ObjectivesPublication bias has the potential to adversely impact clinical decision making and patient health if alternative decisions would have been made had there been complete publication of evidence.MethodsThe objective of our analysis was to determine if earlier publication of the complete evidence on rosiglitazone’s risk of myocardial infarction (MI) would have changed clinical decision making at an earlier point in time. We tested several methods for adjustment of publication bias to assess the impact of potential time delays to identifying the MI effect. We then performed a cumulative meta-analysis (CMA) for both published studies (published-only data set) and all studies performed (comprehensive data set). We then created an adjusted data set using existing methods of adjustment for publication bias (Harbord regression, Peter’s regression, and the nonparametric trim and fill method) applied to the limited data set. Finally, we compared the time to the decision threshold for each data set using CMA.ResultsAlthough published-only and comprehensive data sets did not provide notably different final summary estimates [OR = 1.4 (95 percent confidence interval [CI]: .95–2.05) and 1.42 (95 percent CI: 1.03–1.97)], the comprehensive data set reached the decision threshold 36 months earlier than the published-only data set. All three adjustment methods tested did not show a differential time to decision threshold versus the published-only data set.ConclusionsComplete access to studies capturing MI risk for rosiglitazone would have led to the evidence reaching a clinically meaningful decision threshold 3 years earlier.
      PubDate: 2022-06-28
      DOI: 10.1017/S0266462322000435
       
  • Cost-utility of a web-based intervention to promote maternal mental health
           among postpartum women presenting low risk for postpartum depression

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      Authors: Monteiro; Fabiana, Antunes, Patrícia, Pereira, Marco, Canavarro, Maria C., Fonseca, Ana
      First page: 62
      Abstract: ObjectivesWeb-based interventions for the promotion of maternal mental health could represent a cost-effective strategy to reduce the burden associated with perinatal mental illness. This study aimed to evaluate the cost-utility of Be a Mom, a self-guided web-based cognitive behavioral therapy intervention, compared with a waiting-list control.MethodsThe economic evaluation alongside a randomized controlled trial was conducted from a societal perspective over a 14-month time frame. Postpartum women presenting low risk for postpartum depression were randomized to the intervention (n = 191) or control (n = 176) group and assessed at baseline, postintervention and 4 and 12 months after postintervention. Data regarding healthcare use, productive losses and quality-adjusted life years (QALYs) were collected and used to calculate incremental cost-effectiveness ratios (ICERs). Uncertainty was accounted for with nonparametric bootstrapping and sensitivity analyses.ResultsAt 14 months, and after accounting for a 3.5 percent discount rate, the intervention resulted in a yearly cost-saving of EUR 165.47 (−361.77, 28.51) and a QALY gain of 0.0064 (−0.0116, 0.0244). Bootstrapping results revealed a dominant ICER for the intervention group. Although results were statistically nonsignificant, cost-effectiveness acceptability curves showed that at a EUR 0 willingness to pay threshold, there is a 96 percent probability that the intervention is cost-effective when compared with the control group. The sensitivity analyses generally supported the acceptable likelihood of the intervention being more cost-effective than the control group.ConclusionsFrom a societal perspective, the implementation of Be a Mom among low-risk postpartum women could be a cost-effective way to improve perinatal mental health.
      PubDate: 2022-07-21
      DOI: 10.1017/S0266462322000447
       
  • Learning and practicing more value-reflective, problem-setting health
           technology assessment: experiences and lessons from the VALIDATE project

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      Authors: Grin; John, Bloemen, Bart, Gutierrez-Ibarluzea, Iñaki, Hofmann, Bjørn, Oortwijn, Wija, Refolo, Pietro, Sacchini, Dario, Sampietro-Colom, Laura, Sandman, Lars, van der Wilt, Gert Jan
      First page: 63
      Abstract: ObjectivesTo conduct a formative evaluation of applying the VALIDATE approach in practice by (i) assessing how students appreciated the e-learning course, (ii) exploring how, for what purposes and with what outcomes the acquired VALIDATE competences subsequently were used in internships in different institutional contexts, and how this was shaped by these contexts, and (iii) what this shows on real-world use of VALIDATE.MethodsComparative discussion of experiences of applying the VALIDATE approach via a semistructured survey and oral feedback from e-course students; final reports on internships in health technology assessment (HTA) practice, followed by semistructured interviews with interns and supervisors to complement and interpret results.ResultsAll students considered the VALIDATE approach an enlightening and important addition to current HTA knowledge, especially regarding understanding the relation between empirical analysis and normative inquiry, identifying policy relevant questions and using the method of reconstructing interpretive frames for scoping. The latter appeared intellectually challenging and requiring some prior HTA knowledge. The use the VALIDATE approach in practice shows that interns productively redefined the HTA problem, based on appreciation of different stakeholders’ definition of the issue; they experienced constraints from retrieving all relevant perspectives from existing literature as well as from institutional rules and routines.ConclusionsSome challenges in applying the VALIDATE approach deserve attention for its future use: currently used research approaches often assume a problem as “given”; and the data needed on different perspectives is often not reported in scientific literature. Finally, data gathering on and evaluation of value dimensions was experienced as challenging.
      PubDate: 2022-06-21
      DOI: 10.1017/S0266462322000204
       
