Opinion: I’m a rare disease mom, and I finally have new hope for my son’s future
The FDA published a draft "plausible mechanism" framework to enable platform-based approvals for individualized genetic therapies. The proposal would allow developers to extend an approved therapeutic approach to patients with different mutations without repeating full new approvals, while keeping safety and efficacy standards.
Why it mattersWhen the FDA released its draft plausible mechanism framework, individualized therapies could be authorized under the same approval.
Entities Mentioned
Food and Drug AdministrationDepartment of Health and Human ServicesDuke-Margolis Institute for Health PolicyRare Disease Innovation Hub
People Mentioned
Marty MakaryVinay Prasad