FDA wants participants for CMC readiness pilot for expedited drugs

The US Food and Drug Administration (FDA) announced on 11 September a limited chemistry, manufacturing and controls (CMC) development and readiness pilot (CDRP) program to facilitate the development of breakthrough or accelerated review products.

The pilot was highlighted in the Prescription Drug User Fee Act (PDUFA) VII commitment letter for fiscal years 2023 through 2027. The pilot is now in its second year; the first phase of the pilot was announced last November. (RELATED: FDA announces CMC review pilot for drugs with expedited development, Regulatory Focus 1 November 2022).

The pilot aims to facilitate CMC readiness for selected products regulated by the Center for Biologics Evaluation and Research (CBER) and the Center for Drug Evaluation and Research (CDER) with accelerated clinical development timelines. It is also meant to increase communication between FDA and sponsors, and provide patients with earlier access to these products, said FDA.

FDA will select nine applications, with two-thirds of these products from CBER and one-third from CDER.

Under the pilot, FDA will provide product-specific CMC advice during product development, as well as two additional CMC-focused Type B meetings, and a limited number of additional CMC-focused discussions.

Interested sponsors should submit a request to participate as an amendment to their IND. The cover letter should state “Request to participate in the CMC Development and Readiness Pilot.”

The plan should describe the current state of CMC development, including any ongoing activities not already addressed in the IND. Applicants should also submit a projected timeline for product development, showing any outstanding CMC tasks and activities that need to be completed before a marketing application is submitted. The request should also list any additional CMC challenges that may require FDA input.

Sponsors with INDs that have not yet reached the end of Phase 2 are eligible to participate “to allow the pilot to have sufficient time to have an impact on CMC readiness.” Yet exceptions will be considered for clinical development programs incorporating an innovative trial design or for products intended to treat a rare disease.

FDA will consider the clinical benefits of facilitating earlier patient access to the product, the novelty of the product, complexity of the product or its manufacturing process, the sponsor's overall manufacturing experience. FDA may also consider less-experienced sponsors in an effort to “seek balance and diversity in product types, sponsors, and therapeutic indications to obtain a variety of relevant experience and learnings from the pilot.”

The agency will notify sponsors of their decision within 180 days of receipt of the applications.

FDA plans to hold a public workshop and issue a strategy document to focus on some of the lessons from the CDRP.

CMC pilot

FDA wants participants for CMC readiness pilot for expedited drugs

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