Parexel Introduces FDA Expedited Designation Trends For Advanced Therapies

By Mark Mathieu, Executive Director, Strategic Research

The analysis conducted by Parexel’s Cell & Gene Therapy Center of Excellence delves into the evolving trends of FDA expedited development designations for advanced therapies overseen by the Center for Biologics Evaluation and Research (CBER). It highlights the strategic landscape of three pivotal designations: breakthrough therapy designation (BTD), regenerative medicine advanced therapy (RMAT), and fast track (FT) designations, which are integral to innovation and the research and development (R&D) pipeline.

The Breakthrough Therapy Designation (BTD), established in 2012, is noted for its rigorous qualification criteria and grant success rate of 33% from 2012 to 2023. Although BTD requests declined during the pandemic, they saw a resurgence in 2023, albeit with only a 29% success rate. BTD is applicable to all CBER-regulated products, including vaccines and cell and gene therapies (CGTs).

Introduced in 2016, the RMAT designation has rapidly gained traction, especially for CGTs, due to its relatively lower qualifying threshold compared to BTD. RMAT provides similar benefits to 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 BTD.

The Fast Track (FT) designation, characterized by its minimal qualifying criteria, has become the most sought-after expedited designation, with requests reaching unprecedented levels. Despite offering limited regulatory advantages, FT is valued for its validation potential and increased interactions with the FDA.

This analysis underscores the strategic decision-making involved for developers in selecting these designations, with RMAT and FT offering more accessible alternatives compared to the stringent BTD, thereby influencing the trajectory of the R&D pipeline.