- FDA Leaders Outline New “Plausible Mechanism Pathway”
- The Agency’s Plans to Hire More Staff
- FDA Introduces Pilot Aimed at Faster Meeting Minutes Clarifications
FDA Leaders Outline New “Plausible Mechanism Pathway”
This month, FDA Commissioner, Dr. Martin A. Makary, M.D., M.P.H., and Dr. Vinay Prasad, M.D., M.P.H., director of the Center for Biologics Evaluation and Research (CBER), published an article in The New England Journal of Medicine, detailing “a path to market entry for products where a randomized trial is not feasible” called the “plausible mechanism pathway.” Drs. Makary and Prasad outline the following factors that will support approval of a product under the plausible mechanism pathway:
- A specific molecular or cellular abnormality is identified, rather than a broad set of consensus diagnostic criteria.
- The therapy targets the underlying or proximate biological alterations.
- A well-characterized natural history of the disease in the untreated population is established.
- It is confirmed that the target was successfully drugged, edited, or both.
- There is “improvement in clinical outcomes or course.”
The article states that the FDA will weigh granting marketing authorization “once a manufacturer has demonstrated success with several consecutive patients with different bespoke therapies.” Further, as part of their postmarketing requirements, sponsors will have certain commitments, which include collecting “real-world evidence to confirm continued preservation of efficacy.”
The Agency’s Plans to Hire More Staff
Recently, in an interview with Bloomberg Law, FDA Commissioner, Dr. Marty Makary, M.D., M.P.H., discussed the FDA’s intent to hire almost 1,000 scientists and inspectors. This news follows the high turnover the FDA has experienced this year due to staffing cuts, resignations, and retirements. According to Dr. Makary, the hiring will help the FDA carry out its “aggressive plans” for reform.
FDA Introduces Pilot Aimed at Faster Meeting Minutes Clarifications
On November 19, the FDA announced the launch of a pilot program intended to streamline communications with sponsors following formal meetings. The program, which is being piloted by the Office of New Drugs, provides sponsors with a “Meeting Minute Clarification Opportunity.” The Meeting Minutes Clarification Opportunity allows sponsors “to obtain a quick clarification of a response from a single discipline” by submitting a question via email. According to the press release, the Agency’s staff will aim to provide an email response within three business days.