Application of patient preference information in premarketing decision of medical products in the United States and its implications

REN Xiaoyue; YAN Jianzhou; DING Ruilin; SHAO Rong

doi:10.6039/j.issn.1001-0408.2024.19.01

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Application of patient preference information in premarketing decision of medical products in the United States and its implications

更新时间：2025-09-26

- Application of patient preference information in premarketing decision of medical products in the United States and its implications
- ZHONGGUO YAOFANG Vol. 35, Issue 19, Pages: 2321-2327(2024)
- 作者机构：
  
  中国药科大学国家药物政策与医药产业经济研究中心，南京　211198
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2024.19.01
  CLC： R95
- Received：18 May 2024，
  
  Revised：2024-08-26，
  
  Accepted：02 September 2024，
  
  Published：15 October 2024
- 稿件说明：
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任晓悦,颜建周,丁瑞琳,等.患者偏好信息在美国医疗产品上市前决策中的应用及启示[J].中国药房,2024,35(19):2321-2327.

REN Xiaoyue,YAN Jianzhou,DING Ruilin,et al.Application of patient preference information in premarketing decision of medical products in the United States and its implications[J].ZHONGGUO YAOFANG,2024,35(19):2321-2327.
任晓悦,颜建周,丁瑞琳,等.患者偏好信息在美国医疗产品上市前决策中的应用及启示[J].中国药房,2024,35(19):2321-2327. DOI： 10.6039/j.issn.1001-0408.2024.19.01.

REN Xiaoyue,YAN Jianzhou,DING Ruilin,et al.Application of patient preference information in premarketing decision of medical products in the United States and its implications[J].ZHONGGUO YAOFANG,2024,35(19):2321-2327. DOI： 10.6039/j.issn.1001-0408.2024.19.01.

摘要

目的

通过研究患者偏好信息在美国医疗产品上市前决策中的应用，为我国患者偏好信息的应用提供借鉴。

方法

采用文献研究法探究美国患者偏好信息收集与应用的概况、法律依据和参与主体，详细分析患者偏好信息在医疗产品上市前决策中的应用情况，分析我国患者偏好信息的应用现状与挑战，并提出建议。

结果与结论

美国通过开展多项患者参与项目以及立法推动多主体进行患者偏好信息的收集和应用，将患者偏好信息用于为医疗产品上市前决策提供参考：在研发环节为医疗产品研发和设计提供信息、辅助临床试验设计；在上市审批环节作为FDA上市审批决策的证据之一，用于识别获益大于风险的患者群体，并纳入医疗产品说明书。我国患者偏好信息的应用缺乏更高层级法律文件的指导，尚无针对性的指南文件。建议我国借鉴美国经验，在更高层级的法律文件中明确鼓励患者偏好的研究与应用；针对患者偏好信息的收集与应用制定专门的指导文件；根据我国国情确定监管决策中患者偏好信息纳入的权重和形式。

Abstract

OBJECTIVE

To provide reference for the application of patient preference information in China by studying the application of patient preference information in the premarketing decision-making of medical products in the United States.

METHODS

The literature research method was used to explore the general situation， legal basis， and participants of the collection and application of patient preference information in the United States， analyze the application of patient preference information in premarketing decision-making of medical products in detail， and analyze the application status and challenges of patient preference information in China， so as to put forward suggestions.

RESULTS & CONCLUSIONS

United States has promoted the collection and application of patient preference information through several patient participation projects and legislation. The patient preference information is used to support premarketing decision-making of medical products： providing information for medical product development and design， and assisting clinical trial design in the research and development process； helping to support FDA’s marketing approval decisions， identifying patient groups whose benefits outweigh risks， and included in medical product descriptions in the marketing approval process. The application of patient preference information in China lacks the guidance of higher-level legal documents， and there are no targeted guidance documents. It is suggested that China should learn from the experience of the United States and clearly encourage the research and application of patient preference in higher-level legal documents； develop specific guidance documents for the collection and application of patient preference information； determine the weight and form of patient preference information to be considered in regulatory decision-making according to national conditions.

关键词

Keywords

references

国家药品监督管理局药品审评中心 . 国家药监局药审中心关于发布《以患者为中心的药物临床试验设计技术指导原则（试行）》《以患者为中心的药物临床试验实施技术指导原则（试行）》《以患者为中心的药物获益-风险评估技术指导原则（试行）》的通告：2023年第44号 [EB/OL ] .（ 2023-07-27 ）[ 2024-03-03 ] . https：//www.cde.org.cn/main/news/viewInfoCommon/42c008e28f7004cd19b7394- 9142380bd https://www.cde.org.cn/main/news/viewInfoCommon/42c008e28f7004cd19b7394-9142380bd .

