Accelerating Innovation: FDA Expedited Programs 101

In the fast-paced world of biotech innovation, time is of the essence. For European biotech ventures eyeing entry into the US market, navigating the regulatory landscape can often feel like a daunting hurdle. However, the FDA offers expedited pathways designed to accelerate the development and approval of promising therapies, providing a beacon of hope for companies eager to bring their innovations to patients in need.  

In this blog post, we’ll delve into the FDA’s expedited pathways, exploring the various options available, eligibility criteria, benefits, and key considerations for European biotech ventures looking to leverage these pathways to streamline their path to market.  

The FDA has established five programs intended to facilitate and expedite development and review of new drugs and biologics to address unmet medical need in the treatment of a serious or life-threatening condition:

1. Fast Track Designation: Designed for therapies addressing unmet medical needs, Fast Track designation expedites the review process by facilitating more frequent interactions with the FDA and providing opportunities for rolling reviews of data. 
2. Breakthrough Therapy Designation (BTD): Legislated in 2012 to shorten the development and review time of new therapies for serious conditions when available therapy exists but early clinical evidence demonstrates that the new potential therapy may offer substantial improvement over available therapies. 
3. Regenerative Medicine Advanced Therapy Designation (RMAT): Enacted in the 21st Century Cures Act in 2016 to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse or cure a serious condition. 
4. Priority Review (PR): Designation accelerates the review timeline for therapies that offer significant improvements in safety or efficacy, reducing the standard review period from 10 months to 6 months. 
5. Accelerated Approval (AA): This pathway allows for the approval of therapies based on surrogate endpoints or intermediate clinical endpoints that are reasonably likely to predict clinical benefit, enabling faster access to patients while further data is collected post-approval. 

While FDA expedited pathways offer promising opportunities for accelerated development and approval, European biotech ventures must navigate several considerations:

• Eligibility Criteria: Understanding the specific eligibility criteria for each expedited pathway is crucial. Ventures must demonstrate the potential for significant clinical benefit or address unmet medical needs to qualify for these designations. In this webinar, we talked in detail about the types of situations to which these tools are applicable and the differences in requirements to obtain each tool.  
• Early Engagement with the FDA: Proactive engagement with the FDA is essential for maximizing the benefits of expedited pathways. Seeking FDA guidance early in the development process can help streamline regulatory interactions and optimize development strategies. 
• Robust Clinical Data: Despite the expedited nature of these pathways, robust clinical data remain paramount. You must prioritize the generation of high-quality data to support the safety and efficacy of your therapies.

The following table summarizes the criteria and situations:

You must have noticed that “Serious disease or condition” and “Unmet medical needs” are often cited in the criteria. It’s necessary to shed some lights on the definitions.

• Serious disease or condition is associated with morbidity that has substantial impact on day-to-day functioning. Short-lived and self-limiting morbidity will usually not be sufficient, but the morbidity need not be irreversible, provided it is persistent or recurrent.  Whether a disease or condition is serious is a matter of clinical judgment, based on its impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one (21CFR312.300). 

• Unmet Medical Needs are conditions whose treatment or diagnosis is not addressed adequately by available therapy​ 

1. Immediate need for a defined population (i.e., to treat a serious condition with no or limited treatment) ​ 
2. Address a longer-term need for society (e.g., to address the development of resistance to antibacterial drugs)​ 
3. In some disease settings, a drug that is not shown to provide a direct efficacy or safety advantage over available therapy may nonetheless provide an advantage that would be of sufficient public health benefit to qualify as meeting an unmet medical need

It’s important to know that these programs can be used as a stand-alone program or in combination.  

In Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021, Overall, of 581 drug-indication pairs: 

• 363 (62.5%) used at least 1 expedited program 
• 257 (44.2%) used 2 or more programs 
• 97 (16.7%) used 3 or more programs 
• 13 (2.2%) used all 4 programs 
• 218 (37.5%) did not use any expedited programs.  
• 97 of the 139 biologic drug–indication pairs (69.8%) and 266 of the 442 small-molecule drug–indication pairs (60.2%) used at least 1 program. 

Priority Review was the most frequently used program, 330 drug-indication pairs (56.8%) used it either alone or in combination with other programs. This was followed by Fast Track (203 pairs [34.9%]), Breakthrough Therapy (115 pairs [19.8%]), and Accelerated Approval (82 pairs [14.1%]) 

The key is to have a thorough understanding of the criteria around each expedited program and evaluate eligibility early on to ensure full use of the benefits offered and engage with the FDA early on to ensure the Agency’s requirements and expectation are fully understood. In the next blog post of this series, we will talk about how to communicate with the FDA and Dos & Don’ts.

Published on: Apr 30th, 2024