Matthias Hunger, Céline Taveau, and Ankit Pahwa present the challenges and opportunities when using external control arm studies in submissions to HTA agencies.
Randomized controlled trials (RCTs) are considered the gold standard to determine the efficacy of a new therapy or to evaluate its comparative effectiveness against standard of care. However, regulators acknowledge situations when a RCT may not be operationally or ethically feasible, such as in rare disease patient populations. In those cases, data from single arm trials complemented by external control arms are becoming more commonly accepted as evidence by both regulatory and Health Technology Assessment (HTA) bodies.
Due to the lack of randomization, submissions based on external control arm studies should apply rigorous analytical methods to adjust for potential confounding caused by imbalances between treatment groups. While regulatory bodies have published guidance on the use of non-randomized evidence, recommendations from HTA agencies have long been limited.
Key Topics Include:
- Current guidance regarding the use of external control arms from selected HTA agencies in Europe, including Germany and France
- Analysis and comparison of critical assessments made by HTA agencies on a number of recent submissions using external control arm trials
- Illustration and comparison of statistical methods to adjust for imbalances in key prognostic variables between treatment groups
- Summarization of recommendations and critical assessments from selected HTA agencies on statistical methods used in external control arm submissions
Who Should Attend?
This program will be beneficial for pharmaceutical, biotech, and medical device professionals involved in:
- Rare diseases
- Health technology assessment
- Health economics & outcomes research
- Market access, pricing and reimbursement
- Real world evidence
Global Health Economics, Outcomes Research and Epidemiology
Global Health Economics and Outcomes Research
Real World Evidence Strategy and Analytics