Co-authored by three life sciences leaders with direct FDA regulatory experience, the paper builds on a presentation delivered at DIA 2026

BLUEBELL, Pa.: Slipstream, a technology consulting partner exclusively focused on life sciences, today announced the publication of a new peer-reviewed paper in Clinical Pharmacology and Therapeutics outlining practical guidance for pharmaceutical and biotech companies seeking to use real-world data to support FDA drug approval or label expansion.

The paper, A Practical Guide to Implementing Real-World Evidence as Primary Basis for Approval and Label Expansion, is co-authored by Dr. Tracy Mayne, SVP of Regulatory and Life Sciences Research at Slipstream Life Sciences; Dr. Alan Brookhart of Duke University and Headwater Science; and Dr. Nancy Dreyer, President of Dreyer Strategies. Drawing on decades of combined experience bringing real-world evidence to the FDA, the authors lay out a practical, experience-based approach to applying the FDA RWD/RWE guidances to real-world evidence for drug approval and label expansion.

“The FDA has provided road maps on how real-world evidence can be used, but there remain numerous gaps when it comes to their practical application,” said Tracy Mayne, SVP of Regulatory and Life Sciences Research at Slipstream.

The publication serves as a companion piece to a session the authors presented at the DIA 2026 Innovation Theater, where they shared early insights on this topic. The full DIA presentation is available on Slipstream’s website.

"Real-world data can provide credible evidence when randomized trials are infeasible or unethical, but only when the study is designed and documented with the same rigor we expect of a trial. This guide is meant to help sponsors get those details right early, when course corrections are still inexpensive," said Dr. Brookhart.

“This piece addresses many practical questions about whether real-world data, study design and execution are good enough for submission in a regulatory evidence package, all lessons learned through the school of hard knocks,” said Dr. Dreyer.

The paper is available via open access at https://ascpt.onlinelibrary.wiley.com/doi/10.1002/cpt.70487.

The FDA’s use of real-world data to support regulatory decisions is rapidly evolving, and the RWD/RWE guidances specifying how those data should be generated, documented, and submitted are complex. Only through practical experience does one learn of the challenges and impediments to meeting FDA’s requirements and how to address them.

For sponsors, the paper offers a practical way to reduce the risk and guesswork involved in bringing a real-world evidence package to the FDA, whether the goal is an initial approval or label expansion. While the majority of use cases involve oncology and rare disease, any indication where a randomized placebo-controlled trial is infeasible or unethical provides an opportunity to integrate real-world data into the evidence package.

The three authors have been directly involved in multiple FDA submissions utilizing real-world evidence, helping establish the standards in this rapidly evolving environment, reinforcing their leadership in this space.

About Slipstream: Slipstream is a technology consulting partner exclusively focused on life sciences. Its teams operate as an extension of their clients, bringing deep domain expertise across R&D, enterprise platforms, data, and operations to help organizations accelerate research, optimize operations, and improve patient outcomes. Learn more about Slipstream Life Sciences at slipstreamls.com.

Fonte: Business Wire