ECA Pilot
Informing the Use of External Control Arms in Clinical Development and Regulatory Decision-Making
Application of External Control Arms in Oncology Drug Development
Friends of Cancer Research (Friends) is holding a public meeting to explore opportunities for advancing the use of external control arms (ECAs) to enhance oncology drug development.
Tuesday, April 7, 2026
10:00AM – 2:00PM ET
How can we reliably leverage external data to support the evaluation of new treatments, particularly in situations where traditional randomized controlled trials (RCTs) are not feasible or ethical?
What is the unmet need and why does it matter?
Traditional randomized controlled trials (RCTs) are the gold standard for evaluating new therapies but in many rare diseases and cancers, they are not always feasible or ethical. Small patient populations make it difficult to enroll enough trial participants for robust statistical analysis. In some cases, it can be ethically complex to randomize patients to a control arm, particularly when effective options are limited or nonexistent.
This creates a critical need for reliable alternative approaches that generate scientifically valid and meaningful data. Without solutions to overcome these challenges, there may be insufficient evidence to make informed decisions about new therapies and advance the development of potentially life-saving treatments for patients. This contributes to delays in patient access and hinders innovation in areas of high unmet need, particularly for populations affected by rare or aggressive diseases.
How are we helping to find solutions?
The External Control Arm (ECA) pilot project builds upon Friends’ RWE Portfolio to advance oncology clinical research and regulatory decision-making. By leveraging insights from past RWD initiatives, this project aims to identify and refine best practices for using historical clinical trial data and RWD to construct rigorous ECAs. These ECAs are compiled from patients who received standard-of-care treatments and can serve as a comparator to augment single arm trials, improving the efficiency of oncology drug development.
Since 2017, Friends has led collaborations between RWD developers, industry, government agencies, and academic researchers to address challenges in using RWD/E for clinical development. This project builds on prior initiatives—RWE Pilot 1.0, Pilot 2.0, rw-Response, and rw-Care—which advanced methods for endpoint alignment, trial emulation, real-world response measurement, and clinical decision-making. Visit our RWE portfolio site for additional details.
How does this impact patients?
Developing effective and ethical approaches to clinical research can significantly benefit patients, especially those with rare or hard-to-treat conditions where RCTs are challenging or impractical. Using RWD and historical clinical trial data to create an ECA has the potential to accelerate evidence generation and regulatory decision-making for promising therapies, bringing them to patients faster.
In situations where traditional RCTs are not possible, ECAs provide additional insights for evaluating and approving new treatments, ensuring those with urgent medical needs gain access to life-saving options sooner. Use of external data also has the potential to provide additional context for single-arm studies. Additionally, ECAs may be used to supplement traditional control arms, enabling more trial participants to receive experimental therapies.
Friends’ collaborative approach and the support of our partners will align best practices for using RWD or historical clinical trial data to construct ECAs, enabling evidence generation and the delivery of innovative treatments to patients quickly and safely.
Project Approach
Project Partners
ECA Pilot Project: AbbVie Inc., Aetion, American Society of Clinical Oncology (ASCO), Amgen Inc., AstraZeneca, BeOne Medicines USA, Bristol Myers Squibb, Canal Row, ConcertAI, COTA, Eisai Co., Ltd., Flatiron Health, Friends Advisory Advocates, Genentech, Inc., iOMEDICO, IQVIA Holdings, Inc./Guardian Research Network, Johnson & Johnson Innovative Medicine, Medidata Solutions, Merck & Co., Inc., Merck KGaA, N-Power Medicine, Inc., Ontada/McKesson Corporation, Pancreatic Cancer Action Network (PanCAN), Patient Advocates, Tempus AI, Inc., and Truveta.