Webinar: Using external control arm studies in HTA submissions

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 studies complemented by external control arms are becoming more commonly accepted as evidence by both regulatory and 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.

Join us to learn about the challenges and opportunities when using external control arm studies in submissions to HTA agencies. In this webinar, we will:

• Describe current guidance regarding the use of external control arms from selected HTA agencies in Europe, including, for example, Germany and France
• Analyze and compare critical assessments made by HTA agencies on a number of recent submissions using external control arm trials
• Illustrate and compare statistical methods to adjust for imbalances in key prognostic variables between treatment groups
• Summarize recommendations and critical assessments from selected HTA agencies on statistical methods used in external control arm submissions

Audience

This program will be beneficial for pharmaceutical, medical device, and biotechnology professionals working in the following areas:

• Rare disease
• Epidemiology
• Health technology assessment
• Health economics and outcomes research
• Marketing
• Market access, pricing and reimbursement
• Statistics
• Real world evidence