  • A multi-criteria decision analysis on the value of nintedanib for
           interstitial lung diseases

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      Authors: Zozaya; Néboa, Arrizubieta Basterrechea, Maria Iciar, Bollo, Elena, Castellví, Iván, Espín, Jaime, Ortego, Norberto, Poveda-Andrés, José Luis, Rodríguez Portal, José Antonio, Rivero, Agustín, Marcos-Rodríguez, José Antonio, Verde, Luis
      First page: 64
      Abstract: ObjectivesOur aim was to assess the value of nintedanib for non-idiopathic progressive fibrosing interstitial lung disease (non-IPF PF-ILD) and systemic sclerosis-associated ILD (SSc-ILD) in the Spanish context, using a multi-criteria decision analysis (MCDA).MethodsFollowing an adaptation of the Evidence and Value: Impact on DEcision Making (EVIDEM) MCDA methodology, the estimated value of nintedanib was obtained by means of an additive linear model that combined individual weights (100-points distribution) of criteria with the individual scoring of nintedanib in each criterion for every indication, assigned by a multidisciplinary committee of twelve clinicians, patients, pharmacists, and decision-makers. To assess the reproducibility, an alternative weighting method was applied, as well as a re-test of weights and scores at a different moment of time.ResultsThe experts committee recognized nintedanib as an intervention with a positive value contribution in comparison to placebo for the treatment of non-IPF PF-ILD (0.50 ± 0.16, on a scale from −1 to 1) and SSc-ILD (0.40 ± 0.12), diseases which were considered as very severe and with high unmet needs. The drug was perceived as a treatment that provides an added therapeutic benefit for patients (0.06–0.07), given its proven clinical efficacy (0.05–0.06), slight improvements in patient-reported outcomes (0.01–0.02), and similar safety profile than placebo (−0.04–0.00), which will likely be positioned as a recommended therapy in the next clinical practice guidelines updates.ConclusionsUnder this increasingly used methodology, nintedanib has shown to provide a positive value estimate for non-IPF PF-ILD and SSc-ILD when compared to placebo in Spain.
      PubDate: 2022-08-01
      DOI: 10.1017/S0266462322000459
       
  • Test–retest reliability of the Cost for Patients Questionnaire

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      Authors: Poder; Thomas G., Coulibaly, Lucien P., Hassan, Abakar Idriss, Conombo, Blanchard, Laberge, Maude
      First page: 65
      Abstract: ObjectivesTo investigate the test–retest reliability of the Costs for Patients Questionnaire (CoPaQ).MethodsThrough an online survey, individuals were invited to participate in a two-step study to assess the test–retest reliability of the CoPaQ. Participants to the first step were invited to complete the questionnaire a second time 2 weeks after. Reliability was assessed by calculating Cohen’s Kappa coefficients and intraclass correlation coefficients (ICC) for discrete and continuous data, respectively. A sensitivity analysis was carried out.ResultsFrom a total of 1,200 participants who completed the first test, 403 completed the second test. The ICC varied from −0.00 to 0.98 with poor, moderate, good, and excellent results. The Kappa coefficients varied from −0.004 to 0.65 and were poor, slight, fair, moderate, and substantial. The sensitivity analysis showed the median value of ICC and Kappa coefficients for each category before and after the outliers’ exclusion. The median value of ICC changed from 0.30 (before) to 0.70 (after), and from 0.12 (before) to 0.04 (after), respectively, for each category. The median value of the Cohen’s Kappa coefficient increased from 0.44 (before) to 0.46 (after) and decreased from 0.32 (before) to 0.30 (after), respectively.ConclusionsTest–retest reliability results indicated that the CoPaQ has a moderate reliability in terms of ICC and Kappa coefficients. The moderate reliability observed gives additional support for the applicability of this tool in economic evaluations of health interventions. Additional studies including on other properties and a cultural adaptation could further enhance the use of the tool.
      PubDate: 2022-07-11
      DOI: 10.1017/S0266462322000460
       
