JournalTOCs

PharmacoEconomics
Journal Prestige (SJR): 1.998

Citation Impact (citeScore): 3
Number of Followers: 21

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ISSN (Print) 1170-7690 - ISSN (Online) 1179-2027
Published by Adis

[21 journals]

Correction: A Framework for Using Cost‑effectiveness Analysis to Support
Pricing and Reimbursement Decisions for New Pharmaceuticals in a Context
of Evolving Treatments, Prices, and Evidence
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  PubDate: 2025-07-04
The Psychometric Performance of Generic Preference-Based Measures in
Informal Carers: A Systematic Review of Validation Studies
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  Abstract: Background and Objective A growing number of health technology assessment agencies recommend inclusion of informal carer outcomes in health economic evaluations. While generic preference-based measures (GPBMs) are favoured, the evidence regarding their performance in measuring the health-related quality of life of informal carers has not been synthesised. The aim of this systematic review was to synthesise the psychometric evidence of GPBMs in informal carers. Methods Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, a literature search (indexed through October 2024) was conducted in CINAHL, PsycInfo, Embase and MEDLINE databases, supplemented with forward and backward citation searches. Publications were included that reported the psychometric performance of GPBMs in informal carers, regardless of care recipients’ condition. Narrative synthesis was used to summarise the evidence. Quality of studies was evaluated using the COSMIN risk of bias checklist. International Prospective Register of Systematic Reviews (PROSPERO) registration is CRD42023434651. Results Twenty-one studies (published between 2001 and 2024) were identified, with nine evaluating multiple GPBMs (head-to-head comparisons). The EQ-5D 3-level (EQ-5D-3L) [n = 9] and EQ-5D 5-level (EQ-5D-5L) [n = 7] were the most frequently evaluated, followed by the Short-form 6-Dimension version 1 (SF-6Dv1) [n = 4], EuroQol Health and Wellbeing Short Form (EQ-HWB-9) [n = 4], Health Utilities Index (HUI) marks 2/3 (n = 3), Health-related Quality of Life Instrument with 8 Items (HINT-8) [n = 1] and Quality of Well Being Self-Administered (QWB-SA) [n = 1]. Studies were conducted in the USA (n = 6), UK (n = 4), China (n = 4), Australia (n = 3), Italy (n = 1), Iran (n = 1) and South Korea (n = 1), including a multi-country study (UK, Germany and France) study (n = 1). Care recipient conditions included carers of unspecified conditions, adults using long-term care, Alzheimer’s disease or dementia, autism, cancer, leukaemia, craniofacial malformations, meningitis and multiple sclerosis. The EQ-5D-3L and EQ-5D-5L had evidence of ceiling effects at the index level. The EQ-5D-3L, EQ-5D-5L and EQ-HWB-9 demonstrated at least ‘good’ (intraclass correlation coefficient> 0.60) test–retest reliability. Known-group validity evidence was available for the EQ-5D-3L, EQ-5D-5L, EQ-HWB-9, HUI3 and SF-6Dv1 where each GPBM was able to discriminate over 60% of the groups (known or exploratory). Convergent validity studies reported that the EQ-5D-3L, EQ-5D-5L, EQ-HWB-9, HUI3, SF-6Dv1 and QWB-SA had moderate correlations with at least one care-specific preference-based measure (Adult Social Care Outcomes Toolkit for Carers [ASCOT-Carer], Care-Related Quality of Life [CarerQol] and Carer Experience Scale [CES]). Responsiveness was evaluated for the EQ-5D-5L, EQ-HWB-9 and SF-6Dv1 where mixed evidence was reported for the two EuroQol instruments, whereas the SF-6Dv1 was not found to be responsive. The studies identified were generally of adequate quality. Conclusions Current literature supports the use of GPBMs for informal carers; however, evidence on individual psychometric indicators is still limited. Further research is still needed, preferably involving head-to-head comparison and content validity studies in carers of people with various conditions and across countries that utilise cost-effectiveness evidence in resource allocation decisions, ideally employing longitudinal study designs.
