Advanced therapy medicinal product (ATMP) development is on the rise. According to the American Society of Gene + Cell Therapy, there were 1,745 gene therapies in development in May 2021, 70% of which were in preclinical studies, and more than 1,300 of these candidates were in development for oncology, the most active therapeutic area.
Given that ATMPs—and the patient journeys associated with them—are fundamentally different from traditional biopharmaceuticals, designing and conducting trials of these novel therapeutics involves unique regulatory and clinical considerations. In this article, we explore strategies for successful start-up and execution of ATMP studies.
As the regulatory framework for ATMP development can be complicated, early and proactive engagement with regulators is essential. Requesting feedback on data and biomarker requirements, the need for long-term follow-up (LTFU), and opportunities for expedited review and approval will help sponsors shape their clinical development programs. In the U.S., for example, the regenerative medicine advanced therapy (RMAT) designation offers a streamlined approval pathway for ATMPs that address unmet medical needs for serious or life-threatening conditions.
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