Parexel's Cell & Gene Therapy Center of Excellence has released an analysis detailing the evolving trends in FDA expedited development designations for advanced therapies. The analysis focuses on three key designations overseen by the Center for Biologics Evaluation and Research (CBER): Breakthrough Therapy Designation (BTD), Regenerative Medicine Advanced Therapy (RMAT), and Fast Track (FT) designations.
Breakthrough Therapy Designation (BTD)
Established in 2012, the BTD is known for its rigorous qualification criteria. From 2012 to 2023, the grant success rate for BTD was 33%. While BTD requests experienced a decline during the pandemic, they rebounded in 2023, albeit with a lower success rate of 29%. The BTD is applicable to all CBER-regulated products, including vaccines and cell and gene therapies (CGTs).
Regenerative Medicine Advanced Therapy (RMAT)
Introduced in 2016, the RMAT designation has rapidly gained traction, particularly for CGTs. This is attributed to its relatively lower qualifying threshold compared to the BTD. The RMAT designation offers similar benefits to the BTD, such as early FDA insights and opportunities for discussions on clinical development programs, leading to a higher success rate and frequent requests surpassing those for the BTD.
Fast Track (FT) Designation
The Fast Track (FT) designation, characterized by its minimal qualifying criteria, has become the most sought-after expedited designation. Requests for FT designation have reached unprecedented levels. Despite offering limited regulatory advantages, the FT designation is valued for its validation potential and the opportunity for increased interactions with the FDA.
The analysis underscores the strategic decision-making involved for developers in selecting these designations. RMAT and FT offer more accessible alternatives compared to the stringent BTD, thereby influencing the trajectory of the R&D pipeline.
