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International Journal of Technology Assessment in Health Care
Journal Prestige (SJR): 0.714
Citation Impact (citeScore): 1
Number of Followers: 18  
 
  Hybrid Journal Hybrid journal (It can contain Open Access articles)
ISSN (Print) 0266-4623 - ISSN (Online) 1471-6348
Published by Cambridge University Press Homepage  [353 journals]
  • Patient-reported outcomes using a wearable cardioverter-defibrillator:
           results from a systematic review

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      Authors: Aidelsburger; Pamela, Seyed-Ghaemi, Janine, Bonderman, Diana
      First page: 1
      Abstract: ObjectivesThe aim of this study was to assess the effect of the wearable cardioverter-defibrillator (WCD) on patient-reported outcomes (PRO) in adult patients with high risk for sudden cardiac arrest.MethodsWe performed a systematic literature search in Medline (via PubMed) and Cochrane Library in February 2022 and included studies with a study population ≥18 years and prescribed WCD. PRO include health-related quality of life (QoL), symptoms, utilities, or satisfaction ratings. Study selection was done by two reviewers independently using predefined inclusion and exclusion criteria. Quality assessment of studies as well as data extraction was performed by one author and approved by a second author. Results of the included studies are presented quantitatively.ResultsOne randomized controlled trial (RCT), one comparative non-randomized trial, and three single-arm trials were included. QoL was assessed in four studies, but with different assessment tools. One study additionally evaluated the change in depressive symptoms and anxiety and one study focused on acceptability of WCD but evaluated items that are closely related to QoL. Results of the RCT show no statistically significant difference in QoL assessed by SF-36 and EQ-5D comparing WCD and Guideline-Directed Medical Therapy (GDMT) versus GDMT alone. One comparative study reports an improvement in depressive symptoms and anxiety within groups but no significant difference between groups. Further, one single-arm study reported improvement in QoL between baseline and day 90 and day 180.ConclusionsThe available evidence demonstrates that the usage of WCD is not affecting PRO, like QoL, depressive symptoms or anxiety negatively.
      PubDate: 2023-01-06
      DOI: 10.1017/S0266462322003300
       
  • Direct healthcare costs of non-metastatic castration-resistant prostate
           cancer in Italy

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      Authors: Borsoi; Ludovica, Ciani, Oriana, Fornarini, Giuseppe, Oderda, Marco, Sciarra, Alessandro, Vetrini, Damir, Luccarini, Irene
      First page: 2
      Abstract: ObjectivesThe management of non-metastatic castration-resistant prostate cancer (nmCRPC) is rapidly evolving; however, little is known about the direct healthcare costs of nmCRPC. We aimed to estimate the cost-of-illness (COI) of nmCRPC from the Italian National Health Service perspective.MethodsStructured, individual qualitative interviews were carried out with clinical experts to identify what healthcare resources are consumed in clinical practice. To collect quantitative estimates of healthcare resource consumption, a structured expert elicitation was performed with clinical experts using a modified version of a previously validated interactive Excel-based tool, EXPLICIT (EXPert eLICItation Tool). For each parameter, experts were asked to provide the lowest, highest, and most likely value. Deterministic and probabilistic sensitivity analyses (PSA) were carried out to test the robustness of the results.ResultsTen clinical experts were interviewed, and six of them participated in the expert elicitation exercise. According to the most likely estimate, the yearly cost per nmCRPC patient is €4,710 (range, €2,243 to €8,243). Diagnostic imaging (i.e., number/type of PET scans performed) had the highest impact on cost. The PSA showed a 50 percent chance for the yearly cost per nmCRPC patient to be within €5,048 using a triangular distribution for parameters, and similar results were found using a beta-PERT distribution.ConclusionsThis study estimated the direct healthcare costs of nmCRPC in Italy based on a mixed-methods approach. Delaying metastases may be a reasonable goal also from an economic standpoint. These findings can inform decision-making about treatments at the juncture between non-metastatic and metastatic prostate cancer disease.
      PubDate: 2023-01-06
      DOI: 10.1017/S0266462322003336
       
  • Genuine policy learning is fundamental: the journey of the United Arab
           Emirates toward the establishment of health technology assessment

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      Authors: Ahmad; Maiss, Akhras, Kasem S., Saleh, Shadi
      First page: 3
      Abstract: The launch of innovative technologies has been credited with significant improvements in health indicators, but it comes at a high financial impact, and the value of certain innovations may not be well documented. Health technology assessment (HTA) is a universally established process to assess the incremental value of innovations. Despite its acknowledged value, almost one-third of the countries around the globe have not established yet a formal HTA in their health systems. The UAE is one of the pioneering countries worldwide in adopting innovative health technologies. This emphasizes the importance of exploring the key elements in the UAE’s journey toward the establishment of HTA. Our study aims to articulate an academic insight that can support the ongoing endeavors to establish the HTA in the UAE. This case study was guided by an analytical framework. Data was collected from document review and semistructured interviews, then analyzed by applying the codebook thematic analysis technique. The findings outline multiple facilitators and challenges in the perspective process, as they show a multidimensional interlink between all identified elements. Markedly, leveraging the role of specialized academia and building HTA genuine knowledge are the areas that need the most attention. The originality of this research is associated with analyzing the three health policy pillars: the context, actors, and content in a prospective HTA establishment process. The main practical implications generated from this study are supporting global health organizations, HTA policy entrepreneurs, and academics in improving their strategies and designing more effective HTA policy learning programs.
      PubDate: 2023-01-09
      DOI: 10.1017/S0266462322003348
       
  • Using early health economic modeling to inform medical innovation
           development: a soft robotic sock in poststroke patients in Singapore

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      Authors: Wang; Yi, Low, Fan-Zhe, Low, Yin-Yi, Lai, Hwa-Sen, Lim, Jeong-Hoon, Yeow, Chen-Hua, Teerawattananon, Yot
      First page: 4
      Abstract: ObjectivesBased on a real-world collaboration with innovators in applying early health economic modeling, we aimed to offer practical steps that health technology assessment (HTA) researchers and innovators can follow and promote the usage of early HTA among research and development (R&D) communities.MethodsThe HTA researcher was approached by the innovator to carry out an early HTA ahead of the first clinical trial of the technology, a soft robotic sock for poststroke patients. Early health economic modeling was selected to understand the potential value of the technology and to help uncover the information gap. Threshold analysis was used to identify the target product profiles. Value-of-information analysis was conducted to understand the uncertainties and the need for further research.ResultsBased on the expected price and clinical effectiveness by the innovator, the new technology was found to be cost-saving compared to the current practice. Risk reduction in deep vein thrombosis and ankle contracture, the incidence rate of ankle contracture, the compliance rate of the new technology, and utility scores were found to have high impacts on the value-for-money of the new technology. The value of information was low if the new technology can achieve the expected clinical effectiveness. A list of parameters was recommended for data collection in the impending clinical trial.ConclusionsThis work, based on a real-world collaboration, has illustrated that early health economic modeling can inform medical innovation development. We provided practical steps in order to achieve more efficient R&D investment in medical innovation moving forward.
      PubDate: 2023-01-11
      DOI: 10.1017/S026646232200335X
       
  • Informing decision makers about public preferences for different
           modalities of cancer treatment in the Rhône–Alps region in France

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      Authors: Margier; Jennifer, Gafni, Amiram, Moumjid, Nora
      First page: 5
      Abstract: BackgroundAlternative options to hospital care like home care or local health centers (LHCs) are being advocated. However, no study has measured citizens’ preferences (who will finance these services via taxation) for these options.ObjectivesWe measured (i) citizens’ preferences for these services, that is, respondents stated where they would like to get the treatment; (ii) the strength of their preference.MethodsA computerized survey composed of (i) a decision aid to inform respondents about the three options; (ii) three scenarios, from light-to-heavy care, that respondents should rank from the most to the least preferred option of care. (iii) a contingent valuation survey (CVS) to assess how much respondents were willing to pay for their preferred option (except for hospital care if chosen, because it is the default option and free). (iv) a socio-demographic questionnaire.ResultsData were collected from a representative sample of citizens living in the Rhône–Alps Region (n = 800). The heavier the care was, the more respondents preferred hospital care. Willingness to pay for additional taxation per household/month varied from €13.9 for light care in LHC to €19.1 for heavy home care. The small number of protesting respondents and outliers, and the close correlation between preferences, income, and WTP supports the validity of the CVS.ConclusionIn France, for cancer, not all citizens would prefer to be treated at home rather than in a hospital. Only less than a quarter would prefer LHC. These results show the mismatch between public health policies and the citizens’ preferences.
      PubDate: 2023-01-16
      DOI: 10.1017/S0266462322000599
       
  • A model-based economic analysis of the CFHealthHub intervention to support
           adherence to inhaled medications for people with cystic fibrosis in the UK
           

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      Authors: Tappenden; Paul, Navega Biz, Aline, Hernández Alava, Mónica, Sasso, Alessandro, Sutton, Laura, Ennis, Kate, Elliott, Rachel, Wildman, Martin
      First page: 6
      Abstract: BackgroundAdherence to preventative inhaled therapies in people with cystic fibrosis (CF) is low, resulting in potentially avoidable health losses and the need for costly rescue therapies.ObjectivesTo estimate the cost-effectiveness of the CFHealthHub (CFHH) intervention to support adherence to inhaled medications.MethodsA state transition model was developed to assess the cost-effectiveness of the CFHH intervention versus usual care from the perspective of the UK National Health Service and Personal Social Services over a lifetime horizon. Costs and health outcomes were discounted at a rate of 3.5 percent per annum. Costs were valued at 2021/22 prices. The model structure includes health states defined by survival status, level of lung function, and transplant history. Treatment effects were modeled by changing the probabilities of transitioning between lung function states and reducing exacerbation rates. Model parameters were informed by the CFHH trial, CF Registry data, routine cost databases, literature, and expert opinion. Deterministic and probabilistic sensitivity analyses were undertaken to assess uncertainty.ResultsThe CFHH intervention is expected to generate additional health gains and cost savings compared with usual care. Assuming that it is delivered for 10 years, the CFHH intervention is expected to generate 0.17 additional quality-adjusted life years and cost savings of GBP 1,600 (EUR 1,662) per patient.ConclusionsThe CFHH intervention is expected to dominate usual care, irrespective of the duration over which the intervention is delivered. The modeled benefits and cost savings are smaller than initially expected and are sensitive to relative treatment effects on lung function.
      PubDate: 2023-01-17
      DOI: 10.1017/S0266462322003373
       
  • The value of reducing arthroscopic partial meniscectomy in the treatment
           of degenerative meniscus tears: a budget impact analysis

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      Authors: van Well; Evelien B., Wijn, Stan R.W., Hannink, Gerjon, Grutters, Janneke P.C., Rovers, Maroeska M.
      First page: 7
      Abstract: AimsNumerous studies have shown that arthroscopic partial meniscectomy (APM) is not (cost-) effective in patients with symptoms attributed to a degenerative meniscus tear. We aimed to assess the budget impact of reducing APM in routine clinical practice in this population.Materials and methodsA patient-level state transition model was developed to simulate patients recently diagnosed with a degenerative meniscus tear. Three strategies were compared: “current guideline” (i.e., postpone surgery to at least 3 months after diagnosis), “APM at any time” (i.e., APM available directly after diagnosis), and “nonsurgical” (i.e., APM no longer performed). Total societal costs over 5 years were calculated to determine the budget impact. Probabilistic and deterministic sensitivity analyses were conducted to address uncertainty.ResultsThe average cost per patient over 5 years were EUR 5,077, EUR 4,577, and EUR 4,218, for the “APM at any time,” “current guideline,” and “nonsurgical” strategy, respectively. Removing APM from the treatment mix (i.e., 30,000 patients per year) in the Netherlands, resulted in a reduction in health care expenditures of EUR 54 million (95 percent confidence interval [CI] EUR 38 million–EUR 70 million) compared to the “current guideline strategy” and EUR 129 million (95 percent CI EUR 102 million–EUR 156 million) compared to the “APM at any time” strategy. Sensitivity analyses showed that uncertainty did not alter our conclusions.ConclusionsSubstantial costs can be saved when APM is no longer performed to treat symptoms attributed to degenerative meniscus tears in the Netherlands. It is therefore recommended to further reduce the use of APM to treat degenerative meniscus tears.
      PubDate: 2023-01-18
      DOI: 10.1017/S0266462322003361
       
  • A technical review of the ISPOR Presentations Database: an update on
           changes to the database from the authors and ISPOR

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      Authors: Cooper; Chris, Brown, Anna, Court, Rachel, Schauberger, Ute, Pizzi, Laura, Willke, Richard
      First page: 8
      PubDate: 2023-01-24
      DOI: 10.1017/S0266462322003324
       
  • Scaling up health technology assessment capacities in selected African
           countries – A conceivable route ahead

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      Authors: Mueller; Debjani, Alouane, Leila, Jameleddine, Mouna, Lenoir-Wijnkoop, Irene
      First page: 9
      Abstract: BackgroundThis study aimed to provide a structured description of the commonalities and differences in healthcare structures across Africa to establish a reliable basis for the health technology assessment (HTA) of nutrition and nutrition interventions. A ranking of current nutrition conditions in the general population of the participating countries was included to gain a better understanding of the factors influencing hospital malnutrition (HMN), which will inform future multi-country research.MethodA questionnaire on the structure of the health systems was distributed among ten African countries. Subsections were included that inquired about the drivers or barriers to using principles of HTA to assess nutritional care. Analysis and ranking of malnutrition data were based on data from the Global Hunger Index report and two poverty indicators used by the World Bank.ResultsThe health system structure of each country was identified and described, whereas questions about HTA could not always be analyzed due to a lack of adequate in-depth knowledge and skills in most countries. Early experience from some countries demonstrates a conceivable route ahead for African countries in strengthening the capacity for and implementing HTA in accordance with distinct national healthcare contexts and social determinants of health.ConclusionProblems related to nutritional care represent one of the major priorities in the surveyed countries. A future HMN multi-country study will provide valuable insight into the potential of low-cost primary prevention orientations.
      PubDate: 2023-01-30
      DOI: 10.1017/S0266462323000016
       
  • Novel approach to decision making for orphan drugs

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      Authors: Decker; Barbora, Mlcoch, Tomas, Pustovalova, Anastasie, Dolezal, Tomas
      First page: 10
      Abstract: BackgroundOut of 185 orphan medicinal products (OMPs) registered in 2015–2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia.MethodologyThe innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients’ organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial.ConclusionWe believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of “value-based” decision making.
      PubDate: 2023-02-07
      DOI: 10.1017/S0266462323000053
       
  • A decision-support tool for funding health innovations at a tertiary
           academic medical center

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      Authors: Cai; Yiying, Nazeha, Nuraini, Perera, Shamira, Thiery, Alexandre H., Girard, Michaël J.A., Lee, Chen E., Hong, Weiwei, Graves, Nicholas
      First page: 11
      Abstract: ObjectivesTo report the processes used to design and implement an assessment tool to inform funding decisions for competing health innovations in a tertiary hospital.MethodsWe designed an assessment tool for health innovation proposals with three components: “value to the institution,” “novelty,” and “potential for adoption and scaling.” The “value to the institution” component consisted of twelve weighted value attributes identified from the host institution’s annual report; weights were allocated based on a survey of the hospital’s leaders. The second and third components consisted of open-ended questions on “novelty” and “barriers to implementation” to support further dialogue. Purposive literature review was performed independently by two researchers for each assessment. The assessment tool was piloted during an institutional health innovation funding cycle.ResultsWe used 17 days to evaluate ten proposals. The completed assessments were shared with an independent group of panellists, who selected five projects for funding. Proposals with the lowest scores for “value to the institution” had less perceived impact on the patient-related value attributes of “access,” “patient centeredness,” “health outcomes,” “prevention,” and “safety.” Similar innovations were reported in literature in seven proposals; potential barriers to implementation were identified in six proposals. We included a worked example to illustrate the assessment process.ConclusionsWe developed an assessment tool that is aligned with local institutional priorities. Our tool can augment the decision-making process when funding health innovation projects. The tool can be adapted by others facing similar challenges of trying to choose the best health innovations to fund.
      PubDate: 2023-02-13
      DOI: 10.1017/S0266462323000028
       
  • Stakeholder preferences for attributes of digital health technologies to
           consider in health service funding

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      Authors: von Huben; Amy, Howell, Martin, Norris, Sarah, Wong, Kam Cheong, Tang, James, Kazi, Samia, Laranjo, Liliana, Chow, Clara K., Howard, Kirsten
      First page: 12
      Abstract: ObjectivesHealth service providers are currently making decisions on the public funding of digital health technologies (DHTs) for managing chronic diseases with limited understanding of stakeholder preferences for DHT attributes. This study aims to understand the community, patient/carer, and health professionals’ preferences to help inform a prioritized list of evaluation criteria.MethodsAn online best–worst scaling survey was conducted in Australia, New Zealand, Canada, and the United Kingdom to ascertain the relative importance of twenty-four DHT attributes among stakeholder groups using an efficient incomplete block design. The attributes were identified from a systematic review of DHT evaluation frameworks for consideration in a health technology assessment. Results were analyzed with multinomial models by stakeholder group and latent class.ResultsA total of 1,251 participants completed the survey (576 general community members, 543 patients/carers, and 132 health professionals). Twelve attributes achieved a preference score above 50 percent in the stakeholder group model, predominantly related to safety but also covering technical features, effectiveness, ethics, and economics. Results from the latent class model supported this prioritization. Overall, connectedness with the patient’s healthcare team seemed the most important; with “Helps health professionals respond quickly when changes in patient care are needed” as the most highly prioritized of all attributes.ConclusionsIt is proposed that these prioritized twelve attributes be considered in all evaluations of DHTs that manage chronic disease, supplemented with a limited number of attributes that reflect the specific perspective of funders, such as equity of access, cost, and system-level implementation considerations.
      PubDate: 2023-02-14
      DOI: 10.1017/S0266462323000089
       
  • Environmental impact assessment in health technology assessment:
           principles, approaches, and challenges

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      Authors: Toolan; Michael, Walpole, Sarah, Shah, Koonal, Kenny, Juliet, Jónsson, Páll, Crabb, Nick, Greaves, Felix
      First page: 13
      Abstract: To reduce harm to the environment resulting from the production, use, and disposal of health technologies, there are different options for how health technology assessment (HTA) agencies can consider environmental information. We identified four approaches that HTA agencies can use to take environmental information into account in healthcare decision making and the challenges associated with each approach. Republishing data that is in the public domain or has been submitted to an HTA agency we term the “information conduit” approach. Analyzing and presenting environmental data separately from established health economic analyses is described as “parallel evaluation.” Integrating environmental impact into HTAs by identifying or creating new methods that allow clinical, financial, and environmental information to be combined in a single quantitative analysis is “integrated evaluation.” Finally, evidence synthesis and analysis of health technologies that are not expected to improve health-related outcomes but claim to have relative environmental benefits are termed “environment-focused evaluation.”
      PubDate: 2023-02-23
      DOI: 10.1017/S0266462323000041
       
  • Health technology assessment of diagnostic tests: a state of the art
           review of methods guidance from international organizations

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      Authors: Ferrante di Ruffano; Lavinia, Harris, Isobel M., Zhelev, Zhivko, Davenport, Clare, Mallett, Sue, Peters, Jamie, Takwoingi, Yemisi, Deeks, Jon, Hyde, Chris
      First page: 14
      Abstract: ObjectivesTo identify which international health technology assessment (HTA) agencies are undertaking evaluations of medical tests, summarize commonalities and differences in methodological approach, and highlight examples of good practice.MethodsA methodological review incorporating: systematic identification of HTA guidance documents mentioning evaluation of tests; identification of key contributing organizations and abstraction of approaches to all essential HTA steps; summary of similarities and differences between organizations; and identification of important emergent themes which define the current state of the art and frontiers where further development is needed.ResultsSeven key organizations were identified from 216 screened. The main themes were: elucidation of claims of test benefits; attitude to direct and indirect evidence of clinical effectiveness (including evidence linkage); searching; quality assessment; and health economic evaluation. With the exception of dealing with test accuracy data, approaches were largely based on general approaches to HTA with few test-specific modifications. Elucidation of test claims and attitude to direct and indirect evidence are where we identified the biggest dissimilarities in approach.ConclusionsThere is consensus on some aspects of HTA of tests, such as dealing with test accuracy, and examples of good practice which HTA organizations new to test evaluation can emulate. The focus on test accuracy contrasts with universal acknowledgment that it is not a sufficient evidence base for test evaluation. There are frontiers where methodological development is urgently required, notably integrating direct and indirect evidence and standardizing approaches to evidence linkage.
      PubDate: 2023-02-21
      DOI: 10.1017/S0266462323000065
       
  • Health technology assessment 2025 and beyond: lifecycle approaches to
           promote engagement and efficiency in health technology assessment

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      Authors: Trowman; Rebecca, Migliore, Antonio, Ollendorf, Daniel A.
      First page: 15
      Abstract: Lifecycle considerations have always been part of health technology assessment (HTA). However, the concept of taking a fuller, more holistic “lifecycle approach” is gaining interest in the HTA community. The 2022 HTAi Global Policy Forum (GPF) discussed how adopting a lifecycle approach could promote stakeholder engagement and robust evidence generation, and whether it could enhance information sharing and transparency across stakeholder groups. This article summarizes the discussions held at the 2022 HTAi GPF and subsequent HTAi Annual Meeting panel session that debated some of the key challenges and opportunities, with particular focus on the pre- and postmarket and disinvestment phase activities. Core themes and recommendations identified that collaboration and patient involvement are happening but still needs to be strengthened, and moving to disease-based approaches may help, although individual contexts still need to be considered. Appropriately developed and mandated core outcome sets may help with information sharing and efficiency in all lifecycle activities. Further, methods for the appropriate use of big data and digital data collection should be developed and driven by the HTA community. The value of lifecycle activities should be reviewed; in particular, scientific advice appears valuable, but the magnitude of effect is somewhat unknown due to the challenges around the confidential nature of these activities. Not all lifecycle activities can be conducted for every technology, and while there is a move away from disinvestment phase activities, more structured prioritization criteria are required. This article ends with suggested next steps to bring forward some of the priority recommendations.
      PubDate: 2023-02-23
      DOI: 10.1017/S0266462323000090
       
  • Clinical effectiveness reporting of novel cancer drugs in the context of
           non-proportional hazards: a review of nice single technology appraisals

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      Authors: Salmon; David, Melendez-Torres, G. J.
      First page: 16
      Abstract: ObjectivesThe hazard ratio (HR) is a commonly used summary statistic when comparing time to event (TTE) data between trial arms, but assumes the presence of proportional hazards (PH). Non-proportional hazards (NPH) are increasingly common in NICE technology appraisals (TAs) due to an abundance of novel cancer treatments, which have differing mechanisms of action compared with traditional chemotherapies. The goal of this study is to understand how pharmaceutical companies, evidence review groups (ERGs) and appraisal committees (ACs) test for PH and report clinical effectiveness in the context of NPH.MethodsA thematic analysis of NICE TAs concerning novel cancer treatments published between 1 January 2020 and 31 December 2021 was undertaken. Data on PH testing and clinical effectiveness reporting for overall survival (OS) and progression-free survival (PFS) were obtained from company submissions, ERG reports, and final appraisal determinations (FADs).ResultsNPH were present for OS or PFS in 28/40 appraisals, with log-cumulative hazard plots the most common testing methodology (40/40), supplemented by Schoenfeld residuals (20/40) and/or other statistical methods (6/40). In the context of NPH, the HR was ubiquitously reported by companies, inconsistently critiqued by ERGs (10/28), and commonly reported in FADs (23/28).ConclusionsThere is inconsistency in PH testing methodology used in TAs. ERGs are inconsistent in critiquing use of the HR in the context of NPH, and even when critiqued it remains a commonly reported outcome measure in FADs. Other measures of clinical effectiveness should be considered, along with guidance on clinical effectiveness reporting when NPH are present.
      PubDate: 2023-03-08
      DOI: 10.1017/S0266462323000119
       
