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# Woodcock: Ways to Make Gene Therapies More Viable
- URL: https://www.fdaweb.com/woodcock-ways-to-make-gene-therapies-more-viable/
- Published: 2026-10-29T22:00:00.000Z
- Updated: 2026-10-30T00:04:56.000Z
- Author: David McFarland
- Tags: Drugs

Former FDA principal deputy commissioner, acting commissioner, and CDER director **Janet Woodcock** says reforms are needed to make individualized gene therapies a viable development opportunity for rare diseases. Writing in a *BioCentury* online commentary, Woodcock [says](https://www.biocentury.com/article/661302/woodcock-new-paradigm-needed-to-unlock-therapies-for-rare-diseases?ref=fdaweb.com) FDA already has the statutory authority to address several of the barriers preventing the treatments from reaching patients.

“FDA’s Plausible Mechanism proposal contained useful ideas but did not define a regulatory pathway to approval,” she writes, “and its totality of evidence approach to evaluating product performance is contradicted by the position taken in the recent draft guidance on Demonstrating Substantial Evidence of Effectiveness. Similarly, the Platform Designation enacted by Congress represents a good direction but does not take the concept to completion.”

She recommends these five reforms:

- define indications by molecular defect;
- approve “platforms” that accommodate individualized treatments;
- match clinical evaluation to the patient population and intervention;
- make manufacturing requirements proportionate; and
- enable shared learning through data-sharing.

 “These recommended changes are modular; their appropriateness would depend on the rare disease and therapy in question,” Woodcock concludes. “But taken together, they could change the landscape of therapeutics and open up rare genetic diseases to commercial interest. Families and patients know these technologies have been developed. Are we willing to tell them scientific advances won’t be turned into medicines because we are reluctant to change?”