PharmacoEconomicsPub Date : 2024-12-01Epub Date: 2024-08-29DOI: 10.1007/s40273-024-01425-4
Ash Bullement, Mark Edmondson-Jones, Patricia Guyot, Nicky J Welton, Gianluca Baio, Matthew Stevenson, Nicholas R Latimer
{"title":"MPES-R: Multi-Parameter Evidence Synthesis in R for Survival Extrapolation-A Tutorial.","authors":"Ash Bullement, Mark Edmondson-Jones, Patricia Guyot, Nicky J Welton, Gianluca Baio, Matthew Stevenson, Nicholas R Latimer","doi":"10.1007/s40273-024-01425-4","DOIUrl":"10.1007/s40273-024-01425-4","url":null,"abstract":"<p><p>Survival extrapolation often plays an important role in health technology assessment (HTA), and there are a range of different approaches available. Approaches that can leverage external evidence (i.e. data or information collected outside the main data source of interest) may be helpful, given the extent of uncertainty often present when determining a suitable survival extrapolation. One of these methods is the multi-parameter evidence synthesis (MPES) approach, first proposed for use in HTA by Guyot et al., and more recently by Jackson. While MPES has potential benefits over conventional extrapolation approaches (such as simple or flexible parametric models), it is more computationally complex and requires use of specialist software. This tutorial presents an introduction to MPES for HTA, alongside a user-friendly, publicly available operationalisation of Guyot's original MPES that can be executed using the statistical software package R. Through two case studies, both Guyot's and Jackson's MPES approaches are explored, along with sensitivity analyses relevant to HTA. Finally, the discussion section of the tutorial details important considerations for analysts considering use of an MPES approach, along with potential further developments. MPES has not been used often in HTA, and so there are limited examples of how it has been used and perceived. However, this tutorial may aid future research efforts exploring the use of MPES further.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1317-1327"},"PeriodicalIF":4.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142110706","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 : 2024-12-01Epub Date: 2024-09-02DOI: 10.1007/s40273-024-01424-5
Meiyu Wu, Jing Ma, Sini Li, Shuxia Qin, Chongqing Tan, Ouyang Xie, Andong Li, Aaron G Lim, Xiaomin Wan
{"title":"Effects and Costs of Hepatitis C Virus Elimination for the Whole Population in China: A Modelling Study.","authors":"Meiyu Wu, Jing Ma, Sini Li, Shuxia Qin, Chongqing Tan, Ouyang Xie, Andong Li, Aaron G Lim, Xiaomin Wan","doi":"10.1007/s40273-024-01424-5","DOIUrl":"10.1007/s40273-024-01424-5","url":null,"abstract":"<p><strong>Background and objective: </strong>China has the highest number of hepatitis C virus (HCV) infections in the world. However, it is unclear what levels of screening and treatment are needed to achieve the WHO 2030 hepatitis C elimination targets. We aimed to evaluate the impact of scaling up interventions on the hepatitis C epidemic and determine how and at what cost these elimination targets could be achieved for the whole population in China.</p><p><strong>Methods: </strong>We developed a compartmental model incorporating HCV transmission, disease progression, and care cascade for the whole population in China, calibrated with data on demographics, injecting drug use, HCV prevalence, and treatments. Five different scenarios were evaluated for effects and costs for 2022-2030. All costs were converted to 2021 US dollar (USD) and discounted at an annual rate of 5%. One-way sensitivity analyses were conducted to assess the robustness of the model.</p><p><strong>Results: </strong>Under the status quo scenario, the incidence of hepatitis C is projected to increase from 60.39 (57.60-63.45) per 100,000 person-years in 2022 to 68.72 (65.3-73.97) per 100,000 person-years in 2030, and 2.52 million (1.94-3.07 million) infected patients are projected to die between 2022 and 2030, of which 0.76 (0.61-1.08) million will die due to hepatitis C. By increasing primary screening to 10%, conducting regular rescreening (annually for PWID and every 5 years for the general population) and treating 90% of patients diagnosed, the incidence would be reduced by 88.15% (86.61-89.45%) and hepatitis C-related mortality by 60.5% (52.62-65.54%) by 2030, compared with 2015 levels. This strategy would cost USD 52.78 (USD 43.93-58.53) billion.</p><p><strong>Conclusions: </strong>Without changes in HCV prevention and control policy, the disease burden of HCV in China will increase dramatically. To achieve the hepatitis C elimination targets, China needs to sufficiently scale up screening and treatment.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1345-1357"},"PeriodicalIF":4.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142110705","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 : 2024-12-01Epub Date: 2024-08-24DOI: 10.1007/s40273-024-01428-1