  • The added value of applying a disinvestment approach to the process of
           health technology assessment in Italy

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      Authors: Cadeddu; Chiara, Regazzi, Luca, Di Brino, Eugenio, Basile, Michele, Cascini, Fidelia, Paladini, Andrea, Rumi, Filippo, Cicchetti, Americo, Ricciardi, Walter
      First page: 17
      Abstract: ObjectivesThe objective of the present policy analysis was to understand how a disinvestment approach to the process of health technology assessment (HTA), applied to the field of medical devices, might help Italian policymakers to properly spend the resources in healthcare.MethodsPrevious international and national experiences in disinvestment for medical devices were reviewed. Precious insights for the rational expenditure of the resources were derived by assessing the evidence available.ResultsThe disinvestment of ineffective or inappropriate technologies or interventions with an inadequate value-for-money ratio has become a growing priority for National Health Systems. Different international disinvestment experiences of medical devices were identified and described through a rapid review. Although most of them have a strong theoretical framework, their practical application remains difficult. In Italy, there are no examples of large and complex HTA-based disinvestment practices, but their importance is becoming increasingly acknowledged, especially given the need to prioritize the funds provided by Recovery and Resilience Plan.ConclusionsAnchoring decisions on health technologies without reassessing the current technological landscape through a robust HTA model might expose to the risk of not ensuring the best employment of the resources available. Thus, it is necessary to develop a strong HTA ecosystem in Italy through adequate consultation with stakeholders to enable a data-driven and evidence-based prioritization of resources toward choices characterized by high value for both patients and society as a whole.
      PubDate: 2023-03-02
      DOI: 10.1017/S0266462323000107
       
  • Health technology assessment for oral health in the past decade: a scoping
           review

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      Authors: Bhatia; Sonal, Mohanty, Vikrant, Balappanavar, Aswini Y., Chahar, Puneet, Rijhwani, Kavita, Gupta, Radhika
      First page: 18
      Abstract: BackgroundHealth technology assessment (HTA) is the systematic evaluation of various properties and effects of a health technology. HTA can serve as a bridge between the world of knowledge and that of decision making, offering decision makers the best summary of scientific evidence. Scoping HTA reports in the context of dentistry can help researchers identify grey areas; help practitioners make evidence-based decisions and further initiate better policy making.AimTo provide an overview on HTAs pertaining to oral health and dentistry in the past decade, map the extension and scope of the methodological practices, key findings, and limitations.MethodologyA scoping review was conducted using the Joanna Briggs Institute framework. A comprehensive search for HTA reports was done through the International Network of Agencies for Health Technology Assessment Database from January 2010 to December 2020. Consecutively, electronic databases (PubMed and Google Scholar) were searched. Finally, thirty-six reports were included in this review and analyzed.ResultsA total of 709 articles were initially identified, of which thirty-six met the inclusion criteria. Reviewed HTAs focused on various specialties of dentistry worldwide. Maximum number of reports (N = 5) were related to “prosthodontics and dental implants” and technologies related to preventive dentistry were most commonly assessed (N = 4).ConclusionFunctional, appropriate, and evidence-based information provided through HTA pertaining to oral health on a regular basis will enable decision makers to have enough data to make decisions on the future use of new technology, modify existing policies, accelerate its translation into practice, and ensure provision of robust dental healthcare services.
      PubDate: 2023-03-27
      DOI: 10.1017/S0266462322003312
       
  • Patient-based evidence: its role in decision making on end-of-life,
           orphan, and ultra-orphan medicines

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      Authors: Hems; Sharon, Taylor, Louise, Jones, Jan, Holmes, Eileen
      First page: 19
      Abstract: ObjectivesThe Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of NHSScotland. Assessment of end-of-life (EoL), orphan, and ultra-orphan medicines includes a process to gather evidence from patients and carers during Patient and Clinician Engagement (PACE) meetings. The output of PACE meetings is a consensus statement describing the medicine’s added value from the perspective of patients/carers and clinicians. The PACE statement is used by SMC committee members in decision making. This study compared how PACE participants and SMC committee members rate the importance of information in PACE statements for these medicines.MethodsA survey was undertaken of patient group (PG) representatives and clinicians who participated in PACE meetings, and SMC committee members.ResultsPACE participants who responded (26 PG representatives and 14 clinicians) rated health benefits and ability to take part in normal life as important/very important. Convenience of administration and treatment choice received the lowest rating. Hope for the future received the most diverse response. PACE participants generally rated the importance of quality of life themes higher than committee members (n = 20) but the rank order was similar. Differences between the proportion of PACE participants and committee members who rated themes as important/very important were greatest for treatment choice and hope for the future.ConclusionsIn general, PACE themes and subthemes that were rated highly by PACE participants were also considered important by SMC committee members, indicating that information captured during PACE meetings is relevant when making decisions on EoL, orphan, and ultra-orphan medicines.
      PubDate: 2023-04-11
      DOI: 10.1017/S026646232300003X
       
  • Regulatory, health technology assessment and company interactions: the
           current landscape and future ecosystem for drug development, review and
           reimbursement

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      Authors: Wang; Ting, McAuslane, Neil, Goettsch, Wim G., Leufkens, Hubert G.M., De Bruin, Marie L.
      First page: 20
      Abstract: BackgroundMulti-stakeholder interactions have evolved at product and policy levels. There is a need to assess the current and future landscape of interactions between companies, and regulatory and HTA agencies to address challenges and identify areas for improvement.ObjectivesThe aims of this study were to review the current interactions within and across regulatory and HTA agencies, and companies’ experiences in engaging in these activities; to assess the added value of interactions as well as limitations; to explore the future ecosystem for stakeholder interactions.MethodThree separate questionnaires were developed for companies, regulators and HTA agencies, respectively, to assess their experiences and perceptions. The responses were analyzed using descriptive statistics and discussed at a multi-stakeholder workshop. Key outcomes from the surveys and workshop discussion were reported.ResultsAll seven regulators and seven HTA agencies in the survey indicated that they had stakeholder interactions. More formal collaboration occurred with regulators compared with HTA agencies. All nine companies have taken early advice but indicated the need for future prioritization. Success indicators can be built at the product and therapy levels, with the added value of faster patient access. Four principles were proposed for the future ecosystem: separate remit and functions between regulators and HTA; align processes; converge evidence requirements where possible; increase transparency.ConclusionsThis research brought together regulators, HTA agencies, companies to examine how they interact with one another. We propose measures of value and make recommendations on future evolution to enable better evidence generation and improve regulatory and HTA decision-making.
      PubDate: 2023-04-11
      DOI: 10.1017/S0266462323000144
       
  • Patient preferences do matter: a discrete choice experiment conducted with
           breast cancer patients in six European countries, with latent class
           analysis

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      Authors: Stamuli; Eugena, Corry, Sorcha, Foss, Petter
      First page: 21
      Abstract: ObjectivesThe evolution of breast cancer (BC) treatments has resulted in tailored therapies for the different types and stages of BC. Each treatment has a profile of benefits and adverse effects which are taken into consideration when planning a treatment pathway. This study examines whether patients’ preferences are in line with what is considered important from decision makers viewpoint.MethodsAn online discrete choice experiment was conducted in six European countries (France, Germany, Ireland, Poland, Spain, UK) with BC patients. Six attributes were included: overall survival (OS), hyperglycemia, rash, pain, functional well-being (FWB), and out-of-pocket payment (OOP). Sixteen choice sets with two hypothetical treatments and a “No treatment” option were presented. Data were analyzed with the use of heteroscedastic conditional, mixed logistic, and latent class models. Marginal rate of substitution (MRS) were estimated for OOP versus the rest of attributes to establish the ranking of preferences for each attribute.ResultsTwo hundred and forty-seven patients with advanced or metastatic BC and 314 with early-stage BC responded. Forty-nine percent of patients were 
      PubDate: 2023-04-19
      DOI: 10.1017/S0266462323000168
       
  • Involvement of information specialists and statisticians in systematic
           reviews

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      Authors: Waffenschmidt; Siw, Bender, Ralf
      First page: 22
      Abstract: BackgroundSystematic reviews (SRs) are usually conducted by a highly specialized group of researchers. The routine involvement of methodological experts is a core methodological recommendation. The present commentary describes the qualifications required for information specialists and statisticians involved in SRs, as well as their tasks, the methodological challenges they face, and potential future areas of involvement.Tasks and qualificationsInformation specialists select the information sources, develop search strategies, conduct the searches, and report the results. Statisticians select the methods for evidence synthesis, assess the risk of bias, and interpret the results. The minimum requirements for their involvement in SRs are a suitable university degree (e.g., in statistics or librarian/information science or an equivalent degree), methodological and content expertise, and several years of experience.Key argumentsThe complexity of conducting SRs has greatly increased due to a massive rise in the amount of available evidence and the number and complexity of SR methods, largely statistical and information retrieval methods. Additional challenges exist in the actual conduct of an SR, such as judging how complex the research question could become and what hurdles could arise during the course of the project.ConclusionSRs are becoming more and more complex to conduct and information specialists and statisticians should routinely be involved right from the start of the SR. This increases the trustworthiness of SRs as the basis for reliable, unbiased and reproducible health policy, and clinical decision making.
      PubDate: 2023-04-25
      DOI: 10.1017/S026646232300020X
       
  • Beyond clinical and cost-effectiveness: The contribution of qualitative
           research to health technology assessment

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      Authors: Germeni; Evi, Szabo, Shelagh
      First page: 23
      Abstract: Recent developments in health technology assessment (HTA), including the promotion of a new and internationally accepted definition of HTA, have highlighted the need to go beyond clinical and cost-effectiveness to fully understand the potential value of health technologies. Multidisciplinary efforts to generate patient-focused evidence relevant to HTA, using both quantitative and qualitative approaches, are needed. Although it has been more than 20 years since opportunities for qualitative methods to inform HTA were first discussed, their use remains infrequent. The goal of this article is to resurrect the debate about the value of qualitative research in HTA. Drawing on examples from published literature, we propose five key areas where qualitative methods can contribute to HTA, complementary to studies of clinical and cost-effectiveness: (i) assessing acceptability and subjective value; (ii) understanding perspectives and providing context; (iii) reaching the groups other methods cannot reach; (iv) laying the groundwork for subsequent quantitative exercises; and (v) contributing to economic model development.
      PubDate: 2023-04-24
      DOI: 10.1017/S0266462323000211
       
  • Health technology assessment of tests for SARS-CoV-2 and treatments for
           COVID-19: A proposed approach and best-practice recommendations

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      Authors: Elvidge; Jamie, Summerfield, Ashley, Knies, Saskia, Németh, Bertalan, Kaló, Zoltán, Goettsch, Wim, Dawoud, Dalia M.
      First page: 24
      Abstract: ObjectivesTo develop best-practice guidance for health technology assessment (HTA) agencies when appraising diagnostic tests for SARS-CoV-2 and treatments for COVID-19.MethodsWe used a policy sandbox approach to develop best-practice guidance for HTA agencies to approach known challenges associated with assessing tests and treatments for COVID-19. The guidance was developed by a multi-stakeholder workshop of twenty-one participants representing HTA agencies, clinical and patient experts, academia, industry, and a payer, from across Europe and North America. The workshop was supported by extensive background work to identify the key challenges, including: targeted reviews of existing COVID-related methods guidance for assessing interventions and clinical guidelines, engagement with clinical experts, a survey and workshop of HTA agencies, a systematic review of published economic evaluations, and a workshop of health economic modelers.ResultsWe suggest HTA agencies should consider using other types of evidence (e.g., real world) where high-quality randomized controlled trials may be lacking and healthcare systems would value timely HTA outputs. A “living” HTA approach may be useful, given the context of an evolving disease, scientific understanding and evidence base, allowing for decisions to be efficiently revisited in response to new information; particularly, if supported by a common “disease model” for COVID-19. Innovative ways of engaging with the public and clinicians, and early engagement with regulators and payers, are recommended.ConclusionsHTA agencies should consider the elements of this guidance that are most suited to their existing processes to enable them to assess the effectiveness and value of interventions for COVID-19.
      PubDate: 2023-04-24
      DOI: 10.1017/S0266462323000223
       