Center for Drug Evaluation，NMPA . Notice of the Center for Drug Evaluation of the National Medical Products Administration on the issuance of The Technical Guidelines for Patient-Centered Drug Clinical Trial Design （Trial）， The Technical Guidelines for Patient-Centered Drug Clinical Trial Implementation （Trial），and The Technical Guidelines for Patient-Centered Drug Benefit-Risk Assessment （ Trial ）：No. 44，2023 [EB/OL ] .（ 2023-07-27 ）[ 2024-03-03 ] . https：//www.cde.org.cn/main/news/viewInfoCommon/42c008e28f700-4cd19b73949142380bd https://www.cde.org.cn/main/news/viewInfoCommon/42c008e28f700-4cd19b73949142380bd .

FDA . Patient preference information：voluntary submission，review in premarket approval applications，humanitarian device exemption applications，and De Novo requests，and inclusion in decision summaries and device labeling：guidance for industry，Food and Drug Administration staff，and other stakeholders [EB/OL ] .（ 2016-08-24 ）[ 2024-03-03 ] . https：//www.fda.gov/media/92593/download?attachment https://www.fda.gov/media/92593/download?attachment .

吴晓蕾，史梦龙，张晨瑶，等 . 以患者为中心药物研发相关概念介绍 [J ] . 中国循证医学杂志， 2023 ， 23 （ 12 ）： 1472 - 1477 .

WU X L ， SHI M L ， ZHANG C Y ， et al . Introduction of patient-focused drug development-related concepts [J ] . Chin J Evid Based Med ， 2023 ， 23 （ 12 ）： 1472 - 1477 .

张渊 . 患者偏好与医患共同决策 [J ] . 协和医学杂志， 2019 ， 10 （ 6 ）： 679 - 684 .

ZHANG Y . Patient preference and shared decision making [J ] . Med J Peking Union Med Coll Hosp ， 2019 ， 10 （ 6 ）： 679 - 684 .

CHACHOUA L ， DABBOUS M ， FRANÇOIS C ， et al . Use of patient preference information in benefit-risk assessment，health technology assessment，and pricing and reimbursement decisions：a systematic literature review of attempts and initiatives [J ] . Front Med ， 2020 ， 7 ： 543046 .

HO M P ， GONZALEZ J M ， LERNER H P ， et al . Incorporating patient-preference evidence into regulatory decision making [J ] . Surg Endosc ， 2015 ， 29 （ 10 ）： 2984 - 2993 .

FDA . Division of patient-centered development [EB/OL ] .（ 2024-01-24 ）[ 2024-05-05 ] . https：//www.fda.gov/about-fda/center-devices-and-radiological-health/division-patient-centered-development https://www.fda.gov/about-fda/center-devices-and-radiological-health/division-patient-centered-development .

FDA . Center for Biologics Evaluation and Research patient engagement program [EB/OL ] .（ 2023-09-08 ）[ 2024-05-05 ] . https：//www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/center-biologics-eva-luation-and-research-patient-engagement-program https://www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/center-biologics-eva-luation-and-research-patient-engagement-program .

FDA . FDA patient engagement partnerships [EB/OL ] .（ 2022-05-16 ）[ 2024-05-05 ] . https：//www.fda.gov/patients/learn-about-fda-patient-engagement/fda-patient-engage-ment-partnerships https://www.fda.gov/patients/learn-about-fda-patient-engagement/fda-patient-engage-ment-partnerships .

FDA . CDER patient-focused drug development [EB/OL ] .（ 2024-02-14 ）[ 2024-03-14 ] . https：//www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development .

FDA . Center for biologics evaluation and research（CBER） 2021 - 2025 strategic plan[EB/OL ] .（ 2021-03-19 ）[ 2024-04-21 ] . https：//www.fda.gov/media/81152/download?attachment https://www.fda.gov/media/81152/download?attachment .

FDA . How patient preferences contribute to regulatory decisions for medical devices [EB/OL ] .（ 2017-09-25 ）[ 2024-03-20 ] . https：//wayback.archive-it.org/8521/20180724174-811/https：/blogs.fda.gov/fdavoice/index.php/2017/09/how-patient-preferences-contribute-to-regulatory-deci-sions-for-medical-devices/ https://wayback.archive-it.org/8521/20180724174-811/https:/blogs.fda.gov/fdavoice/index.php/2017/09/how-patient-preferences-contribute-to-regulatory-deci-sions-for-medical-devices/ .

FDA . Public law 114 - 255 ：Dec. 13 ， 2016 [EB/OL ] .（ 2016-12-13 ）[ 2024-04-21 ] . https：//www.congress.gov/114/plaws/publ255/PLAW-114publ255.pdf https://www.congress.gov/114/plaws/publ255/PLAW-114publ255.pdf .