Ippazio Cosimo Antonazzo, Giorgia Gribaudo, Adriano La Vecchia, Pietro Ferrara, Alexandra Piraino, Paolo Angelo Cortesi, Lorenzo Giovanni Mantovani
{"title":"Cost and Cost Effectiveness of Treatments for Psoriatic Arthritis: An Updated Systematic Literature Review.","authors":"Ippazio Cosimo Antonazzo, Giorgia Gribaudo, Adriano La Vecchia, Pietro Ferrara, Alexandra Piraino, Paolo Angelo Cortesi, Lorenzo Giovanni Mantovani","doi":"10.1007/s40273-024-01428-1","DOIUrl":"10.1007/s40273-024-01428-1","url":null,"abstract":"<p><strong>Background: </strong>Psoriatic arthritis (PsA) is an inflammatory disease characterised by a variety of clinical manifestations. Considering the economic burden posed by PsA and the increasing number of treatment options, economic evaluations are required to better allocate available resources. This work aims to update a previous published literature review on PsA cost-of-illness and cost-effectiveness analysis.</p><p><strong>Methods: </strong>A search was performed of English-language literature between January 2017 and March 20, 2024 in Medline/PubMed, Embase and Cochrane library using the terms 'psoriatic arthritis', 'cost of illness' and 'cost effectiveness'. Data on decision model, time horizon, population, treatment options, perspective, type of costs, relevant results and authors' conclusion were extracted from the reviewed articles. Finally, the quality of the included studies was evaluated.</p><p><strong>Results: </strong>Twenty-seven studies met the inclusion criteria: 16 cost-of-illness and 11 cost-effectiveness/cost-utility analyses. PsA is characterised by high direct and indirect costs. Drug costs as well as hospitalisation and absenteeism were the major drivers of the observed costs. The cost-effectiveness analyses reported the dominance or the cost effectiveness of biologic therapies compared with non-biologic PsA treatment. Biological options like bimekizumab and ixekizumab have demonstrated a better cost-effectiveness profile in PsA patients compared with other treatments (i.e., other biological treatments).</p><p><strong>Conclusions: </strong>There was an increased number of economic evaluations compared with the previous review. PsA is still associated with significant economic burden worldwide. The main cost was represented by therapies, specifically biological therapies. Amongst the biological therapies, bimekizumab and ixekizumab appear to provide the most economic benefit. Finally, new economic studies are needed to enrich knowledge on the economic burden of subgroups of PsA patients as well as early treatment of PsA with new therapies.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1329-1343"},"PeriodicalIF":4.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142056248","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 : 2024-12-01Epub Date: 2024-09-26DOI: 10.1007/s40273-024-01432-5
Mihir Gandhi, Ravindran Kanesvaran, Mohamad Farid Bin Harunal Rashid, Dawn Qingqing Chong, Wen-Yee Chay, Rachel Lee-Yin Tan, Richard Norman, Madeleine T King, Nan Luo
{"title":"Valuation of the EORTC Quality of Life Utility Core 10 Dimensions (QLU-C10D) in a Multi-ethnic Asian Setting: How Does Having Cancer Matter?","authors":"Mihir Gandhi, Ravindran Kanesvaran, Mohamad Farid Bin Harunal Rashid, Dawn Qingqing Chong, Wen-Yee Chay, Rachel Lee-Yin Tan, Richard Norman, Madeleine T King, Nan Luo","doi":"10.1007/s40273-024-01432-5","DOIUrl":"10.1007/s40273-024-01432-5","url":null,"abstract":"<p><strong>Objectives: </strong>The aim of the study was to develop and compare utility value sets for the EORTC QLU-C10D, a cancer-specific utility instrument based on the EORTC QLQ-C30, using the preferences of the general public and cancer patients in Singapore, and to assess their measurement properties.</p><p><strong>Methods: </strong>A total of 600 individuals from the general public were recruited using a multi-stage random sampling, along with 626 cancer patients with clinically confirmed diagnoses from outpatient clinics of the largest tertiary cancer hospital. Each participant valued 16 pairs of EORTC QLU-C10D health states using a discrete choice experiment (DCE). Conditional logit models were used to analyze the DCE responses of the general public and cancer patients separately. Utility values were assessed for known-group validity and responsiveness in the cancer patients by comparing mean values across subgroups of patients and calculating standardized response means using longitudinal EORTC QLQ-C30 data, respectively.