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FDA Issues More Policies to Facilitate Innovation in Regenerative Medicine

December 19, 2017
As part of the Food and Drug Administration's 21st Century Cures Act, two more policy documents were released to accelerate development of regenerative medicine technologies quickly and safely.

Interview with Jay Joshi, MD

On November 16, 2017, two new guidance documents were issued to build a policy framework on regenerative medicine designed to “spur innovation, efficient access to potentially transformative products, while ensuring safety and efficacy,”1-4 according to a Food and Drug Administration (FDA) press release.

These regulatory guidelines continue to expand the FDA’s risk-based regulatory approach. The documents propose a process for ensuring the safety and effectiveness of regenerative medicine therapies and aim to support development in this growing area of medicine to improve patient care.1-4 These guidance documents also define the process in which the FDA intends to enforce safety measures on products that have significant safety concerns.

Stems cells and tissues can be engineered to replaced diseased tissues and organs.Stems cells reflect the potential of regenerative medicine.

“We’re at the beginning of a paradigm change in medicine with the promise of being able to facilitate regeneration of parts of the human body, where cells and tissues can be engineered to grow healthy, functional organs to replace diseased ones, new genes can be introduced into the body to combat disease, and adult stem cells can generate replacements for cells that are lost to injury or disease. This is no longer the stuff of science fiction. This is the practical promise of modern applications of regenerative medicine,” said Scott Gottlieb, MD, FDA Commissioner, in a press release.

“The current FDA Commissioner seems to understand that stem cells and regenerative medicine are the future of medicine, and he is moving quickly to enact appropriate guidelines,” Jay Joshi, MD CEO and medical director of the National Pain Centers in Vernon Hills, Illinois, told Practical Pain Management.

Finalizing 2 Policies on Regenerative Practices 

The two new final guidance documents address stem cell and tissue products taken from and then used in the same person and more clearly define “minimal manipulation” and “homologous use.”1,2

The first guidance addresses human tissues that are intended for implantation, transplantation, infusion, or transfer into a human recipient. The FDA clarifies which regulations must be followed for autologous use of cells or tissues. In particular, the FDA is detailing the exceptions that exist for tissues or cells that are used in the same individual during the same procedure.1

The second guidance is directed to manufacturers, healthcare providers and the FDA staff that work with human cells, tissues, and cellular and tissue-based products. This guidance clarifies the definitions of “minimal manipulation” and “homologous use” so that stakeholders know which regulation their product falls under and whether they need certain approvals.2

Draft Guidance for Advanced Therapy Devices

The two new draft guidance documents address the regulatory application process for devices used in regenerative medicine advanced therapies (RMATs) and the expedited programs available for RMAT designation.3.4

The first draft guidance, “Evaluation of devices used with regenerative medicine advanced therapies,” clarifies how the FDA will evaluate devices used in the recovery, isolation or delivery of RMATs. It is meant to help streamline the process of regulatory approval for combination device and cell or tissue products used in procedures with RMATs.3

The 21st Century Cures Act created the regenerative medicine advanced therapy designation. This designation can be used to speed up the review of cell therapies, therapeutic tissue engineering products, human cell and tissue products, and combination products. The second draft guidance released addresses the expedited approval process for regenerative medicine therapies for serious or life-threatening diseases or conditions. It clarifies details to expedite the application for regenerative medicine therapies and the new RMAT designation program.4

Both of these draft guidance documents have a 90-day comment period prior to the FDA issues final guidance documents.

Regenerative Medicine is the Future of Treatment

“Given [regenerative medicine’s] rapid growth, dynamism and complexity, this field has also presented unique challenges to researchers, health care providers, and the FDA. We need to provide a clear, efficient pathway for product developers while making sure that we meet our obligation to help ensure the safety and efficacy of these medical products so that patients can benefit from these novel therapies,” explains Dr. Gottleib.

He goes on to say, “but the rapid growth and promise of this field have increasingly sowed the ground for the entry of some unscrupulous actors, who have opportunistically seized on the clinical potential of regenerative medicine to make deceptive claims to patients about unproven and, in some cases, dangerous products. By exploiting the lack of consumer understanding of this area, as well as the fear and uncertainties posed by the diseases these bad actors claim to treat, they’re jeopardizing the legitimacy and advancement of the entire field.”

Dr. Joshi said, “These comments are spot on and, and I could not have said it better. Our stem cell program was the first of its kind in Illinois, and I have been very outspoken about my concerns for the potential charlatans in this field. We are now seeing non-physicians taking out full-page ads in newspapers and 30-minute infomercials on television promoting stem cells, and they are not even harvesting stem cells. This situation is completely delegitimizing the field of regenerative medicine."

"It is so crucial that the FDA provides some type of guideline to prohibit this practice," he said. Dr. Joshi went on to urge the regenerative medicine community to learn from the past mistakes of the pain management community.

Challenge Will Come In Regulating Practitioners

“In the 1990’s, various societies and accrediting bodies had the opportunity to require advanced education and training in Pain Management, such as an accredited fellowship. Instead, they made the fatal mistake, literally and figuratively, of opening up the pain management field to anyone," Dr. Joshi said. 

“This laxity has contributed to irresponsible, careless, and fraudulent procedures and prescribing and it has created problems for legitimate pain practitioners and patients.” In addition, “while these guidelines are helpful, and many pain practitioners are already following them, the biggest source of the problem has arisen from an area not directly addressed by the FDA, which is that there are non-physicians offering regenerative treatments that are tantamount to counterfeit products and services,” he said.

“Pain management is an amazing field of medicine, as is regenerative medicine. Both fields have already suffered credibility issues. Going forward, we need to be very specific about who is permitted to provide these services or the future of both fields will be in jeopardy,” Dr. Joshi said.

Last updated on: December 19, 2017
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