Mitigation of Immature and Variable Survival Data In Oncology With Additional Endpoints During Health Technology Assessment in Germany and France
Author(s)
Isabelle Petit, PharmD1, Nicolas Plommet, MSc2, Henry Calver, MSc3, Martina Sattlecker, MSc4, Jonathan Morris, MD5, Balazs Vamosi, MSc6.
1Debiopharm International SA, Lausanne, Switzerland, 2Senior Manager, Global Value & Access, Debiopharm International SA, Lausanne, Switzerland, 3IQVIA, London, United Kingdom, 4IQVIA, Bromley, United Kingdom, 5IQVIA, Devon, PA, USA, 6IQVIA, Basel, Switzerland.
1Debiopharm International SA, Lausanne, Switzerland, 2Senior Manager, Global Value & Access, Debiopharm International SA, Lausanne, Switzerland, 3IQVIA, London, United Kingdom, 4IQVIA, Bromley, United Kingdom, 5IQVIA, Devon, PA, USA, 6IQVIA, Basel, Switzerland.
OBJECTIVES: In oncology, Overall Survival (OS) remains the gold standard for assessing clinical benefit. However, in the context of hard-to-treat cancers and rapid therapeutic innovation, OS data are often immature or variable, complicating benefit appraisals. This study aimed to explore the influence of complementary endpoints on HTA Outcomes alongside OS data.
METHODS: Univariate analysis considered all drugs that underwent HTA assessments from the G-BA and HAS between January 2014 and October 2024 in Lung, Breast and Prostate cancer, captured in the IQVIA Market Access Insights database. HTA outcomes were classified, scored and analysed in relation with the magnitude of OS Hazard Ratio, Progression-Free Survival (PFS), safety and Health-Related Quality of Life (HR-QoL).
RESULTS: In Germany and France submissions with an OS HR ≥ 0.7 had a 50% rate achieving a positive HTA outcome. In France, drugs achieving an OS HR < 0.75 and PFS < 0.55 obtained an ASMR I-III rating in 73% of the cases. In Germany, drugs achieving an OS HR < 0.75 with a favourable safety profile achieved in 86% of the times a considerable / major benefit rating while the probability decreases to 36% when the OS HR ≥ 0.75. Meanwhile, submissions with a favourable safety profile in France had 50% of obtaining ASMR I-III when OS HR < 0.75; there were no examples found in database with ASMR I-III when OS HR ≥ 0.75. When pairing OS (HR < 0.75) with positive and accepted QoL data, 78% of drugs achieve a considerable / major benefit rating in Germany compared to 38% when the OS HR is ≥ 0.75.
CONCLUSIONS: OS data can be mitigated with additional endpoints to optimize HTA outcome. Additional endpoints should be anticipated from early-on to ensure robust conclusions during benefit appraisals. Nevertheless, the magnitude of OS remains key to maximize HTA outcomes.
METHODS: Univariate analysis considered all drugs that underwent HTA assessments from the G-BA and HAS between January 2014 and October 2024 in Lung, Breast and Prostate cancer, captured in the IQVIA Market Access Insights database. HTA outcomes were classified, scored and analysed in relation with the magnitude of OS Hazard Ratio, Progression-Free Survival (PFS), safety and Health-Related Quality of Life (HR-QoL).
RESULTS: In Germany and France submissions with an OS HR ≥ 0.7 had a 50% rate achieving a positive HTA outcome. In France, drugs achieving an OS HR < 0.75 and PFS < 0.55 obtained an ASMR I-III rating in 73% of the cases. In Germany, drugs achieving an OS HR < 0.75 with a favourable safety profile achieved in 86% of the times a considerable / major benefit rating while the probability decreases to 36% when the OS HR ≥ 0.75. Meanwhile, submissions with a favourable safety profile in France had 50% of obtaining ASMR I-III when OS HR < 0.75; there were no examples found in database with ASMR I-III when OS HR ≥ 0.75. When pairing OS (HR < 0.75) with positive and accepted QoL data, 78% of drugs achieve a considerable / major benefit rating in Germany compared to 38% when the OS HR is ≥ 0.75.
CONCLUSIONS: OS data can be mitigated with additional endpoints to optimize HTA outcome. Additional endpoints should be anticipated from early-on to ensure robust conclusions during benefit appraisals. Nevertheless, the magnitude of OS remains key to maximize HTA outcomes.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA236
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Oncology