  • Economic feasibility of a novel tool to assist extubation decision-making:
           an early health economic modeling

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      Authors: Zheng; Katina, Kumar, Srishti, Sarti, Aimee J., Herry, Christophe L., Seely, Andrew J. E., Thavorn, Kednapa
      First page: 66
      Abstract: ObjectivesTo estimate the minimum percent change in failed extubation to make a tool designed to reduce extubation failure (Extubation Advisor [EA]) economically viable.MethodsWe conducted an early return on investment (ROI) analysis using data from intubated intensive care unit (ICU) patients at a large Canadian tertiary care hospital. We obtained input parameters from the hospital database and published literature. We ran generalized linear models to estimate the attributable length of stay, total hospital cost, and time to subsequent extubation attempt following failure. We developed a Markov model to estimate the expected ROI and performed probabilistic sensitivity analyses to assess the robustness of findings. Costs were presented in 2020 Canadian dollars (C$).ResultsThe model estimated a 1 percent reduction in failed extubation could save the hospital C$289 per intubated patient (95 percent CI: 197, 459). A large center seeing 2,500 intubated ICU patients per year could save C$723,124/year/percent reduction in failed extubation. At the current annual price of C$164,221, the EA tool must reduce extubation failure by at least 0.24 percent (95 percent CI: .14, .41) to make the tool cost-effective at our site.ConclusionsClinical decision-support tools like the EA may play an important role in reducing healthcare costs by reducing the rate of extubation failure, a costly event in the ICU.
      PubDate: 2022-07-11
      DOI: 10.1017/S0266462322000472
       
  • Economic evaluation of genomic/genetic tests: a review and future
           directions

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      Authors: Bouttell; Janet, Heggie, Robert, Oien, Karin, Romaniuk, Amy, VanSteenhouse, Harper, von Delft, Stephan, Hawkins, Neil
      First page: 67
      Abstract: It has been suggested that health economists need to improve their methods in order to meet the challenges of evaluating genomic/genetic tests. In this article, we set out twelve challenges identified from a rapid review of the literature and suggest solutions to the challenges identified. Two challenges were common to all economic evaluations: choice of perspective and time-horizon. Five challenges were relevant for all diagnostic technologies: complexity of analysis; range of costs; under-developed evidence base; behavioral aspects; and choice of outcome metrics. The final five challenges were pertinent for genomic tests and only these may require methodological development: heterogeneity of tests and platforms, increasing stratification, capturing personal utility; incidental findings; and spillover effects. Current methods of economic evaluation are generally able to cope with genomic/genetic tests, although a renewed focus on specific decision-makers’ needs and a willingness to move away from cost-utility analysis may be required. Certain analysts may be constrained by reference cases developed primarily for the assessment of pharmaceuticals. The combined impact of multiple challenges may require analysts to be particularly careful in setting the scope of their analysis in order to ensure that feasibility is balanced with usefulness to the decision maker. A key issue is the under-developed evidence-base and it may be necessary to rethink translation processes to ensure sufficient, relevant evidence is available to support economic evaluation and adoption of genomic/genetic tests.
      PubDate: 2022-08-01
      DOI: 10.1017/S0266462322000484
       
  • Implementation of a new Digi-HTA process for digital health technologies
           in Finland

