Requirements for Patient-Reported Outcome and Data Analytics in Health Technology Assessment in England, France and Germany, and Opportunities for Methods Harmonization across European Markets
Author(s)
On Yee Wong, MSc1, Laurie DiModica, PhD1, Sarah Knight, MSc1, Melanie Calvert, BSc, PhD2, Olalekan Lee Aiyegbusi, PhD2, Saeid SHAHRAZ, MD, PhD3, Yanni Hao, PhD4, Denise Globe, PhD4.
1Clarivate, London, United Kingdom, 2University of Birmingham, London, United Kingdom, 3DIRECTOR-HEOR, Gilead Sciences, Foster City, CA, USA, 4Gilead Sciences, Foster City, CA, USA.
1Clarivate, London, United Kingdom, 2University of Birmingham, London, United Kingdom, 3DIRECTOR-HEOR, Gilead Sciences, Foster City, CA, USA, 4Gilead Sciences, Foster City, CA, USA.
OBJECTIVES: To compare methods for collection, reporting, and analysis of patient-reported outcome (PRO) data in health technology assessment (HTA) in France, Germany, and England and identify opportunities for HTA harmonization across European markets.
METHODS: Interviews with key opinion leaders (KOLs) with HTA- and PRO-related expertise in Europe were conducted to gather feedback on PRO data collection, analysis, and reporting preferences in HTA.
RESULTS: KOLs from England (n=4), France (n=3), and Germany (n=5) were interviewed. All KOLs from Germany gave the highest rating regarding their perceived impact of PRO data on HTA decision-making. KOLs highlighted that PROs are particularly impactful in highly symptomatic and burdensome conditions. The availability of PRO data collection, analysis, and reporting guidance in HTA varies among HTAs. Most guidance, where available, is country-specific and, therefore, lacks harmonization across markets. HTAs in included markets require validated instruments to collect PRO data. Preferred instruments for HTA are specified in England (EQ-5D-3L) and France (EQ-5D-5L). There is a <30% allowance for missing data in HTA in Germany. Transparency is lacking for the missing data threshold accepted in England and France. Minimal clinically important differences (MCIDs) for PROs are specified in Germany (≥15% of the PRO scale range), while other markets have non-standardized MCIDs reflecting disease-specific expectations. Post-progression data collection is expected for oncology submissions in Germany and England.KOLs reported that cross-market PRO standards could be supported by Joint European HTA regulation, but uncertainties and challenges remain.
CONCLUSIONS: There is a lack of transparency and harmonization regarding the requirements for PRO data collection, analysis, and reporting for HTA in key European markets. Enhanced transparency of HTA requirements will help harmonization efforts and may be supported by Joint Clinical Assessment.
METHODS: Interviews with key opinion leaders (KOLs) with HTA- and PRO-related expertise in Europe were conducted to gather feedback on PRO data collection, analysis, and reporting preferences in HTA.
RESULTS: KOLs from England (n=4), France (n=3), and Germany (n=5) were interviewed. All KOLs from Germany gave the highest rating regarding their perceived impact of PRO data on HTA decision-making. KOLs highlighted that PROs are particularly impactful in highly symptomatic and burdensome conditions. The availability of PRO data collection, analysis, and reporting guidance in HTA varies among HTAs. Most guidance, where available, is country-specific and, therefore, lacks harmonization across markets. HTAs in included markets require validated instruments to collect PRO data. Preferred instruments for HTA are specified in England (EQ-5D-3L) and France (EQ-5D-5L). There is a <30% allowance for missing data in HTA in Germany. Transparency is lacking for the missing data threshold accepted in England and France. Minimal clinically important differences (MCIDs) for PROs are specified in Germany (≥15% of the PRO scale range), while other markets have non-standardized MCIDs reflecting disease-specific expectations. Post-progression data collection is expected for oncology submissions in Germany and England.KOLs reported that cross-market PRO standards could be supported by Joint European HTA regulation, but uncertainties and challenges remain.
CONCLUSIONS: There is a lack of transparency and harmonization regarding the requirements for PRO data collection, analysis, and reporting for HTA in key European markets. Enhanced transparency of HTA requirements will help harmonization efforts and may be supported by Joint Clinical Assessment.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
P8
Topic
Patient-Centered Research
Disease
No Additional Disease & Conditions/Specialized Treatment Areas