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status of the most recent attempts to redefine and conceptualize these changes in the United States, we will examine 4 draft guidance documents implemented by the Food and Drug Administration in interpreting relevant concepts and terminology pertaining to HCT/Ps: the Bipartisan Policy Center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.
Highlights
The nascent, exponentially growing regenerative medicine field has introduced new classes of therapies and treatments that have outgrown the existing legislative parameters internationally
This review will outline the four draft guidance documents released by the Food and Drug Administration (FDA) before the enactment of the 21st Century Cures Act and the accompanying regenerative medicine advanced therapy (RMAT) draft guidance documents; a recent report with recommendations on cell therapy policy released by a U.S think tank known as the Bipartisan Policy Center (BPC); and the Reliable and Effective Growth for Regenerative Health Options that Improve Wellness Act of 2016 (REGROW Act), a body of legislation proposed during the previous session of the U.S Congress
The 21st Century Cures Act, enacted in December 2016 (Public Law 114‐255 114th Congress—Dec. 13, 2016), is seen by many as the replacement legislation to the REGROW Act as it includes provisions to ease the approval requirements for “regenerative medicine advanced therapy (RMAT)” category for drugs (FDA, 2017l) that encompass (a) “regenerative medicine therapies, including cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products,” aside from those that are regulated as 361 products; (b) drugs that “treat, modify, reverse, or cure a serious or life‐threatening disease or condition”; or (c) “preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such a disease or condition” (Public Law 114‐255 114th Congress—Dec. 13, 2016)
Summary
The nascent, exponentially growing regenerative medicine field has introduced new classes of therapies and treatments that have outgrown the existing legislative parameters internationally. This bill could be the first to restrict the FDA's current control over the regulation of cell therapies (FDA, 2017d) Given this political context, this article will focus on providing an overview of the regulatory momentum that has been gathering over the past 5 years in the United States to reframe the translation policies for HCT/Ps. This review will outline the four draft guidance documents released by the FDA before the enactment of the 21st Century Cures Act and the accompanying regenerative medicine advanced therapy (RMAT) draft guidance documents; a recent report with recommendations on cell therapy policy released by a U.S think tank known as the Bipartisan Policy Center (BPC); and the Reliable and Effective Growth for Regenerative Health Options that Improve Wellness Act of 2016 (REGROW Act), a body of legislation proposed during the previous session of the U.S Congress. Extraction and concentration of haematopoietic stem/ progenitor cells from peripheral blood for transplantation would generally be considered minimal manipulation procedures because the TABLE 1 Four recently issued draft guidance documents on human cells, tissues, and cellular and tissue‐based products from the Food and Drug Administration. Draft Guidance Relating to the Regulation of Human Cells, Tissues, and Cellular and Tissue‐Based Products; Extension of Comment Periods (Federal Register, 81(78), 23664–23666, Friday, April 22, 2016): Due date for submitting either electronic or written comments was extended by September 27, 2016
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