Friends is leading a research partnership, the External Control Arm (ECA) Pilot Project, to inform the use of ECAs in clinical development and regulatory decision-making. The project will evaluate the use of external data from 8 different sources—including both historical clinical trial data and real-world data (RWD)—to construct a series of ECAs to inform practical strategies and optimal data characteristics for future application.
Randomized controlled trials (RCTs) remain the gold standard for evaluating new therapies, but in many rare diseases and cancers, novel evidence-generation strategies may be required to advance development and expand patient access. Small patient populations make it challenging to enroll enough participants for robust statistical analyses, and in some cases, randomizing patients to a control arm raises ethical concerns when effective treatment options are limited or unavailable. Without alternative approaches that generate reliable, meaningful data, barriers in trial feasibility and evidence generation could delay patient access and slow innovation.
Friends has a long track record of convening RWD developers, industry, government agencies, patient advocates and academic researchers to identify opportunities and optimize use of RWD/ Real World Evidence (RWE) for clinical development. The ECA Pilot Project extends this work by generating insights to support the reliable use of external data in situations where traditional RCTs may not be feasible, accelerating evidence generation in areas of unmet need, and helping bring promising therapies to patients safely.
ECA Project Timeline
To learn more about the project visit: https://friendsofcancerresearch.org/eca/
ECA Pilot Project Partners
AbbVie, Aetion, American Society of Clinical Oncology (ASCO), AstraZeneca, BeOne Medicines, Bristol Myers Squibb, ConcertAI, COTA, Eisai Inc., Flatiron Health, Gemini, Genentech Inc, IQVIA/ Guardian Research Network, Inc., Johnson and Johnson Innovative Medicine, Medidata Solutions, Merck & Co., Inc., N-Power Medicine, Ontada, Pancreatic Cancer Action Network (PanCAN), Tempus AI, and Truveta.