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What it does 

Commercialization of regenerative medicine technologies, a life science area that aims to restore and/or replace damaged tissue or organs, has been hindered by uncertainty regarding the classification of cellular therapies (*Note – for the purposes of this bill, the terms “therapeutic”, “therapy”, and “product” are interchangeable). This bill creates a 5-year conditional use period for products that have demonstrated preclinical safety and a reasonable expectation of effectiveness. The goals of the legislation are to (1) offer patients early access to new treatments, (2) build in time for companies to file for official biologic approval while continuing post-market surveillance, (3) and clarify industry ambiguity regarding oversight.

S. 2689/H.R. 4762 amends the Federal Food, Drug, and Cosmetic Act of 1938 and the Public Health Service (PHS) Act of 1944 to create a conditional approval pathway for cellular therapeutics. While the final approval process of the Food and Drug Administration (FDA) would remain the same, S. 2689 would:

  • Require the Secretary of Health and Human Services (the “Secretary”) to establish a program for conditional approval of cellular therapeutics within one year of the legislation’s enactment, so long as a product sponsor demonstrates preclinical safety evidence and a reasonable expectation of of efficacy, without initiation of Phase 3 investigations.
  • Allow such regenerative medicine products to enter the market without first receiving official FDA approval as a biologic, provided that:
    • The cells and tissues are:
      • Adult and have been evaluated and determined not to provoke significant unintended immune response;
      • Not genetically modified;
      • Produced for a specific use and perform the same or similiar biological function in a recipient as in the donor;
    • The sponsor submits:
      • A biological license application (PHS Act, Section 351[a]) within the 5-year conditional use period (The Secretary may permit continued use of product until a final outcome is determined; the Secretary’s disapproval disallows continued use of the product);
      • Annual and adverse event reports to the Secretary;
      • An application for treatment of patients during the 5-year conditional use period and has not previously applied for conditional use of the product for the same purpose.
  • Mandate that those administering conditional use technologies inform users of the product’s provisional status and requirement for continued study.
  • Require public and private repositories of cord blood, tissues and bone marrow providing material for cell therapy products conditionally approved for use to comply with good tissue practice requirements.

The bill also:

  • Adds conditional use cell and tissue therapy product reporting requirements to existing registration standards that must occur preceeding introduction of devices into interstate commerce.
  • Codifies a new cellular therapeutics classification of and clarifies that conditional use products shall be based on in vitro performance testing (simulated lab environments); not in vivo human clinical trials.
  • Codifies that combination products with cellular components shall fall under the jurisdiction of those with biological products oversight.
  • Authorizes the Secretary to issue guidance regarding legislative provisions and amend regulations, as necessary.
  • Requires the Secretary to hold a public meeting relating to cell and tissue products within 90 days of legislative enactment and work with relevant stakeholders to promote regulatory transparency and consistency.
Relevant Science 

Regenerative medicine is a life science area that aims to restore (e.g., using stem cells) and/or replace (e.g., using skin grafts) damaged tissue or organs. These treatments work like biological Band-Aids. A donor provides a source of cells (e.g. bone marrow), which are implanted into a patient to promote the healing of damaged tissues. The cellular therapies eligible for conditional use have important implications for human health, most notably for blood-based cancers.

While these treatments previously required clinical (human) studies, the bill now allows for conditional market use based solely on preclinical data. Products still require a strong scientific evidence base, with a specific emphasis on immune response, which relates to whether a person’s body will successfully accept foreign tissue. However, opponents of the bill argue that the current lack of clinical data would endanger patients.

Importantly, this legislation limits therapies to “homologous use”, which means that the function of the transplanted cells or tissues may not change. For example, chemotherapy often destroys both cancer cells and blood-forming cells (which produce bone marrow) in leukemia patients. As the body has difficult regenerating its own marrow after radiation, patients receive new blood cells from donors. The most common source, peripheral blood stem cells, have the same biological function in both the patient and donor. This approach differes from experimental therapies that obtain one cell type from a donor (e.g. a skin cell) and convert into another cell type (e.g. a heart cell) before treating the patient.

Under this legislation, adult human stem cells (such as those found in bone marrow) qualify for conditional use, and have been previously approved by the FDA. However, the other two types of stem cells, those derived from embryos and those that are reprogrammed into specific cells, are ineligible, restricting therapeutic options.

Relevant Experts 

Joanne Kurtzberg, MD. Dr. Kurtzberg contributed as an expert panelist to the Bipartisan Policy Center’s recent report, “Advancing Regenerative Cellular Therapy: Medical Innovation for Healthier Americans.”

Endorsements & Opposition 

Like many FDA reforms, this bill has bipartisan support in Congress. Several advocacy groups have spoken publicly about the legislation.

Endorsements:

  • The Alliance for the Advancement of Cellular Therapies supports the REGROW Act, with an online statement from the Chairman citing the regulatory approach of Japan as proof that the legislation “is a good model that has real promise”.
  • A Congressional press release also cited support from 40 health groups including National Stroke Association, Alliance for Aging Research, and the Regenerative Medicine Foundation.

Opposition:

  • The Alliance for Regenerative Medicine lauded Congress’s efforts to accelerate research and development for regenerative medicine, but refused to support the legislation. In an open letter to the bill’s sponsor, Senator Mark Kirk (R-IL), the organization stated that “the bill contains other provisions that we believe leave patients unnecessarily vulnerable and exposed to exploitation.”
  • The President of the National Center for Health Research criticized the bill in an interview, stating that the de-regulation of the FDA indicates “a lack of understanding of the importance of well-designed research.”
Status 

The bill was introduced and referred to the Senate committee on Health, Education, Labor, and Pensions (HELP) on March 16, 2016. 

Sponsors 

The Senate bill was introduced by Mark Kirk (R-IL) and co-sponsored by Joe Manchin (D-WV) and Susan Collins (R-ME). The identical House version was introduced by Mike Coffman (R-CO-6) and co-sponsored by Mark Takai (D-HI-1) and Morgan Griffith (R-VA-9).

Primary Author 
Kushal Kadakia
Editor(s) 
Misha Angrist, PhD, Allison Roder, PhD Candidate, Thomas Williams, JD, MBE & Aubrey Incorvaia, MPP
Recommended Citation 

Duke SciPol, “Reliable and Effective Growth for Regenerative Health Options that Improve Wellness (REGROW) Act (S 2689/ HR 4762, 114th Congress)” available at http://scipol.duke.edu/content/reliable-and-effective-growth-regenerative-health-options-improve-wellness-regrow-act-s-2689 (05/27/2016).