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      Authors: Haverinen; Jari, Turpeinen, Miia, Falkenbach, Petra, Reponen, Jarmo
      First page: 68
      Abstract: ObjectivesThere has been a lack of health technology assessment (HTA) methods for novel digital health technologies (DHTs) such as mHealth, artificial intelligence, and robotics in Finland. The Digi-HTA method has been developed for this purpose. The aim of this study is to determine whether it would be possible to use Digi-HTA recommendations to support healthcare decision-makers. Secondly, from the perspective of companies offering different types of DHT products, this study assesses the suitability of using the Digi-HTA framework to perform HTAs for their products.MethodsFeedback about Digi-HTA recommendations was collected from healthcare professionals. DHT companies provided input about the Digi-HTA framework. Data were collected via a web-based survey and were analyzed using qualitative methods.ResultsOf the twenty-four healthcare professional respondents, twenty said that the Digi-HTA recommendations contained all the necessary information, and twenty-one found them useful for their work. Respondents hoped that the Digi-HTA recommendations would be better integrated into the decision-making processes and healthcare professionals would be more informed about this new HTA process. The questions of the Digi-HTA framework were applicable for different DHT products based on the responses from DHT companies (n = 8).ConclusionsAccording to the study participants, although the Digi-HTA recommendations include clear and beneficial information, their integration into healthcare decision-making processes should be improved. Responses from DHT companies indicate that the Digi-HTA framework would be an appropriate tool for performing assessments for their products. To generalize the findings of this study, more comprehensive studies will be needed.
      PubDate: 2022-08-19
      DOI: 10.1017/S0266462322000502
       
  • Disinvestment in healthcare: a scoping review of systematic reviews

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      Authors: Kamaruzaman; Hanin Farhana, Grieve, Eleanor, Wu, Olivia
      First page: 69
      Abstract: ObjectivesDisinvestment from low value health technologies is growing globally. Diverse evidence gathering and assessment methods were used to implement disinvestment initiatives, however, less than half of the empirical studies report reduced use of the low-value services. This scoping review aimed to synthesize the information from available reviews on the concepts and purposes of disinvestment in healthcare, the approaches and methods used, the role of stakeholders and facilitators and barriers in its implementation.MethodsThis scoping review was guided by the Joanna Briggs Institute Manual for Evidence Synthesis and PRISMA statement for scoping review. Published reviews on disinvestment were identified from scientific databases including health technology assessment (HTA) Web sites using the terms “disinvestment,” “health technology reassessment,” and “healthcare.” The data obtained was synthesized narratively to identify similarities and differences across the approaches based on the prespecified categories.ResultsSeventeen reviews were included with thirty-four initiatives identified across sixteen countries at various levels of implementation and responsible agencies for the activities. Two most used methods to facilitate disinvestment decisions are Programme Budgeting and Marginal Analysis (PBMA) and HTA. Stakeholder involvement is the most important aspect to be addressed, as it acts as both facilitator and barrier in disinvestment initiatives implementation.ConclusionsDisinvestment programs have been implemented at multilevel, involving multistakeholders and using multiple methods such as PBMA and HTA. However, there is a lack of clarity on the additional dimensions of technical analysis related to these tools. Further research could focus on technology optimization in healthcare as part of overall health technology management.
      PubDate: 2022-07-20
      DOI: 10.1017/S0266462322000514
       
  • Disruptive technologies in health care disenchanted: a systematic review
           of concepts and examples

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      Authors: Perleth; Matthias, Di Bidino, Rossella, Huang, Li-Ying, Jones, Lydia, Mujoomdar, Michelle, Myles, Susan, Pichon-Riviere, Andres, Sabirin, Junainah, Schuller, Tara, Washington, Jennifer
      First page: 70
      Abstract: ObjectivesTo clarify the concept of disruptive technologies in health care, provide examples and consider implications of potentially disruptive technologies for health technology assessment (HTA).MethodsWe conducted a systematic review of conceptual and empirical papers on healthcare technologies that are described as “disruptive.” We searched MEDLINE and Embase from 2013 to April 2019 (updated in December 2021). Data extraction was done in duplicate by pairs of reviewers utilizing a data extraction form. A qualitative data analysis was undertaken based on an analytic framework for analysis of the concept and examples. Key arguments and a number of potential predictors of disruptive technologies were derived and implications for HTA organizations were discussed.ResultsOf 4,107 records, 28 were included in the review. Most of the papers included conceptual discussions and business models for disruptive technologies; only few papers presented empirical evidence. The majority of the evidence is related to the US healthcare system. Key arguments for describing a technology as disruptive include improvement of outcomes for patients, improved access to health care, reduction of costs and better affordability, shift in responsibilities between providers, and change in the organization of health care. A number of possible predictors for disruption were identified to distinguish these from “sustaining” innovations.ConclusionsSince truly disruptive technologies could radically change technology uptake and may modify provision of care patterns or treatment paths, they require a thorough evaluation of the consequences of using these technologies, including economic and organizational impact assessment and careful monitoring.
      PubDate: 2022-05-16
      DOI: 10.1017/S0266462322000307
       
  • Health technology assessment of whole genome sequencing in the diagnosis
           of genetic disorders: a scoping review of the literature

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      Authors: Nurchis; Mario Cesare, Riccardi, Maria Teresa, Damiani, Gianfranco
      First page: 71
      Abstract: ObjectiveThe aim of this scoping review is to map the available evidence about the use of health technology assessment (HTA) in the assessment of whole genome sequencing (WGS).MethodsA scoping review methodology was adopted. The population, concept, and context framework was used to build up the research question and to establish the eligibility criteria. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews was adopted to implement a comprehensive search strategy. Evidence was retrieved from scientific databases and HTA organizations Web sites. Reports were classified as full HTA, mini-HTA, rapid reviews or other.ResultsThe search strategy identified seven reports. Five HTA organizations from five countries elaborated the reports: one full HTA, four rapid reviews, and two classified as others. The reports were mainly focused on the evaluation of the clinical utility and cost-effectiveness of genome-wide sequencing as well as informing policy questions by providing analyses of organizational and ethical considerations.ConclusionsFew HTA organizations are drafting reports for WGS. It is essential to stimulate a critical reflection during the elaboration of HTA reports for WGS to steer choices of decision makers in the establishment of priorities for research and policy and reimbursement rates.
      PubDate: 2022-08-26
      DOI: 10.1017/S0266462322000496
       
  • PP112 Review On Change Management Models In Multi-Lateral,
           Multi-Stakeholder Contexts To Engage Stakeholders – Erratum

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      Authors: Lokhorst; Ayla, Hogervorst, Milou
      First page: 72
      PubDate: 2022-09-28
      DOI: 10.1017/S0266462322000320
       
  • Nonsurgical periodontal treatment in type II diabetics in a middle-income
           country: a cost-effectiveness analysis

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      Authors: Rojas-Gómez; Ana M., Serrano, Juan J., Rosselli, Diego
      First page: 73
      Abstract: ObjectivesTo evaluate the cost-effectiveness of the nonsurgical periodontal treatment (NSPT) compared with supragingival therapy in type II diabetics with periodontitis.MethodsA decision tree analysis was used to estimate the costs and health outcomes of two periodontal therapies in a hypothetical cohort of type II diabetics with periodontitis. The analysis was developed from the perspective of a third-party payer at 1 year and 5 years. Probabilities were derived from two systematic reviews. The costs and resource use were validated by a Delphi expert panel. All costs were expressed in USD, using the 25 May 2021 Colombian pesos market exchange rate (USD 1 = COP 3,350).ResultsNSPT was a dominant alternative compared with subsidized supragingival therapy in type II diabetics with periodontitis, generating savings of USD 87 and 400, during the first year or up to 5 years, respectively, and improving dental survival from 32 to 69 percent.ConclusionsNSPT can generate savings by reducing the complications derived from uncontrolled periodontitis and tooth loss.
      PubDate: 2022-09-28
      DOI: 10.1017/S0266462322000563
       
  • Model for ASsessing the value of Artificial Intelligence in medical
           imaging (MAS-AI)

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      Authors: Fasterholdt; Iben, Kjølhede, Tue, Naghavi-Behzad, Mohammad, Schmidt, Thomas, Rautalammi, Quinnie T.S., Hildebrandt, Malene G., Gerdes, Anne, Barkler, Astrid, Kidholm, Kristian, Rac, Valeria E., Rasmussen, Benjamin S.B.
      First page: 74
      Abstract: ObjectivesArtificial intelligence (AI) is seen as a major disrupting force in the future healthcare system. However, the assessment of the value of AI technologies is still unclear. Therefore, a multidisciplinary group of experts and patients developed a Model for ASsessing the value of AI (MAS-AI) in medical imaging. Medical imaging is chosen due to the maturity of AI in this area, ensuring a robust evidence-based model.MethodsMAS-AI was developed in three phases. First, a literature review of existing guides, evaluations, and assessments of the value of AI in the field of medical imaging. Next, we interviewed leading researchers in AI in Denmark. The third phase consisted of two workshops where decision makers, patient organizations, and researchers discussed crucial topics for evaluating AI. The multidisciplinary team revised the model between workshops according to comments.ResultsThe MAS-AI guideline consists of two steps covering nine domains and five process factors supporting the assessment. Step 1 contains a description of patients, how the AI model was developed, and initial ethical and legal considerations. In step 2, a multidisciplinary assessment of outcomes of the AI application is done for the five remaining domains: safety, clinical aspects, economics, organizational aspects, and patient aspects.ConclusionsWe have developed an health technology assessment-based framework to support the introduction of AI technologies into healthcare in medical imaging. It is essential to ensure informed and valid decisions regarding the adoption of AI with a structured process and tool. MAS-AI can help support decision making and provide greater transparency for all parties.
      PubDate: 2022-10-03
      DOI: 10.1017/S0266462322000551
       
