Value in Health最新文献

{"title":"Guiding Principles for Evaluating Vaccines in Joint Health Technology Assessment in the European Union: Preparing for the EU's Regulation on HTA for Vaccines.","authors":"Nathalie Largeron, Paolo D'Agostino, Ruth Chapman, David Danko, Juhani Eskola, Philippe Godfroid, Gyorgyi Feldmajer, Riona Hanley, Gerard de Pouvourville, Maarten Postma, Joan Puig-Barberà, Kassandra Schaible, Ugne Sabale, Joe Schmitt, Chiara de Waure, Anna Vicere, Ekkehard Beck","doi":"10.1016/j.jval.2024.06.011","DOIUrl":"https://doi.org/10.1016/j.jval.2024.06.011","url":null,"abstract":"<p><strong>Objectives: </strong>The appraisal of vaccines in the European Union (EU) currently involves many different decision-making bodies and processes. The objective of this study was to help inform the development of standardized methodology and vaccine-specific processes for use in the EU Regulation on health technology assessment (HTA).</p><p><strong>Methods: </strong>Literature reviews and expert consultation were conducted to identify current practices and gaps related to vaccine appraisals and to develop guiding principles for the joint clinical assessment of vaccines.</p><p><strong>Results: </strong>We found that significant variation exists across the EU Member States in the decision-making processes when clinically evaluating vaccines. Three guiding principles consisting of 13 recommendations were developed to help inform the development of decision-making frameworks for the joint clinical assessment of vaccines in the EU: (1) Support the creation of appropriate terminology and measurements for clinical appraisals of vaccines; (2) Develop inclusive, timely, and transparent vaccine appraisal processes to support stronger evidence generation for vaccine decision-making and appraisal; and (3) Improve the collection and interoperability of real-world data, including robust surveillance, to foster evidence generation and support the standardization of vaccine clinical appraisals.</p><p><strong>Conclusions: </strong>Given the significance of vaccines for public health, there is an urgency to develop standardized and vaccine-specific methodologies and processes for use in the EU joint HTA framework. The proposed guiding principles could support the effective implementation of the EU Regulation on HTA for vaccines and have the potential to ensure consistent, transparent, and timely access to new vaccines in the EU.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559835","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Methods for Measuring and Valuing Informal Care: A Systematic Review and Meta-Analysis in Stroke.","authors":"Neha Das, Phuong Nguyen, Thi Quynh Anh Ho, Peter Lee, Suzanne Robinson, Lan Gao","doi":"10.1016/j.jval.2024.06.007","DOIUrl":"10.1016/j.jval.2024.06.007","url":null,"abstract":"<p><strong>Objectives: </strong>To accurately capture informal care in healthcare evaluations, rigorous approaches are required to measure and value this important care component. In this systematic review and meta-analysis, we intended to summarize the current methods of measuring and valuing informal care costs in healthcare evaluations (full and partial healthcare evaluations, including cost of illness and cost analysis) in stroke.</p><p><strong>Methods: </strong>A systematic search was conducted in MEDLINE, Embase, EconLit, and CINAHL. We used EndNote 20, Research Screener, and Covidence platforms for screening and data extraction. A meta-analysis was performed on informal care hours, and a subgroup meta-analysis was conducted based on stroke severity.</p><p><strong>Results: </strong>A total of 31 articles were included in the qualitative synthesis. There was variation among the studies in the informal care measurement and valuation approaches. The meta-analysis of studies where data on informal care hours were available showed an estimate of informal care hours of 25.76 per week (95% CI 13.36-38.16) with a high heterogeneity (I² = 99.97%). The overall risk of bias in the studies was assessed as low.</p><p><strong>Conclusions: </strong>Standardizing the measurement and valuation of informal care costs is essential for improving the consistency and comparability of economic evaluations. Pilot studies that incorporate standardized informal care cost valuation methods can help identify any practical challenges and capture the impact of informal care more accurately.