  • Economic evaluation and costs of remote patient monitoring for
           cardiovascular disease in the United States: a systematic review

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      Authors: Zhang; Yunxi, Peña, Maria T., Fletcher, Lauren M., Lal, Lincy, Swint, J. Michael, Reneker, Jennifer C.
      First page: 25
      Abstract: BackgroundRemote patient monitoring (RPM) has emerged as a viable and valuable care delivery method to improve chronic disease management. In light of the high prevalence and substantial economic burden of cardiovascular disease (CVD), this systematic review examines the cost and cost-effectiveness of using RPM to manage CVD in the United States.MethodsWe systematically searched databases to identify potentially relevant research. Findings were synthesized for cost and cost-effectiveness by economic study type with consideration of study perspective, intervention, clinical outcome, and time horizon. The methodological quality was assessed using the Joanna Briggs Institute Checklist for Economic Evaluations.ResultsThirteen articles with fourteen studies published between 2011 and 2021 were included in the final review. Studies from the provider perspective with a narrow scope of cost components identified higher costs and similar effectiveness for the RPM group relative to the usual care group. However, studies from payer and healthcare sector perspectives indicate better clinical effectiveness of RPM relative to usual care, with two cost-utility analysis studies suggesting that RPM relative to usual care is a cost-effective tool for CVD management even at the conservative $50,000 per Quality-Adjusted Life-Year threshold. Additionally, all model-based studies revealed that RPM is cost-effective in the long run.ConclusionsFull economic evaluations identified RPM as a potentially cost-effective tool, particularly for long-term CVD management. In addition to the current literature, rigorous economic analysis with a broader perspective is needed in evaluating the value and economic sustainability of RPM.
      PubDate: 2023-04-28
      DOI: 10.1017/S0266462323000156
       
  • Expanding health technology assessment towards broader value: Ireland as a
           case study

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      Authors: Kinchin; Irina, Walshe, Valerie, Normand, Charles, Coast, Joanna, Elliott, Rachel, Kroll, Thilo, Kinghorn, Philip, Thompson, Alexander, Viney, Rosalie, Currow, David, O’Mahony, James F.
      First page: 26
      Abstract: Healthcare innovations often represent important improvements in population welfare, but at what cost, and to whom' Health technology assessment (HTA) is a multidisciplinary process to inform resource allocation. HTA is conventionally anchored on health maximization as the only relevant output of health services. If we accept the proposition that health technologies can generate value outside the healthcare system, resource allocation decisions could be suboptimal from a societal perspective. Incorporating “broader value” in HTA as derived from social values and patient experience could provide a richer evaluative space for informing resource allocation decisions. This article considers how HTA is practiced and what its current context implies for adopting “broader value” to evaluating health technologies. Methodological challenges are highlighted, as is a future research agenda. Ireland serves as an example of a healthcare system that both has an explicit role for HTA and is evolving under a current program of reform to offer universal, single-tier access to public services. There are various ways in which HTA processes could move beyond health, including considering the processes of care delivery and/or expanding the evaluative space to some broader concept of well-being. Methods to facilitate the latter exist, but their adaptation to HTA is still emerging. We recommend a multi-stakeholder working group to develop and advance an international agenda for HTA that captures welfare/benefit beyond health.
      PubDate: 2023-05-02
      DOI: 10.1017/S0266462323000235
       
  • A HTA of what' Reframing through including patient perspectives in health
           technology assessment processes

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      Authors: Gunn; Callum J., Regeer, Barbara J., Zuiderent-Jerak, Teun
      First page: 27
      Abstract: ObjectivesDespite increasing emphasis on the inclusion of patient input in health technology assessment (HTA) in Europe in particular, questions remain as to the integration of patient insight alongside other HTA inputs. This paper aims to explore how HTA processes, while ensuring the scientific quality of assessments, “make do” with patient knowledge elicited through patients’ involvement mechanisms.MethodsThe qualitative study analyzed institutional HTA and patient involvement in four European country contexts. We combined documentary analysis with interviews with HTA professionals, patient organizations, and health technology industry representatives, complemented with observational findings made during a research stay at an HTA agency.ResultsWe present three vignettes which showcase how different parameters of assessment become reframed upon the positioning of patient knowledge alongside other forms of evidence and expertise. Each vignette explores patients’ involvement during an assessment of a different type of technology and at a different stage of the HTA process. First, cost-effectiveness considerations were reframed during an appraisal of a rare disease medicine based on patient and clinician input regarding its treatment pathway; in the second vignette reframing amounted to what counts as a meaningful outcome measure for a glucose monitoring device; in the third, evaluating pediatric transplantation services involved reframing an option’s appropriateness from a question of moral to one of legal acceptability.ConclusionsMaking do with patient knowledge in HTA involves reframing of what is being assessed. Conceptualizing patients’ involvement in this way helps us to consider the inclusion of patient knowledge not as complementary to, but as something that can transform the assessment process.
      PubDate: 2023-05-18
      DOI: 10.1017/S0266462323000132
       
  • Is the quality of evidence in health technology assessment deteriorating
           over time' A case study on cancer drugs in Australia

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      Authors: Gao; Yuan, Laka, Mah, Merlin, Tracy
      First page: 28
      Abstract: ObjectiveThis study aimed to assess whether there have been changes in the quality of clinical evidence submitted for government subsidy decisions on cancer medicines over the past 15 years.MethodsWe reviewed public summary documents (PSDs) reporting on subsidy decisions made by the Pharmaceutical Benefits Advisory Committee (PBAC) from July 2005 to July 2020. Information was extracted on the study design, directness of comparison, sample size, and risk of bias (RoB). Changes in the quality of evidence were assessed using regression analysis.ResultsOverall, 214 PSDs were included in the analysis. Thirty-seven percent lacked direct comparative evidence. Thirteen percent presented observational or single-arm studies as the basis for decisions. Among PSDs presenting indirect comparisons, 78 percent reported transitivity issues. Nearly half (41 percent) of PSDs reporting on medicines supported by head-to-head studies noted there was a moderate/high/unclear RoB. PSDs reporting concerns with RoB increased by a third over the past 7 years, even after adjusting for disease rarity and trial data maturity (OR 1.30, 95% CI: 0.99, 1.70). No time trends were observed regarding the directness of clinical evidence, study design, transitivity issues, or sample size during any of the analyzed periods.ConclusionOur findings indicate that the clinical evidence supplied to inform funding decisions for cancer medicines is often of poor quality and has been deteriorating over time. This is concerning as it introduces greater uncertainty in decision making. This is particularly important as the evidence supplied to the PBAC is often the same as that supplied to other global decision-making bodies.
      PubDate: 2023-05-18
      DOI: 10.1017/S0266462323000259
       
  • Stakeholder perspectives on cooperation in the clinical and nonclinical
           health technology assessment domains

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      Authors: Zimmermann; Gina, Michelmore, Sandra, Hiligsmann, Mickaël
      First page: 29
      Abstract: ObjectivesThe aim of this study was to deliver insights from multiple stakeholders into actual and future collaboration for health technology assessment (HTA) in general and in oncology in particular.MethodsEighteen semi-structured interviews were conducted with experts from European HTA bodies (HTAbs), former board members of the European Network for Health Technology Assessment (EUnetHTA), and representatives from the pharmaceutical industry, a regulatory agency, academia, and patient organizations. The stakeholders were asked about their support of the EUnetHTA’s intent, about the general strengths and challenges of the EUnetHTA and its Joint Action 3 (JA 3), the strengths and challenges of the clinically oriented HTA collaboration in oncology during JA 3 across the technology life cycle, about future challenges to HTA in oncology with consequences for collaboration, and about collaboration in the economic domains of HTA. The transcribed interviews were analyzed qualitatively.ResultsThe participants perceived the intention and work quality of the EUnetHTA as positive. The experts described methodological, procedural, and capacity challenges in early dialogues (EDs) and rapid relative effectiveness assessments (REAs) meant to analyze clinical effectiveness in oncology. The majority attached increasing importance to collaboration in the future to cope with the uncertainty of HTA. Several stakeholders also proposed the incorporation of joint postlaunch evidence generation (PLEG) activities. Some gave sporadic suggestions for voluntary nonclinical collaboration as well.ConclusionStakeholders’ continued readiness to discuss the remaining challenges to and sufficient resources for implementing HTA regulation, as well as further cooperative expansion along the technology life cycle, are necessary for improved HTA collaboration in Europe.
      PubDate: 2023-05-22
      DOI: 10.1017/S0266462323000077
       
  • Demonstrating proof of concept for value-based agreements in Europe: two
           real-world cases

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      Authors: Griffiths; Elizabeth A., Odelade, Onivefu E., Gostkorzewicz, Joana, Cordero, Luis
      First page: 30
      Abstract: ObjectivesValue-based agreements (VBAs) link access, reimbursement, or price to the real-world usage and impact of a medicine, thereby enabling patient access while reducing clinical or financial uncertainty for the payer. VBAs have the potential to support improved patient outcomes, given the value-oriented approach to care, and lead to overall savings, while enabling payers to share risk and reduce uncertainty.MethodsThis commentary outlines the key challenges, enablers, and a framework for successful implementation by comparing the experience of two VBAs for AstraZeneca medicines, aiming to increase confidence in their future use.ResultsEngagement by payers, manufacturers, physicians, and provider institutions, and robust data collection systems that are accessible, simple to use, and add little burden to physicians were key to successfully negotiating a VBA that worked for all stakeholders. In both country systems, a legal/policy framework enabled innovative contracting.ConclusionsThese examples demonstrate proof of concept for VBA implementation in different settings, and may inform future VBAs.
      PubDate: 2023-05-22
      DOI: 10.1017/S0266462323000260
       
  • A review of HTA guidelines on societal and novel value elements

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      Authors: Breslau; Rachel Milstein, Cohen, Joshua T., Diaz, Jose, Malcolm, Bill, Neumann, Peter J.
      First page: 31
      Abstract: ObjectivesHealth technology assessment (HTA) organizations vary in terms of how they conduct assessments. We assess whether and to what extent HTA bodies have adopted societal and novel elements of value in their economic evaluations.MethodsAfter categorizing “societal” and “novel” elements of value, we reviewed fifty-three HTA guidelines. We collected data on whether each guideline mentioned each societal or novel element of value, and if so, whether the guideline recommended the element’s inclusion in the base case, sensitivity analysis, or qualitative discussion in the HTA.ResultsThe HTA guidelines mention on average 5.9 of the twenty-one societal and novel value elements we identified (range 0–16), including 2.3 of the ten societal elements and 3.3 of the eleven novel value elements. Only four value elements (productivity, family spillover, equity, and transportation) appear in over half of the HTA guidelines, whereas thirteen value elements are mentioned in fewer than one-sixth of the guidelines, and two elements receive no mention. Most guidelines do not recommend value element inclusion in the base case, sensitivity analysis, or qualitative discussion in the HTA.ConclusionsIdeally, more HTA organizations will adopt guidelines for measuring societal and novel value elements, including analytic considerations. Importantly, simply recommending in guidelines that HTA bodies consider novel elements may not lead to their incorporation into assessments or ultimate decision making.
      PubDate: 2023-05-25
      DOI: 10.1017/S026646232300017X
       
