Speed your time to market with expedited programs from the FDA
Todd Phillips, PharmD
Director, Regulatory Affairs and Product Development
Cardinal Health Regulatory Sciences
The FDA offers five expedited programs for products that address unmet medical needs for serious or life-threatening conditions – and the goal of these programs is to ensure therapies are made available to seriously ill patients, as soon as possible. One of the most recent and high-profile products to receive “Fast Track” designation from the FDA was mRNA-1273, Moderna’s potential vaccine for COVID-19, which was granted the designation in May of 2020. As an increasing number of drugs in development focus on areas of unmet medical need, it’s important for developers to understand FDA expedited approval programs; the degree to which each one can compress development and marketing application review timelines; and the most current utilization rates for each. Armed with a better understanding of these programs, sponsors are in a better position to accelerate the development and regulatory review process and get therapies to patients, faster.
Fast Track designation: With 151 requests granted by the Center for Drug Evaluation and Research (CDER) in 2019, the Fast Track designation was the most utilized of the FDA’s expedited programs last year. It is intended for drugs that treat serious conditions and have the nonclinical or clinical data to demonstrate that they have the potential to address unmet medical needs. FDA takes actions to expedite development and review of these products, which are also eligible for a rolling review. Requests for Fast Track designation should be submitted with the original IND or thereafter.
Breakthrough Therapy designation: CDER granted 67 “Breakthrough Therapy” designations in 2019, making this the second most utilized of its expedited programs last year. This designation is intended for therapies that address a serious condition and have preliminary clinical evidence that indicates that they may demonstrate substantial improvement over other available therapies. Sponsors can request this designation with the original IND or anytime thereafter – but should ideally do so prior to the initiation of clinical trials that are intended to demonstrate efficacy. Submissions must include preliminary clinical evidence; and each indication requires a separate Breakthrough Therapy designation request. This designation benefits from an organizational commitment from FDA and intensive guidance from FDA on how to develop the therapy most efficiently. Breakthrough Therapies are also eligible for rolling review.
Accelerated Approval pathway: Of the 48 novel drugs approved by CDER in 2019, nine were under the “Accelerated Approval” pathway. To qualify, atherapy must treat a serious condition and provide a meaningful advantage over available therapies. FDA bases its approval on surrogate endpoints or intermediate clinical endpoints that are reasonably likely to predict the product’s clinical benefit.
Priority Review designation: Of the 48 novel drugs approved by CDER in 2019, 28 earned “Priority Review.” This designation is specific to a marketing application for a therapy that meets one of the following four criteria: it must meet a serious condition and, if approved, provide a significant improvement on safety or effectiveness; it must propose a labeling change pursuant to a report on a pediatric study under 505A; it must be designated as a qualified infectious disease product; or it must be submitted with a priority review voucher. This designation’s key benefit is that the product’s marketing application is reviewed in a more compressed timeframe. FDA informs the applicant if it has received Priority Review designation by day 60 of the original review timeline.
Regenerative Medicine Advanced Therapy (RMAT) designation: A product is eligible for this designation if it’s a regenerative medicine therapy; if it is intended to treat, modify, reverse or cure a serious condition; and if preliminary clinical evidence indicates that the therapy has potential to address an unmet medical need. Requests for RMAT designation must be submitted with the original RND or thereafter; and this designation features all of the benefits associated with both Fast Track and Breakthrough Therapy designations. The Center for Biologics Evaluation and Research (CBER) granted 16 RMAT designations in 2019.
Cardinal Health Regulatory Sciences (CHRS) has more than four decades of experience supporting pharmaceutical and biotech companies in getting their products to market quickly – CHRS associates have supported approximately 30 original marketing applications utilizing one or more expedited program application. Our Expedited Pathways Center of Excellence features cross-functional experts in non-clinical, clinical, chemistry, manufacturing and controls and regulatory affairs – each of whom is committed to helping developers realize the full benefits of these expedited programs. Learn more about our tailored approaches to guide expedited programs at our Expedited Pathways Center of Excellence landing page.
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