Our Contribution to FDA Consultation on Individualised Therapies

The International Collaborative Network for N-of-1 Trials and Single-Case Designs (ICN) has submitted a public comment in response to the U.S. Food and Drug Administration’s (FDA) draft guidance, Considerations for the Use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological Cause. The draft guidance represents an important step towards recognising that flexible evidentiary approaches are needed for rare and ultra-rare conditions where conventional clinical trials are often not feasible.

In its submission, ICN welcomed the direction of the draft guidance and highlighted the important role that established methodologies, including N-of-1 trials and single-case experimental designs (SCEDs), can play in generating rigorous evidence for individualised therapies. The submission also encourages continued collaboration between regulators and methodological experts to support the development of clear, fit-for-purpose expectations for these study designs.

As an international network dedicated to advancing the science and application of N-of-1 trials and SCEDs, ICN is pleased to contribute to discussions that help shape the future of personalised medicine and regulatory science. We look forward to continuing to engage with regulators and the broader research community as this field evolves.

Read the FDA draft guidance: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-plausible-mechanism-framework-develop-individualized-therapies-target-specific

Read ICN’s full submission: https://www.regulations.gov/comment/FDA-2026-D-1256-0134