Medical product development can be both challenging and time-consuming and while FDA reviews are often more rapid than international peers, every day that your product is not on the market is a missed opportunity for revenue and for helping patients. Timing is especially critical for products addressing serious conditions. Fortunately, FDA has a number of programs that offer opportunities for accelerated product development and/or expedited review of the marketing application (some of these programs are addressed in an FDA Guidance for Industry here). In this post, you’ll be introduced to the whats, whens, and whys of six FDA programs that could help speed your product to market.
Fast Track Designation
What: To qualify for Fast Track designation, the product must be intended to treat a serious disease and have the potential to meet an unmet medical need or must be designated as a qualified infectious disease product (QIDP; QIDP products are defined under Title VIII of the Food and Drug Administration Safety and Innovation Act [FDASIA] and in addition to Fast Track, also qualify for Priority Review, described below).
When: Fast Track designation can be requested at any time, but ideally no later than the pre-NDA or pre-BLA meeting.
Why: Among the key advantages is better access to FDA during the drug development process, including more frequent meetings and written communications to facilitate efficient product development, data collection, and clinical trial design. A designated product may also be eligible for Accelerated Approval, Priority Review, and has the option for Rolling Review, meaning the sponsor can submit sections of its marketing application for review by FDA as they are completed rather than having to wait until every section of the application is complete for review to begin.
Breakthrough Therapy Designation
What: Breakthrough Therapy designation is for products intended to treat a serious medical condition that also have the potential to show substantial improvement over existing therapies on a clinically significant endpoint(s).
When: Breakthrough Therapy designation should be requested no later than the end-of-phase 2 meeting. If a sponsor has not requested designation, FDA may suggest that the sponsor submit a request if upon review of submitted data the FDA believes that the product may meet the requirements for designation and feels that the remaining drug development program may benefit from designation.
Why: Products garnering Breakthrough Therapy designation receive the benefit of intensive guidance from FDA on efficient drug development, involvement of FDA senior managers and experienced project management staff to facilitate efficient review, the opportunity for rolling review, and may also qualify for Priority Review.
What: Accelerated Approval allows drugs intended to treat a serious medical condition and that fill an unmet medical need to be approved based on a surrogate or intermediate endpoint (e.g., tumor shrinkage for a cancer drug) rather than requiring pre-approval evidence of actual clinical benefit (e.g., extending patient survival), with confirmatory trials to verify clinical benefit being run post-approval (phase 4).
When: Accelerated approval should be discussed with FDA during development and generally as early as feasible.
Why: Because it can sometimes take years to determine a drug’s effect on patient survival or some other long-term benefit parameter, accelerated approval allows promising drugs to get to market much faster than they otherwise could, and often considerably so.
Priority Review Designation
What: Priority Review designation is for therapies that are likely to provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious medical condition, those designated as a QIDP (discussed above), those submitted with a priority review voucher, or those for which the submission is a supplement proposing a labeling change based upon the results of a pediatric study under Section 505A of the FD&C Act.
When: Priority Review designation may be requested by the sponsor, although FDA automatically considers all marketing applications for this, assigning designation to qualifying applications within 60 days of submission (i.e., by the filing decision date).
Why: Designation indicates that FDA intends to take action on an application within 6 months of filing compared to the 10-month review clock for a standard submission. Priority review can be especially critical for products in competitive fields where first-to-market therapies often enjoy significant advantages, including early adoption by physicians, marketing exclusivity, and advantaged drug pricing.
Regenerative Medicine Advanced Therapy Designation
What: Regenerative Medicine Advanced Therapy designation was introduced as part of the 21st Century Cures Act in December 2016. A regenerative medicine therapy is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products (with a few exceptions), that is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and for which preliminary clinical evidence suggests the therapy’s potential to address an unmet medical need for that indication.
When: A request for designation should be made at the time of IND submission or as an amendment to an existing IND.
Why: Designation conveys eligibility for early interactions with FDA to support product development, as well as access to expedited approval pathways including Priority Review and Accelerated Approval.
Expedited Access Pathway
What: The only program addressed in this post that is specifically geared toward medical devices, the Expedited Access Pathway (EAP) program applies to devices that demonstrate the potential to address unmet medical needs for life-threatening or irreversibly debilitating diseases or conditions and that are subject to premarket approval applications (PMA), premarket notification [510(k)], or requests for “de novo” classification.
When: Sponsors should contact FDA early in the development program and in most cases should request EAP designation prior to beginning an Investigational Device Exemption (IDE) pivotal study.
Why: EAP designation includes Priority Review, earlier and more frequent interactions with FDA, involvement of senior FDA management, and an assigned case manager, all of which combine to facilitate efficient device development, assessment, and review and have the potential to save significant time and money for the sponsor.
For therapies aimed at addressing serious conditions and unmet medical needs, there are several FDA programs that may expedite product development and approval. Understanding what these programs are, when action needs to be taken, and why these programs are advantageous can help get innovative medical products to market faster and at lower cost.
Looking for ways to speed your product’s development? We’d love the opportunity to discuss with you how Nuventra can help put your prod