The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) has launched a voluntary novel excipient review pilot program, which is intended to allow excipient manufacturers to obtain FDA review of certain novel excipients prior to their use in drug formulations.

The pilot program is intended to foster development of excipients that may be useful in scenarios in which excipient manufacturers and drug developers have cited difficulty in using existing excipients. The pilot program will initially be available for novel excipients that: (1) have not been previously used in FDA-approved drug products; and (2) do not have an established use in food.

The program will consist of two stages. The first stage is an initial proposal stage for excipient manufacturers to provide a high-level overview of their novel excipient. CDER says it intends to accept approximately four initial proposals but will consider accepting more proposals as resources allow. Excipient manufacturers whose initial proposals are accepted would then enter the second stage, during which they would provide a full data package consisting of toxicology and quality data. 

At the initial proposal stage, excipient manufacturers will submit brief summaries describing the novel excipient, its proposed use, and the public health or drug development need addressed by the excipient. The initial proposal would include a summary of the supportive data generated or collected so far and some indication of the timing of any subsequent data needed for submission of the full data package. Details of the initial proposal model content outline can be found here.

The FDA will review the initial proposals and select approximately four proposals (two for the first year and two for the second year) to proceed to Stage 2 of the program. The FDA says it will consider the following factors, among other considerations, in determining which proposals it selects:

• Potential public health benefit of the novel excipient (for example, excipients that may facilitate opioid abuse-deterrent formulations or excipients that may promote development of new therapies for serious diseases);
• Likelihood of the novel excipient manufacturer’s ability to submit a complete data package within the established time frame;
• Overall potential of the novel excipient to meaningfully improve pharmacokinetic characteristics that may lead to novel drug development.

Proposals for the pilot will be accepted through December 7, 2021.

Source: US Food and Drug Administration