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559814","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Does Willingness to Pay Differ for Mental and Physical Health?","authors":"Eleanor Eaton, Alistair Hunt","doi":"10.1016/j.jval.2024.06.009","DOIUrl":"10.1016/j.jval.2024.06.009","url":null,"abstract":"<p><strong>Objectives: </strong>The urban environment can have a significant impact on mental and physical health. Health impact appraisal of new developments should address these issues. However, transferable economic valuation evidence for urban planners in the United Kingdom is thin, especially around mental health, making it harder to estimate the cost-efficiency of public health interventions to address these conditions. A further complication is that mental health may be perceived differently from physical health. This study examines willingness to pay (WTP) to avoid depression and lower back pain.</p><p><strong>Methods: </strong>WTP estimates were obtained by applying contingent valuation tasks in an online survey with a representative sample in the United Kingdom (N = 1553). Interval regression models were used to estimate the effects of disease severity, payment frequency, and respondent characteristics on WTP.</p><p><strong>Results: </strong>Respondents' WTP to avoid both conditions was relatively high (around 5%-6% of stated income to return to current health state). Depression was rated as being twice as burdensome on quality of life than pain, and bids to avoid depression were 20% to 30% more than pain. Analysis of motivation responses suggests mental health treatment is perceived as less easy to access and less effective than the equivalent for pain, and respondents expect a larger burden on their family and relationships as they try to manage their condition themselves.</p><p><strong>Conclusions: </strong>Results suggest that depression bids may be affected by uncertainty around access to effective treatment in the healthcare system. This has implications for how mental illness may be prioritized in resource allocation toward public health interventions.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559832","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sales Revenues for New Therapeutic Agents Approved by the United States Food and Drug Administration From 1995 to 2014.","authors":"Olivier J Wouters, Aaron S Kesselheim, Jouni Kuha, Jeroen Luyten","doi":"10.1016/j.jval.2024.06.015","DOIUrl":"10.1016/j.jval.2024.06.015","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to analyze worldwide sales of new therapeutic agents and to estimate the time it takes for product sales to exceed industry-wide average drug development costs.</p><p><strong>Methods: </strong>Data obtained from company reports were analyzed to track worldwide sales of new medicines approved by the US Food and Drug Administration from 1995 to 2014. All sales figures were reported in 2019 US dollars. Kaplan-Meier curves were used to evaluate the time it took for discounted product sales to exceed the average costs associated with developing 1 new drug (accounting for the costs of failed trials), using published estimates of these costs.</p><p><strong>Results: </strong>Based on data for 361 of 558 new therapeutic agents approved over the study period (median follow-up 13.2 years), mean sales revenue per product was $15.2 billion through the end of 2019; the median was $6.7 billion. These products jointly generated global sales of $5.5 trillion since approval. Revenues were highly skewed, with the 25 best selling products (7%, 25 of 361) accounting for 38% of this amount ($2.1 trillion of $5.5 trillion). Approximately 47% of products had discounted sales that exceeded the estimated industry-wide average costs of development within 5 years of approval, and 75% within 10 years. After attributing potential production, marketing, and other costs, these numbers dropped to 21% of products within 5 years of approval, and 46% within 10 years.</p><p><strong>Conclusions: </strong>Sales of new medicines approved from 1995 to 2014 were highly skewed, but many products had net discounted sales that exceeded the industry-wide average costs of development within 10 years of approval. An understanding of how sales revenues accrue in the years after initial approval, alongside data on business costs, can inform discussions about how to incentivize private investment in innovation while ensuring affordable prices for patients and the healthcare system.