</p><p><strong>Results: </strong>Physical functioning and pain had the most impact on utility for both cancer patients and general public groups. Worst health state utility values were -0.821 and -0.463 for the general public and cancer patients, respectively. Cancer patients' values were lower for mild-to-moderate health states but higher for moderately-to-highly impaired states compared with the general public's values. Both value sets discriminated between patients with differing characteristics and responded equally well to improved health status, but the cancer patients' value set was slightly more responsive to deteriorated health.</p><p><strong>Conclusions: </strong>EORTC QLU-C10D value sets based on the preferences of the Singaporean general public and cancer patients exhibited differences in values but similar psychometric properties.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1413-1425"},"PeriodicalIF":4.4,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142351616","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 : 2024-11-01Epub Date: 2024-08-02DOI: 10.1007/s40273-024-01422-7
Brendan Collins, Gregory Y H Lip
{"title":"Delaying Oral Anticoagulants: A False Economy?","authors":"Brendan Collins, Gregory Y H Lip","doi":"10.1007/s40273-024-01422-7","DOIUrl":"10.1007/s40273-024-01422-7","url":null,"abstract":"","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1237-1239"},"PeriodicalIF":4.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141875587","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 : 2024-11-01Epub Date: 2024-08-07DOI: 10.1007/s40273-024-01410-x
Ewan M Tomeny, Thomas Hampton, Phuong Bich Tran, Laura Rosu, Mphatso D Phiri, Kathryn A Haigh, Jasper Nidoi, Tom Wingfield, Eve Worrall
{"title":"Rethinking Tuberculosis Morbidity Quantification: A Systematic Review and Critical Appraisal of TB Disability Weights in Cost-Effectiveness Analyses.","authors":"Ewan M Tomeny, Thomas Hampton, Phuong Bich Tran, Laura Rosu, Mphatso D Phiri, Kathryn A Haigh, Jasper Nidoi, Tom Wingfield, Eve Worrall","doi":"10.1007/s40273-024-01410-x","DOIUrl":"10.1007/s40273-024-01410-x","url":null,"abstract":"<p><strong>Background: </strong>The disability-adjusted life year (DALY), a key metric for health resource allocation, encompasses morbidity through disability weights. Widely used in tuberculosis cost-effectiveness analysis (CEAs), DALYs play a significant role in informing intervention adopt/reject decisions. This study reviews the values and consistency of disability weights applied in tuberculosis-related CEAs.</p><p><strong>Methods: </strong>We conducted a systematic review using the Tufts CEA database, updated to July 2023 with searches in Embase, Scopus and PubMed. Eligible studies needed to have included a cost-per-DALY ratio, and additionally either evaluated a tuberculosis (TB) intervention or included tuberculosis-related weights. We considered all tuberculosis health states: with/without human immunodeficiency virus (HIV) coinfection, TB treatments and treatment side effects. Data were screened and extracted independently by combinations of two authors.</p><p><strong>Findings: </strong>A total of 105 studies spanning 2002-2023 across 50 countries (mainly low- and middle-income countries) were extracted. Disability weights were sourced primarily from the Global Burden of Disease (GBD; 100/165; 61%), with 17 non-GBD studies additionally referenced, along with primary derivation. Inconsistencies in the utilisation of weights were evident: of the 100 usages of GBD-sourced weights, only in 47 instances (47%) had the weight value been explicitly specified with an appropriate up-to-date reference cited (constituting 28% of all weight usages, 47/165). Sensitivity analyses on weight values had been conducted in 30% of studies (31/105). Twelve studies did not clearly specify weights or their sources; nine further calculated DALYs without morbidity. The review suggests methodological gaps in current approaches for representing important aspects of TB, including TB-HIV coinfection, treatment, drug-resistance, extrapulmonary TB and psychological impacts. We propose a set of best practice recommendations.</p><p><strong>Interpretation: </strong>There is a need for increased rigour in the application, sensitivity testing and reporting of TB disability weights. Furthermore, there appears a desire among researchers to reflect elements of the tuberculosis experience beyond those allowed for by GBD disability weights.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1209-1236"},"PeriodicalIF":4.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499453/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897963","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}
