PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-11-25DOI: 10.1007/s40273-024-01439-y
Hiral Anil Shah, Ginita Jutlla, Oscar Herrera-Restrepo, Jonathan Graham, Katherine A Hicks, Justin Carrico, Mei Grace, Diana E Clements, Cindy Burman, Woo-Yun Sohn, Elise Kuylen, Shahina Begum, Zeki Kocaata
{"title":"Public Health Impact of Introducing a Pentavalent Vaccine Against Invasive Meningococcal Disease in the United States.","authors":"Hiral Anil Shah, Ginita Jutlla, Oscar Herrera-Restrepo, Jonathan Graham, Katherine A Hicks, Justin Carrico, Mei Grace, Diana E Clements, Cindy Burman, Woo-Yun Sohn, Elise Kuylen, Shahina Begum, Zeki Kocaata","doi":"10.1007/s40273-024-01439-y","DOIUrl":"10.1007/s40273-024-01439-y","url":null,"abstract":"<p><strong>Background: </strong>Invasive meningococcal disease (IMD) is primarily associated with five Neisseria meningitidis serogroups: A, B, C, W, or Y. In the United States (US), available vaccines protect against serogroups B (MenB), A, C, W, and Y (MenACWY), and A, B, C, W, and Y (MenABCWY). The Advisory Committee on Immunization Practices is re-evaluating the adolescent meningococcal vaccination schedule with varying recommendation formats. This analysis aimed to predict which schedule could avert the most IMD cases and have the most positive public health impact (PHI).</p><p><strong>Methods: </strong>An epidemiological model compared the 15-year PHI of vaccination schedules using MenB, MenACWY, and/or MenABCWY vaccines versus current US standard of care (SoC). Varying coverage rates reflected routine, shared clinical decision making, and risk-based recommendations. Sensitivity analyses assessed robustness of the results to different inputs/assumptions.</p><p><strong>Results: </strong>The most positive PHI compared with SoC was observed with one dose of MenACWY at 11 years of age and two doses of MenABCWY (6 months apart) at 16 years of age, assuming routine recommendation and coverage reflecting real-world uptake of MenACWY. This strategy resulted in 123 IMD cases averted (MenB: 59, MenACWY: 64), 17 deaths prevented, 574 life-years saved, and 757 quality-adjusted life-years gained versus SoC. Eliminating MenACWY vaccination at 11 years was found to result in an additional IMD burden.</p><p><strong>Conclusion: </strong>A routinely recommended two-dose pentavalent vaccine, with doses administered 6 months apart at 16 years of age, alongside the routinely recommended MenACWY vaccine at 11 years of age, would improve the PHI and benefits of IMD vaccination to society.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"311-329"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825582/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142710883","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-11-15DOI: 10.1007/s40273-024-01453-0
Xiaomo Xiong, Jeff Jianfei Guo
{"title":"Cost Effectiveness of Tremelimumab Plus Durvalumab for Unresectable Hepatocellular Carcinoma in the USA.","authors":"Xiaomo Xiong, Jeff Jianfei Guo","doi":"10.1007/s40273-024-01453-0","DOIUrl":"10.1007/s40273-024-01453-0","url":null,"abstract":"<p><strong>Background: </strong>Treating unresectable hepatocellular carcinoma (uHCC) is challenging. Clinical trials have shown that Single Tremelimumab Regular Interval Durvalumab (STRIDE) offers clinical benefits as a first-line treatment for uHCC, but its cost effectiveness remains unknown in the USA.</p><p><strong>Objective: </strong>We aimed to assess the cost effectiveness of STRIDE (tremelimumab plus durvalumab) versus sorafenib and durvalumab monotherapy as the first-line treatment for uHCC in the USA.</p><p><strong>Methods: </strong>A partitioned survival model was constructed to assess the cost effectiveness of STRIDE compared to sorafenib and durvalumab monotherapy as the first-line treatment for uHCC from the US societal perspective. The time horizon was 48 months with 1-month cycles. Seven parametric survival functions replicated survival curves from clinical trials, with the best-fitting model used to calculate survival probabilities. Costs, health utilities, and adverse events were included, with quality-adjusted life-years (QALYs) as the primary effectiveness measure. Both costs and effectiveness were discounted at 3%. In the base-case analysis, the incremental cost-effectiveness ratio was compared to a willingness-to-pay threshold of $150,000 per QALY gained. Deterministic and probabilistic sensitivity analyses were conducted to examine the uncertainty of the model.