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FDA issues a statement on new policy and enforcement steps to ensure proper oversight of stem cell therapies and regenerative medicine in the wake of two incidents involving unauthorized stem-cell treatment activity.
On August 28, 2017, FDA Commissioner Scott Gottlieb, MD, issued a statement on FDA’s new policy steps and enforcement efforts to ensure proper oversight of stem cell therapies and regenerative medicines. The statement comes after two incidents of unauthorized stem-cell treatments investigated by the agency.
In his statement, Dr. Gottlieb acknowledged that cell therapies and their use in regenerative medicine is one of the most promising new fields of science and medicine. These new technologies, which are largely in early stages of development, offer the advantage of transforming the treatment landscape for some of the most difficult-to-treat diseases. Recent advances in the basic knowledge of the pathways involved in tissue damage and regeneration have combined with progress in adult stem cell biology, bringing the development of medicines to a crucial point.
“The prospect of clinical tissue repair strategies is a tangible reality. This promise is reinforced by the strong commitment of the investment and scientific communities in exploring the potential applications across a wide range of vexing diseases and conditions, such as cancer, Parkinson’s disease, and diabetes, among many others,” Dr. Gottlieb said in his statement.
The FDA commissioner also pointed out the risks associated with the development of these types of therapies. “However, with all of the medical potential, also comes novelty and uncertainty as this field matures. There are a small number of unscrupulous actors who have seized on the clinical promise of regenerative medicine, while exploiting the uncertainty, in order to make deceptive, and sometimes corrupt, assurances to patients based on unproven and, in some cases, dangerously dubious products. These dishonest actors exploit the sincere reports of the significant clinical potential of properly developed products as a way of deceiving patients and preying on the optimism of patients facing bad illnesses. This puts the entire field at risk,” he said in his statement, referencing two recent incidents involving the unauthorized use of unproved medicines to treat patients.
“In the last few days alone, [FDA] has taken steps in Florida and California to address a number of especially troubling products being marketed,” Dr. Gottlieb said in his statement.
FDA is currently investigating two separate incidences of unauthorized use of stem-cell products. In Florida, the agency issued a warning letter to US Stem Cell Clinic (Sunrise, FL) for marketing stem cell products without FDA approval and for significant deviations from current good manufacturing practice (CGMP) requirements. In California, the agency halted unproved stem-cell cancer treatments administered by StemImmune (San Diego, CA) to California patients. FDA seized military-grade vaccines from StemImmune that are unavailable commercially and which were being used to create the stem cell product.
“[These] are examples of a larger pool of actors who claim that their unproven and unsafe products will address a serious disease, but instead put patients at significant risk. We will seek to take additional actions in the coming months as we address this field, and target those who are clearly stepping over the line, at the same time that they create a potential danger to patients. We have examples where some of these unproven treatments have clearly harmed patients,” Dr. Gottlieb said in his statement.
“As the agency responsible for ensuring these therapies are safe and effective, I will not allow these activities to go unchecked. I’ve directed [FDA] to launch a new working group to pursue unscrupulous clinics through whatever legally enforceable means are necessary to protect the public health,” he added.
Properly developed products will be harder to advance if bad actors can assert unproven claims and market products based on unsafe science, he noted. In this environment, bad actors can promote unproven, clearly illegal, and often expensive treatments that may pose health and safety risks and which operate against FDA’s legal and regulatory framework.
“At the same time, it’s incumbent upon [FDA] to make sure that this existing framework is properly defined, with bright lines separating new treatments that are medical products subject to FDA’s regulation from those therapies that are individualized by surgeons in such a way that they are not subject to FDA regulation. The field of regenerative medicine, because of the very nature of the science and the rapidly evolving clinical developments, not infrequently lends itself to often close calls between what constitutes an individualized treatment being performed by a doctor within the scope of his medical practice on the one hand, and what constitutes a medical product that is currently subject to the authorities Congress has already charged FDA with exercising,” Dr. Gottlieb said in his statement.
“[While] we take steps to prevent unscrupulous actors from being able to deceive patients and potentially harm their health, we also need to make sure that the vast majority of responsible product developers know where the regulatory lines governing this new field are drawn. FDA must advance an efficient and least burdensome framework as a way to help new products remain compliant with the law through a regulatory structure that does not become a barrier to beneficial new innovation,” he added.
FDA is stepping up its enforcement activity in this area to ensure that it is separating the promise of these newer therapies from unscrupulous claims. The agency will advance a new comprehensive policy framework this autumn that will establish clearer lines around when these regenerative medicine products have sufficient complexity to fall under the agency’s current authority. The agency will also advance a new policy that will define an efficient process for how these products should be evaluated for safety and effectiveness. The policies will be set forth in a series of guidance documents based on a public process.
“We want to facilitate innovation. We seek a regulatory process that accommodates the complexity of developing these therapies, and takes measure of their tremendous and near-term potential,” said Dr. Gottlieb in his statement. “As we work to protect Americans from the bad actors, I’m equally committed to doing all we can to help bring to patients more quickly innovative, scientifically proven regenerative cell therapies. For this reason, we’re developing a comprehensive and efficient, science-based policy with the aim of accelerating the proper development of these products.”
FDA’s new policy will build upon the agency’s current risk-based, flexible regulatory framework and will also serve to implement provisions related to regenerative medicine in the 21st Century Cures Act.
FDA will also issue a compliance policy that will give current product developers a reasonable timeframe to interact with FDA to determine if a marketing authorization application needs to be submitted. The policy will also give developers the opportunity to come into the agency to work on a regulatory pathway toward approval. FDA will also be developing a new approach to product approval that will allow small product developers to gain all the benefits of the agency’s approval with less burden and cost.
In addition, FDA will continue to work with industry to find other ways to facilitate bringing new therapies to patients as quickly and as safely as possible. This will include a continued commitment to fully implement the regenerative medicine advanced therapy (RMAT) designation. This pathway enables regenerative cell therapies to access FDA’s existing expedited programs to help foster the development and approval of these products. Among other things, FDA plans to include certain gene-therapy products that permanently alter tissue and produce a sustained therapeutic benefit among the products that will meet eligibility requirements under the RMAT pathway.
“This is part of our broader commitment to pursue efforts that will advance innovation in this space. We encourage sponsors who are seeking FDA approval of their product to consider this pathway,” said Dr. Gottlieb in his statement.