FDA Funding for Orphan Drug Clinical Trials
(Thursday, September 7, 2023)
The US FDA offers a grant of up to $3.6 million in non-dilutive funding to researchers developing new treatments for orphan drugs with an unmet medical need through a competitive selection process. Although the orphan drug development grant is not discussed much in funding circles, it offers several benefits, additional to the funds, to the developers, which should be considered for the overall development strategies. There are a few factors that cause the relative obscurity of this grant.
This is an RO1 grant which means that it can only be awarded to individual investigator(s) at academic centers rather than companies. The RO1 grants are traditionally NIH-funded programs intended to support academic researchers. In that regard, the FDA grant is identical to the NIH grant. However, there is no restriction on the collaboration of academic researchers with industry partners. FDA touts that this grant has enabled more than 80 orphan drug approvals but did not elaborate on how. One can assume that the FDA is counting trials it funded which in turn led to the design of subsequent clinical development plans used for the market approval of those products. Since all clinical data, including early-stage trials are critical for drug approvals, particularly those for orphan and rare diseases, FDA has a good point to claim the credit for products that got approval post-funding from the FDA.
The grant amount could be $650,000 to 900,000 per year for up to 4 years and could total a maximum of $3.6 million over 4 years. Unlike the traditional NIH-funded R01 programs, the FDA grant is designed to support clinical trials only and since those would require IND clearance, it automatically culls out programs not ready for clinical trial due to lack of pre-clinical data. FDA recommends that the applicants organize pre-IND and similar pre-submission meetings with the FDA to discuss their clinical trial programs, prior to applying for this grant.
Overall, the grant processes are similar to other grants administered by US government agencies and have similar conditions such as the work be conducted in the US only and there are extensive reporting and compliance expectations.
The industry frequently works with academic researchers to develop new products either via collaborative agreements or intellectual property development and licensing programs. As such, grants like the FDA grants might not directly benefit the companies but could provide significant funding for collaborating academic researchers and opportunities for other collaborative relationships with the FDA. Academics can benefit from industry collaborations for their orphan drug development programs for which the grant could be a credible attraction. The size of the grants assures that they can be useful to support most early-stage clinical trials either completely or be a major funding component. Most importantly, an FDA-funded clinical trial program could provide significant credibility to the funded program for later development and fundraises. However, just because it is funded by an FDA grant, does not mean that these clinical trials would get a pass from the FDA. Rather FDA's expectation that grant applicants arrange pre-submission meetings with the FDA for their programs indicates a pre-selection process for orphan grant applicants, without using specific terms to describe such meetings.
Overall, the FDA’s orphan drug grant is worth considering in the strategies for new product development.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC