Piers Gillett, Robert K Mahar, Nancy R Tran, Mark Rosenthal, Maarten IJzerman
{"title":"开发和验证一种多标准决策分析工具来评估癌症临床试验的价值:评估癌症临床试验的价值。","authors":"Piers Gillett, Robert K Mahar, Nancy R Tran, Mark Rosenthal, Maarten IJzerman","doi":"10.1186/s12962-023-00496-y","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Demonstrating safety and efficacy of new medical treatments requires clinical trials but clinical trials are costly and may not provide value proportionate to their costs. As most health systems have limited resources, it is therefore important to identify the trials with the highest value. Tools exist to assess elements of a clinical trial such as statistical validity but are not wholistic in their valuation of a clinical trial. This study aims to develop a measure of clinical trials value and provide an online tool for clinical trial prioritisation.</p><p><strong>Methods: </strong>A search of the academic and grey literature and stakeholder consultation was undertaken to identify a set of criteria to aid clinical trial valuation using multi-criteria decision analysis. Swing weighting and ranking exercises were used to calculate appropriate weights of each of the included criteria and to estimate the partial-value function for each underlying metric. The set of criteria and their respective weights were applied to the results of six different clinical trials to calculate their value.</p><p><strong>Results: </strong>Seven criteria were identified: 'unmet need', 'size of target population', 'eligible participants can access the trial', 'patient outcomes', 'total trial cost', 'academic impact' and 'use of trial results'. The survey had 80 complete sets of responses (51% response rate). A trial designed to address an 'Unmet Need' was most commonly ranked as the most important with a weight of 24.4%, followed by trials demonstrating improved 'Patient Outcomes' with a weight of 21.2%. The value calculated for each trial allowed for their clear delineation and thus a final value ranking for each of the six trials.</p><p><strong>Conclusion: </strong>We confirmed that the use of the decision tool for valuing clinical trials is feasible and that the results are face valid based on the evaluation of six trials. A proof-of-concept applying this tool to a larger set of trials with an external validation is currently underway.</p>","PeriodicalId":47054,"journal":{"name":"Cost Effectiveness and Resource Allocation","volume":"21 1","pages":"87"},"PeriodicalIF":1.7000,"publicationDate":"2023-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10647033/pdf/","citationCount":"0","resultStr":"{\"title\":\"Developing and validating a multi-criteria decision analytic tool to assess the value of cancer clinical trials: evaluating cancer clinical trial value.\",\"authors\":\"Piers Gillett, Robert K Mahar, Nancy R Tran, Mark Rosenthal, Maarten IJzerman\",\"doi\":\"10.1186/s12962-023-00496-y\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Demonstrating safety and efficacy of new medical treatments requires clinical trials but clinical trials are costly and may not provide value proportionate to their costs. As most health systems have limited resources, it is therefore important to identify the trials with the highest value. Tools exist to assess elements of a clinical trial such as statistical validity but are not wholistic in their valuation of a clinical trial. This study aims to develop a measure of clinical trials value and provide an online tool for clinical trial prioritisation.</p><p><strong>Methods: </strong>A search of the academic and grey literature and stakeholder consultation was undertaken to identify a set of criteria to aid clinical trial valuation using multi-criteria decision analysis. Swing weighting and ranking exercises were used to calculate appropriate weights of each of the included criteria and to estimate the partial-value function for each underlying metric. The set of criteria and their respective weights were applied to the results of six different clinical trials to calculate their value.</p><p><strong>Results: </strong>Seven criteria were identified: 'unmet need', 'size of target population', 'eligible participants can access the trial', 'patient outcomes', 'total trial cost', 'academic impact' and 'use of trial results'. The survey had 80 complete sets of responses (51% response rate). A trial designed to address an 'Unmet Need' was most commonly ranked as the most important with a weight of 24.4%, followed by trials demonstrating improved 'Patient Outcomes' with a weight of 21.2%. The value calculated for each trial allowed for their clear delineation and thus a final value ranking for each of the six trials.</p><p><strong>Conclusion: </strong>We confirmed that the use of the decision tool for valuing clinical trials is feasible and that the results are face valid based on the evaluation of six trials. A proof-of-concept applying this tool to a larger set of trials with an external validation is currently underway.</p>\",\"PeriodicalId\":47054,\"journal\":{\"name\":\"Cost Effectiveness and Resource Allocation\",\"volume\":\"21 1\",\"pages\":\"87\"},\"PeriodicalIF\":1.7000,\"publicationDate\":\"2023-11-14\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10647033/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Cost Effectiveness and Resource Allocation\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1186/s12962-023-00496-y\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"HEALTH POLICY & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cost Effectiveness and Resource Allocation","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1186/s12962-023-00496-y","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"HEALTH POLICY & SERVICES","Score":null,"Total":0}
Developing and validating a multi-criteria decision analytic tool to assess the value of cancer clinical trials: evaluating cancer clinical trial value.
Background: Demonstrating safety and efficacy of new medical treatments requires clinical trials but clinical trials are costly and may not provide value proportionate to their costs. As most health systems have limited resources, it is therefore important to identify the trials with the highest value. Tools exist to assess elements of a clinical trial such as statistical validity but are not wholistic in their valuation of a clinical trial. This study aims to develop a measure of clinical trials value and provide an online tool for clinical trial prioritisation.
Methods: A search of the academic and grey literature and stakeholder consultation was undertaken to identify a set of criteria to aid clinical trial valuation using multi-criteria decision analysis. Swing weighting and ranking exercises were used to calculate appropriate weights of each of the included criteria and to estimate the partial-value function for each underlying metric. The set of criteria and their respective weights were applied to the results of six different clinical trials to calculate their value.
Results: Seven criteria were identified: 'unmet need', 'size of target population', 'eligible participants can access the trial', 'patient outcomes', 'total trial cost', 'academic impact' and 'use of trial results'. The survey had 80 complete sets of responses (51% response rate). A trial designed to address an 'Unmet Need' was most commonly ranked as the most important with a weight of 24.4%, followed by trials demonstrating improved 'Patient Outcomes' with a weight of 21.2%. The value calculated for each trial allowed for their clear delineation and thus a final value ranking for each of the six trials.
Conclusion: We confirmed that the use of the decision tool for valuing clinical trials is feasible and that the results are face valid based on the evaluation of six trials. A proof-of-concept applying this tool to a larger set of trials with an external validation is currently underway.
期刊介绍：
Cost Effectiveness and Resource Allocation is an Open Access, peer-reviewed, online journal that considers manuscripts on all aspects of cost-effectiveness analysis, including conceptual or methodological work, economic evaluations, and policy analysis related to resource allocation at a national or international level. Cost Effectiveness and Resource Allocation is aimed at health economists, health services researchers, and policy-makers with an interest in enhancing the flow and transfer of knowledge relating to efficiency in the health sector. Manuscripts are encouraged from researchers based in low- and middle-income countries, with a view to increasing the international economic evidence base for health.
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