Draft Guidance from the FDA Suggests Important Changes to Drug Development

On February 1, 2023, FDA issued a Draft Guidance Document, called “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (Docket ID: FDA-2022-D-2983).  The Draft Guidance considers the use of “Real World Data” (RWD) as the comparator for new medical treatments in clinical trials, rather than requiring patients to be assigned to placebo groups within the trials. The FDA has requested public comments on the Draft Guidance Document until May 2, 2023.

The Draft Guidance Document deserves considerable praise, as the use of RWD to support evidence of product effectiveness and safety in applications for drug and biological product approvals is forward-thinking and responsive to patient wishes to avoid needlessly being placed in placebo groups in clinical trials, especially in diseases that predictably have the bleakest outcomes.

There is a provision of the Draft Guidance Document that, as currently written, could effectively prevent this new Guidance from being usable for many diseases – especially rare diseases.  The Draft Guidance says that clinical trial “sponsors must include in their marketing applications relevant patient-level data” (line 507) for each of the external controls… But, for many diseases – especially orphan and rare diseases – such individual patient-level data is not available at present.

Collections of medical data are growing, but are not yet large enough to provide individual patient matching data, and do not yet cover many diseases.  While technology has advanced such that “Big Data” solutions are possible in certain disease areas, the breadth of comprehensive patient data in the rare disease world is still relatively sparse.  Without some in-built degree of flexibility regarding the provision of patient-level data, the RWD innovation in this Draft Guidance Document is illusory.

To access and review this FDA Draft Guidance document, please follow this link.  If this RWD topic and potential innovation is important to your disease community, or to you personally, please consider making a public comment by the May 2, 2023 deadline in support of this Guidance.  Comments can be submitted as electronic attachments (e.g., a typewritten letter, in Word or PDF documents), or, entered as free-form text (up to 5,000 characters) within the comment portal.  For additional tips on submitting comments, please review the FDA website, or be sure to click on the “Commenter’s Checklist” within the comment portal.

Share this post

Share on facebook
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email