  • Cost-effectiveness of neuromuscular electrical stimulation for the
           treatment of mild obstructive sleep apnea: an exploratory analysis

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      Authors: Liu; Shan, Cao, Khoa N., Garner, Abigail M., Punjabi, Naresh M., Pietzsch, Jan B.
      First page: 32
      Abstract: ObjectivesTo assess the potential cost-effectiveness of neuromuscular electrical stimulation (NMES) for treatment of mild obstructive sleep apnea (OSA).MethodsA decision-analytic Markov model was developed to estimate health state progression, incremental cost, and quality-adjusted life year (QALY) gain of NMES compared to no treatment, continuous airway pressure (CPAP), or oral appliance (OA) treatment. The base case assumed no cardiovascular (CV) benefit for any of the interventions, while potential CV benefit was considered in scenario analyses. Therapy effectiveness was based on a recent multi-center trial for NMES, and on the TOMADO and MERGE studies for OA and CPAP. Costs, considered from a United States payer perspective, were projected over lifetime for a 48-year-old cohort, 68% of whom were male. An incremental cost-effectiveness ratio (ICER) threshold of USD150,000 per QALY gained was applied.ResultsFrom a baseline AHI of 10.2 events/hour, NMES, OA and CPAP reduced the AHI to 6.9, 7.0 and 1.4 events/hour respectively. Long-term therapy adherence was estimated at 65-75% for NMES and 55% for both OA and CPAP. Compared to no treatment, NMES added between 0.268 and 0.536 QALYs and between USD7,481 and USD17,445 in cost, resulting in ICERs between USD15,436 and USD57,844 per QALY gained. Depending on long-term adherence assumptions, either NMES or CPAP were found to be the preferred treatment option, with NMES becoming more attractive with younger age and assuming CPAP was not used for the full night in all patients.ConclusionsNMES might be a cost-effective treatment option for patients with mild OSA.
      PubDate: 2023-06-06
      DOI: 10.1017/S0266462323000272
       
  • Expert opinion on a consensus-based checklist for the critical appraisal
           of cost-of-illness (COI) studies: qualitative interviews

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      Authors: Schnitzler; Lena, Paulus, Aggie T.G., Evers, Silvia M.A.A., Roberts, Tracy E., Jackson, Louise J.
      First page: 33
      Abstract: ObjectivesThis study explored experts’ views on the development of a proposed checklist for cost-of-illness (COI) studies. It also investigated experts’ perspectives on the use of COI studies and quality/critical appraisal tools used for COI studies as well as their experiences with the use of these tools.MethodsSemi-structured, open-ended interviews were conducted with health economists and other experts working with COI studies and with experience of developing health economic guidelines or checklists. Participants were selected purposively using network and snowball sampling. A framework approach was applied for the thematic data analysis. Findings were reported narratively.ResultsTwenty-one experts from eleven different countries were interviewed. COI studies were found to be relevant to estimate the overall burden of a disease, to draw attention to disease areas, to understand different cost components, to explain cost variability, to inform decision making, and to provide input for full economic evaluations. Experts reported a lack of a standardized critical appraisal tool for COI studies. Their experience related predominantly to guidelines and checklists designed for full economic evaluations to review and assess COI studies. The following themes emerged when discussing the checklist: (i) the need for a critical appraisal tool, (ii) format and practicality, (iii) assessing the questions, (iv) addressing subjectivity, and (v) guidance requirements.ConclusionsThe interviews provided relevant input for the development of a checklist for COI studies that could be used as a minimum standard and for international application. The interviews confirmed the important need for a checklist for the critical appraisal of COI studies.
      PubDate: 2023-06-09
      DOI: 10.1017/S0266462323000181
       
  • A consensus-based checklist for the critical appraisal of cost-of-illness
           (COI) studies

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      Authors: Schnitzler; Lena, Roberts, Tracy E., Jackson, Louise J., Paulus, Aggie T. G., Evers, Silvia M. A. A.
      First page: 34
      Abstract: ObjectivesTo develop a consensus-based checklist that can be used as a minimum standard to appraise the comprehensiveness, transparency and consistency of cost-of-illness (COI) studies. This is important when, for instance, reviewing and assessing COI studies as part of a systematic review or when building an economic model.MethodsThe development process of the consensus-based checklist involved six steps: (i) a scoping review, (ii) an assessment and comparison of the different checklists and their questions, (iii) the development of a (preliminary) checklist, (iv) expert interviews, (v) the finalization of the checklist, and (vi) the development of guidance statements explaining each question.ResultsThe result was a consensus-based checklist for the critical appraisal of COI studies, comprising seventeen main questions (and some additional subquestions) across three domains: (i) study characteristics; (ii) methodology and cost analysis; and (iii) results and reporting. Guidance statements were developed describing the purpose and meaning behind each question and listing examples of best practice. The following answer categories were suggested to be applied when answering the questions in the checklist: Yes, Partially, No, Not Applicable, or Unclear.ConclusionsThe consensus-based checklist for COI studies is a first step toward standardizing the critical appraisal of COI studies and is one that could be considered a minimum standard. The checklist can help to improve comprehensiveness, transparency and consistency in COI studies, to address heterogeneity, and to enable better comparability of methodological approaches across international studies.
      PubDate: 2023-06-16
      DOI: 10.1017/S0266462323000193
       
  • Clinical effectiveness of fluticasone furoate nasal spray for perennial
           allergic rhinitis in children: a comprehensive review

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      Authors: Rivera; Paola Andrea
      First page: 35
      Abstract: ObjectiveTo assess the clinical effectiveness of fluticasone furoate nasal spray (FFNS) versus placebo on nasal symptoms and safety in children with perennial allergic rhinitis (AR).MethodsA comprehensive review was conducted with data obtained from Medline and Embase databases up to April 2023. The population of interest was patients aged 2–12 years with perennial AR. The selection was limited to randomized controlled trials (RCTs), comparing FFNS with placebo. Outcomes of interest included the reflective total nasal symptoms scores (rTNSS) and safety. To assess the minimal clinically important difference for rTNSS, the Cohen’s guideline was used. If the pooled standardized mean difference (SMD) and the lower limit of the 95 percent confidence interval (CI) exceeded the threshold of −0.20, effects were considered clinically significant.ResultsThree RCTs (959 pediatric patients) were selected. One study evaluated the short-term use of FFNS, another evaluated the long-term use of FFNS, and another evaluated both the short-term and long-term use of FFNS. FFNS produced a statistically significant reduction over placebo in rTNSS (SMD −0.18; 95 percent CI −0.35 to −0.01, p = 0.03) in long-term treatment studies, but not in short-term treatment studies. However, since the mean reduction did not reach the minimum clinically important difference (SMD −0.20), these results were considered clinically not relevant. Safety outcomes with FFNS were similar to placebo.ConclusionsThe currently available evidence suggests that FFNS, 110 μg once daily, compared to placebo, does not produce a meaningful clinical effect on nasal symptom in children with perennial AR.
      PubDate: 2023-06-16
      DOI: 10.1017/S026646232300034X
       
  • Emerging healthcare interventions: Patient-Centered Outcomes Research
           Institute’s programmatic initiative

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      Authors: Raman; Gowri, Vijaysimha, Meghana, Kopleff, Emma, Dunham, Kelly, Martin, Greg, Slutsky, Jean, Lawrence, William
      First page: 36
      Abstract: The Patient-Centered Outcomes Research Institute (PCORI) is a nonprofit, nongovernmental organization established by the U.S. Congress to fund comparative clinical effectiveness research focusing on patient-centered outcomes through the engagement of stakeholders. Evaluation of emerging healthcare innovations is one of PCORI’s five National Priorities for Health. One such initiative is PCORI’s Emerging Technologies and Therapeutics Reports program, established to provide timely overviews of evidence on new drugs and other healthcare technologies. This article provides an overview of completed and ongoing Emerging Technologies and Therapeutics Reports including lessons learned to date. In addition to systematic searches, systematic selection of studies, and transparent reporting of the available evidence, informed by a select number of stakeholders (i.e., key informants), these reports focus on contextual factors shaping the diffusion of emerging technologies that are often not reported in the medical literature. This article also compares processes and methodologies of health technology assessments (HTAs) from a selected number of national and international publicly funded agencies with a goal toward potential future enhancement of PCORI’s Emerging Technologies and Therapeutics Reports program. HTAs vary considerably in terms of funding, types of assessments, the role of manufacturers, stakeholder engagement, timeline to complete from the start to the finish of a draft report publication, and communication of uncertainty for informed decision making. Future Emerging Technologies and Therapeutics Reports may focus on rapid reports to support a more expedient development of evidence. Future research could explore the role of contextual factors identified in these reports on targeted evidence generation.
      PubDate: 2023-06-20
      DOI: 10.1017/S0266462323000284
       
  • Healthcare priority-setting criteria and social values in Iran: an
           investigation of local evidence

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      Authors: Goudarzi; Zahra, Bijlmakers, Leon, Nouhi, Mojtaba, Jahangiri, Reza, Heydari, Majid, Simangolwa, Warren, Hakimzadeh, Seyyed Mostafa, Jara, Karen Trujillo
      First page: 37
      Abstract: IntroductionIntegrating social values into health technology assessment processes is an important component of proper healthcare priority setting. This study aims to identify social values related to healthcare priority setting in Iran.MethodA scoping review was conducted on original studies that investigating social values in the healthcare system in Iran. The databases of PubMed, EMBASE, and EBSCO were searched with no restrictions on time and language. The reported criteria were clustered using Sham’s framework of social value analysis in health policy.ResultsTwenty-one studies published between 2008 and 2022 met the inclusion criteria. Fourteen of the included studies followed a quantitative approach with different methods to identify criteria, and the remaining seven studies used a qualitative approach. A total of fifty-five criteria were extracted and clustered into necessity, quality, sustainability, and process categories. Only six studies found criteria that were related to processes. Only three studies used public opinions as a source of value identification and eleven studies investigated the weight of criteria. None of the included studies explored the interdependency of the criteria.ConclusionEvidence suggests that several criteria other than cost per health unit also need to be considered in healthcare priority setting. Previous studies have paid little attention to the social values that underlie priority setting and policy-making processes. To reach consensus on social values related to healthcare priority setting, future researches need to involve broader stakeholders’ perspectives as a valuable source of social values in a fair process.
      PubDate: 2023-06-19
      DOI: 10.1017/S0266462323000302
       
  • A systematic review of economic evaluations for RPE65-mediated inherited
           retinal disease including HTA assessment of broader value

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      Authors: Farris; Maria, Goodall, Stephen, De Abreu Lourenco, Richard
      First page: 38
      Abstract: ObjectiveTo summarize the key methodological challenges identified by health technology assessment (HTA) agencies assessing gene therapy (GT) and consideration of broad elements of value.MethodEconomic evaluations (EEs) of voretigene neparvovec (VN) in RPE65-mediated inherited retinal disease (IRD) published in English were selected. HTA evaluations from Australia, Canada, Ireland, Scotland, England, and the United States were reviewed. An existing methodological framework was used to identify the challenges and considerations.ResultsEight unique EEs were identified of which six were evaluated by HTA agencies. Incremental cost-effectiveness ratios ranged from $68,951 to $643,813 per quality-adjusted life-years (QALY) gained (healthcare perspective) and dominant to $480,130 per QALY gained (societal perspective). The key challenges were the lack of validated surrogate outcome, utility values and indirect costs from IRD patients, and limited evidence of the long-term treatment effect. Two HTA agencies reviewed a range of novel broader elements of value and whether they were associated with VN while other agencies discussed some elements of broader value. Caregiver disutility was included in some, but not all, evaluations.ConclusionThe methodological challenges were consistent with innovative interventions for rare diseases and managed using standard methods. Broader value was important to decision-makers but inconsistently applied across agencies. Possible reasons are limitations in the evidence available of the broader benefits that VN offers and how to incorporate these within an EE. A need exists for greater guidance and consistency across jurisdictions regarding the consideration of broader value that considers latest best practice.
      PubDate: 2023-06-14
      DOI: 10.1017/S0266462323000326
       
