Maximizing Acceptance of Patient-Reported Outcome Data in German HTA Decision Making: Results From a Horizon Scan
Author(s)
Kristi Bertzos, PhD1, Nicole Alt, MSc2, Peter Coyle, PhD, DPT, PT3, Katharine Gries, PharmD, PhD3, Anke Kummer, PhD2, Anna Lieb, MS2, Sophie Thiemann, PhD2, Daniel Wirth, PhD2, Shivani Shah, N/A4, Chad Gibson, N/A5, Markus Frosien, MSc2.
1Director, Patient-Reported Outcomes, Johnson and Johnson, Horsham, PA, USA, 2Johnson & Johnson, Neuss, Germany, 3Johnson & Johnson, Raritan, NJ, USA, 4Eversana, Mumbai, India, 5EVERSANA, Raleigh, NC, USA.
1Director, Patient-Reported Outcomes, Johnson and Johnson, Horsham, PA, USA, 2Johnson & Johnson, Neuss, Germany, 3Johnson & Johnson, Raritan, NJ, USA, 4Eversana, Mumbai, India, 5EVERSANA, Raleigh, NC, USA.
OBJECTIVES: To demonstrate clinical effectiveness in the German benefit assessment, clinical trial endpoints must be patient relevant. Therefore, patient-reported outcome (PRO) data can be a key driver for added benefit assessment in the morbidity and quality of life outcome categories. The objective of this research was to review recent IQWiG assessments/G-BA decisions to develop best practice recommendations for inclusion of PROs in clinical studies that would maximize success in G-BA benefit assessment.
METHODS: A systematic search of G-BA decisions from 2018-2024 was conducted using the NAVLIN database and G-BA/IQWiG websites. Dossiers, assessments, and decision documents were reviewed for submissions that included PRO data in the areas of oncology, cardiovascular medicine, immunology, and neurology. Key product details, PRO information (PRO measures implemented, PRO administration schedule, PRO completion rates, etc.), and G-BA outcomes were extracted and summarized. Six case studies were also selected for in-depth analysis to further understand nuances of G-BA decision-making relative to PRO design strategies (3) and use of flexible PRO instruments such as item libraries (3).
RESULTS: 281 G-BA decisions including PRO data were reviewed (76.3% oncology). PRO considerations during the review process focused on adequacy or PRO measures, frequency and timing of PRO measure collection, length of follow-up, and data quality and completeness. Aside from study design elements (e.g., single arm trial), common PRO criticisms were PRO completion rates below 70% / missing data, lack of validation of PRO instruments and scores, inappropriate analysis conducted, and too short or incongruent observation periods across treatment arms. Case studies provided detailed insights into overall design considerations and PRO measure selection associated with a positive benefit assessment.
CONCLUSIONS: Analysis of recent G-BA decisions can serve to develop a framework for the PRO evidence needed to obtain an added benefit rating in Germany and propose actions that would maximize HTA success.
METHODS: A systematic search of G-BA decisions from 2018-2024 was conducted using the NAVLIN database and G-BA/IQWiG websites. Dossiers, assessments, and decision documents were reviewed for submissions that included PRO data in the areas of oncology, cardiovascular medicine, immunology, and neurology. Key product details, PRO information (PRO measures implemented, PRO administration schedule, PRO completion rates, etc.), and G-BA outcomes were extracted and summarized. Six case studies were also selected for in-depth analysis to further understand nuances of G-BA decision-making relative to PRO design strategies (3) and use of flexible PRO instruments such as item libraries (3).
RESULTS: 281 G-BA decisions including PRO data were reviewed (76.3% oncology). PRO considerations during the review process focused on adequacy or PRO measures, frequency and timing of PRO measure collection, length of follow-up, and data quality and completeness. Aside from study design elements (e.g., single arm trial), common PRO criticisms were PRO completion rates below 70% / missing data, lack of validation of PRO instruments and scores, inappropriate analysis conducted, and too short or incongruent observation periods across treatment arms. Case studies provided detailed insights into overall design considerations and PRO measure selection associated with a positive benefit assessment.
CONCLUSIONS: Analysis of recent G-BA decisions can serve to develop a framework for the PRO evidence needed to obtain an added benefit rating in Germany and propose actions that would maximize HTA success.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA229
Topic
Clinical Outcomes, Health Technology Assessment, Patient-Centered Research
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas