External Control Arms

The Power of Real-World Data to Support Clinical Development

Randomized controlled trials (RCTs) are the accepted gold standard for medical product clinical development programs, however, there are times where RCTs are not feasible or ethical. In those circumstances, external control arms (ECAs) may be the right solution to augment trial data.

Advances such as gene and mRNA therapies, and the increasing focus on rare disease and new cancer indication treatments have led to trials in indications for which there is a high unmet medical need and corresponding questions about the suitability of traditional RCTs. In these cases, a single-arm study becomes necessary, and ECAs become an important and viable option to generate comparator data.

An external control arm uses existing patient-level data, either clinical trial or real-world data, to create a cohort of patients to use as a comparator arm – or supplement an existing randomly allocated control arm – that mimics the characteristics of an RCT control arm.

When to Consider an External Control Arm

Rare indications with high need, urgency, or poor outcomes
Therapies with large benefit versus risk
RCTs are unethical or unfeasible, resulting in significant delays to patient access
Scientific concerns about treatment switching/cross-over or other barriers to RCTs

The application of ECAs can be for regulatory approvals, label expansion or changes, medical affairs, or market access and reimbursement.

Key Benefits of an External Control Arm

Provides comparator data to strengthen regulatory submissions when an RCT is not possible

Eliminates or reduces the need to enroll control participants

Increases efficiency and product development time

Enables quicker patient access

We Can Help You

Evidera’s dedicated team of epidemiologists, real-world data scientists and analysts, in combination with our global regulatory experts, are well positioned to help you navigate the use and implementation of ECAs, including:

Strategize if an ECA is right for your study
Support your regulatory strategy and interactions, as well as broader product strategy
Prioritize key criteria for selection
Validate real-world outcomes
Identify real-world data sources that are fit-for-purpose to meet the study’s needs
Design and validate state-of-the-art ECA methods
Fully implement and operationalize the ECA whether leveraging existing databases or requiring data collection via chart review or prospective patient enrollment

Related Resources

Webinar

Clinical and Real-World Study Challenges in the COVID-19 Era: Scientific and Operational Strategies for External Control Arms using Real-World Data

Apr 28, 2020 10:00 AM - 11:00 AM

Watch Webinar

Publication

Considerations for Regulatory Application of RWD-Generated External Comparators

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