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Optimizing Patient-Reported Outcomes for HTA Success: Lessons from German Early Benefit Assessments
Kristi A Bertzos1, Nicole Alt2, Peter C Coyle3
1Johnson and Johnson, 850 Ridgeview Drive, Horsham, PA, 19044, USA. kbertzos@its.jnj.com.
Advances in Therapy
|July 20, 2026
Summary
Optimizing patient-reported outcomes (PROs) in clinical trials is crucial for health technology assessment (HTA) acceptance. Methodological and operational design issues, not lack of relevance, often lead to PRO rejection in HTA processes.
Area of Science:
- Health Technology Assessment (HTA)
- Patient-Centered Outcomes Research (PCOR)
- Clinical Trial Design
Background:
- Patient-reported outcomes (PROs) are increasingly vital in health technology assessment (HTA), reflecting a shift towards patient-centered evaluations.
- Germany's HTA process, involving the Institute for Quality and Efficiency in Health Care (IQWiG) and the Federal Joint Committee (G-BA), prioritizes patient-relevant endpoints.
- Limited guidance exists on optimally designing and positioning PROs in clinical trials to meet HTA requirements.
Purpose of the Study:
- To review German HTA reports and identify methodological expectations and guidance for PRO evidence generation.
- To analyze common critiques and themes related to PRO design, conduct, and analysis within HTA.
- To understand factors influencing the acceptance or rejection of PRO data in HTA.
Main Methods:
- A targeted review of 249 German benefit assessments from January 2019 to August 2024.
- Inclusion of studies in oncology, cardiovascular, immunology/dermatology, and neurology.
- Quantitative and qualitative analyses of HTA feedback, critiques, and recurring themes on PROs.
Main Results:
- Common PRO critiques included low response rates (<70%), failure to apply the 15% scale-span responder definition, open-label designs, and statistical analysis issues.
- PRO rejection was most strongly associated with inadequate instrument validation, low/imbalanced response rates, differing observation periods, and incomplete follow-up.
- The frequency of a critique did not always correlate with PRO data rejection.
Conclusions:
- Early optimization of PRO selection, trial design, and analysis can enhance PRO acceptance in HTA.
- Nonacceptance of PRO results stemmed primarily from methodological and operational design misalignment, not a lack of patient relevance.
- While early strategy can address many factors, operational challenges like data completeness remain difficult to mitigate.
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