</p><p><strong>Results: </strong>In the base-case analysis, STRIDE was cost effective compared to sorafenib, with an incremental cost-effectiveness ratio of $97,995.51 per QALY gained, based on a willingness-to-pay threshold of $150,000 per QALY gained. However, STRIDE was not cost effective compared to durvalumab monotherapy at the same threshold, with an incremental cost-effectiveness ratio of $754,408.92 per QALY gained. Deterministic sensitivity analyses were consistent with the base-case analysis. A probabilistic sensitivity analysis indicated that STRIDE was more likely to be cost effective than sorafenib and durvalumab monotherapy when the willingness-to-pay exceeded $101,000 and $713,000, respectively.</p><p><strong>Conclusions: </strong>The STRIDE regimen appears to be cost effective compared to sorafenib but not compared to durvalumab for first-line uHCC treatment in the USA. However, durvalumab has not yet been approved for uHCC in the USA. Future research should focus on long-term data and economic evaluations of other recommended biologics.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"271-282"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638768","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-12-02DOI: 10.1007/s40273-024-01454-z
Jedidiah I Morton, Danny Liew, Gerald F Watts, Sophia Zoungas, Stephen J Nicholls, Christopher M Reid, Zanfina Ademi
{"title":"Immediate Versus 5-Year Risk-Guided Initiation of Treatment for Primary Prevention of Cardiovascular Disease for Australians Aged 40 Years: A Health Economic Analysis.","authors":"Jedidiah I Morton, Danny Liew, Gerald F Watts, Sophia Zoungas, Stephen J Nicholls, Christopher M Reid, Zanfina Ademi","doi":"10.1007/s40273-024-01454-z","DOIUrl":"10.1007/s40273-024-01454-z","url":null,"abstract":"<p><strong>Background and objective: </strong>Current Australian cardiovascular disease (CVD) prevention guidelines calculate 5-year CVD risk and recommend treatment when risk crosses specific thresholds. This may leave risk factors untreated for people with a low short-term (i.e. 5 years), but high long-term (i.e. lifetime), risk of CVD. We aimed to evaluate the cost effectiveness of intervention for risk factor control at age 40 years (regardless of calculated risk) compared to intervention for risk factor control at the age recommended by contemporary Australian CVD prevention guidelines (when the 5-year CVD risk reaches 10%) across a range of individual risk factor profiles.</p><p><strong>Methods: </strong>We used a causal microsimulation model populated with 108 different risk factor profiles, each replicated 10,000 times. Model data were derived from the UK Biobank study and published sources. The primary causal relationships factored in were those of low-density lipoprotein-cholesterol and systolic blood pressure with CVD (defined as myocardial infarction or stroke). The model simulated the ageing of individuals from 40 to 85 years. We calculated years of life lived, quality-adjusted life-years gained, incremental healthcare costs and the incremental cost-effectiveness ratio when low-density lipoprotein-cholesterol and blood pressure were controlled from age 40 years compared to initiation of treatment as recommended by Australian guidelines. The main side effect in the model was an increased risk of type 2 diabetes mellitus from statin use. The trade-off between reduced CVD and increased type 2 diabetes was summarised via quality-adjust life-years. Incremental cost-effectiveness ratios were compared to the Australian willingness-to-pay threshold of AU$28,000 per quality-adjust life-year gained. We adopted a healthcare perspective (2022 AUD) and discounted results at 3% annually.</p><p><strong>Results: </strong>An earlier intervention meaningfully prevented CVD in all but the lowest risk individuals. Intervention at age 40 years versus age when the 5-year CVD risk reaches 10% led to an increase in quality-adjust life-years for 37/54 female individuals and 44/54 male individuals simulated and an increase in years of life lived (i.e. life expectancy) for 46/54 female individuals and 47/54 male individuals simulated. Earlier intervention was also cost effective in 5/54 female individuals and 17/54 male individuals.</p><p><strong>Conclusions: </strong>Current guidelines may result in certain individuals with a lower 5-year, but higher lifetime, risk of CVD being overlooked for earlier cost-effective interventions to prevent CVD.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"331-349"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142771142","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-11-25DOI: 10.1007/s40273-024-01457-w
Enoch Yi-Tung Chen, Paul W Dickman, Mark S Clements
{"title":"A Multistate Model Incorporating Relative Survival Extrapolation and Mixed Time Scales for Health Technology Assessment.","authors":"Enoch Yi-Tung Chen, Paul W Dickman, Mark S Clements","doi":"10.1007/s40273-024-01457-w","DOIUrl":"10.1007/s40273-024-01457-w","url":null,"abstract":"<p><strong>Background: </strong>Multistate models have been widely applied in health technology assessment. However, extrapolating survival in a multistate model setting presents challenges in terms of precision and bias. In this article, we develop an individual-level continuous-time multistate model that integrates relative survival extrapolation and mixed time scales.</p><p><strong>Methods: </strong>We illustrate our proposed model using an illness-death model. We model the transition rates using flexible parametric models. We update the hesim package and the microsimulation package in R to simulate event times from models with mixed time scales. This feature allows us to incorporate relative survival extrapolation in a multistate setting. We compare several multistate settings with different parametric models (standard vs. flexible parametric models), and survival frameworks (all-cause vs. relative survival framework) using a previous clinical trial as an illustrative example.</p><p><strong>Results: </strong>Our proposed approach allows relative survival extrapolation to be carried out in a multistate model. In the example case study, the results agreed better with the observed data than did the commonly applied approach using standard parametric models within an all-cause survival framework.</p><p><strong>Conclusions: </strong>We introduce a multistate model that uses flexible parametric models and integrates relative survival extrapolation with mixed time scales. It provides an alternative to combine short-term trial data with long-term external data within a multistate model context in health technology assessment.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"297-310"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825556/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142716834","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-03-01Epub Date: 2024-12-03DOI: 10.1007/s40273-024-01460-1
Sarah J Nevitt, David M Phillippo, Robert Hodgson, Nicky J Welton, Sofia Dias
{"title":"Application of Multi-level Network Meta-Regression in the NICE Technology Appraisal of Quizartinib for Induction, Consolidation and Maintenance Treatment of Newly Diagnosed FLT3-ITD-Positive Acute Myeloid Leukaemia: An External Assessment Group Perspective.","authors":"Sarah J Nevitt, David M Phillippo, Robert Hodgson, Nicky J Welton, Sofia Dias","doi":"10.1007/s40273-024-01460-1","DOIUrl":"10.1007/s40273-024-01460-1","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"243-247"},"PeriodicalIF":4.4,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11825565/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142771140","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-02-26DOI: 10.1007/s40273-025-01471-6
Michał Jakubczyk, Bram Roudijk, Stefan A Lipman, Peep Stalmeier
{"title":"Making Composite Time Trade-Off Sensitive for Worse-than-Dead Health States.","authors":"Michał Jakubczyk, Bram Roudijk, Stefan A Lipman, Peep Stalmeier","doi":"10.1007/s40273-025-01471-6","DOIUrl":"https://doi.org/10.1007/s40273-025-01471-6","url":null,"abstract":"<p><strong>Objective: </strong>The utilities elicited with the composite time trade-off (cTTO) method for health states worse-than-dead (WTD) often correlate poorly with other severity measures, indicating a poor sensitivity of cTTO. We aimed to explore modifications to cTTO to better understand this phenomenon and identify potential improvements.