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559816","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Cost-Effectiveness of Germline BReast CAncer Gene Testing in Metastatic Prostate Cancer Followed by Cascade Testing of First-Degree Relatives of Mutation Carriers.","authors":"Srinivas Teppala, Paul Scuffham, Kim Edmunds, Matthew J Roberts, David Fairbairn, David P Smith, Lisa Horvath, Haitham Tuffaha","doi":"10.1016/j.jval.2024.06.019","DOIUrl":"10.1016/j.jval.2024.06.019","url":null,"abstract":"<p><strong>Objectives: </strong>Patients with metastatic prostate cancer (mPCa) with BReast CAncer gene (BRCA) mutations benefit from targeted treatments (eg, olaparib). In addition, family members of affected patients have increased risk of hereditary cancers and benefit from early detection and prevention. International guidelines recommend genetic testing in mPCa; however, the value for money of testing patients with mPCa and cascade testing of blood-related family members has not been assessed. In this context, we evaluated the cost-effectiveness of germline BRCA testing in patients with mPCa followed by cascade testing of first-degree relatives (FDRs) of mutation carriers.</p><p><strong>Methods: </strong>We conducted a cost-utility analysis of germline BRCA testing using 2 scenarios: (1) testing patients with mPCa only and (2) testing patients with mPCa and FDRs of those who test positive. A semi-Markov multi-health-state transition model was constructed using a lifetime time horizon. The analyses were performed from an Australian payer perspective. Decision uncertainty was characterized using probabilistic analyses.</p><p><strong>Results: </strong>Compared with no testing, BRCA testing in mPCa was associated with an incremental cost of AU$3731 and a gain of 0.014 quality-adjusted life-years (QALYs), resulting in an incremental cost-effectiveness ratio of AU$265 942/QALY. Extending testing to FDRs of variant-positive patients resulted in an incremental cost-effectiveness ratio of AU$16 392/QALY. Probability of cost-effectiveness at a willingness-to-pay of AU$75 000/QALY was 0% in the standalone mPCa analysis and 100% in the cascade testing analysis.</p><p><strong>Conclusion: </strong>BRCA testing when performed as a standalone strategy in patients with mPCa may not be cost-effective but demonstrates significant value for money after the inclusion of cascade testing of FDRs of mutation carriers.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559817","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Traditional Methods Hold Their Ground Against Machine Learning in Predicting Potentially Inappropriate Medication Use in Older Adults.","authors":"Yohann Moanahere Chiu, Caroline Sirois, Marc Simard, Marie-Eve Gagnon, Denis Talbot","doi":"10.1016/j.jval.2024.06.005","DOIUrl":"10.1016/j.jval.2024.06.005","url":null,"abstract":"<p><strong>Objectives: </strong>Machine learning methods have gained much attention in health sciences for predicting various health outcomes but are scarcely used in pharmacoepidemiology. The ability to identify predictors of suboptimal medication use is essential for conducting interventions aimed at improving medication outcomes. It remains uncertain whether machine learning methods could enhance the identification of potentially inappropriate medication use among older adults compared with traditional methods. This study aimed to (1) to compare the performances of machine learning models in predicting use of potentially inappropriate medications and (2) to quantify and compare the relative importance of predictors in a population of community-dwelling older adults (>65 years) in the province of Québec, Canada.</p><p><strong>Methods: </strong>We used the Québec Integrated Chronic Disease Surveillance System and selected a cohort of 1 105 295 older adults of whom 533 719 were potentially inappropriate medication users. Potentially inappropriate medications were defined according to the Beers list. We compared performances between 5 popular machine learning models (gradient boosting machines, logistic regression, naive Bayes, neural networks, and random forests) based on receiver operating characteristic curves and other performance criteria, using a set of sociodemographic and medical predictors.</p><p><strong>Results: </strong>No model clearly outperformed the others. All models except neural networks were in agreement regarding the top predictors (sex and anxiety-depressive disorders and schizophrenia) and the bottom predictors (rurality and social and material deprivation indices).</p><p><strong>Conclusions: </strong>Including other types of predictors (eg, unstructured data) may be more useful for increasing performance in prediction of potentially inappropriate medication use.