Expediting Drug Development: FDA's New Regenerative Medicine Advanced Therapy Designation

Area of Science:

• Regenerative medicine
• Advanced therapy medicinal products
• Regulatory science

Background:

• Regenerative medicine offers significant therapeutic potential but faces development challenges, particularly for small and medium-sized enterprises.
• The US Food and Drug Administration (FDA) recognized the need for accelerated development pathways for these complex therapies.

Purpose of the Study:

• To introduce the Regenerative Medicine Advanced Therapy (RMAT) designation established by the US FDA in March 2017.
• To outline existing FDA regulatory tools for expediting the approval of regenerative medicines.
• To provide recommendations for companies on integrating regulatory strategies for advanced therapies.

Main Methods:

• Review of the FDA's RMAT designation criteria and benefits.
• Analysis of current FDA expedited programs relevant to regenerative medicine.
• Discussion of strategic considerations for companies pursuing advanced therapy development.

Main Results:

• The RMAT designation provides intensive FDA support for developing cell-, tissue-based, and tissue-engineered products, including those using genome editing technologies like CRISPR-Cas9.
• Existing FDA programs offer pathways to accelerate the development and commercialization of innovative therapies.

Conclusions:

• The RMAT designation and other FDA expedited programs are valuable tools for advancing regenerative medicine.
• Companies should develop integrated strategies to leverage these regulatory pathways effectively for complex advanced therapies.