  PubDate: 2025-06-28
Key Considerations for Assessing Real-World Comparative Effectiveness in
the Context of the Drug Price Negotiation Program: A Case Study of
Pembrolizumab
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  Abstract: Background and Objective The Centers for Medicare and Medicaid Services increasingly rely on real-world evidence to inform drug price negotiations under the Inflation Reduction Act. This study aims to evaluate methodological decisions that impact real-world comparative effectiveness outcomes using a case example of first-line pembrolizumab versus therapeutic alternatives in advanced non-small cell lung cancer among the Medicare population. Methods This study used a deidentified, electronic health record-derived, advanced non-small cell lung cancer dataset (2011–23) to analyze Medicare-eligible stage IV patients in three indications: (1) non-squamous, epidermal growth factor receptor, and anaplastic lymphoma kinase negative; (2) squamous; and (3) epidermal growth factor receptor and anaplastic lymphoma kinase negative with programmed death ligand-1 expression ≥1%. Indications (1)–(2) involved pembrolizumab combinations, while (3) referred to pembrolizumab monotherapy. Comparators included common non-platinum-based chemotherapy regimens. Propensity score-based inverse probability weighting was applied. The primary outcomes were real-world progression-free survival and overall survival. Scenario analyses examined the influence of time period selection, programmed death ligand-1 inclusion, therapeutic alternatives, and treatment switching on comparative effectiveness estimates. Results In the non-squamous cohort (1), overall survival benefits of pembrolizumab therapies compared to alternatives varied from a non-significant difference to an improvement of 2.7 months (95% confidence interval 1.2, 4.8), depending on analytical choices. In the squamous cohort (2), pembrolizumab combinations consistently demonstrated overall survival benefits, which ranged from 1.4 months (95% confidence interval 0.1, 3.0) to up to 3.6 months (95% confidence interval 0.1, 5.9). However, for pembrolizumab monotherapy (3), overall survival differences were statistically non-significant. Scenario analyses indicated substantial variability in outcomes based on methodological choices. Conclusions This study underscores the importance of transparent reporting and scenario analyses in real-world evidence to support Centers for Medicare & Medicaid Services decision making during drug price negotiations. Findings highlight the need for rigorous methodological standards to ensure the external validity of real-world evidence and its alignment with clinical practice.
  PubDate: 2025-06-23
Innovation Headroom for a Highly Accurate PD-L1 Companion Diagnostic in
Non-small Cell Lung Cancer
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  Abstract: Background and Objective Companion diagnostics (CDx) are critical to precision medicine. Developing and commercializing new CDx faces regulatory and economic challenges. This study aims to illustrate the utility of an early health technology assessment in quantifying the unmet clinical need and commercial opportunity created by the limited accuracy of existing programmed cell death ligand 1 CDx. Methods The study uses an early health technology assessment and market sizing to assess the potential value of a novel programmed cell death ligand 1 CDx for non-small cell lung cancer (NSCLC). Decision tree-based cost-effectiveness models were used to evaluate clinical and economic outcomes for improved programmed cell death ligand 1 testing in atezolizumab-treated patients with stage II–IIIA and metastatic NSCLC from a US payer perspective in 2023 US Dollars. Three strategies were examined: standard care, new CDx for cytology specimens only, and new CDx for all patients. Commercial opportunities from the perspectives of diagnostics and pharmaceutical manufacturers were assessed using headroom and threshold analyses. Results Headroom analyses indicated that a new CDx is not cost effective for metastatic NSCLC but holds significant value for stage II–IIIA NSCLC. Assuming perfect sensitivity and specificity, the incremental cost-effectiveness ratio for the new CDx in stage II–IIIA NSCLC was $57,650/quality-adjusted life-year and $54,950/quality-adjusted life-year for cytology specimens only and all patients, respectively. A threshold analysis showed that at a $500 price point, the new CDx is cost effective at sensitivity levels of 0.9 for all patients and 0.8 for cytology only. The total addressable US market for the CDx manufacturer was estimated at $2.6 million per year with a $500/test kit price. Conclusions A novel, highly accurate CDx for stage II–IIIA NSCLC could provide significant value to patients, payers, and manufacturers.