{"title":"Considerations Around the Inclusion of Children and Young People's Time in Economic Evaluation: Findings from an International Delphi Study.","authors":"Cameron Morgan, Cam Donaldson, Emily Lancsar, Stavros Petrou, Lazaros Andronis","doi":"10.1007/s40273-024-01411-w","DOIUrl":"10.1007/s40273-024-01411-w","url":null,"abstract":"<p><strong>Background: </strong>People's time is a finite resource and a valuable input that ought to be considered in economic evaluations taking a broad, societal perspective. Yet, evaluations of interventions focusing on children and young people (CYP) rarely account for the opportunity cost of time in this population. As a key reason for this, health economists have pointed to uncertainty around when it is appropriate to include CYP time-related costs in an economic evaluation and highlighted the lack of clear guidance on the topic.</p><p><strong>Methods: </strong>With this in mind, we carried out a Delphi study to establish a list of relevant considerations for researchers to utilise whilst making decisions about whether and when to include CYP time in their economic evaluations. Delphi panellists were asked to propose and rate a set of possible considerations and provide additional thoughts on their ratings. Ratings were summarised using descriptive statistics, and text comments were interrogated through thematic analysis.</p><p><strong>Findings: </strong>A total of 73 panellists across 16 countries completed both rounds of a two-round Delphi study. Panellists' ratings showed that, when thinking about whether to include displaced CYP time in an economic evaluation, it is very important to consider whether: (1) inclusion would be in line with specified perspective(s) (median score: 9), (2) CYP's time may already be accounted for in other parts of the evaluation (median score: 8), (3) the amount of forgone time is substantial, either in absolute or relative terms (median score: 7) and (4) inclusion of CYP's time costs would be of interest to decision-makers (median score: 7). Respondents thought that considerations such as (1) whether inclusion would be of interest to the research community (median score: 6), (2) whether CYP's time displaced by receiving treatment is 'school' or 'play' time (median score: 5), and (3) whether CYP's are old enough for their time to be considered valuable (median score: 5) are moderately important. A range of views was offered to support beliefs and ratings, many of which were underpinned by compelling normative questions.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1267-1277"},"PeriodicalIF":4.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499441/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141996244","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 : 2024-11-01Epub Date: 2024-08-24DOI: 10.1007/s40273-024-01420-9
Zachary Tirrell, Alicia Norman, Martin Hoyle, Sean Lybrand, Bonny Parkinson
{"title":"Bring Out Your Dead: A Review of the Cost Minimisation Approach in Health Technology Assessment Submissions to the Australian Pharmaceutical Benefits Advisory Committee.","authors":"Zachary Tirrell, Alicia Norman, Martin Hoyle, Sean Lybrand, Bonny Parkinson","doi":"10.1007/s40273-024-01420-9","DOIUrl":"10.1007/s40273-024-01420-9","url":null,"abstract":"<p><strong>Objectives: </strong>Published literature has levied criticism against the cost-minimisation analysis (CMA) approach to economic evaluation over the past two decades, with multiple papers declaring its 'death'. However, since introducing the requirements for economic evaluations as part of health technology (HTA) decision-making in 1992, the cost-minimisation analysis (CMA) approach has been widely used to inform recommendations about the public subsidy of medicines in Australia. This research aimed to highlight the breadth of use of CMA in Australia and assess the influence of preconditions for the approach on subsidy recommendations METHODS: Relevant information was extracted from Public Summary Documents of Pharmaceutical Benefits Advisory Committee (PBAC) meetings in Australia considering submissions for the subsidy of medicines that included a CMA and were assessed between July 2005 and December 2022. A generalised linear model was used to explore the relationship between whether medicines were recommended and variables that reflected the primary preconditions for using CMA set out in the published PBAC Methodology Guidelines. Other control variables were selected through the Bolasso Method. Subgroup analysis was undertaken which replicated this modelling process.</p><p><strong>Results: </strong>While the potential for inferior safety or efficacy reduced the likelihood of recommendation (p < 0.01), the effect sizes suggest that the requirements for CMA were not requisite for recommendation.</p><p><strong>Conclusion: </strong>The Australian practice of CMA does not strictly align with the PBAC Methodology Guidelines and the theoretically appropriate application of CMA. However, within the confines of a deliberative HTA decision-making process that balances values and judgement with available evidence, this may be considered acceptable, particularly if stakeholders consider the current approach delivers sufficient clarity of process and enables patients to access medicines at an affordable cost.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1287-1300"},"PeriodicalIF":4.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499440/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142056247","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 : 2024-11-01Epub Date: 2024-08-06DOI: 10.1007/s40273-024-01414-7
