According to the Alliance for Regenerative Medicine, there were 1,085 active advanced therapy medicinal product (ATMP) developers and more than 150 phase III trials underway at the end of 2020. These numbers are not small, and they make it clear that ATMPs have much to offer in helping us overcome as-yet undefeated diseases.
ATMPs are different from biopharmaceuticals, and these differences influence the regulatory and clinical approaches that sponsors must consider when designing and conducting trials. In our view, there are four key considerations when it comes to trials. Let’s take a look.
First, the need for early, proactive engagement with regulators is critical. This is an emerging field; regulations can change. Because ATMPs are complex biological products, the current regulations around them are also complex. We would advise all sponsors to initiate discussions with regulators early in their development planning. This is an opportunity to get clarification on topics such as data requirements, the need for biomarkers as outcome measures, the necessity of long-term follow-up (LTFU), and the possibility of accelerated approval. In the US, the “regenerative medicine advanced therapy” designation offers an expedited path to market for ATMPs that target serious or life-threatening conditions with unmet medical needs.