  PubDate: 2025-06-20
An Updated Systematic Literature Review of the Economic Costs of
Loneliness and Social Isolation and the Cost Effectiveness of
Interventions
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  Abstract: Purpose There has been growing interest in understanding the economic impacts of loneliness and social isolation. This study updates a previous review on the economic costs of loneliness and social isolation and the cost effectiveness of related interventions. Methods We conducted a systematic search in the MEDLINE, PsycInfo, CINAHL, and Embase databases from 2018 to 13 August 2024, supplemented by a search of the grey literature. Studies included cost-of-illness studies, economic evaluations, and social return on investment (SROI) analyses published in the English language. All studies were evaluated for quality and summarised using a narrative approach. Costs reported were converted into US$, year 2024 values. Results In total, 15 studies were included: six cost-of-illness studies, four economic evaluations, and five SROI studies. Cost-of-illness studies primarily examined healthcare and productivity costs. All but one study reported excess costs linked to loneliness and social isolation, ranging from US$2 billion to US$25.2 billion per annum. Among four economic evaluations, three were model-based cost-utility or cost-effectiveness analyses (targeting older adults and the general population), and one was trial based (focusing on low-income individuals with health issues). One study found an intervention cost effective, whereas cost-effectiveness probabilities in others ranged from 54% to 68%. One study concluded that an intervention to reduce severe loneliness in older adults was cost effective but unlikely to be cost saving. All SROI studies reported positive returns, with SROI ratios ranging from US$2.28 to US$13.72. Conclusion This review highlights additional evidence on the economic burden of loneliness and social isolation. Future research should explore broader cost impacts beyond healthcare and expand cost-effectiveness studies to younger populations.
  PubDate: 2025-06-16
Inequalities in Quality-Adjusted Life Expectancy in Australia by
Educational Attainment
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  Abstract: Introduction Summary measures such as quality-adjusted life expectancy (QALE) are increasingly used to monitor health inequalities. Socioeconomic inequalities in health are well documented in Australia, including inequalities by education. However, estimates for QALE by level of education are lacking for Australia. We aimed to provide QALE stratified by age and sex across levels of educational attainment for the Australian population aged 25 years and above. Methods We categorized educational attainment as low (completed year 11 or below), intermediate (completed year 12 and/or other non-tertiary or vocational qualification) or high (completed a bachelor’s degree or above). Mean Short-Form Six-Dimension health utility was estimated for sex- and education-specific subgroups from the Household, Income and Labour Dynamics in Australia survey (2022). We constructed life tables using age-sex-education-specific mortality rates for 2019 obtained from linked 2016 Census and Death Registrations data. Health utility was incorporated into the life tables to derive age- and sex-specific QALE across education levels. Results At age 25 years, males with high education had 7.3 years greater life expectancy than those with low education (61.0 versus 53.7 years undiscounted) and larger QALE (39.9 versus 28.8 years undiscounted), a gap of 11.1 years (39% relative difference). Females aged 25 years with a high level of education experienced 3.9 years greater life expectancy (LE; 63.1 versus 59.2 years, undiscounted) and an additional 7.6 years of QALE (36.9 versus 29.3 years, undiscounted), compared with those with low education, a 26% relative difference in QALE. Conclusions Significant disparities in QALE by educational attainment exist in Australia. These findings can inform policies aimed at reducing health inequity by guiding resource allocation and supporting future equity-informative economic evaluations.
