The Food and Drug Administration recently issued draft guidance recommending steps drug companies should take to evaluate the safety of gene-editing technologies when seeking approval for new treatments for genetic diseases. The guidance is part of a broader FDA initiative to build on the success of CRISPR-based gene-editing therapy, allowing similar treatments to move forward. The agency previously introduced a new regulatory framework intended to speed approval of similar treatments. Known as the “plausible mechanism framework,” it will allow researchers to support an intervention’s effectiveness using evidence from a small group of patients, rather than relying on a large clinical study.
Dominick DiSabatino, a member of Sheppard’s Life Sciences industry team, noted to Law360 that the guidance is likely to be a net positive for developers of individualized therapies, though the impact on specific products may vary. “There is obviously a link between this guidance and the administration's goals with the plausible mechanism pathway framework, and the goal here was movement toward technical enablement of that framework. If FDA provides you an opportunity to engage — as it is doing here — then take the opportunity, document everything, and hold the agency to its guidance,” he said.
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