  • Best practice considerations on the assessment of robotic assisted
           surgical systems: results from an international consensus expert panel

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      Authors: Erskine; Jamie, Abrishami, Payam, Charter, Richard, Cicchetti, Americo, Culbertson, Richard, Faria, Eliney, Hiatt, Jo Carol, Khan, Jim, Maddern, Guy, Patel, Anita, Rha, Koon Ho, Shah, Paresh, Sooriakumaran, Prasanna, Tackett, Scott, Turchetti, Giuseppe, Chalkidou, Anastasia
      First page: 39
      Abstract: BackgroundHealth technology assessments (HTAs) of robotic assisted surgery (RAS) face several challenges in assessing the value of robotic surgical platforms. As a result of using different assessment methods, previous HTAs have reached different conclusions when evaluating RAS. While the number of available systems and surgical procedures is rapidly growing, existing frameworks for assessing MedTech provide a starting point, but specific considerations are needed for HTAs of RAS to ensure consistent results. This work aimed to discuss different approaches and produce guidance on evaluating RAS.MethodsA consensus conference research methodology was adopted. A panel of 14 experts was assembled with international experience and representing relevant stakeholders: clinicians, health economists, HTA practitioners, policy makers, and industry. A review of previous HTAs was performed and seven key themes were extracted from the literature for consideration. Over five meetings, the panel discussed the key themes and formulated consensus statements.ResultsA total of ninety-eight previous HTAs were identified from twenty-five total countries. The seven key themes were evidence inclusion and exclusion, patient- and clinician-reported outcomes, the learning curve, allocation of costs, appropriate time horizons, economic analysis methods, and robotic ecosystem/wider benefits.ConclusionsRobotic surgical platforms are tools, not therapies. Their value varies according to context and should be considered across therapeutic areas and stakeholders. The principles set out in this paper should help HTA bodies at all levels to evaluate RAS. This work may serve as a case study for rapidly developing areas in MedTech that require particular consideration for HTAs.
      PubDate: 2023-06-05
      DOI: 10.1017/S0266462323000314
       
  • Uncertainty management in regulatory and health technology assessment
           decision-making on drugs: guidance of the HTAi-DIA Working Group

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      Authors: Hogervorst; Milou Amber, Vreman, Rick, Heikkinen, Inkatuuli, Bagchi, Indranil, Gutierrez-Ibarluzea, Inaki, Ryll, Bettina, Eichler, Hans-Georg, Petelos, Elena, Tunis, Sean, Sapede, Claudine, Goettsch, Wim, Janssens, Rosanne, Huys, Isabelle, Barbier, Liese, DeJean, Deirdre, Strammiello, Valentina, Lingri, Dimitra, Goodall, Melinda, Papadaki, Magdalini, Toussi, Massoud, Voulgaraki, Despina, Mitan, Ania, Oortwijn, Wija
      First page: 40
      Abstract: ObjectivesUncertainty is a fundamental component of decision making regarding access to and pricing and reimbursement of drugs. The context-specific interpretation and mitigation of uncertainty remain major challenges for decision makers. Following the 2021 HTAi Global Policy Forum, a cross-sectoral, interdisciplinary HTAi-DIA Working Group (WG) was initiated to develop guidance to support stakeholder deliberation on the systematic identification and mitigation of uncertainties in the regulatory-HTA interface.MethodsSix online discussions among WG members (Dec 2021–Sep 2022) who examined the output of a scoping review, two literature-based case studies and a survey; application of the initial guidance to a real-world case study; and two international conference panel discussions.ResultsThe WG identified key concepts, clustered into twelve building blocks that were collectively perceived to define uncertainty: “unavailable,” “inaccurate,” “conflicting,” “not understandable,” “random variation,” “information,” “prediction,” “impact,” “risk,” “relevance,” “context,” and “judgment.” These were converted into a checklist to explain and define whether any issue constitutes a decision-relevant uncertainty. A taxonomy of domains in which uncertainty may exist within the regulatory-HTA interface was developed to facilitate categorization. The real-world case study was used to demonstrate how the guidance may facilitate deliberation between stakeholders and where additional guidance development may be needed.ConclusionsThe systematic approach taken for the identification of uncertainties in this guidance has the potential to facilitate understanding of uncertainty and its management across different stakeholders involved in drug development and evaluation. This can improve consistency and transparency throughout decision processes. To further support uncertainty management, linkage to suitable mitigation strategies is necessary.
      PubDate: 2023-06-16
      DOI: 10.1017/S0266462323000375
       
  • Decision making for early surgical technology adoption into Canada’s
           healthcare system: a scoping review of the decision-making criteria,
           challenges, and opportunities

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      Authors: Shoman; Haitham, Tanzer, Michael
      First page: 41
      Abstract: ObjectivesIn 2020, Canada spent 12.9 percent of its GDP on healthcare, of which 3 percent was on medical devices. Early adoption of innovative surgical devices is mostly driven by physicians and delaying adoption can deprive patients of important medical treatments. This study aimed to identify the criteria in Canada used to decide on the adoption of a surgical device and identify challenges and opportunities.MethodsThis scoping review was guided by the Joanna Briggs Institute Manual for Evidence Synthesis and PRISMA-ScR reporting guidelines. The search strategy included Canada’s provinces, different surgical fields, and adoption. Embase, Medline, and provincial databases were searched. Grey literature was also searched. Data were analyzed by reporting the criteria that were used for technology adoption. Finally, a thematic analysis by subthematic categorization was conducted to arrange the criteria found.ResultsOverall, 155 studies were found. Seven were hospital-specific studies and 148 studies were from four provinces with publicly available Web sites for technology assessment committees (Alberta, British Columbia, Ontario, and Quebec). Seven main themes of criteria were identified: economic, hospital-specific, technology-specific, patients/public, clinical outcomes, policies and procedures, and physician specific. However, standardization and specific weighted criteria for decision making in the early adoption stage of novel technologies are lacking in Canada.ConclusionsSpecific criteria for decision making in the early adoption stage of novel surgical technologies are lacking. These criteria need to be identified, standardized, and applied in order to provide innovative, and the most effective healthcare to Canadians.
      PubDate: 2023-06-19
      DOI: 10.1017/S0266462323000363
       
  • Appropriateness of strategy comparisons in cost-effectiveness analyses of
           infant pneumococcal vaccination: a systematic review

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      Authors: Scheffer; Mariska M. J., Coffeng, Luc E., O’Mahony, James F.
      First page: 42
      Abstract: ObjectivesCost-effectiveness analysis (CEA) is the standard framework for informing the efficient allocation of scarce healthcare resources. The importance of considering all relevant intervention strategies and appropriate incremental comparisons have both long been recognized in CEA. Failure to apply methods correctly can lead to suboptimal policies. Our objective is to assess if CEAs of infant pneumococcal vaccination apply appropriate methods with respect to the completeness of strategies assessed and incremental comparisons between them.MethodsWe conducted a systematic search of the PubMed, Scopus, Embase, and Web of Science databases and performed a comparative analysis of the retrieved pneumococcal vaccination CEAs. We checked the appropriateness of the incremental analyses by attempting to replicate the published incremental cost-effectiveness (CE) ratios from the reported costs and health effects.ResultsOur search returned twenty-nine eligible articles. Most studies failed to recognize one or more intervention strategies (n = 21). Incremental comparisons were questionable in four CEAs and insufficient reporting of cost and health effect estimates was identified in three studies. Overall, we only found four studies that made appropriate comparisons between all strategies. Lastly, study findings appear to be strongly associated with manufacturer sponsorship.ConclusionsWe found considerable scope for improvement regarding strategy comparison in the infant pneumococcal vaccination literature. To prevent overestimation of the CE of new vaccines, we urge greater adherence to existing guidelines recommending that all available strategies are evaluated to capture relevant comparators for CE evaluation. Closer adherence to existing guidelines will generate better evidence, leading to more effective vaccination policies.
      PubDate: 2023-07-12
      DOI: 10.1017/S0266462323000351
       
  • Ethics information retrieval in HTA: state of current practice

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      Authors: Horton; Jennifer, DeJean, Deirdre, Farrah, Kelly, Hodgson, Amanda, Kaunelis, David, Walter, Melissa
      First page: 43
      Abstract: ObjectivesThough there have been longstanding discussions on the value of ethics in health technology assessment (HTA), less awareness exists on ethics information retrieval methods. This study aimed to scope available evidence and determine current practices for ethics information retrieval in HTA.MethodsLiterature searches were conducted in Ovid MEDLINE, LISTA, Scopus, and Google Scholar. Once a list of relevant articles was determined, citation tracking was conducted via Scopus. HTA agency websites were searched for published guidance on ethics searching, and for reports which included ethical analyses. Methods sections of each report were analyzed to determine the databases, subject headings, and keywords used in search strategies. The team also reached out to information specialists for insight into current search practices.ResultsFindings from this study indicate that there is still little published guidance from HTA agencies, few HTAs that contain substantial ethical analysis, and even less information on the methodology for ethics information retrieval. The researchers identified twenty-five relevant HTAs. Ten of these reports did not utilize subject-specific databases outside health sciences. Eight reports published ethics searches, with significant overlap in subject headings and text words.ConclusionsThis scoping study of current practice in HTA ethics information retrieval highlights findings of previous studies—while ethics analysis plays a crucial role in HTA, methods for literature searching remain relatively unclear. These findings provide insight into the current state of ethics searching, and will inform continued work on filter development, database selection, and grey literature searching.
      PubDate: 2023-07-19
      DOI: 10.1017/S0266462323000247
       
  • The Beneluxa Initiative domain task force health technology assessment: a
           comparison of member countries’ past health technology assessments

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      Authors: Vreman; Rick A., van Hoof, Daan, Nachtnebel, Anna, Daems, Joël, van de Casteele, Marc, Fogarty, Emer, Adams, Roisin, Timmers, Lonneke
      First page: 44
      Abstract: ObjectiveThis study aimed to compare assessments between Beneluxa Initiative member countries’ assessments and identify alignments and divergences.MethodsA retrospective comparative analysis was performed that investigated (i) number and type of assessed indications (for Austria (AT), Belgium (BE), Ireland (IE), and the Netherlands (NL)); (ii) added benefit conclusions (for BE, IE, and NL); and (iii) the main arguments underlying differences in conclusions (for BE, IE, and NL). Data were retrieved directly from agency representatives and from public HTA reports. European Medicines Agency approved indications were included for drugs assessed between 2016 and 2020, excluding veterinary drugs, generics, and biosimilars.ResultsOnly 44 (10 percent) of the 444 included indications were assessed by all four member countries. Between any pair of two countries, the overlap was higher, from 63 (AT–NL) to 188 (BE–IE). Added benefit conclusions matched exactly in 62–74 percent of the indications, depending on the countries compared. In the remaining cases, most often a difference of one added benefit level was observed (e.g., higher vs. equal relative effect). Contradictory outcomes were very rare: only three cases were observed (lower vs. higher effect). When assessing the underlying arguments for seven cases with different outcomes, differences were attributable to slight differences in weighing of evidence and uncertainties rather than disagreement on aspects within the assessment itself.ConclusionsDespite high variability in European HTA procedures, collaboration on HTA between the Beneluxa Initiative member countries is very feasible and would likely not result in added benefit conclusions that would be very different from added benefit conclusions in national procedures.
      PubDate: 2023-06-15
      DOI: 10.1017/S0266462323000338
       
  • Early access schemes for innovative health technologies: the views of
           international stakeholders