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559819","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Valuation of Multi-Dimensional Health States With a Bolt-On: Is There a Shortcut?","authors":"Zhihao Yang, Kim Rand, Nan Luo","doi":"10.1016/j.jval.2024.06.018","DOIUrl":"10.1016/j.jval.2024.06.018","url":null,"abstract":"<p><strong>Objectives: </strong>To use the EQ-5D questionnaire with bolt-on dimensions in economic evaluation studies, new value sets are needed. In this study, we explored the feasibility of a new approach called the scaling factor model, which estimates bolt-on value sets using estimated EQ-5D dimensional weights.</p><p><strong>Methods: </strong>We designed a 2-arm study, inviting university students to value health states with and without bolt-on items using the composite time trade-off method. We selected 25 health states from an orthogonal array and added the 5 mildest EQ-5D states in the design. In arm 1, EQ-5D without self-care and standard EQ-5D states were valued, and in arm 2, standard EQ-5D states and EQ-5D with vision were valued. By arm, we compared the mean observed values of health states with and without bolt-on item. Next, by arm, we estimated value sets for the EQ-5D with bolt-on states using both standard model and scaling factor model. Model performances were compared in terms of prediction accuracy and correlation with likelihood-based mean values.</p><p><strong>Results: </strong>Adding a five-level bolt-on to EQ-5D resulted in statistically lower values. This effect was consistent across 2 arms and bolt-on items. The scaling factor models outperformed the standard models in all statistics.</p><p><strong>Conclusions: </strong>The scaling factor model offers a methodologically viable and low-cost option for producing value sets for EQ-5D supplemented with bolt-on items. Future studies should further test this method using other bolt-on items and more relevant study populations.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559820","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Tutorial on Value-Based Adaptive Designs: Could a Value-Based Sequential 2-Arm Design Have Created More Health Economic Value for the Big CACTUS Trial?","authors":"Laura Flight, Alan Brennan, Isabelle Wilson, Stephen E Chick","doi":"10.1016/j.jval.2024.06.004","DOIUrl":"10.1016/j.jval.2024.06.004","url":null,"abstract":"<p><strong>Objectives: </strong>Value-based trials aim to maximize the expected net benefit by balancing technology adoption decisions and clinical trial costs. Adaptive trials offer additional efficiency. This article provides guidance on determining whether a value-based sequential design is the best option for an adaptive 2-arm trial, illustrated through a case study.</p><p><strong>Methods: </strong>We outlined 4 steps for the value-based sequential approach. The case study re-evaluates the Big CACTUS trial design using pilot trial data and a model-based health economic analysis. Expected net benefit is computed for (1) original fixed design, (2) value-based design with fixed sample size, and (3) optimal value-based sequential design with adaptive stopping. We compare pretrial modeling with the actual Big CACTUS trial results.</p><p><strong>Results: </strong>Over 10 years, the adoption decision would affect approximately 215 378 patients. Pretrial modeling shows that the expected net benefit minus costs are (1) £102 million for the original fixed design, (2) £107 million (+5.3% higher) for the value-based design with optimal fixed sample size, and (3) £109 million (+6.7% higher) for the optimal value-based sequential design with maximum sample size of 435 per arm. Post hoc analysis using actual Big CACTUS trial data indicates that the value-adaptive trial with a maximum sample size of 95 participant pairs would not have stopped early. Bootstrap simulations reveal a 9.76% probability of early completion with n = 95 pairs compared with 31.50% with n = 435 pairs.</p><p><strong>Conclusions: </strong>The 4-step approach to value-based sequential 2-arm design with adaptive stopping was successfully implemented. Further application of value-based adaptive approaches could be useful to assess the efficiency of alternative study designs.