This article examines US regulations and guidance documents for regenerative medicine advanced therapies (RMATs). The author describes the field of regenerative medicine, noting that it is expanding at an accelerated pace, and outlines some of the common terms associated with it. He also addresses the application process for these therapies, accelerated regulatory pathways, market access, and the outlook for RMATs. The author cautions that, as exciting as these therapies are, they require a rigorous and carefully planned approach to ensure a seamless progression to regulatory approval and commercial success.
Regenerative medicine is a rapidly expanding field, offering the potential to treat serious and life-threatening conditions by replacing, or “regenerating,” human cells, tissues, or organs that have been damaged by disease, trauma, or congenital defects.1 With more than 200 investigational new drug (IND) applications anticipated by the US Food and Drug Administration (FDA) in 2020,2 regenerative medicine should yield many new therapies with enormous benefits to patients, especially those with unmet medical needs.
Navigating the complex regulatory environment of regenerative medicine requires companies to engage with the FDA early and often throughout the drug development process to identify and overcome potential obstacles to approval. Many of these therapies are developed by scientific institutions and medical research groups with limited inhouse regulatory resources, so it is advisable to seek external regulatory support early in the planning process…