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 understand the impact and challenges associated with using PRO data in HTA. 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. 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 KOLs 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. 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. For reimbursement of new therapies, health technology assessment (HTA) bodies need to understand the impact of treatment on patients’ quality of life, symptoms, and daily functioning. This information comes from patient-reported outcomes (PRO) data. However, requirements for collecting and reporting this information for HTA processes can differ between regions. This lack of consistent guidelines creates uncertainty when developing new treatments and may delay patient access to new therapies. Published guidance about PRO requirements for HTA from England, France, and Germany was reviewed. Semi-structured qualitative interviews were conducted with 12 experts with HTA- and PRO-related expertise in these countries. They were asked about current practices and challenges with using PRO data in HTA, and opportunities to make the requirements more consistent across countries. The availability of guidance varies between England, France, and Germany. German experts reported that PRO data had a high impact on HTA decisions, whereas English and French experts reported more mixed impact, depending on factors such as the therapy area. Experts cited challenges related to methodological considerations, such as measurement tools that don’t capture what’s important to patients, and inconsistencies between European-level guidance and country requirements. European health systems need clearer and more consistent requirements for using PRO data. This would improve understanding of HTA expectations when planning for HTA submissions, ensure patient voices are heard in HTA decisions, and may enable patient access to new treatments. The new European Joint Clinical Assessment process could help achieve this harmonization if country-specific PRO guidance is available and consistent.
Dimodica et al. (Fri,) studied this question.