  • Neglected impacts of patient decision-making associated with genetic
           testing

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      Authors: Milverton; Joanne, Carter, Drew
      First page: 75
      Abstract: We highlight non-health-related impacts associated with genetic testing (GT) and knowing one’s genetic status so that health technology assessment (HTA) analysts and HTA audiences may more appropriately consider the pros and cons of GT. Whereas health-related impacts of GT (e.g., increased healthy behaviors and avoidance of harms of unnecessary treatment) are frequently assessed in HTA, some non-health-related impacts are less often considered and are more difficult to measure. This presents a challenge for accurately assessing whether a genetic test should be funded. In health systems where HTA understandably places emphasis on measurable clinical outcomes, there is a risk of creating a GT culture that is pro-testing without sufficient recognition of the burdens of GT. There is also a risk of not funding a genetic test that provides little clinical benefit but nonetheless may be seen by some as autonomy enhancing. The recent development of expanded HTA frameworks that include ethics analyses helps to address this gap in the evidence and bring awareness to non-health-related impacts of GT. The HTA analyst should be aware of these impacts, choose appropriate frameworks for assessing genetic tests, and use methods for evaluating impacts. A new reporting tool presented here may assist in such evaluations.
      PubDate: 2022-10-17
      DOI: 10.1017/S0266462322000575
       
  • Patient-centered health technology assessment: a perspective on engagement
           in health technology assessment by three patient organizations and a
           health technology assessment body

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      Authors: Arntsen; Kathleen A., Blount, Linda G., Dickerson, Bradley J., Koola, Catherine P., Venable, Yvette, Wildman, Patrick
      First page: 76
      Abstract: Patient engagement in health technology assessment (HTA) has become increasingly important over the past 20 years. Academic and practitioner literature has produced numerous case studies and best practice accounts of patient involvement practices around the world. This text analyzes the experience of being involved in an Institute for Clinical and Economic Review (ICER) HTA review in the United States. The analysis comes from the joint perspective of three patient organizations: Lupus and Allied Diseases Association, Inc.; Lupus Foundation of America; and Black Women’s Health Imperative, as well as ICER. We suggest that meaningful, patient-centered engagement, where patient communities are systematically integrated throughout the review, can be a way of returning to the discipline’s roots focusing on technologies’ societal and ethical impact. It is a process that requires robust commitment from all involved but produces assessments relevant to those directly affected by them.
      PubDate: 2022-10-20
      DOI: 10.1017/S0266462322000587
       
  • Using discrete-choice experiments to elicit preferences for digital
           wearable health technology for self-management of chronic kidney disease

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      Authors: Gc; Vijay S, Iglesias, Cynthia P, Erdem, Seda, Hassan, Lamiece, Peek, Niels, Manca, Andrea
      First page: 77
      Abstract: ObjectivesWearable digital health technologies (DHTs) have the potential to improve chronic kidney disease (CKD) management through patient engagement. This study aimed to investigate and elicit preferences of individuals with CKD toward wearable DHTs designed to support self-management of their condition.MethodsUsing the results of our review of the published literature and after conducting qualitative patient interviews, five-choice attributes were identified and included in a discrete-choice experiment. The design consisted of 10-choice tasks, each comprising two hypothetical technologies and one opt-out scenario. We collected data from 113 adult patients with CKD stages 3–5 not on dialysis and analyzed their responses via a latent class model to explore preference heterogeneity.ResultsTwo patient segments were identified. In all preference segments, the most important attributes were the device appearance, format, and type of information provided. Patients within the largest preference class (70 percent) favored information provided in any format except the audio, while individuals in the other class preferred information in text format. In terms of the style of engagement with the device, both classes wanted a device that provides options rather than telling them what to do.ConclusionsOur analysis indicates that user preferences differ between patient subgroups, supporting the case for offering a different design of the device for different patients’ strata, thus moving away from a one-size-fits-all service provision. Furthermore, we showed how to leverage the information from user preferences early in the R&D process to inform and support the provision of nuanced person-centered wearable DHTs.
      PubDate: 2022-10-26
      DOI: 10.1017/S0266462322003233
       