  PubDate: 2025-06-16
Bayesian Meta-Analysis: A Practical Introduction
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  PubDate: 2025-06-09
Health Care Resource Utilization and Costs Associated with US Medicaid
Sobriety Restrictions on Direct-Acting Antivirals for Hepatitis C Virus: A
Retrospective Claims Database Analysis
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  Abstract: Background and Aims Many state Medicaid programs implemented sobriety restrictions that delay timely initiation of direct-acting antivirals (DAAs) for patients with hepatitis C virus (HCV) infections. This claims database study examined the economic impact of sobriety restrictions on DAAs among Medicaid-insured patients with HCV. Methods A retrospective database analysis of the Anlitiks All Payor Claims data (APCD) during the period January 1, 2020 to June 30, 2022 was conducted. Continuously enrolled adult (aged 18–64 years) Medicaid-insured patients with HCV who initiated DAAs (i.e., index date) during the period January 1, 2021 to December 31, 2021 with ≥ 12 months pre-index and ≥ 6 months post-index follow-up were categorized into two cohorts (states with sobriety restriction [SR] and states with no sobriety restriction [NSR]) based on the sobriety restriction status in the state of residence on the index date. Measures analyzed were the proportion of patients with one or more all-cause medical health care resource utilization (HCRU) (inpatient hospitalization [IP], emergency department [ED], outpatient [OP], professional office [PV], and other [OV] visits) and mean per-patient medical, pharmacy, and overall costs. HCRU and cost differences were compared using adjusted multivariable logistic and gamma-log link regression models, respectively. Results Patients in the SR (n = 2,295) versus NSR (n = 4,623) cohort had a higher mean age (45 ± 12.02 vs. 43 ± 11.51 years), fewer males (50.28% vs. 58.1%), and they had lower substance use rates (44.10% vs. 59.68%), all significant at p
  PubDate: 2025-05-28
Are Economic Evaluations of Task Shifting Too Narrow in Focus' A Rapid
Review
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  Abstract: Background and Objectives Task shifting between different cadres of health worker has been proposed as an approach to address workforce shortages. Whether such reallocation is a useful strategy for a health system depends on the potential costs and consequences. Too narrow a focus has implications for population health as resources could be incorrectly directed towards inefficient activities owing to important costs and/or benefits being omitted from the evaluation. We aim to identify the key issues when evaluating the value for money of task shifting and review the applied literature to determine whether it is fit for purpose. Methods We developed an a priori logic model of task shifting and searched five databases (MEDLINE, Embase, EconLit, Social Sciences Citation Index and CEA Registry) for economic evaluations of task shifting published between 2014 and 2024. We performed forwards and backwards citation searching. We considered the scope of the evaluations with respect to the ability to capture key costs and outcomes of task shifting from the logic model. Reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. Results The rapid review identified 26 studies for inclusion covering 16 countries. Studies evaluated task shifting to community health workers and lay health workers as well as from doctors to radiographers, non-physician clinicians and nurse-midwives. The studies included health costs and outcomes but few included changes in the capacity of the workforce to undertake tasks, access, waiting times, productivity, burden on other staff, patient satisfaction, patient productivity and health equity concerns. There was a predominance for cost-effectiveness analysis to be used to assess the value for money of task shifting but the literature did include a cost-benefit analysis, a cost-consequence analysis and an extended cost-effectiveness analysis. Conclusions The majority of studies identified a range of costs and consequences that may only be appropriate for resource allocation under the strong assumption that all longer term costs and consequences would be unaffected by the task shift.
  PubDate: 2025-05-23
Anchoring the Hemophilia Joint Health Score for Conventional Value
Assessment in the Care of People Living with Severe Hemophilia A
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  PubDate: 2025-05-23
Correction: Health-Related Quality-of-Life Outcomes of Very Preterm or
Very Low Birth Weight Adults: Evidence From an Individual Participant Data
Meta-Analysis
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  PubDate: 2025-05-23
Global Assessment of Health Utilities Associated with Pneumococcal Disease
in Children—Targeted Literature Reviews
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  Abstract: Background Pneumococcal disease can significantly impact the quality of life (QoL) of children. Health utilities are used to measure the disease burden and calculate quality-adjusted life year (QALY) estimates. These estimates provide critical inputs in economic evaluations of pneumococcal vaccines in children. Objectives This study aimed to synthesize utility values used in cost-utility analyses (CUAs) of pediatric pneumococcal vaccines and to summarize published utility studies on pneumococcal disease and post-meningitis sequelae (PMS) in children on a global scale. Methods Two targeted literature reviews were conducted to identify CUAs of pediatric pneumococcal vaccines and original studies on health utilities of pneumococcal disease and PMS. Both literature reviews identified relevant studies using published reviews, supplemented by de novo searches conducted in MEDLINE in June 2024 to cover periods not included in those reviews. References from published literature reviews on QoL of pneumococcal disease and CUAs were screened to identify additional original utility studies. Health utility values applied in the CUAs were summarized and the source studies for these utilities were reviewed. For original utility studies, methods and utility estimates were summarized for each condition. Results The study identified 45 CUAs of pediatric pneumococcal vaccines in North America and Europe published from 2004 to 2024, and 21 original utility studies on pneumococcal disease or PMS in children published globally from 1994 to 2017. QALY decrement