</p><p><strong>Methods: </strong>A total of 480 respondents completed an online TTO interview, each valuing 12 EQ-5D-5L health states. The participants were randomized into four arms, A-D. Arm A followed the standard cTTO, serving as a reference. In arm B, we removed the sorting question comparing immediate death versus 10 years in a valued state. Arm C allowed for utility values <math><mrow><mo><</mo> <mo>-</mo> <mn>1</mn></mrow> </math> by reducing the time in the valued state in the lead-time TTO (LT-TTO) part of cTTO. In arm D, we randomly selected the starting negative utility in LT-TTO. Utility value distributions, correlations between utilities and level sum score (LSS), and inconsistencies between Pareto-ordered states were analyzed.</p><p><strong>Results: </strong>Arm A replicated the lack of significant correlation between LSS and the negative utility observed in previous work. Of the experimental arms, only arm B exhibited a significant negative correlation. Compared with arm A, arm B produced a higher proportion of WTD states ( <math><mrow><mn>46.5</mn> <mo>%</mo></mrow> </math> versus <math><mrow><mn>26.3</mn> <mo>%</mo></mrow> </math> ), less negative utility for WTD states on average ( <math><mrow><mo>-</mo> <mn>0.571</mn></mrow> </math> versus <math><mrow><mo>-</mo> <mn>0.752</mn></mrow> </math> ), and a lower mean censored utility for 55555 ( <math><mrow><mo>-</mo> <mn>0.486</mn></mrow> </math> versus <math><mrow><mo>-</mo> <mn>0.406</mn></mrow> </math> ).</p><p><strong>Conclusions: </strong>The observed lack of correlation between LSS and utility for WTD states appears linked to the use of comparison with immediate death in the sorting question. LT-TTO is capable of eliciting utility values in a way that is sensitive to severity. Modifying the initial questions in cTTO to identify whether health states are BTD or WTD should be considered.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143502145","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-02-21DOI: 10.1007/s40273-025-01474-3
Antal Zemplenyi, Jim Leonard, Garth C Wright, Michael J DiStefano, Kavita Nair, Kelly E Anderson, R Brett McQueen
{"title":"Innovative Payment Models for Sickle-Cell Disease Gene Therapies in Medicaid: Leveraging Real-World Data and Insights from CMMI's Gene Therapy Access Model.","authors":"Antal Zemplenyi, Jim Leonard, Garth C Wright, Michael J DiStefano, Kavita Nair, Kelly E Anderson, R Brett McQueen","doi":"10.1007/s40273-025-01474-3","DOIUrl":"https://doi.org/10.1007/s40273-025-01474-3","url":null,"abstract":"<p><strong>Objective: </strong>This study aims to evaluate the financial implications of implementing various payment models, including outcome-based agreements (OBAs), volume-based rebates, and guaranteed rebates, for the newly approved gene therapies, exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel), in the treatment of sickle-cell disease (SCD) from the perspective of Colorado Medicaid. The analysis specifically examines the cost of standard of care (SoC) for severe SCD, the impact of different eligibility criteria based on vaso-occlusive events (VOEs), and the potential financial impacts associated with rebate structures.</p><p><strong>Methods: </strong>Data from the Colorado Department of Health Care Policy & Financing (HCPF) database was used to estimate the annual costs for Medicaid-enrolled patients with severe SCD from 2018 to 2023. Patients were selected based on various eligibility criteria, including the number of VOEs, acute chest syndrome events, and stroke diagnoses. Three-state Markov models (SCD, stable, and dead) were constructed to compare the costs of SoC and gene therapies. The durability of gene therapy effectiveness and the financial impact of OBAs, volume-based rebates, and guaranteed rebates were evaluated over a 6-year contract period, with scenarios reflecting different VOE criteria and treatment durability.</p><p><strong>Results: </strong>The average annual SoC cost for severe SCD patients (N = 138) was US$45,941 (SD US$59,653), with higher costs associated with more frequent VOEs. Gene therapies exa-cel and lovo-cel, with one-off list prices of US$2.2 million and US$3.1 million, respectively, exhibited high upfront costs, resulting in a negative cumulative balance averaging - US$2.11 million for exa-cel and - US$3.00 million for lovo-cel per patient over 6 years compared with SoC. Outcome-based rebates could potentially save Medicaid approximately US$260K (uncertainty interval 88K-772K) per patient on average for exa-cel and US$367K (uncertainty interval 122K-1111K) for lovo-cel after they pay the full up-front cost. Volume-based and guaranteed rebates also offered potential savings but varied in impact based on contract duration and effectiveness of gene therapy.