As a follow-up to the 2009 Guidance on development of products under the Animal Rule, the most recent draft guidance issued by the FDA provides greater detail on the FDA expectations regarding the data used to prove product efficacy and how to extrapolate that information to effective human doses when pivotal human efficacy studies are not ethical or feasible. Foremost amongst those expectations is that the FDA should be consulted and that agreement be reached on study design before the conduct of any studies that will be offered as pivotal in establishing product efficacy. These interactions are encouraged to begin early in development and to take advantage of programs intended to enhance FDA collaboration with Sponsors. Fast Track and Orphan Designations and Special Protocol Assessments are among the programs the FDA suggests that may be applicable to facilitate Agency interactions and optimize product development.
The new guidance does more than just recommend that Sponsors obtain agreement on study design and conduct. It also provides many suggestions for program and study design elements to be considered by Sponsors. Since the considerations in the design of studies intended to prove efficacy under the Animal Rule are in many substantive ways different than those for typical pivotal human efficacy trials, the FDA has provided in the Guidance both suggestions that address unique design elements and an openness to consider alternative approaches provided there is good scientific justification. As evidence of their concern that Sponsors fully appreciate the importance of these unique features, the Agency has gone so far as to develop animal model and study design checklists for consideration by Sponsors during product development.
Sponsors of products under the Animal Rule, whether they have extensive experience in the traditional drug development realm or are less familiar with the FDA, will find a thoughtful treatment of issues based on the FDA’s accumulated experience with Sponsors of these critically important products.
Carrie Rabe is a Senior Consultant at The Weinberg Group, the world’s leading food and drug consulting firm. If you have any questions or thoughts on this blog post or others, please reach out to us by email.