Califf: Decades Of Follow-Up Necessary For Gene Therapy
FDA Commissioner Califf acknowledges that regulatory pathways for gene therapies are still being developed and that many factors outside of the modality impact agency requirements as he emphasized the need for long-term follow up due to the unknowns of gene modification.
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Agency officials don’t think a new definition or incentives will fix the issues related to development in rare diseases with only a handful of patients, as advocates continue pushing the idea.
In a Pink Sheet interview Emil Kakkis contends the agency’s recruitment challenges, high turnover rates and a lack of expertise on the diseases for which reviewers are evaluating drugs have created a disconnect between FDA leadership’s stated willingness to permit accelerated approval for gene therapy and what review divisions are doing on the ground.
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