Regenerative Medicine Therapies for Rare Diseases

Abstract

The field of regenerative medicine is growing rapidly with the introduction of new therapies that have the potential to treat and cure serious medical conditions, including rare diseases, for which there are no available treatments. In the United States, the development of novel medical products is regulated and guided by the Food and Drug Administration (FDA). As scientific and technological advances are discovered and adopted by the medical industrial enterprise, the FDA's implementation of policies that create a climate conducive to safe development and rapid availability of novel medical products is one of the pillars which support the Agency's mission of protecting and promoting the public health. With advancements in cell modifications and tissue engineering, innovative creation of biomaterials, adoption of three-dimensional bioprinting, and rapid development of human genome editing technologies, the need for Agency's work in ensuring that its science-based policies remain relevant and helpful in facilitating the availability of new treatments to the most vulnerable populations of patients becomes more pressing than ever before. In December 2016, Congress amended section 506 of the Food, Drug, and Cosmetic (FD&C) Act [21 U.S.C. 356] by adding a new section 506(g), which defines the categories of products considered to be regenerative medicine therapies. As further described by FDA [1], regenerative medicine therapies are considered to include cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies, as well as gene therapies, including genetically modified cells that lead to a durable modification of cells or tissues. The development and approval of regenerative medicine therapies are regulated by FDA's Office of Tissues and Advanced Therapies (OTAT) in the Center for Biologics Evaluation and Research (CBER). In this review article, we present practical considerations for investigating regenerative medicine therapies intended for the treatment of rare diseases. The material presented may be useful to researchers who are undertaking the challenging task of finding and delivering new treatments for those in need.

Keywords: Center for Biologics Evaluation and Research; FDA; OTAT; Office of Tissues and Advanced Therapies; RMAT; Regenerative Medicine Advanced Therapy designation; medical product development; natural history study; patient engagement; rare disease; regenerative medicine therapy.

Fig. 1.. Product Development Program: Building Blocks.

B: R – benefit: risk.

Fig. 2.

Investigational New Drug (IND) applications with development programs that included study populations with rare diseases. The graph depicts the rising numbers of new IND applications with rare disease populations for cell, gene, tissue-based, and other biological products submitted to OTAT (formerly Office of Cellular, Tissues, and Gene Therapies – OCTGT) in calendar years 2002–2017.