FDA . Patient preference-sensitive areas：using patient preference information in medical device evaluation [EB/OL ] .（ 2020-09-29 ）[ 2024-06-14 ] . https：//www.fda.gov/about-fda/division-patient-centered-development/patient-preference-sensitive-areas-using-patient-preference-infor-mation-medical-device-evaluation https://www.fda.gov/about-fda/division-patient-centered-development/patient-preference-sensitive-areas-using-patient-preference-infor-mation-medical-device-evaluation .

FDA . FDA patient-focused drug development guidance series for enhancing the incorporation of the patient’s voice in medical product development and regulatory decision making [EB/OL ] .（ 2024-02-14 ）[ 2024-03-14 ] . https：//www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhan-cing-incorporation-patients-voice-medical https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhan-cing-incorporation-patients-voice-medical .

FDA . Factors to consider when making benefit-risk determinations in medical device premarket approval and De Novo classifications：guidance for industry and Food and Drug Administration staff [EB/OL ] .（ 2019-08-29 ）[ 2024-04-10 ] . https：//www.fda.gov/regulatory-information/search-fda-guidance-documents/factors-consider-when-making-benefit-risk-determinations-medical-device-pre-market-approval-and-de https://www.fda.gov/regulatory-information/search-fda-guidance-documents/factors-consider-when-making-benefit-risk-determinations-medical-device-pre-market-approval-and-de .

FDA . Patient preference information （PPI） in medical device decision making [EB/OL ] .（ 2023-03-07 ）[ 2024-04-24 ] . https：//www.fda.gov/about-fda/division-patient-centered-development/patient-preference-information-ppi-medical-device-decision-making https://www.fda.gov/about-fda/division-patient-centered-development/patient-preference-information-ppi-medical-device-decision-making .

WHICHELLO C ， BYWALL K S ， MAUER J ， et al . An overview of critical decision-points in the medical product lifecycle：where to include patient preference information in the decision-making process? [J ] . Health Policy ， 2020 ， 124 （ 12 ）： 1325 - 1332 .

EMA . CHMP & EUnetHTA parallel scientific advice：qualification of a framework and “points to consider” for method selection along with five methods for performing patient preference studies to inform regulatory and HTA body medical product decision-making [EB/OL ] .（ 2021-10-18 ）[ 2024-04-14 ] . https：//www.ema.europa.eu/en/documents/regulatory-procedural-guideline/chmp-eunethta-parallel-scientific-advice-qualification-framework-points-consider-method-selection-methods-performing-patient-preference-studies-inform-regulatory-htabody-medical-product-decision_en.pdf https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/chmp-eunethta-parallel-scientific-advice-qualification-framework-points-consider-method-selection-methods-performing-patient-preference-studies-inform-regulatory-htabody-medical-product-decision_en.pdf .

Medical Device Innovation Consortium . Patient centered outcomes research [EB/OL ] .（ 2019-10-28 ）[ 2024-04-08 ] . https：//mdic.org/project/patient-centered-outcomes-research https://mdic.org/project/patient-centered-outcomes-research .

BENZ H L ， CALDWELL B ， RUIZ J P ， et al . Patient-centered identification of meaningful regulatory endpoints for medical devices to treat Parkinson’s disease [J ] . MDM Policy Pract ， 2021 ， 6 （ 1 ）： 23814683211021380 .

JANSSENS R ， HUYS I ， VAN OVERBEEKE E ， et al . Opportunities and challenges for the inclusion of patient pre- ferences in the medical product life cycle：a systematic review [J ] . BMC Med Inform Decis Mak ， 2019 ， 19 （ 1 ）： 189 .

FDA . Patient experience in oncology [EB/OL ] .（ 2017-11-13 ）[ 2024-04-26 ] . https：//www.fda.gov/media/109731/download https://www.fda.gov/media/109731/download .

RUMMEL M ， KIM T M ， AVERSA F ， et al . Preference for subcutaneous or intravenous administration of rituximab among patients with untreated CD20 + diffuse large B-cell lymphoma or follicular lymphoma：results from a prospective，randomized，open-label，crossover study （PrefMab） [J ] . Ann Oncol ， 2017 ， 28 （ 4 ）： 836 - 842 .

中国医药创新促进会 . “以患者为中心”引领药物研发新方向：全球进展与趋势展望 [EB/OL ] .（ 2022-11-03 ）[ 2024-04-28 ] . http：//www.phirda.com/artilce_29455.html http://www.phirda.com/artilce_29455.html .

China Pharmaceutical Innovation and Research Development Association . “Patient-centered” leads the new direction of drug research and development：global progress and trend prospects [EB/OL ] .（ 2022-11-03 ）[ 2024-04-28 ] . http：//www.phirda.com/artilce_29455.html http://www.phirda.com/artilce_29455.html .

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