was the most common utility input in CUAs. Most CUAs referenced an earlier CUA for utility inputs, which were often sourced from one or two original utility studies for each health state. Most source studies were published more than two decades ago; some common source studies were conducted in adults. Utility estimates from original studies showed considerable variability, with ranges of −0.330 to 0.6882 for meningitis, −0.331 to 0.93 for non-meningitis invasive pneumococcal disease (IPD), −0.054 to 0.71 for inpatient pneumonia, 0.412–0.82 for outpatient pneumonia, 0.389–0.97 for acute otitis media (AOM)/simple AOM, 0.434–0.540 for recurrent AOM, −0.33 to 0.89 for neurological deficits, and 0.217–0.97 for hearing loss. Variability in methods, including in the surveyed population, utility elicitation method, and use of different country-specific preference weights, substantially impacted utility values. Overall, the methods were not suitable for temporary health states. Additionally, many studies used instruments that have not been validated in children. Conclusions Original utility studies demonstrated that pneumococcal disease and PMS are associated with impaired QoL in children; however, there was considerable variability in utility estimates across studies, reflecting the inherent methodological challenges in estimating utilities for acute diseases in children. Most CUAs referenced previous CUAs for health utility values, which were sourced from a limited number of outdated original utility studies. Contemporary data and methods adapted for acute diseases in children are needed for future studies. Given the significance of health utilities in the economic valuations of new pneumococcal vaccines, utility values should be carefully selected in CUAs, considering alternative sources and assumptions.
  PubDate: 2025-05-23
Reply to Comment on “Examining Consistency Across NICE Single Technology
Appraisals: A Review of Appraisals for Paroxysmal Nocturnal
Haemoglobinuria”
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  PubDate: 2025-05-22
Comment on “Examining Consistency Across NICE Single Technology
Appraisals: A Review of Appraisals for Paroxysmal Nocturnal
Haemoglobinuria”
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  PubDate: 2025-05-22
The Evolving Landscape of Discrete Choice Experiments in Health Economics:
A Systematic Review
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  Abstract: Introduction Stakeholder preference evaluations are increasingly emphasized in healthcare policy and health technology assessment. Discrete choice experiments (DCEs) are the most common method for quantifying preferences among patients, the public, and healthcare professionals. While prior reviews (1990–2017) have examined DCE trends, no comprehensive synthesis exists for studies published since 2018. This updated review (2018–2023) provides critical insights into evolving methodologies and global trends in health-related DCEs. Methods A systematic search (2018–2023) of Medline, Embase, and Web of Science identified relevant studies. Studies were screened for inclusion and data were extracted, including details on DCE design and analysis. To enable trend comparisons, the search strategy and extraction items aligned with previous reviews. Results Of 2663 identified papers, 1279 met the inclusion criteria, reflecting a significant rise in published DCEs over time. DCEs were conducted globally, with a remarkable increase in publications from Asia and Africa compared with previous reviews. Experimental designs and econometric models have advanced, continuing prior trends. Notably, most recent DCEs were administered online. Discussion The rapid growth of DCE applications underscores their importance in health research. While the methodology is advancing rapidly, it is crucial that researchers provide full transparency in reporting their methods, particularly in detailing experimental designs and validity tests, which are too often overlooked. Key recommendations include improving reporting of experimental designs, applying validity tests, following good practices for presenting benefit–risk attributes, and adopting open science practices. Ensuring methodological rigor will maximize the impact and reproducibility of DCE research in health economics.
  PubDate: 2025-05-21
Does the Informal Carer Identification Method Matter' Evidence from
Self-Declaration and Time Diary Approaches
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  Abstract: Objectives Impacts on informal carers are increasingly being incorporated into cost-of-illness and cost-effectiveness analyses. However, little is known about whether the method used to identify carers affects the estimated impacts. We compare a novel time diary technique to a common self-declaration question for identifying carers. We investigate whether it: (1) detects more and different carers, and (2) if carers across identification techniques have different mental health outcomes. Methods We use the Innovation Panel component of the UK Household Longitudinal Study, which records all activities performed in two 24-h periods and contains a rich set of individual characteristics. We use regression analysis to compare the number and characteristics of carers identified across the two methods. We then use the doubly robust approach of entropy balancing combined with regression adjustment to estimate the mental health impacts of caregiving across both methods. Results Among 1055 individuals, we identify 261 carers by at least one method. The self-declaration method fails to classify 16% of individuals identified as carers through time diary data. We find that carers identified by the time diary have a 1.24 (p < 0.05) higher score on the 36-point General Health Questionnaire (GHQ) scale in the subsequent survey wave compared with similar non-carers. For self-declared carers, the estimated difference in GHQ score is 0.36 (p> 0.1), a smaller and statistically non-significant association compared with that observed among time diary-identified carers Conclusions The mental health impacts of caregiving may be underestimated when carers are identified by self-declaration. Supplementing self-declaration with time diaries may offer a means of including more carers. Future research, if only one method is applied, should more carefully consider the means of identifying informal carers and the implications that the use of one method may have on conclusions.