</p><p><strong>Conclusions: </strong>The study highlights critical considerations for Medicaid in negotiating OBAs for SCD gene therapies. Achieving budget neutrality over 6 years is unlikely due to low SoC costs. However, payment models can enhance value-based spending by linking high therapy costs and potential rebates to the health gains these treatments may offer. OBAs offer offsets contingent on therapy effectiveness durability and contract terms (such as length and price), while varying eligibility criteria impact budgets and outcomes. Medicaid real-world data is crucial for navigating complexities in defining eligible populations and structuring OBAs.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-02-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143468544","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-02-20DOI: 10.1007/s40273-025-01469-0
Florian Zeevat, Simon van der Pol, Alexia Kieffer, Maarten J Postma, Cornelis Boersma
{"title":"Cost-Effectiveness Analysis of Nirsevimab for Preventing Respiratory Syncytial Virus-Related Lower Respiratory Tract Disease in Dutch Infants: An Analysis Including All-Infant Protection.","authors":"Florian Zeevat, Simon van der Pol, Alexia Kieffer, Maarten J Postma, Cornelis Boersma","doi":"10.1007/s40273-025-01469-0","DOIUrl":"https://doi.org/10.1007/s40273-025-01469-0","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to assess the cost effectiveness of nirsevimab, a recently authorized monoclonal antibody (mAb) for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV), in comparison with the standard practice involving palivizumab for high-risk infants during their first RSV season in the Netherlands.</p><p><strong>Methods: </strong>A static cost-effectiveness model was populated for the Netherlands to evaluate different immunization strategies for nirsevimab over a single RSV season from a societal perspective. The model considered the most recently published RSV incidence data (average incidence from 2006 to2018), costs (adjusted to the 2023 price year), and associated health effects. Extensive scenario analyses were conducted to explore various strategies, and sensitivity analysis was performed to assess the model's robustness.</p><p><strong>Results: </strong>In the base-case scenario, all-infant protection-a strategy of in-season with catch-up immunization for all infants-nirsevimab has the potential to prevent numerous RSV-related cases, including 2333 hospitalizations and 150 intensive-care admissions, in the overall population compared with the standard of care. Nirsevimab appears to be cost effective under this strategy with an economically justifiable acquisition price for nirsevimab of €220 at a willingness-to-pay threshold of €50,000 per quality-adjusted life-year. Sensitivity analyses indicate a 52% probability that nirsevimab is cost effective at this threshold. Comparison of different vaccination strategies revealed that the all-infant protection approach was the one that prevented the higher number of cases.</p><p><strong>Conclusions: </strong>This study indicates that universal infant immunization with nirsevimab has the potential to be cost effective and significantly reduces the burden of RSV among Dutch infants. These findings underscore the importance of implementing effective protective measures against RSV-LRTD, reducing the pressure on the healthcare system during the RSV season.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-02-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143458849","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"A Taxonomy for Assessing Whether HRQoL Value Sets Are Obsolete.","authors":"Richard Norman, Bram Roudijk, Marcel Jonker, Elly Stolk, Saskia Knies, Raoh-Fang Pwu, Ciaran O'Neill, Kirsten Howard, Nancy Devlin","doi":"10.1007/s40273-025-01476-1","DOIUrl":"https://doi.org/10.1007/s40273-025-01476-1","url":null,"abstract":"<p><p>Providing health-related quality of life (HRQoL) value sets to enable estimation of quality adjusted life years (QALYs) is important in facilitating economic evaluation and in supporting reliable decision-making about healthcare. However, as the field matures, many value sets across a range of HRQoL instruments are now old, based on potentially outdated valuation methodologies and preference data from samples that no longer represent the contemporary population. Having a clear strategy for identification and mitigation of obsolescence is important to ensure policy makers retain confidence in their country-specific value sets. In this Current Opinion, we develop a taxonomy of value set obsolescence. We then explore how the different types of obsolescence might be identified and how methodologists might work with local policymakers to address obsolescence and therefore ensure HRQoL instruments remain relevant for use. The taxonomy of obsolescence consists of four main areas: (1) the value set no longer aligns with current normative health technology assessment (HTA) requirements; (2) the methods used to generate it are no longer considered robust or adequately close to best practice; (3) the population composition has moved too far from the characteristics of the sample in which the original value set was derived; and (4) even after controlling for population differences, preferences are likely to have changed since the original data collection. Through identification of the type of obsolescence that applies in a particular setting, we then suggest a range of possible solutions to each, ranging from recommending particular sensitivity analyses, through reweighting of existing data to better account for population differences, to collecting new data for an updated value set. Obsolescence of existing value sets is driven by more than just time since data collection is often a matter of judgment rather than based on a clear definition. The taxonomy presented here provides a tool for assessing whether value sets are obsolete and what the appropriate response to this obsolescence should be. Working closely with local policymakers and involving discussions regarding the ongoing appropriateness of existing value sets should form an important part of future activities. This should include the consideration of updating value sets in contemporary populations using current best-practice methods. However, the benefits of updating value sets have to be balanced against the cost of doing so, including the challenges faced by policymakers when new values sets require a transition to new local decision-making processes.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143441378","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
PharmacoEconomicsPub Date : 2025-02-12DOI: 10.1007/s40273-025-01472-5
Jeremiah Donoghue, Matthew Youngs, Alex Reeve, Krishna Vydyula, Natalia Kunst, Roochi Trikha, Daniel Gallacher
{"title":"Examining Consistency Across NICE Single Technology Appraisals: A Review of Appraisals for Paroxysmal Nocturnal Haemoglobinuria.","authors":"Jeremiah Donoghue, Matthew Youngs, Alex Reeve, Krishna Vydyula, Natalia Kunst, Roochi Trikha, Daniel Gallacher","doi":"10.1007/s40273-025-01472-5","DOIUrl":"https://doi.org/10.1007/s40273-025-01472-5","url":null,"abstract":"<p><p>In 2024, the National Institute for Health and Care Excellence (NICE) recommended two new health technologies for paroxysmal nocturnal haemoglobinuria. This review systematically compares the clinical and cost-effectiveness evidence considered within the NICE single technology appraisals of iptacopan, danicopan and pegcetacoplan, examines the consistency of the clinical evidence and economic modelling, and considers whether single technology appraisals are a suitable apparatus for consistent decision making. The studies used different follow-up lengths and used different definitions for reporting breakthrough haemolysis (BTH), but otherwise reported similar outcomes and found a significant benefit for their interventions. A lack of direct evidence and unreliable indirect comparisons meant that naïve comparisons across trials were carried into the economic modelling despite differences in their control arms. Approaches to modelling BTH and associated dose escalation differed across appraisals, despite information for pegcetacoplan coming from the same source in each appraisal, which had a large impact on the economic results. This review raises the question of whether NICE should implement multiple technology appraisals more frequently to reduce these inconsistences. Additionally, we recommend the development of a framework for revisiting positive recommendations when the implementation of health technologies deviates from assumptions made in the economic modelling to ensure cost-effective healthcare is preserved.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399709","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}