The Center for Drug Evaluation and Research (CDER) has launched the voluntary Novel Excipient Review Pilot Program (Pilot Program), which is intended to allow excipient manufacturers to obtain FDA review of certain novel excipients prior to their use in drug formulations. This Pilot Program will 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. It 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 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 is anticipated to 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 Package. A link to the initial proposal model content outline can be found below under “Resources.”
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 two of the program. FDA will consider the following factors, among other considerations, in determining which proposals to select:
- 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 and life-threatening diseases).
- Likelihood of the novel excipient manufacturer’s ability to submit a complete package within the established timeframe.
- Overall potential of the novel excipient to meaningfully improve pharmacokinetic characteristics that may lead to novel drug development.
Interested excipient manufacturers should submit initial proposals via email at Novel-Excipient-Program@fda.hhs.gov. Proposals for the pilot will be accepted through December 7, 2021.
For novel excipients selected into the program, the developer should then submit a full package consisting of toxicology (see CDER Guidance for industry “Nonclinical Studies for the Safety Evaluation of Pharmaceutical Excipients”) and quality data (chemistry, manufacturing, and controls data) similar to that provided in an investigational new drug application.