In late October 2022, FDA announced a new program called the CMC Development and Readiness Pilot, or CDRP (see the Federal Register notice dated October 31, 2022). The purpose of the CDRP is to increase CMC-focused communication between FDA and sponsors “to accelerate CMC development and facilitate CMC readiness” for a biologics license application (BLA). FDA stated that CDRP will include “the use of science- and risk-based regulatory approaches.”
Participants selected for the CDRP will benefit from additional CMC-focused FDA interactions and input intended to accelerate CMC development. Applications to participate in the CDRP will be accepted starting April 1, 2023. FDA intends to choose no more than nine proposals, with approximately 6 being CBER-regulated products and 3 CDER-regulated products.
With the rapid growth in cell and gene therapy and the unique CMC challenges encountered by both sponsors and CBER in CGT, our best guess is that the majority of the 6 participants chosen for CBER will be from the CGT space.
Let’s look at the benefits of the CDRP, the qualifications, the application process, and how FDA will consider the applications.
Benefits of CDRP:
FDA will provide product-specific CMC advice during product development via
Determining eligibility for CBER CDRP:
Applications for the CDRP should be submitted in an amendment to the IND. We recommend clearly labeling the amendment, and including in the cover letter that this amendment is an application for/request to participate in the CMC Development and Readiness Pilot. You may also consider contacting the Regulatory Project Manager (RPM) assigned to the IND to make them aware of your submission of this amendment.
The Federal Register notice lists the required content for applications.
FDA states that they intend to seek balance and diversity in product types, sponsors, and therapeutic indications to obtain a variety of relevant experience and learnings from the CDRP, and that selection criteria will include
We expect the number of applications from CGT product sponsors to be high, and CBER will need to carefully consider the factors listed above to fairly select a balanced group of participants. Novel products for which sponsors have limited experience will have a higher chance of selection, along with products for rare diseases with no available treatments. FDA notes that INDs for combination products are eligible, but that products that require significant cross-Center interactions may be less likely to be selected for the pilot. This was an important clarification for FDA to include in the FR notice, because this is a CBER and CDER pilot under the PDUFA VII agreement, and CDRH was not given resources related to this pilot.
FDA plans to review of requests on a quarterly basis, and suggested this schedule may be altered depending on the volume of applications. FDA intends to issue a letter to notify each sponsor of FDA's decision on their request to participate within 180 days of receipt.
In summary, we would describe the CDRP program as an excellent opportunity for sponsors to obtain the additional CMC-focused FDA interactions they have been seeking. We strongly encourage everyone who fits the eligibility criteria to consider applying. We at DHC are available to help clients evaluate whether their products meet the eligibility criteria and craft their CMC development plans to apply for the CDRP.