患者体验数据作为决策级证据：来自EMA反思文件的监管策略见解

Patient Experience Data asDecision Grade Evidence:Regulatory Strategy Insightsfrom EMA’s Reflection Paper BARBARA MILITZER, MSHI, RAC, Head of Regulatory Strategy, Patient Centered Solutions,Strategic & Scientific Research, IQVIA Table of contents Executive summary1FDA’s Patient-Focused Drug Development (PFDD) framework2Health Technology Assessment (HTA) and the EU HTA regulation3Joint Clinical Assessment (JCA) under the EU HTA regulation3A practical framework for PED planning5References7About the author8 External-facing note: This Insight Brief reflects IQVIA’s perspective on publicly available frameworks and consultationdocuments. It is intended for informational purposes and does not constitute legal or regulatory advice. Regulatory signals andoutstanding questions Executive summary The European Medicines Agency’s (EMA’s) draftReflection Paper on Patient Experience Data (PED) (Ref.EMA/CHMP/PRAC/148869/2025) was published for publicconsultation on 29 September 2025, with commentsaccepted through 31 January 2026. As a reflection paperissued for consultation, it does not establish bindingrequirements, but it does signal that patient experienceevidence is expected to be planned and submitted asdecision-relevant information in the European Union’smedicines development. From a regulatory strategystandpoint, the opportunity now is to translate thesehigh-level principles into clearer expectations thatexplicitly connect PED to regulatory decision points,use consistent scope and terminology, including thefull Clinical Outcome Assessment (COA) taxonomy, andsupport more proactive and fit-for-purpose evidenceplanning while enabling greater consistency in howPED is framed across regulatory, Health TechnologyAssessment (HTA), and access-related evidencecontexts. Over time, stronger evidence planning anddocumentation could also improve the reusability of PEDpackages across major markets, including Europe, theUnited States, Japan, and China, while recognizing thatjurisdiction-specific adaptation will still be required. The EMA’s paper encourages early dialogue withregulators on how PED will be generated, analyzed,and submitted. It recognizes multiple forms of PED,including COAs, qualitative research, patient preferencestudies, and patient-generated digital data collected inclinical trials or in real-world research settings. At thesame time, the paper leaves key questions open thatdirectly affect evidence planning: how PED should beframed for specific regulatory decisions (beyond generalencouragement), whether PED should be interpretednarrowly as Patient-Reported Outcomes (PROs) ormore broadly to encompass other COA types, and whatminimum fit-for-purpose expectations apply acrossdifferent PED types and development stages. Making PED decision-ready For PED to consistently influence regulatory assessment,it must be anchored to the decision context. In practice,this means clarifying how PED informs:1selectionand prioritization of patient-relevant endpoints;2interpretation of meaningful change and difference(including how thresholds are defined and justified);3benefit–risk assessment, including tolerability andpreference-sensitive trade-offs; and4where appropriate,communication in product information. Clear linkagebetween PED and these decision points improvespredictability for sponsors and helps reviewers evaluaterelevance and rigor proportionately. Why this matters now Two forces are converging. First, regulators areincreasingly placing greater emphasis on systematicapproaches for collecting and using patient input toinform product development and regulatory decision-making. Second, Europe’s evidence environmentis becoming more integrated, with Joint ClinicalAssessments (JCAs) under the HTA regulation increasingthe need for coordinated evidence narratives alongsidemarketing authorization. Together, these trends increasethe value of PED that is fit-for-purpose, interpretable,and reusable across jurisdictions. A second implication is the need for terminologicalprecision. PED should not be interpreted as synonymouswith PROs alone. A regulatory grade approach shouldreflect the full COA taxonomy — PROs, Observer-Reported Outcomes (ObsROs), Clinician-ReportedOutcomes (ClinROs), and Performance Outcomes(PerfOs) — so evidence strategies remain inclusive ofpopulations that cannot self report and reflect conceptsthat matter to patients, including when outcome-relevant data are collected passively through DHTs. should be generated, organized, and reviewed. Althoughthese pathways do not yet reflect full convergence,together they underscore the value of more coordinatedevidence planning across development programs.They also provide a foundation for evidence packagesthat may be more readily leveraged in other importantmarkets, including Japan and China, where interest inpatient-centered evidence and more structured use ofsuch data is continuing to evolve. Advancing convergence in PEDand COA strategy Global alignment is in