  PubDate: 2025-05-16
The Use of EQ-5D in the Middle East and North Africa Region: A Systematic
Literature Review
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  Abstract: Introduction The EQ-5D is the most commonly used preference-based measure of health-related quality of life. There is limited evidence about the use of the EQ-5D in the Middle East and North Africa (MENA) region. This study aimed to systematically identify, review, summarize, and synthesize the published literature on using the EQ-5D in this region. Methods A systematic literature review was conducted, according to the PRISMA 2020 guidelines, using PubMed, Cochrane, PsycINFO, and CINAHL and covering the period up to 30 August 2024. Studies using any version of the EQ-5D in adults or youth in the MENA region were included. Pilot studies, guidelines, study protocols, and reviews were excluded. Key study characteristics and outcomes assessed included study design, clinical area, population, type of EQ-5D data reported, reference value set used, and mode of administration. Title/abstract screening was conducted independently by two reviewers to assess eligibility for inclusion. Two researchers completed full-text screening and extracted data using a standardized form. Disagreements were referred to a third reviewer if not resolved by discussion. Results were summarized in systematic evidence tables. Results After removing duplicates, 18,034 references were considered for title/abstract screening. In total, 184 studies were included with a total sample size of 128,164 subjects. Of the included single-country studies, 42% were reported in Iran, 20% in Saudi Arabia, and 11% in Jordan. Patient populations were investigated in 86% of the studies, 23% of which targeted endocrine diseases. Study design was observational in 57% and experimental in 14% of the studies. Only 10% of the included studies applied the EQ-5D in an economic evaluation. The EQ-5D-3L version was used in 40% of the studies. However, the trend is towards a greater use of the 5L version in more recent years. Twenty percent of the studies reported EQ-5D results using the index score, frequencies of severity levels per dimension, and visual analog scale scores. EQ-5D modes of administration and funding sources were not reported in 16% and 20% of the studies, respectively. Conclusion There is an increased use of the EQ-5D in the MENA region, especially since 2020. In the region, the use of the EQ-5D is more prevalent in clinical studies than in economic evaluation studies. The reporting heterogeneity indicates the need for guidance in reporting EQ-5D study results in this region.
  PubDate: 2025-05-10
When Evidence Falls Short—Belzutifan for Treating Tumours Associated
with von Hippel-Lindau Disease: An External Assessment Group Perspective
on a NICE Single Technology Appraisal
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  PubDate: 2025-05-07
Societal Versus Healthcare Perspectives on the Cost Effectiveness of
Ocrelizumab for Treatment of Primary Progressive Multiple Sclerosis in
Aotearoa New Zealand
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  Abstract: Objectives Multiple sclerosis (MS) is a progressive, degenerative, autoimmune neuronal disease. This study compares the impact of a societal versus a healthcare perspective on the cost effectiveness of treatment of primary progressive MS (PPMS) with ocrelizumab (OCR) versus best supportive care (BSC) in New Zealand. Methods The analysis utilises a lifetime cohort Markov model based on ten Expanded Disability Status Scale (EDSS) states, plus death. It has two structurally identical arms, with forward transition probabilities in the treatment arm obtained by multiplying forward transition probabilities (converted to rates, and back to probabilities) in the control arm by the 12-week disability progression hazard ratio in the clinical trial ORATORIO. Direct and indirect costs of MS were estimated from a 2017 Australian survey and converted to 2024 NZ dollars using purchasing power parity and the NZ consumer price index. Future costs and benefits were discounted at 3.5% per annum. The model is calibrated to NZ mortality for PPMS, and therapy ends when wheelchair dependence (EDSS7) is reached. Results For a modelled cohort 40 years of age starting at the ORATORIO distribution of EDSS, at 50% of the list price (viz. 50% rebate on $NZ37,384 per patient per annum), the incremental cost-effectiveness ratio (ICER) of OCR versus BSC is $NZ114,427 per QALY ($US64,651) from a