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      Authors: Farmer; Caroline, O’Toole, Brian, Barnish, Maxwell S., Trigg, Laura A., Hayward, Samuel, Crathorne, Louise, Kasten, Zelie, Spoors, John, Melendez-Torres, G. J.
      First page: 45
      Abstract: ObjectivesEarly access schemes (EASs) are approaches used by payers to balance and facilitate earlier patient access to innovative health technologies while evidence generation is ongoing. Schemes require investment from payers and are associated with significant risk since not all technologies will be routinely reimbursed. The purpose of this study was to gain the perspectives of policy experts about the key challenges for EASs and potential solutions for their optimal design and implementation.MethodsTwo virtual workshops were convened including (i) UK-based policy experts (England, Wales, and Scotland) and (ii) representatives from multiple healthcare systems (England, France, Sweden, Canada, Poland, and Norway). Participants were encouraged to share their experiences with EASs in their healthcare system and highlight key challenges for policy makers. Discussions were transcribed and analyzed using framework analysis.ResultsParticipants agreed that EASs have value when targeted toward innovative technologies with the potential for significant clinical benefit in an area of high unmet need. Participants discussed potential solutions to the challenges faced by payers implementing EASs, including defining eligibility criteria, supporting evidence generation, and approaches to reimbursement.ConclusionsParticipants agreed that EASs are one possible solution for their healthcare systems and have the potential to deliver significant clinical value to patients. However, widespread adoption of EASs is limited due to concerns about the risks for patients and healthcare budgets, further solutions are needed to deliver EASs for targeted therapies.
      PubDate: 2023-07-06
      DOI: 10.1017/S0266462323000429
       
  • A first exploration of the economic consequences of an autonomous surgical
           robot for lateral skull base surgery: an early health technology
           assessment

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      Authors: Nabuurs; Cindy H., Kievit, Wietske, Haegens, Lex, Grutters, Janneke P.C., Kunst, Henricus P.M.
      First page: 46
      Abstract: ObjectivesLateral skull base procedures, such as translabyrinthine approach (TLA), are challenging. An autonomous surgical robot might be a solution to these challenges. Our aim is to explore in an early phase the economic consequences of an autonomous surgical robot compared with conventional TLA.MethodsAn early decision analytic model was constructed in order to perform a step-wise threshold analyses and a sensitivity analysis to analyze the impact of the several factors on the incremental costs.ResultsUsing surgical robot results in incremental costs – EUR 5,562 per procedure – compared to conventional TLA. These costs are most reduced by higher number of procedures, followed by lower price of the robot, saved operation time, and reduced risk of complication, respectively.ConclusionsThe incremental costs of using an autonomous surgical robot can be decreased by choosing applications with a high turnover rate, a long operation time, and a high complication rate.
      PubDate: 2023-07-31
      DOI: 10.1017/S0266462323000430
       
  • A systematic review of the accessibility, acceptability, safety,
           efficiency, clinical effectiveness, and cost-effectiveness of private
           cataract and orthopedic surgery clinics

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      Authors: Akpinar; Ilke, Kirwin, Erin, Tjosvold, Lisa, Chojecki, Dagmara, Round, Jeff
      First page: 47
      Abstract: ObjectivesMany publicly funded health systems use a mix of privately and publicly operated providers of care to deliver elective surgical services. The aim of this systematic review was to assess the role of privately operated but publicly funded provision of surgical services for adult patients who had cataract or orthopedic surgery within publicly funded health systems in high-income countries.MethodsElectronic databases (Ovid MEDLINE, OVID Embase, and EBSCO EconLit) were searched on 26 March 2021, and gray literature sources were searched on 6 April 2021. Two reviewers independently applied inclusion and exclusion criteria to identify studies, and extracted data. The outcomes evaluated include accessibility, acceptability, safety, clinical effectiveness, efficiency, and cost/cost-effectiveness.ResultsTwenty-nine primary studies met the inclusion criteria and were synthesized narratively. We found mixed results across each of our reported outcomes. Wait times were shorter for patients treated in private facilities. There was evidence that some private facilities cherry-pick or cream-skim by selecting less complex patients, which increases the postoperative length of stay and costs for public facilities, restricts access to private facilities for certain groups of patients, and increases inequality within the health system. Seven studies found improved safety outcomes in private facilities, noting that private patients had a lower preoperative risk of complications. Only two studies reported cost and cost-effectiveness outcomes. One costing study concluded that private facilities’ costs were lower than those of public facilities, and a cost–utility study showed that private contracting to reduce public waiting times for joint replacement was cost-effective.ConclusionsLimited evidence exists that private-sector contracts address existing healthcare delivery problems. Value for money also remains to be evaluated properly.
      PubDate: 2023-08-01
      DOI: 10.1017/S0266462323000120
       
  • Effectiveness and safety of caplacizumab in acquired thrombotic
           thrombocytopenic purpura: health technology assessment and classification
           according to the methodology established in Colombia

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      Authors: Soto-Mora; Jahir A., Gómez-Espitia, Lina M., Lasalvia, Pieralessandro, Castellanos Moreno, Camilo A., Casallas Vanegas, Carol A., Londoño Gutiérrez, Sergio A.
      First page: 48
      Abstract: ObjectivesAcquired thrombotic thrombocytopenic purpura (aTTP) is a rare hematological disease whose clinical management includes caplacizumab along with plasma exchange and immunosuppression, according to international guidelines. Caplacizumab has been available in Colombia since 2022. This study seeks to determine the therapeutic classification of caplacizumab according to the methodology of the Instituto de Evaluación Tecnológica en Salud.MethodsThe classification was carried out through a deliberative process following the modified Delphi technique, with a panel of experts, made up of four hemato-oncologists, a pharmaceutical chemist, and a patient. The results of effectiveness and safety obtained through a systematic review, therapeutic thresholds (clinical significance), and degree of acceptability (willingness to use the technology) were used for the classification.ResultsFourteen effectiveness and safety outcomes were submitted for the classification process. Caplacizumab showed clinical significance for some effectiveness outcomes, was not considered inferior in terms of safety, and displayed acceptability of use. Through consensus, the panel determined that caplacizumab plus the standard regimen is superior to the standard regimen in terms of treatment response and composite outcome, and no different for the other effectiveness and safety outcomes. Likewise, in overall terms, the panel determined that caplacizumab together with the standard regimen is superior to the standard regimen.ConclusionTreatment with caplacizumab together with the standard regimen was considered superior to the standard regimen for the treatment of patients with aTTP, as it showed clinically significant benefits in critical outcomes for decision making, and a safety profile no different to its comparator.
      PubDate: 2023-07-21
      DOI: 10.1017/S0266462323000442
       
  • Institutionalizing health technology assessment in Ethiopia: seizing the
           window of opportunity

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      Authors: Erku; Daniel, Walker, Damian, Caruso, Ana A., Wubishet, Befikadu, Assefa, Yibeltal, Abera, Samuel, Hailu, Alemayehu, Scuffham, Paul
      First page: 49
      Abstract: Ethiopia’s commitment to achieving universal health coverage (UHC) requires an efficient and equitable health priority-setting practice. The Ministry of Health aims to institutionalize health technology assessment (HTA) to support evidence-based decision making. This commentary highlights key considerations for successful formulation, adoption, and implementation of HTA policies and practices in Ethiopia, based on a review of international evidence and published normative principles and guidelines. Stakeholder engagement, transparent policymaking, sustainable financing, workforce education, and political economy analysis and power dynamics are critical factors that need to be considered when developing a national HTA roadmap and implementation strategy. To ensure ownership and sustainability of HTA, effective stakeholder engagement and transparency are crucial. Regulatory embedding and sustainable financing ensure legitimacy and continuity of HTA production, and workforce education and training are essential for conducting and interpreting HTA. Political economy analysis helps identify opportunities and constraints for effective HTA implementation. By addressing these considerations, Ethiopia can establish a well-designed HTA system to inform evidence-based and equitable resource allocation toward achieving UHC and improving health outcomes.
      PubDate: 2023-07-21
      DOI: 10.1017/S0266462323000454
       
  • Deliberative processes in health technology assessment of medicines: the
           case of Spain

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      Authors: Pinilla-Dominguez; Pilar, Pinilla-Dominguez, Jaime
      First page: 50
      Abstract: ObjectivesSpain incorporated in 2020 changes in its health technology assessment (HTA), pricing, and reimbursement system for medicines including publishing reports, development of networks of experts, or consultation with stakeholders. Despite these changes, it is unclear how deliberative frameworks are applied and the process has been criticized for not being sufficiently transparent. This study analyses the level of implementation of deliberative processes in HTA for medicines in Spain.MethodsWe review the grey literature and summarize the Spanish HTA, pricing, and reimbursement process of medicines. We apply the deliberative processes for HTA checklist, developed to assess the overall context of the deliberative process, and identify the stakeholders involved and type of involvement following the framework for evidence-informed deliberative processes, a framework for benefit package design that aims to optimize the legitimacy of decision making.ResultsIn the Spanish HTA, pricing, and reimbursement process deliberation takes place in order to exchange viewpoints and reach common ground, mainly during the prioritization, assessment, and appraisal steps. It is closed to the public, not clearly summarized in published documents and limited to the Ministry of Health, the regulatory agency, other Ministries, and experts with mostly clinical and/or pharmaceutical background. The views of stakeholders are only represented through consultation. Communication is the most commonly used form of stakeholder engagement.ConclusionsDespite improvements in transparency of the Spanish HTA process for evaluating medicines, aspects related to stakeholder involvement and implementation of deliberative frameworks need further attention in order to achieve further legitimacy of the process.
      PubDate: 2023-07-05
      DOI: 10.1017/S0266462323000387
       
  • Patient and citizen participation at the organizational level in health
           technology assessment: an exploratory study in five jurisdictions

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      Authors: Nabarette; Hervé, Chastenay, Marie-Hélène, Dupont, Jean-Claude K., Ganache, Isabelle, Single, Ann N.V.
      First page: 51
      Abstract: ObjectiveWhile patient participation in individual health technology assessments (HTAs) has been frequently described in the literature, patient and citizen participation at the organizational level is less described and may be less understood and practiced in HTA bodies. We aimed to better understand its use by describing current practice.MethodTo elicit descriptive case studies and insights we conducted semi-structured interviews and open-ended questionnaires with HTA body staff and patients and citizens participating at the organizational level in Belgium, France, Quebec, Scotland, and Wales.ResultsWe identified examples of organizational participation in managerial aspects: governance, defining patient involvement processes, evaluation processes and methods, and capacity building. Mechanisms included consultation, collaboration, and membership of standing (permanent) groups. These were sometimes combined. Participants were usually from umbrella patient organizations and patient associations, as well as individual patients and citizens.DiscussionAlthough the concept, participation at the organizational level, is not well-established, we observed a trend toward growth in each jurisdiction. Some goals were shared for this participation, but HTA bodies focused more on instrumental goals, especially improving participation in HTAs, while patients and citizens were more likely to offer democratic and developmental goals beyond improving participation processes.ConclusionOur findings provide rationales for organizational-level participation from the perspectives of HTA bodies and patients. The case studies provide insights into how to involve participants and who may be seen as legitimate participants. These findings may be useful to HTA bodies, the patient sector, and communities when devising an organizational-level participation framework.
      PubDate: 2023-08-08
      DOI: 10.1017/S0266462323000417
       
  • National health technology assessment in Turkiye after a decade: are key
           principles followed'

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      Authors: Avşar; Tuba Saygın, Yıldırım, Hasan Hüseyin
      First page: 52
      Abstract: BackgroundHealth technology assessment (HTA) is growing in low- and middle-income countries (LMICs) to ensure optimal use of limited resources. However, the impact of HTAs on decision making in LMICs has been limited. The study aimed to provide an overview of Turkiye’s progress since establishing the first HTA agency in 2012.MethodsThe web sites of three national HTA agencies in Turkiye were searched for HTA guidelines and national HTA reports. The HTA guidelines were assessed by two researchers independently against the key principles of HTA developed by Drummond et al., and the HTA reports against the national guidelines.ResultsThe study included one HTA guideline and eight national HTA reports. The guideline included very limited technical guidance. Compliance with the principles was poor to moderate, and significant methodological limitations were identified. The reports were inconsistent regarding the scope and the HTA assessment criteria. The link between HTA findings, HTA decision making, and health policies were not clear.DiscussionThe inconsistencies between the reports and the methodological limitations demonstrate the need for national HTA guidelines. Improving the characteristics of the HTA might impact implementation. Among the key issues is transparency regarding priority setting, the HTA process, and decision making.ConclusionEstablishing and adopting national HTA guidelines at international standards is needed. Involving external scientific committees and health economists in the HTA processes might help ensure that the key principles of HTA are followed. The study findings might be helpful for countries that are developing their HTA systems.
      PubDate: 2023-07-24
      DOI: 10.1017/S0266462323000466
       
  • Cost-effectiveness of adding ezetimibe and/or PCSK9 inhibitors to
           high-dose statins for secondary prevention of cardiovascular disease in
           Chinese adults

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      Authors: Xiang; Yuliang, Gan, Lei, Du, Heyue, Hao, Qiukui, Aertgeerts, Bert, Li, Sheyu, Hu, Ming
      First page: 53
      Abstract: ObjectivesThe latest international guideline recommended the add-on therapy of ezetimibe and PCSK9 inhibitors in selected people for the secondary prevention of cardiovascular diseases (CVDs). However, it remains unclear whether these regimens fit the Chinese healthcare system economically.MethodsBased on the Chinese context, this simulation study evaluated four therapeutic strategies including the high-dose statin-only group, ezetimibe plus statin group, PCSK9 inhibitors plus statin group, and PCSK9 inhibitors plus ezetimibe plus statin group. The team developed a Markov model to estimate the incremental cost-effectiveness ratio (ICER). With each 1-yr cycle, the simulation subjects could have nonfatal cardiovascular events (stroke and/or myocardial infarction) or death (vascular or nonvascular death event) with a follow-up duration of 20 yr. Cardiovascular risk reduction was gathered from a network meta-analysis, and cost and utility data were gathered from hospital databases and published research.ResultsFor Chinese adults receiving high-dose statins for secondary prevention of CVDs, the ICER was US$68,910 per quality-adjusted life year (QALY) for adding PCSK9 inhibitors, US$20,242 per QALY for adding ezetimibe, US$51,552 per QALY for adding both drugs. Given a threshold of US$37,655 (three times of Chinese GDP), the probability of cost-effectiveness is 2.9 percent for adding PCSK9 inhibitors, 53.1 percent for adding ezetimibe, and 16.8 percent for adding both drugs. To meet the cost-effectiveness, an acquisition price reduction of PCSK9 inhibitors of 33.6 percent is necessary.ConclusionIn Chinese adults receiving high-dose statins for the secondary prevention of CVDs, adding ezetimibe is cost-effective compared to adding PCSK9 inhibitors and adding both drugs.
      PubDate: 2023-08-31
      DOI: 10.1017/S0266462323000296
       
  • Do France, Germany, and Italy agree on the added therapeutic value of
           medicines'

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      Authors: Casilli; Giorgio, Lidonnici, Dario, Jommi, Claudio, De Nigris, Marika, Genazzani, Armando A.
      First page: 54
      Abstract: ObjectivesThe Health Technology Assessment (HTA) of medicines is performed separately at the country level with some differences, but Italy, France, and Germany have implemented price and reimbursement systems strongly focused on the Added Therapeutic Value (ATV). This study investigates the level of agreement on ATV assessments by Agenzia Italiana del Farmaco (AIFA), Haute Autorité de Santé (HAS), and Gemeinsamer Bundesausschuss (G-BA).MethodsA database was created collecting all information about drugs with innovativeness status requests in Italy from July 2017 to December 2022 and populated with the corresponding HAS and G-BA ATV assessments. The primary comparative analysis was conducted by grouping the ATV ratings into “higher added value” and “lower or no added value”, while a secondary analysis considered the Italian innovativeness status as a criterion to include the quality of evidence assessment. The concordance between ATV assessments was investigated through percentage agreement and unweighted Cohen k-value.Results189 medicines/indications were included. The greatest agreement was found when comparing G-BA versus HAS (82 percent; k = 0.61, substantial agreement). Lower levels of agreements were observed for AIFA versus HAS and AIFA versus G-BA (respectively 52 percent; k = 0.17 and 57 percent; k = 0.25). The secondary analysis led to a reconciliation to moderate agreement for AIFA versus HAS (72 percent; k = 0.45) and AIFA versus G-BA (74 percent; k = 0.47).ConclusionsA high degree of concordance between HTA organizations is reached when considering jointly ATV and quality of evidence, suggesting that the system is extensively mature to make a Joint Clinical Assessment, avoiding duplications and reducing access inequalities.
      PubDate: 2023-08-15
      DOI: 10.1017/S026646232300048X
       
  • An evaluation of managed access agreements in England based on stakeholder
           experience

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      Authors: Farmer; Caroline, Barnish, Maxwell S., Trigg, Laura A., Hayward, Samuel, Shaw, Naomi, Crathorne, Louise, Strong, Thomas, Groves, Brad, Spoors, John, Melendez Torres, G. J.
      First page: 55
      Abstract: ObjectivesThe objective of this research was to evaluate managed access policy in England, drawing upon the expertise of a range of stakeholders involved in its implementation.MethodsSeven focus groups were conducted with payer and health technology assessment representatives, clinicians, and representatives from industry and patient/carer organizations within England. Transcripts were analyzed using framework analysis to identify stakeholders’ views on the successes and challenges of managed access policy.ResultsStakeholders discussed the many aims of managed access within the National Health Service in England, and how competing aims had affected decision making. While stakeholders highlighted a number of priorities within eligibility criteria for managed access agreements (MAAs), stakeholders agreed that strict eligibility criteria would be challenging to implement due to the highly variable nature of innovative technologies and their indications. Participants highlighted challenges faced with implementing MAAs, including evidence generation, supporting patients during and after the end of MAAs, and agreeing and reinforcing contractual agreements with industry.ConclusionsManaged access is one strategy that can be used by payers to resolve uncertainty for innovative technologies that present challenges for reimbursement and can also deliver earlier access to promising technologies for patients. However, participants cautioned that managed access is not a “silver bullet,” and there is a need for greater clarity about the aims of managed access and how these should be prioritized in decision making. Discussions between key stakeholders involved in managed access identified challenges with implementing MAAs and these experiences should be used to inform future managed access policy.
      PubDate: 2023-07-27
      DOI: 10.1017/S0266462323000478
       
  • HTA capacity building in Asia: towards one goal

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      Authors: Mundy; Linda, Maddern, Guy
      First page: 56
      Abstract: ObjectivesThe aim of the 2022 Health Technology Assessment International (HTAi) Asia Policy Forum (APF) was to discuss experiences and challenges around health technology assessment (HTA) capacity building for both HTA agencies and companies in the Asia region and to identify possible solutions as part of a capacity building roadmap.MethodsDiscussions during the 2022 APF, informed by a pre-meeting survey of HTA agencies and industry attendees from the region, form the basis of this paper.ResultsHTA is an essential element of priority-setting in healthcare; however, the scarcity of skilled technical HTA practitioners is a rate-limiting step in the conduct of HTA. The lack of investment in HTA and the political will to mandate the use of HTA in decision-making may be due to a lack of understanding of the value of the HTA process, and how HTA is interpreted and used in the healthcare decision-making process.ConclusionsIncreased demand for HTA is created when the value of HTA is recognized. HTA capacity-building challenges may be mitigated by educating stakeholders, particularly policymakers, on the value of, and the need to invest in, HTA as a transparent process to ensure equitable access to health care for all. Investigating a means of funding and implementing an HTA intern program between agencies, in partnership with industry, to facilitate a supportive environment to foster HTA skills and knowledge, build capacity or strengthen existing capacity should be a priority.
      PubDate: 2023-08-15
      DOI: 10.1017/S0266462323000405
       
  • Emerging healthcare interventions: Patient-Centered Outcomes Research
           Institute’s programmatic initiative – CORRIGENDUM

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      Authors: Raman; Gowri, Vijaysimha, Meghana, Kopleff, Emma, Dunham, Kelly, Martin, Greg, Slutsky, Jean, Lawrence, William
      First page: 57
      PubDate: 2023-09-07
      DOI: 10.1017/S026646232300051X
       
  • Hospital-based health technology assessment of innovative medical devices:
           insights from a nationwide survey in France

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      Authors: Martin; Tess, Guercio, Alessandra, Besseau, Hélène, Huot, Laure, Guerre, Pascale, Atfeh, Jamal, Piazza, Laurent, Pineau, Judith, Sabatier, Brigitte, Borget, Isabelle, Martelli, Nicolas
      First page: 58
      Abstract: ObjectivesTo better understand the process of hospital acquisition of innovative medical devices (MDs) and the hospital-based health technology assessment (HB-HTA) pathways in France, an in-depth study based on a quantitative approach is needed. The aim of the present study was to assess through a national survey how HB-HTA is currently implemented in French hospitals and to identify its level of formalization.MethodsA quantitative online survey was conducted among hospitals performing HB-HTA in France, with a focus on the acquisition of innovative MDs for individual use. The survey, conducted between March and June 2022, was developed by a scientific board composed of members of the French-speaking Society for HB-HTA.ResultsSixty-seven out of 131 surveyed hospitals with HB-HTA activities responded, including 29 university hospitals, 24 nonprofit private hospitals, and 14 local hospitals. Sixty-one respondents (91 percent) reported the existence of a process dedicated to evaluating innovative MDs; of these, 16 declared that their hospitals had a formalized unit with HB-HTA activity. These units were more frequently found in larger hospitals with more than 500 inpatient beds (n = 16, p = 0.0160) and in university hospitals (n = 12, p = 0.0158). No hospital reported any collaboration with HAS, the French national HTA agency.ConclusionA diverse range of HB-HTA organizations with different structural levels exist in France for MD procurement linked to the category of hospitals. The study highlights the need for recognition of HB-HTA activity at the regulatory level in France and for direct collaboration between HTA activities performed at local and national levels.
      PubDate: 2023-09-21
      DOI: 10.1017/S0266462323000521
       
  • Economic evaluation of remote patient monitoring and organizational
           analysis according to patient involvement: a scoping review

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      Authors: Le Goff-Pronost; Myriam, Bongiovanni-Delarozière, Isabelle
      First page: 59
      Abstract: BackgroundA literature review concerning the economic evaluation of telemonitoring was requested by the authority in charge of health evaluation in France, in a context of deployment of remote patient monitoring and identification of its financing. Due to the heterogeneity of existing telemonitoring solutions, it was necessary to stratify the evaluation according to patient involvement. Three levels of patient involvement are considered: weak (automated monitoring), medium (monitoring supported by a professional), and strong (active remote participation).ObjectivesWe performed a scoping review to provide a comprehensive overview of different systems of telemonitoring and their reported cost-effectiveness.MethodsFollowing PRISMA-ScR guidelines, a search was performed in four databases: PubMed, MEDLINE, EMBASE, and Cochrane Library between January 1, 2013 and May 19, 2020. Remote patient monitoring should include the combination of three elements: a connected device, an organizational solution for data analysis and alert management, and a system allowing personalized interactions, and three degrees of involvement.ResultsWe identified 61 eligible studies among the 489 records identified. Heart failure remains the pathology most represented in the studies selected (n = 24). The cost-utility analysis was chosen in a preponderant way (n = 41). Forty-four studies (72 percent) reported that the intervention was expected cost-effective. Heterogeneity has been observed in the remote monitoring solutions but all systems are reported cost-effective. The small number of long-term studies does not allow conclusions to be drawn on the transposability.ConclusionsRemote patient monitoring is reported to be cost-effective whatever the system and patient involvement.
      PubDate: 2023-09-26
      DOI: 10.1017/S0266462323002581
       
 
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