External Control Arms (ECA) in Phase 3 Trials: What Is Needed Now and in the Future to Enable Acceptance With Regulatory and HTA Bodies?

PURPOSE: ECAs are real-world patient cohorts intended to replace or enhance a control arm in clinical trials when randomization to a contemporaneous control arm is not feasible/unethical. However, the conditions for an ECA-controlled trial to be accepted can differ between regulatory and HTA bodies and are beginning to be understood based on publicly available regulatory review use cases. Are all indications appropriate? What are acceptable types of data and methods to build ECAs? What are the circumstances that lead to one design over another? What is the way forward to have regulators and HTA bodies align on these questions to ensure optimal design of a Phase 3 program?

DESCRIPTION: Workshop attendees will obtain a working knowledge of key questions to ask when considering an ECA. Helene will first define ECAs in the context of Phase 3 programs and review the key challenges of using them in lieu of or as augmentation of traditional comparator arms in randomized controlled trials (12min). Emma will detail methodological issues (12min). Nicolle will provide learnings from oncology case examples on how ECAs have been appraised to date and discuss how to approach decision-making for fit-for-purpose study design (12min). Pall will propose a framework for possible alignment between HTA and regulatory bodies (12min). During these 48min, the audience will be invited to real-time polling and verbal comments to identify challenges facing pharma companies internally and in interaction with regulatory and HTA bodies when considering ECAs as part of phase 3 programs. The last 12 min will be dedicated to a guided discussion with the audience on the future of ECAs and how they fit in the evidence-generation framework for new pharmaceutical products. This informative and interactive workshop is intended for an audience of regulators, payers and leaders participating in Phase 3 program design and new pharmaceutical product decisions.