PURPOSE: External control arms (ECAs) created from real-world secondary data sources are increasingly used for rare diseases to support applications to regulatory and health-technology assessment (HTA) agencies. The acceptance of ECA evidence by these agencies will depend heavily on the availability of a well-characterized external cohort and an appropriate justification of the sources and methods used.

In this workshop we will guide the audience through planning considerations for the creation of ECAs for regulatory and reimbursement purposes. Speakers will use real-time polling upfront to get to know the audience (5 min.), will ask each other questions during the presentations and will conclude with an interactive audience discussion (15 min.) to understand the audience’s opinions and experiences.

DESCRIPTION: Carla Vossen will provide a high-level overview of available ECA guidance, examples of ECAs used in regulatory and HTA submissions for rare diseases and the impact of EU Joint Clinical Assessment (10 min.). Raymond Huml will then present how data from different sources can be used for creating ECAs for clinical trials for novel muscular dystrophy treatments, what challenges may be faced and what solutions can be implemented (10 min.). Tracy Mayne will continue the discussion by presenting his recent experience with ECAs created from real-world claims and patient registry data to support regulatory submissions for primary biliary cholangitis (10 min.). Specific topics that Tracy Mayne will address are differences between EMA and FDA requirements (e.g., endpoint definitions, data sources, etc.) and key lessons learned. Lastly, Dawn Lee will discuss how to plan for implementing ECAs created from secondary real-world data sources in economic models for rare cancer indications, how to assess data quality and how to increase the chances of a successful reimbursement application (10 min.). This interactive workshop will be of interest to RWE users planning for ECAs and HTA practitioners.