CAPTURING THE PATIENT PERSPECTIVE: TARGETED LITERATURE REVIEW FINDINGS ON THE CRITICAL ROLE OF PROS IN ONCOLOGIC AMNOG EVALUATIONS
Author(s)
Thomas Brade, Dr.1, Alexandra Carls, Dr.2, Isabel Sohn-Frank, Dr.2, Kathrin Bogner, Dr.2, Alexandra Lauer, PhD3, Maarten Voorhaar, MASc4.
1AMS Advanced Medical Services GmbH, München, Germany, 2AMS Advanced Medical Services GmbH, Mannheim, Germany, 3Boehringer Ingelheim Pharma GmbH & Co. KG, Ingelheim am Rhein, Germany, 4Boehringer Ingelheim International GmbH, Ingelheim, Germany.
1AMS Advanced Medical Services GmbH, München, Germany, 2AMS Advanced Medical Services GmbH, Mannheim, Germany, 3Boehringer Ingelheim Pharma GmbH & Co. KG, Ingelheim am Rhein, Germany, 4Boehringer Ingelheim International GmbH, Ingelheim, Germany.
OBJECTIVES: This study assessed use and acceptance of patient-reported outcome (PRO) data in oncology benefit assessments under the German AMNOG framework. Objective was to identify characteristics of PRO strategies associated with successful acceptance in German benefit assessment.
METHODS: In this exploratory targeted literature review we identified all oncology AMNOG procedures completed between January 01, 2021 and November 15, 2025, through the Federal Joint Committee (G-BA) website. All procedures with comments on PRO data were included. Extraction of relevant data into a structured database, included basic assessment characteristics, PRO instruments, endpoints and operationalization, contribution to additional benefit, and corresponding comments from G-BA and Institute for Quality and Efficiency in Health Care (IQWiG).
RESULTS: Overall 254 completed oncology AMNOG procedures were identified; 100 reported PRO endpoints and underwent evaluation by G-BA/IQWiG. Among these, 29 procedures demonstrated an additional benefit based on PRO evidence. Additional benefit was established through PRO morbidity outcomes in 12 procedures and through quality-of-life (QoL) outcomes in 4 procedures, while 13 procedures demonstrated additional benefit based on morbidity and QoL outcomes.
CONCLUSIONS: Alongside overall survival, PRO based morbidity and QoL outcomes are principally accepted patient-relevant efficacy endpoints and are critical components of evidence packages for therapies undergoing G-BA/IQWiG evaluation. Long-term PRO assessment, supported by balanced data collection across treatment arms (<15% imbalance) and high questionnaire completion rates (≥70%), provides a strong foundation for demonstrating additional benefit in Germany. Long-term PRO evidence acceptance is influenced by combined effects of high assessment frequency (e.g. every visit) and duration (minimum until early survival follow up), data completeness in both treatment arms over time, analytical methodology, statistical reporting, and effective mitigation of potential bias.
METHODS: In this exploratory targeted literature review we identified all oncology AMNOG procedures completed between January 01, 2021 and November 15, 2025, through the Federal Joint Committee (G-BA) website. All procedures with comments on PRO data were included. Extraction of relevant data into a structured database, included basic assessment characteristics, PRO instruments, endpoints and operationalization, contribution to additional benefit, and corresponding comments from G-BA and Institute for Quality and Efficiency in Health Care (IQWiG).
RESULTS: Overall 254 completed oncology AMNOG procedures were identified; 100 reported PRO endpoints and underwent evaluation by G-BA/IQWiG. Among these, 29 procedures demonstrated an additional benefit based on PRO evidence. Additional benefit was established through PRO morbidity outcomes in 12 procedures and through quality-of-life (QoL) outcomes in 4 procedures, while 13 procedures demonstrated additional benefit based on morbidity and QoL outcomes.
CONCLUSIONS: Alongside overall survival, PRO based morbidity and QoL outcomes are principally accepted patient-relevant efficacy endpoints and are critical components of evidence packages for therapies undergoing G-BA/IQWiG evaluation. Long-term PRO assessment, supported by balanced data collection across treatment arms (<15% imbalance) and high questionnaire completion rates (≥70%), provides a strong foundation for demonstrating additional benefit in Germany. Long-term PRO evidence acceptance is influenced by combined effects of high assessment frequency (e.g. every visit) and duration (minimum until early survival follow up), data completeness in both treatment arms over time, analytical methodology, statistical reporting, and effective mitigation of potential bias.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA256
Topic
Clinical Outcomes, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Oncology