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559830","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Key Issues of Economic Evaluations for Health Technology Assessment in China: A Nationwide Expert Survey.","authors":"Gordon G Liu, Haijing Guan, Nan Peng, Shitong Xie, Kang Wang, Larry Z Liu, Yanbing Zhou, Huajie Jin","doi":"10.1016/j.jval.2024.06.020","DOIUrl":"10.1016/j.jval.2024.06.020","url":null,"abstract":"<p><strong>Objectives: </strong>Health technology assessment (HTA) is increasingly crucial in medicine price negotiations in China, yet previous appraisals revealed national discrepancies on key economic evaluation issues: willingness-to-pay threshold, pricing models for multi-indication medicines, and comparator selection principles. This study aimed to collect expert opinions on these issues for future HTA evaluations.</p><p><strong>Methods: </strong>A nationwide anonymous web-based survey encompassing experts across academia, HTA, consultancy/contract research organization/industry, service provider, and payer. In 2023, a generic invitation containing a web link to the questionnaire was disseminated via WeChat using convenience and snowball sampling. Agreement rates for questionnaire views were analyzed using descriptive statistics. The relationship between participants' responses and demographics was examined using appropriate logistic models.</p><p><strong>Results: </strong>A total of 303 responses were received from experts in 34 cities. Key expert views include a suggested base willingness-to-pay threshold ranging from 0.5 to 1.5 times gross domestic product (52.1% agreement); elevated thresholds for childhood diseases, rare diseases, end-of-life diseases, and first-in-class medicines (>78.0% agreement); a single pricing model for multi-indication medicines (60.4% agreement); consideration of multiple medicines as comparators (79.9% agreement); and avoiding the use of centrally procured medicines as comparators for medicines with a time to market of less than 3 years (71.0% agreement). Participants who are service provider had lower odds of selecting higher thresholds (odds ratio 0.26; P < .01) than responders from consultancy/contract research organization/industry.</p><p><strong>Conclusions: </strong>Expert views indicate the need for substantial changes in China's current HTA methods, highlighting the need for increased investment in HTA processes and expertise cultivation.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559837","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Three Pillars of Support for Orphan Drug Programs: Individual Valuations, Societal Valuations, and Anonymous Altruism.","authors":"Darius N Lakdawalla, Charles E Phelps, Hong-Hanh Nguyen","doi":"10.1016/j.jval.2024.06.014","DOIUrl":"10.1016/j.jval.2024.06.014","url":null,"abstract":"<p><strong>Objectives: </strong>To identify and describe potential societal and individual sources of support for orphan drug programs.</p><p><strong>Methods: </strong>The Generalized Risk-Adjusted Cost-Effectiveness method shows that acute illness and disability severity increase individuals' willingness to pay for health gains. We develop a social welfare function (SWF) that incorporates individuals' own values, combined with politically or ethically determined weights. We introduce the concept of horizontal equity-that individuals in similar situations should be treated similarly-into the SWF. Finally, we introduce anonymous altruism into individuals' utility functions-the desire to help others, without knowing their identity.</p><p><strong>Results: </strong>Combined with the empirical link between disease severity and rarity, the Generalized Risk-Adjusted Cost-Effectiveness method demonstrates heightened willingness to pay for health gains for people with rare diseases, leading rational individuals to support orphan drug programs, our first pillar of support. Adding horizontal equity to the SWF further increases societal support for orphan drug programs. Anonymous altruism, focusing most strongly on those in the most-dire circumstances, leads to altruistic support for those with severe disorders. Because innovators' economic incentives lead them to focus on larger markets, anonymous altruistic individuals will increasingly prefer public investments into rare diseases over time, as private markets systematically produce gains for common diseases.</p><p><strong>Conclusions: </strong>We identified 3 supporting pillars for orphan drug programs: (1) individuals' propensity to prefer treatments for severe diseases; (2) the preference for horizontal equity in our social welfare; (3) anonymous altruism, the desire to help strangers, coupled with market incentives that underserve strangers with rare diseases.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559818","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}