  • Considerations for transferability of health technology assessments: a
           scoping review of tools, methods, and practices

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      Authors: Heupink; Lieke Fleur, Peacocke, Elizabeth Fleur, Sæterdal, Ingvil, Chola, Lumbwe, Frønsdal, Katrine
      First page: 78
      Abstract: Health technology assessment (HTA) is commonly used to guide evidence-informed decisions to optimize resource use, prioritize policies, and support countries to achieve universal health coverage. Producing HTAs requires time, scientific expertise, and political commitment, but these are not available in all settings – especially in low- and middle-income countries (LMIC) where HTA processes may be less institutionalized. Transferring and adapting existing HTAs to local settings may offer a solution while reducing duplication efforts. This scoping review aims to provide an overview of tools, methods, approaches, and considerations which can aid HTA transfers. We systematically searched (from 2005 to 2020) six databases and, using predefined inclusion criteria, included twenty-two studies. Data extraction followed a structured process, while synthesis was more iterative. We identified a common approach for HTA transfers. It follows the de novo process of undertaking original HTAs, but with additional steps to assess relevance (applicability), quality, and transferability, as well as steps to adapt parameters where necessary. The EUnetHTA Adaptation Toolkit was the only tool that provided guidance for adapting multiple HTA domains. Other tools were specific to systematic reviews (n = 1) or economic evaluations (n = 12), where one provided guidance for systematic reviews of economic evaluations. Eight papers reported transferring an HTA, with only one transferring to an LMIC. Finally, we reported issues that may facilitate or hinder transferability. In conclusion, we identified fourteen transfer approaches in the form of guidance or checklists, but harmonized and pragmatic guidance for HTA transfers to suit settings with limited HTA capacity seems warranted.
      PubDate: 2022-11-02
      DOI: 10.1017/S026646232200321X
       
  • Can we use existing guidance to support the development of robust
           real-world evidence for health technology assessment/payer
           decision-making'

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      Authors: Capkun; Gorana, Corry, Sorcha, Dowling, Oonagh, Asad Zadeh Vosta Kolaei, Fatemeh, Takyar, Shweta, Furtado, Cláudia, Jónsson, Páll, Kleinermans, Diane, Lambert, Laurie, Schiel, Anja, Facey, Karen
      First page: 79
      Abstract: Advances in the digitization of health systems and expedited regulatory approvals of innovative treatments have led to increased potential for the use of real-world data (RWD) to generate real-world evidence (RWE) to complement evidence from clinical trials. However, health technology assessment (HTA) bodies and payers have concerns about the ability to generate RWE of sufficient quality to be pivotal evidence of relative treatment effectiveness. Consequently, there is a growing need for HTA bodies and payers to develop guidance for the industry and other stakeholders about the use of RWD/RWE to support access, reimbursement, and pricing. We therefore sought to (i) understand barriers to the use of RWD/RWE by HTA bodies and payers; (ii) review potential solutions in the form of published guidance; and (iii) review findings with selected HTA/payer bodies. Four themes considered key to shaping the generation of robust RWE for HTA bodies and payers were identified as: (i) data (availability, governance, and quality); (ii) methodology (design and analytics); (iii) trust (transparency and reproducibility); and (iv) policy and partnerships. A range of guidance documents were found from trusted sources that could address these themes. These were discussed with HTA experts. This commentary summarizes the potential guidance solutions available to help resolve issues faced by HTA decision-makers in the adoption of RWD/RWE. It shows that there is alignment among stakeholders about the areas that need improvement in the development of RWE and that the key priority to move forward is better collaboration to make data usable for multiple purposes.
      PubDate: 2022-11-02
      DOI: 10.1017/S0266462322000605
       
  • Integrating organizational impacts into health technology assessment
           (HTA): an analysis of the content and use of existing evaluation
           frameworks

