{"title":"乳腺癌治疗试验是否充分考虑了与健康相关的生活质量?根据法国卫生技术评估机构2009-2023年的报销意见进行评估","authors":"","doi":"10.1016/j.jcpo.2024.100504","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><p>Breast cancer treatments can impact the patients’ health-related quality of life (HR-QoL). This criterion is relevant for drug reimbursement decisions. We wanted to assess the usage of HR-QoL in health technology assessments (HTA).</p></div><div><h3>Methods</h3><p>All HAS (<em>Haute Autorité de Santé</em>, the French HTA body) opinions published between January 1, 2009 and March 31, 2023 for the reimbursement of breast cancer drugs were analysed.</p></div><div><h3>Results</h3><p>51 distinct appraisals were found during the period, corresponding to 45 product-specific indications, of which 36 (80 %) including clinical studies in which HR-QoL was an endpoint. HAS explicitly rejected HR-QoL data in 25 out of 36 (69 %) indications with such data. Rejections are justified by methodological weaknesses, including lack of adjustment for type I error inflation (n=21 indications), open-label treatment (n=7), lack of a pre-specified clinically relevant HR-QoL threshold (n=6) or missing data (n=6). Regardless of rejection status, HR-QoL results were not mentioned as a determinant of value assessment in 3/36 (8 %) instances (2/25 for rejected data).</p></div><div><h3>Conclusions</h3><p>HR-QoL data are inconsistently present in HTA assessments of new breast cancer drugs. Their methodological quality often hinders their use in determining the drug’s value.</p></div><div><h3>Policy summary</h3><p>A rigorous and acceptable comparative experimental framework is expected for HR-QoL assessments. More detail on the precise impact of the absence or presence of HR-QoL data in the determination of the drug’s added value could help understanding how this dimension is influential in the assessments.</p></div>","PeriodicalId":38212,"journal":{"name":"Journal of Cancer Policy","volume":null,"pages":null},"PeriodicalIF":2.0000,"publicationDate":"2024-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Is health-related quality of life sufficiently addressed in trials for breast cancer treatments? An assessment based on reimbursement opinions from the French health technology assessment body, 2009–2023\",\"authors\":\"\",\"doi\":\"10.1016/j.jcpo.2024.100504\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Background</h3><p>Breast cancer treatments can impact the patients’ health-related quality of life (HR-QoL). This criterion is relevant for drug reimbursement decisions. We wanted to assess the usage of HR-QoL in health technology assessments (HTA).</p></div><div><h3>Methods</h3><p>All HAS (<em>Haute Autorité de Santé</em>, the French HTA body) opinions published between January 1, 2009 and March 31, 2023 for the reimbursement of breast cancer drugs were analysed.</p></div><div><h3>Results</h3><p>51 distinct appraisals were found during the period, corresponding to 45 product-specific indications, of which 36 (80 %) including clinical studies in which HR-QoL was an endpoint. HAS explicitly rejected HR-QoL data in 25 out of 36 (69 %) indications with such data. Rejections are justified by methodological weaknesses, including lack of adjustment for type I error inflation (n=21 indications), open-label treatment (n=7), lack of a pre-specified clinically relevant HR-QoL threshold (n=6) or missing data (n=6). Regardless of rejection status, HR-QoL results were not mentioned as a determinant of value assessment in 3/36 (8 %) instances (2/25 for rejected data).</p></div><div><h3>Conclusions</h3><p>HR-QoL data are inconsistently present in HTA assessments of new breast cancer drugs. Their methodological quality often hinders their use in determining the drug’s value.</p></div><div><h3>Policy summary</h3><p>A rigorous and acceptable comparative experimental framework is expected for HR-QoL assessments. More detail on the precise impact of the absence or presence of HR-QoL data in the determination of the drug’s added value could help understanding how this dimension is influential in the assessments.</p></div>\",\"PeriodicalId\":38212,\"journal\":{\"name\":\"Journal of Cancer Policy\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":2.0000,\"publicationDate\":\"2024-09-10\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Cancer Policy\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2213538324000389\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"HEALTH POLICY & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Cancer Policy","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2213538324000389","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"HEALTH POLICY & SERVICES","Score":null,"Total":0}
Is health-related quality of life sufficiently addressed in trials for breast cancer treatments? An assessment based on reimbursement opinions from the French health technology assessment body, 2009–2023
Background
Breast cancer treatments can impact the patients’ health-related quality of life (HR-QoL). This criterion is relevant for drug reimbursement decisions. We wanted to assess the usage of HR-QoL in health technology assessments (HTA).
Methods
All HAS (Haute Autorité de Santé, the French HTA body) opinions published between January 1, 2009 and March 31, 2023 for the reimbursement of breast cancer drugs were analysed.
Results
51 distinct appraisals were found during the period, corresponding to 45 product-specific indications, of which 36 (80 %) including clinical studies in which HR-QoL was an endpoint. HAS explicitly rejected HR-QoL data in 25 out of 36 (69 %) indications with such data. Rejections are justified by methodological weaknesses, including lack of adjustment for type I error inflation (n=21 indications), open-label treatment (n=7), lack of a pre-specified clinically relevant HR-QoL threshold (n=6) or missing data (n=6). Regardless of rejection status, HR-QoL results were not mentioned as a determinant of value assessment in 3/36 (8 %) instances (2/25 for rejected data).
Conclusions
HR-QoL data are inconsistently present in HTA assessments of new breast cancer drugs. Their methodological quality often hinders their use in determining the drug’s value.
Policy summary
A rigorous and acceptable comparative experimental framework is expected for HR-QoL assessments. More detail on the precise impact of the absence or presence of HR-QoL data in the determination of the drug’s added value could help understanding how this dimension is influential in the assessments.