societal perspective or $NZ146,711 ($US82,892) from a healthcare perspective. From a societal perspective, at Treasury’s willingness to pay (WTP) criterion of $NZ120,200, an acquisition cost up to 56% of list price ($NZ20,935; 44% rebate) is justifiable. From a healthcare payer perspective, at Treasury’s implied WTP of $NZ43,313, an acquisition cost up to 10% of list price ($NZ3738; 90% rebate) is justifiable. Based on this metric, a 5.6-fold higher investment in OCR can be justified from a societal perspective compared with a healthcare perspective. Alternatively, an acquisition cost up to 37.9% of list price (viz. 62.1% rebate or $NZ14,169) could be justified if the criterion of one GDP per capita ($NZ83,011) is used as a societal funding threshold. These results are sensitive to the cost of BSC from a societal perspective but not from a healthcare perspective. From both perspectives the cost effectiveness is sensitive to the acquisition cost and efficacy of OCR, potential waning of efficacy, the age and EDSS state when therapy begins, the cost and timing of entry of a biosimilar pharmaceutical and the discount rate. Conclusions Treatment of PPMS with OCR is more cost effective from a societal than a healthcare perspective, therefore prioritisation of public funding of novel pharmaceuticals for MS and other resource intensive chronic health conditions will depend critically upon the study perspective.
  PubDate: 2025-05-07
Cost-Effectiveness of a Digital Leakage Notification System (Heylo™) for
People with Ileostomies or Colostomies in the United Kingdom
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  Abstract: Background People with stomas report a substantial negative impact of stomal effluent leaking outside the ostomy baseplate and subsequent complications in their professional and social lives, causing immense worry. However, many patients are not able to recognize leakages in a timely manner. We conducted a cost-effectiveness study to evaluate the impact of a digital leakage notification system (DLNS) to reduce leakages outside the baseplate (LOB) and worry about leakage for people with intestinal stomas from a UK National Health Service and Personal Social Services perspective. Methods A Markov model for ostomy care was used to compare health-related quality of life and costs for adults with ileostomies or colostomies using UK standard of care ostomy products with the DLNS (intervention) or without the DLNS (comparator). The base case model used a 3-year time horizon with 1-week cycles and an annual 3.5% discounting of utilities and costs. Patients in all health states experience LOB events and/or worry about leakage as events associated with one-time utility decrements and costs of additional healthcare provider visits and ostomy product use. Probabilities of LOB (DLNS, 46.5%; comparator, 78.6%) and worry about leakage (DLNS, 39.1%; comparator, 78.6%) were based on clinical trial results. Peristomal skin complications were included in the model with the same probabilities of occurrence in the intervention and comparator arms. Sensitivity and scenario analyses were performed to test the robustness of the base case model assumptions. Results In the base case analysis, the DLNS arm had 49.81 fewer LOB events per person than the comparator arm, resulting in 0.309 incremental quality-adjusted life-years (QALYs) and cost savings of British Pound Sterling (GBP) £1703 per person over 3 years (2023/2024 costing year). The DLNS arm had 56.98 fewer worry about leakage events per person, resulting in cost savings of £403 per person. Total costs and QALYs were £18,600 and 1.818 for the intervention arm, respectively, and £18,566 and 1.509 for the comparator arm. Overall, the DLNS provided 0.309 incremental QALYs at an incremental cost of £34 versus the comparator arm for an incremental cost-effectiveness ratio of £110/QALY gained, well below a willingness-to-pay threshold of £20,000/QALY. Sensitivity analyses showed the DLNS was cost-effective in 97.6% of simulations. Conclusions This analysis suggests that the DLNS added to standard of care ostomy products is a cost-effective intervention to help prevent LOB events and reduce worry about leakage for people with stomas in the UK. Results of the present study suggest that timely awareness of leakage has a positive impact on the health-related quality of life of people with stomas and on the direct costs of stoma management in the UK.
  PubDate: 2025-05-06

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