Four Food and Drug Administration draft guidance documents and the REGROW Act: A litmus test for future changes in human cell- and tissue-based products regulatory policy in the United States?

Abstract

Modern regenerative medicine research has expanded well past the development of traditional drugs and medical devices with many promising new therapies encompassing an increasingly diverse range of substances, notably cell-based therapies. These substantial recent developments and the progress in the health care and therapeutics fields necessitate a new regulatory framework agile enough to accommodate these unique therapies and acknowledge their differences with traditional pharmaceuticals. In the United States, recent proposed changes in the regulatory framework for autologous human cells, tissues, and cellular and tissue-based products (HCT/Ps) and their perceived risk-benefit analysis for patients remain controversial in the scientific field. To provide perspective on of the current status of the most recent attempts to redefine and conceptualize these changes in the United States, we will examine 4 draft guidance documents implemented by the Food and Drug Administration in interpreting relevant concepts and terminology pertaining to HCT/Ps: the Bipartisan Policy Center think tank report, "Advancing Regenerative Cellular Therapy: Medical Innovation for Healthier Americans," the proposed REGROW Act for HCT/Ps, and the current 24 Food and Drug Administration-approved HCT/Ps and related products in the United States.

Keywords: Food and Drug Administration (FDA); accelerated approval; conditional and time-limited authorization; conditional market authorization; guidance; human cells, tissues, and cellular and tissue-based products (HCT/Ps); market authorization under exceptional circumstance.

Figure 1

Flowchart to decide categorizations of autologous and allogeneic Human Cells, Tissues, and Cellular and Tissue‐based Products as described by the BPC Report. AIR, induce an adverse immune response; Non‐AIR, do not induce any adverse immune response. Yellow‐coloured boxes indicate new key characteristics of Human Cells, Tissues, and Cellular and Tissue‐based Products (HCT/Ps) under BPC Report Level 2. Reddish‐coloured boxes indicate new key factors of HCT/Ps under BPC Report Level 3. These figures outline the HCT/P categorizations recommended in the Bipartisan Policy Center released in the report titled “Advancing Regenerative Cellular Therapy: Medical Innovation for Healthier Americans.” The figures organize how autologous or allogeneic HCT/Ps would be distributed into Level 1, 2, or 3. HCT/Ps are organized according to source: autologous (a) or allogeneic (b), elicited immune response (AIR or non‐AIR), extent of manipulation (minimal manipulation or more than minimal manipulation), cell character and function after manipulation (retaining the same character and function or not retaining the same character and function), and cell use (homologous use or no homologous use) [Colour figure can be viewed at http://wileyonlinelibrary.com]