Statistical Analysis of Health Technology Assessment Outcomes in Oncology: An Analysis of Key Clinical Value Drivers in Germany and France
Author(s)
Isabelle Petit, PharmD1, Nicolas Plommet, MS2, 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 Germany and France, new innovative medicines are subject to a Health Technology Assessment (HTA), followed by price negotiation. This study aimed to identify key clinical value drivers in oncology in breast, prostate, and lung cancers that influence HTA outcomes. By uncovering consistent patterns across indications, the research seeks to inform future clinical trial design and improve the HTA outcomes for Health Technology Developers (HTD).
METHODS: A multivariate analysis considering all drugs that underwent HTA from the G-BA and HAS between January 2014 and October 2024 in Lung, Breast and Prostate cancer, as captured in the IQVIA Market Access Insights database. HTA outcomes were classified, scored and analysed in relation to PICO (Population, Intervention, Comparator, Outcome) variables to fit a multinomial regression model. The model performance was assessed to establish the validity of the drivers for predicting HTA outcomes.
RESULTS: Two models were developed. The models demonstrated good performance (sensitivity and specificity). In Germany (n=216), three out of the eight variables were statistically significant (p-value<0.05): Overall survival (OS), favorable safety profile and favorable Health-Related Quality of Life profile. Trial design with an active comparator had positive coefficient (active > placebo) but was not significant. In France (n=145), two out of the eight variables were statistically significant (p-value<0.05): Overall survival (OS) and Progression Free Survival (PFS). Favorable safety profiles tend to have a positive impact but the effect was not statistically significant.
CONCLUSIONS: In Germany, OS is the preferred endpoint for the HTA in oncology while in France positive PFS could also help to achieve positive benefit assessment. In Germany a statistically significant favourable safety profile and/or Health-related Quality of Life may also influence in a smaller proportion positively HTA outcome. In France, significant favourable safety profile was also found to have a positive influence on HTA outcome.
METHODS: A multivariate analysis considering all drugs that underwent HTA from the G-BA and HAS between January 2014 and October 2024 in Lung, Breast and Prostate cancer, as captured in the IQVIA Market Access Insights database. HTA outcomes were classified, scored and analysed in relation to PICO (Population, Intervention, Comparator, Outcome) variables to fit a multinomial regression model. The model performance was assessed to establish the validity of the drivers for predicting HTA outcomes.
RESULTS: Two models were developed. The models demonstrated good performance (sensitivity and specificity). In Germany (n=216), three out of the eight variables were statistically significant (p-value<0.05): Overall survival (OS), favorable safety profile and favorable Health-Related Quality of Life profile. Trial design with an active comparator had positive coefficient (active > placebo) but was not significant. In France (n=145), two out of the eight variables were statistically significant (p-value<0.05): Overall survival (OS) and Progression Free Survival (PFS). Favorable safety profiles tend to have a positive impact but the effect was not statistically significant.
CONCLUSIONS: In Germany, OS is the preferred endpoint for the HTA in oncology while in France positive PFS could also help to achieve positive benefit assessment. In Germany a statistically significant favourable safety profile and/or Health-related Quality of Life may also influence in a smaller proportion positively HTA outcome. In France, significant favourable safety profile was also found to have a positive influence on HTA outcome.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA302
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Oncology