Requirements for Patient-Reported Outcomes and Data Analytics in Health Technology Assessment in England, France and Germany, and the Need for Methods Harmonization Across European Markets: A Qualitative Interview Study.

OBJECTIVES: To explore methods for collection, reporting and analysis of patient-reported outcome (PRO) data in health technology assessment (HTA) in England, France, and Germany, and the impact of and challenges with using PRO data in HTA. METHODS: A targeted literature review (TLR) was conducted in November 2023 to identify PRO-related data specifications for HTA in European markets and inform an interview discussion guide. Qualitative, semi-structured 60-minute interviews with key opinion leaders (KOLs) with HTA- and PRO-related expertise in England, France and Germany were conducted to gain expert perspectives on PRO and HTA practices, and to identify opportunities for HTA harmonization across European markets. The interview transcripts were analyzed using content analysis methods and key results extracted by a single analyst into Microsoft ^® Excel. RESULTS: KOLs from England (n=4), France (n=3) and Germany (n=5) were interviewed. Availability of guidance on PRO data collection, analysis and reporting in HTA varies between the HTA bodies included. Guidance, where available, is country specific, and therefore lacks harmonization across included markets. In terms of the perceived impact of PRO data on HTA decision making, KOLs from Germany gave a high rating, while KOLs from England and France gave ratings of low to high impact and moderate to high impact, respectively. It was reported by interviewees in all markets that PROs are expected for submissions in highly symptomatic and burdensome conditions, and that the relative importance of PRO to HTA outcomes varies by disease. KOLs cited challenges with using PRO data in HTA. Commonly cited challenges were related to methodological considerations and included: ‘no/suboptimal PRO data collection/submission (e.g., due to value perceptions)’ and ‘selected instruments not fit-for-purpose/PRO benefits not adequately represented in models’. Adding to the challenges with using PRO data in HTA, there are inconsistencies between Joint Clinical Assessment (JCA) guidance and PRO data requirements for HTA in included markets. CONCLUSIONS: There is a lack of transparency and harmonization in terms of the requirements for PRO data collection, analysis and reporting for HTA in England, Germany and France. Enhanced transparency of HTA requirements will facilitate harmonization efforts and may be supported by JCA.