Frequency and Impact of External Comparator Arms in Oncology HTA Assessments With Single-Arm Trials in the UK, France, and Germany From 2014 to 2024
Author(s)
Mark Chalmers, PhD1, PRATEEK KANADE, M. Pharm2, Vidhyasagari Sundaram, MPH2, Ravina Mathur, M. Pharm2, Rory Dunne, MPH3.
1Partner, EY, Dublin, Ireland, 2Ernst & Young, Bangalore, India, 3Ernst & Young, Dublin, Ireland.
1Partner, EY, Dublin, Ireland, 2Ernst & Young, Bangalore, India, 3Ernst & Young, Dublin, Ireland.
OBJECTIVES: To evaluate the frequency of External Comparator Arms (ECA) in Health Technology Assessment (HTA) outcomes for oncology therapies supported by Single-Arm Trials (SATs) in the UK, France and Germany from 2014 to 2024, and to assess their impact on the HTA outcome.
METHODS: A list of all oncology approved drugs was obtained from the European Medicines Agency (EMA) website from 2014 to 2024. From this list, drugs approved based on SATs were shortlisted. HTA assessments for these drugs were then obtained from the website of NICE (UK), HAS (France), and G-BA (Germany). Based on these assessments, those that included an ECA were identified for further analysis to determine the frequency and types of ECA data sources, their influence on HTA outcomes, cross-agency variability, and trends over time.
RESULTS: Gaining traction in HTA acceptance since 2020, ECAs supported positive HTA recommendations for ~10% of SAT-based oncology therapies in the UK (1%), Germany (4%), and France (6%) from 2014-2024. This highlights their potential for favorable HTA outcomes where randomization is not feasible, with France showing the highest acceptance, followed by Germany and the UK.
CONCLUSIONS: ECAs are increasingly seen as a necessary tool for evidence generation but the HTA agencies approach them with caution and high scrutiny, demanding rigorous methodology, transparency, and robust analyses to address the inherent biases associated with non-randomized evidence such as data quality and comparability issues, and methodological/ statistical limitations. To enhance consistency and predictability in future assessments, clearer guidance and standardized methodologies for ECA design and validation are needed.
METHODS: A list of all oncology approved drugs was obtained from the European Medicines Agency (EMA) website from 2014 to 2024. From this list, drugs approved based on SATs were shortlisted. HTA assessments for these drugs were then obtained from the website of NICE (UK), HAS (France), and G-BA (Germany). Based on these assessments, those that included an ECA were identified for further analysis to determine the frequency and types of ECA data sources, their influence on HTA outcomes, cross-agency variability, and trends over time.
RESULTS: Gaining traction in HTA acceptance since 2020, ECAs supported positive HTA recommendations for ~10% of SAT-based oncology therapies in the UK (1%), Germany (4%), and France (6%) from 2014-2024. This highlights their potential for favorable HTA outcomes where randomization is not feasible, with France showing the highest acceptance, followed by Germany and the UK.
CONCLUSIONS: ECAs are increasingly seen as a necessary tool for evidence generation but the HTA agencies approach them with caution and high scrutiny, demanding rigorous methodology, transparency, and robust analyses to address the inherent biases associated with non-randomized evidence such as data quality and comparability issues, and methodological/ statistical limitations. To enhance consistency and predictability in future assessments, clearer guidance and standardized methodologies for ECA design and validation are needed.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PT2
Topic
Clinical Outcomes, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Oncology