Johanna Lister, Suzy Paisley, Christopher Carroll, Paul Tappenden
{"title":"Empirical Testing of Alternative Search Methods to Retrieve Utility Values for Health Economic Modelling.","authors":"Johanna Lister, Suzy Paisley, Christopher Carroll, Paul Tappenden","doi":"10.1007/s40273-024-01414-7","DOIUrl":"10.1007/s40273-024-01414-7","url":null,"abstract":"<p><strong>Objectives: </strong>The objective of this study is to compare different information retrieval methods that can be used to identify utility inputs for health economic models.</p><p><strong>Methods: </strong>The usual practice of using systematic review methods was compared with two alternatives (iterative searching and rapid review), using a health technology assessment (HTA) case study in ulcerative colitis (UC). We analysed whether there were differences in the utility values identified when using the alternative search methods. Success was evaluated in terms of time, burden and relevance of identified information. The identified utility values were tested in an executable health economic model developed for UC, and the model results were compared.</p><p><strong>Results: </strong>The usual practice of using systematic review search approaches identified the most publications but was also the least precise method and took longest to complete. The inclusion of data from the different search methods in the model did not lead to different conclusions across search methods.</p><p><strong>Conclusions: </strong>In this case study, usual practice was less efficient and resulted in the same health economic model conclusions as the alternative search methods. Further case studies are required to examine whether this conclusion might be generalisable.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1255-1266"},"PeriodicalIF":4.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897961","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 : 2024-11-01Epub Date: 2024-08-23DOI: 10.1007/s40273-024-01423-6
Kurt Taylor, Nicholas R Latimer, Thomas Douglas, Anthony J Hatswell, Sophia Ho, Gabriel Okorogheye, John Borril, Clara Chen, Inkyu Kim, David Bertwistle
{"title":"Treatment Effect Waning in Immuno-oncology Health Technology Assessments: A Review of Assumptions and Supporting Evidence with Proposals to Guide Modelling.","authors":"Kurt Taylor, Nicholas R Latimer, Thomas Douglas, Anthony J Hatswell, Sophia Ho, Gabriel Okorogheye, John Borril, Clara Chen, Inkyu Kim, David Bertwistle","doi":"10.1007/s40273-024-01423-6","DOIUrl":"10.1007/s40273-024-01423-6","url":null,"abstract":"<p><p>Treatment effect waning (TEW) refers to the attenuation of treatment effects over time. Assumptions of a sustained immuno-oncologic treatment effect have been a source of contention in health technology assessment (HTA). We review how TEW has been addressed in HTA and in the wider scientific literature. We analysed company submissions to English language HTA agencies and summarised methods and assumptions used. We subsequently reviewed TEW-related work in the ISPOR Scientific Presentations Database and conducted a targeted literature review (TLR) for evidence of the maintenance of immuno-oncology (IO) treatment effects post-treatment discontinuation. We found no standardised approach adopted by companies in submissions to HTA agencies, with immediate TEW most used in scenario analyses. Independently fitted survival models do however suggest TEW may often be implicitly modelled. Materials in the ISPOR scientific database suggest gradual TEW is more plausible than immediate TEW. The TLR uncovered evidence of durable survival in patients treated with IOs but no evidence that directly addresses the presence or absence of TEW. Our HTA review shows the need for a consistent and appropriate implementation of TEW in oncology appraisals. However, the TLR highlights the absence of direct evidence on TEW in literature, as TEW is defined in terms of relative treatment effects-not absolute survival. We propose a sequence of steps for analysts to use when assessing whether a TEW scenario is necessary and appropriate to present in appraisals of IOs.</p>","PeriodicalId":19807,"journal":{"name":"PharmacoEconomics","volume":" ","pages":"1181-1196"},"PeriodicalIF":4.4,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11499331/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142036603","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}