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      Authors: Pascal; Christophe, Mathy, Caryn, Bongiovanni, Isabelle, Konishi, Mario
      First page: 80
      Abstract: ObjectiveThe French health technology assessment (HTA) agency initiated a research between 2018 and 2019 with the aim of determining whether other HTA organizations (agencies, bodies, institutes, and expert networks) and researchers had developed an evaluation framework of organizational impacts (OIs).MethodsThree types of investigation were carried out: (i) an analysis of documents published by selected HTA organizations, (ii) a rapid review on the OI issues, (iii) a questionnaire survey to experts of the International Network of Agencies for Health Technology Assessment.ResultsThe analyses highlight six key points: (i) there is no explicit conceptual definition of OIs; (ii) OIs are often not included in a specific dimension of the evaluation or in the same dimensions; (iii) three recurring categories emerge from the assessment of OIs: processes, structure, and culture; (iv) despite its limitations, the European Network for Health Technology Assessment framework (Core Model) is the most mature assessment model to date; (v) the question of the scope of OIs to be considered is unresolved (micro-meso-macro); and (vi) the delineation between OI assessment and economic assessment must be addressed.ConclusionsAlthough the issue of considering OI in HTA has been raised for many years, it remains largely unresolved. Defining the concept of OI is a prerequisite for taking the next step toward an evaluation framework. As the question of the impact of innovation goes beyond the health sector, extensive research on how to define and take into account these OIs may be relevant.
      PubDate: 2022-11-02
      DOI: 10.1017/S0266462322003221
       
  • A model to assess the impact of digital technologies on the health-related
           quality of life

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      Authors: Lockl; Jannik, Schick, Doreen, Stoetzer, Jens-Christian, Huff, Katrin
      First page: 81
      Abstract: ObjectivesHealth-related quality of life (HRQoL) is a vital instrument to account for individuals’ well-being in various settings. However, no model of HRQoL allows for examining the effect of digital technology on HRQoL. Therefore, we extend an established HRQoL model by adding a digital technology-related construct. We refer to this extension as the technology-affected health-related quality of life (TA-HRQoL).MethodsWe investigate the extended TA-HRQoL model through a survey. In the survey, we exemplify the use of digital technology through a device for self-managing bladder dysfunction. Hence, we explore whether the model extension proposed is valid and how determinants of the HRQoL affect patients with bladder dysfunction.ResultsThe results indicate that the use of digital technology improves the HRQoL. In our exemplary use scenario, the digital technology decreases bladder-related functional impairments and increases well-being and life satisfaction directly.ConclusionsOur study may provide evidence for the influence of digital technologies on the HRQoL, thus supporting our model extension. We consider our proposed TA-HRQoL model as valid and as useful to account for the influence of digital technology on an individual’s HRQoL. With the TA-HRQoL model, the impact of a digital technology on an individual’s HRQoL can be assessed.
      PubDate: 2022-11-11
      DOI: 10.1017/S0266462322003245
       
  • The role of ethical analysis in conducting a health technology assessment
           of medical treatments for gender dysphoria

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      Authors: Saarni; Samuli I., Uusitalo, Susanne, Autti-Rämö, Ilona
      First page: 82
      Abstract: ObjectivesTreatment seeking for gender dysphoria (GD) has increased manifold in western countries. This has led to increased interest on evidence-base of treatments, but also discussions related to human rights, identity politics, gender-related structures, and medicalization. Combining these discourses into coherent health policy is difficult. Health technology assessment (HTA) is the golden standard for assessing whether a medical intervention should be included in a health system. A comprehensive HTA should include medical, safety, and cost-utility perspectives, but often also ethical, societal, organizational, and legal concerns. Still, ethics is often omitted in practice. This paper aims to demonstrate how integrated ethical analysis influenced a HTA of complex and controversial topics like GD.MethodsA HTA of medical treatments of GD was conducted using integrated ethical analysis based on the EUnetHTA-model. This integrates ethical thinking into the whole HTA, explicitly analyses ethical topics, and balances arguments using several ethical theories.ResultsIntegrating ethics had a significant impact on the HTA process and recommendations. It influenced how the HTA was planned and executed, emphasized autonomy and justice when creating the recommendations, and helped the workgroup to understand the complexity of combining different stakeholders’ discourses. Tensions between scientific evidence, expectations, and values became explicit.ConclusionsComprehensive HTA provides an important, integrative approach to considering complex and controversial topics in health systems. HTA emphasizes multidisciplinary and multi-stakeholder approach but simultaneously forces a pragmatic, results-oriented, and evidence-based approach on all argumentation. Ethical analysis can facilitate interactions between stakeholders, bridge different discourses, and help formulate widely acceptable guidelines and policy decisions.
      PubDate: 2022-11-14
      DOI: 10.1017/S0266462322003257
       
 
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