Carolina Alonzo, Ding Ding, Jiat-Ling Poon, Juergen Zschocke, Lei Zhang, Aranishi Toshihiko, Shane Myrick, Jennifer Hill, Louise Larkin, Nancy Perez, Laure Delbecque
{"title":"PP132 澳大利亚、中国和日本的医疗技术评估机构对患者体验数据的期望","authors":"Carolina Alonzo, Ding Ding, Jiat-Ling Poon, Juergen Zschocke, Lei Zhang, Aranishi Toshihiko, Shane Myrick, Jennifer Hill, Louise Larkin, Nancy Perez, Laure Delbecque","doi":"10.1017/s0266462323002441","DOIUrl":null,"url":null,"abstract":"<span>Introduction</span><p>Health technology assessment (HTA) agencies are increasingly embracing patient experience data (PED) to support reimbursement decisions. This study aimed to review HTA agencies’ expectations with regards to utilizing PED to support drug reimbursement in Australia, Japan, and China.</p><span>Methods</span><p>Published HTA guidance documents were reviewed in 2021 to identify any PED-specific information. If available, recommendations related to the type of PED (e.g., generic vs. disease-specific clinical outcomes assessment (COA)); COA validation, analyses, endpoints and interpretation; and the interest in PED beyond COA in HTA decision-making (e.g., patient preference information) were reviewed. Literature review and semi-structured interviews with key opinion leaders (KOLs) were conducted to further explore these themes and future trends with regards to PED.</p><span>Results</span><p>Australia’s Pharmaceutical Benefits Advisory Committee guidance document includes a dedicated section on patient-reported outcomes (PRO), providing details on preferred PRO instruments; validation expectations; and recommended methods to explore score interpretation, assess and report PRO results and handle missing data. While PED derived from non-PRO sources are not discussed in the guidance, the KOL noted that they should not be ruled out. Japan’s Center for Outcomes Research and Economic Evaluation for Health guideline includes a section dedicated to PROs without details related to instrument validation, analyses and interpretation, however, is focused on the use of PRO to inform health economic assessments. In China, the HTA center of China National Health Commission drafted two disease-specific technical guidance documents recommending the inclusion of PROs in efficacy assessments and use of instruments relevant in the Chinese population; these points were echoed by the KOL interviewed.</p><span>Conclusions</span><p>There are recommendations on PED use included in country-specific guidance documents, however their level of detail varies greatly. Knowing each agency’s expectations with regards to PED is key when submitting HTA evidence dossiers and should be considered early in clinical trial design to integrate market access perspectives and optimize drug development.</p>","PeriodicalId":14467,"journal":{"name":"International Journal of Technology Assessment in Health Care","volume":null,"pages":null},"PeriodicalIF":2.6000,"publicationDate":"2023-12-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"PP132 Health Technology Assessment Agencies’ Expectations Regarding Patient Experience Data in Australia, China, And Japan\",\"authors\":\"Carolina Alonzo, Ding Ding, Jiat-Ling Poon, Juergen Zschocke, Lei Zhang, Aranishi Toshihiko, Shane Myrick, Jennifer Hill, Louise Larkin, Nancy Perez, Laure Delbecque\",\"doi\":\"10.1017/s0266462323002441\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<span>Introduction</span><p>Health technology assessment (HTA) agencies are increasingly embracing patient experience data (PED) to support reimbursement decisions. This study aimed to review HTA agencies’ expectations with regards to utilizing PED to support drug reimbursement in Australia, Japan, and China.</p><span>Methods</span><p>Published HTA guidance documents were reviewed in 2021 to identify any PED-specific information. If available, recommendations related to the type of PED (e.g., generic vs. disease-specific clinical outcomes assessment (COA)); COA validation, analyses, endpoints and interpretation; and the interest in PED beyond COA in HTA decision-making (e.g., patient preference information) were reviewed. Literature review and semi-structured interviews with key opinion leaders (KOLs) were conducted to further explore these themes and future trends with regards to PED.</p><span>Results</span><p>Australia’s Pharmaceutical Benefits Advisory Committee guidance document includes a dedicated section on patient-reported outcomes (PRO), providing details on preferred PRO instruments; validation expectations; and recommended methods to explore score interpretation, assess and report PRO results and handle missing data. While PED derived from non-PRO sources are not discussed in the guidance, the KOL noted that they should not be ruled out. Japan’s Center for Outcomes Research and Economic Evaluation for Health guideline includes a section dedicated to PROs without details related to instrument validation, analyses and interpretation, however, is focused on the use of PRO to inform health economic assessments. In China, the HTA center of China National Health Commission drafted two disease-specific technical guidance documents recommending the inclusion of PROs in efficacy assessments and use of instruments relevant in the Chinese population; these points were echoed by the KOL interviewed.</p><span>Conclusions</span><p>There are recommendations on PED use included in country-specific guidance documents, however their level of detail varies greatly. Knowing each agency’s expectations with regards to PED is key when submitting HTA evidence dossiers and should be considered early in clinical trial design to integrate market access perspectives and optimize drug development.</p>\",\"PeriodicalId\":14467,\"journal\":{\"name\":\"International Journal of Technology Assessment in Health Care\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":2.6000,\"publicationDate\":\"2023-12-14\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"International Journal of Technology Assessment in Health Care\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1017/s0266462323002441\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"HEALTH CARE SCIENCES & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"International Journal of Technology Assessment in Health Care","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1017/s0266462323002441","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
PP132 Health Technology Assessment Agencies’ Expectations Regarding Patient Experience Data in Australia, China, And Japan
Introduction
Health technology assessment (HTA) agencies are increasingly embracing patient experience data (PED) to support reimbursement decisions. This study aimed to review HTA agencies’ expectations with regards to utilizing PED to support drug reimbursement in Australia, Japan, and China.
Methods
Published HTA guidance documents were reviewed in 2021 to identify any PED-specific information. If available, recommendations related to the type of PED (e.g., generic vs. disease-specific clinical outcomes assessment (COA)); COA validation, analyses, endpoints and interpretation; and the interest in PED beyond COA in HTA decision-making (e.g., patient preference information) were reviewed. Literature review and semi-structured interviews with key opinion leaders (KOLs) were conducted to further explore these themes and future trends with regards to PED.
Results
Australia’s Pharmaceutical Benefits Advisory Committee guidance document includes a dedicated section on patient-reported outcomes (PRO), providing details on preferred PRO instruments; validation expectations; and recommended methods to explore score interpretation, assess and report PRO results and handle missing data. While PED derived from non-PRO sources are not discussed in the guidance, the KOL noted that they should not be ruled out. Japan’s Center for Outcomes Research and Economic Evaluation for Health guideline includes a section dedicated to PROs without details related to instrument validation, analyses and interpretation, however, is focused on the use of PRO to inform health economic assessments. In China, the HTA center of China National Health Commission drafted two disease-specific technical guidance documents recommending the inclusion of PROs in efficacy assessments and use of instruments relevant in the Chinese population; these points were echoed by the KOL interviewed.
Conclusions
There are recommendations on PED use included in country-specific guidance documents, however their level of detail varies greatly. Knowing each agency’s expectations with regards to PED is key when submitting HTA evidence dossiers and should be considered early in clinical trial design to integrate market access perspectives and optimize drug development.
期刊介绍:
International Journal of Technology Assessment in Health Care serves as a forum for the wide range of health policy makers and professionals interested in the economic, social, ethical, medical and public health implications of health technology. It covers the development, evaluation, diffusion and use of health technology, as well as its impact on the organization and management of health care systems and public health. In addition to general essays and research reports, regular columns on technology assessment reports and thematic sections are published.