Statement from FDA Commissioner Scott Gottlieb, M.D. on the FDA’s new policy steps and enforcement efforts to ensure proper oversight of stem cell therapies and regenerative medicine

FDA Statement

Statement from FDA Commissioner Scott Gottlieb, M.D. on the FDA’s new policy
steps and enforcement
efforts to ensure proper oversight of stem cell therapies
and regenerative medicine

For Immediate Release

August 28, 2017


One of the most promising new fields of science and medicine is the area of cell therapies and
their use in regenerative medicine. These new technologies, most of which are in early stages of
development, hold significant promise for transformative and potentially curative treatments for
some of humanity’s most troubling and intractable maladies. Recent advances in our basic knowledge
of the pathways involved in tissue damage and regeneration have combined with remarkable progress
in adult stem cell biology to put us at a genuine inflection point in the history of medicine. 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.

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. Products that are reliably and carefully
developed will be harder to advance if bad actors are able to make hollow claims and market unsafe
science. In such an environment a select few, often motivated by greed without regard to
responsible patient care, are able to promote unproven, clearly illegal, and often expensive
treatments that offer little hope, and, even worse, may pose significant risks to the health and
safety of vulnerable patients. These so-called treatments run afoul of the FDA’s
legal and regulatory framework governing this new field.

At the same time, it’s incumbent upon the FDA to make sure that this existing framework is properly
defined, with bright lines separating new treatments that are medical products subject to the 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 the FDA with exercising.

For example, sometimes when cells or tissues are taken from and given back to the same individual
or when the cells or tissues do not undergo significant manufacturing, are intended to perform the
same basic functions, and are not combined with another drug or device, among other factors; their
benefits and risks are well understood. In these circumstances, the products may not require
premarket review under current law. However, when significant manufacturing is performed on the
cells or tissues, or when the cells or tissues are not intended to perform the same basic
functions, far greater uncertainty exists as to the benefits and risks involved. In these cases,
it’s necessary to understand the benefits and risks in clinical trials prior to widespread use of
the products. Therefore, premarket review is required.

At the same time that 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.
The 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.

To make sure the agency is separating the promise from the unscrupulous hype, we are stepping up
our enforcement activity in this area. At the same time, this fall the FDA will advance a
comprehensive policy framework that will more clearly describe the rules of the road for this new
field. This comprehensive policy is based on our existing authority. It will offer responsible
product developers – including individual providers working in clinics and academic hospitals and
advancing their own products as part of regenerative medicine procedures – a way to more
efficiently gain FDA approval for their products through a process that is minimally burdensome and
less costly.  Many of the individualized treatments fall clearly outside the FDA’s pre-market requirements. For
those that currently fall across the line and are subject to the FDA’s existing pre-market review,
we want to make sure the process for gaining FDA approval is efficient. 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.

Stepped Up Enforcement

In terms of compliance, and with regard to our increased oversight and enforcement:
In the last fewdays alone, the FDA has taken steps in
Florida(/NewsEvents/Newsroom/PressAnnouncements/ucm573431.htm) and California
(/NewsEvents/Newsroom/PressAnnouncements/ucm573427.htm) to address a number of
especially troubling products being marketed. But unfortunately, 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.

As the agency responsible for ensuring these therapies are safe and effective, I will not allow
these activities to go unchecked. I’ve directed the FDA to launch a new working group to pursue
unscrupulous clinics through whatever legally enforceable means are necessary to protect the public
health. Late last week, FDA worked with the United States Attorney to ask a court to seize the components
of a product that involved the use of vaccinia virus vaccine as part of a purported treatment for cancer that
FDA believes created the potential for substantial risks to patients. The product posed significant public health concerns
for the agency.

Efficient Regulation

With regard to our regulation of these products, I want to expand on the need for bright lines and
appropriate oversight to accommodate the good actors working on genuine science.

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.

The FDA will advance the new framework this fall. This comprehensive policy will establish clearer
lines around when these regenerative medicine products have sufficient complexity to fall under the
agency’s current authority, and then 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 that are the result of a public process we have held in recent years. The new policy will
build upon the agency’s current risk-based, flexible regulatory framework. It will also serve to
implement provisions of the 21st Century Cures Act related to regenerative medicine. The FDA has
already held public meetings to inform its thinking in these areas, so much of the agency’s
approach is already part of the public record. We’ll continue to work with industry and the
scientific community to perfect the process for bringing safe and effective treatments to patients.

At the same time, we will also issue a compliance policy that, with the exception of outliers
potentially harming public health in a significant way right now, will give current product
developers a very reasonable period of time to interact with the FDA in order to determine if they
need to submit an application for marketing authorization and to come into the agency and work on a
path toward approval. And we will also be developing a novel approach to FDA approval that we
believe will allow very small product developers to gain all the benefits of FDA approval through a
process that is minimally burdensome and less costly. We’re mindful of the significant promise
offered by regenerative medicine, the cost of innovation in this industry, the small companies
engaging in these enterprises, and the difficulty of doing FDA registration trials in this field.
Our framework will take measure of all of these challenges.

In addition, the FDA will continue to work closely with industry to find other ways to aid in the
effort to bring novel therapies to patients as quickly, and as safely, as possible. One of these
will include our continued commitment to fully implement the Regenerative Medicine Advanced Therapy
(RMAT) designation(/BiologicsBloodVaccines/CellularGeneTherapyProducts/ucm537670.htm). This pathway enables
regenerative cell therapies to access the FDA’s existing expedited programs to help foster the
development and approval of these novel products. Among other things, we plan to include certain
gene therapy products that permanently alter tissue and produce a sustained therapeutic benefit as
part of the products that will meet the definition of being eligible to come under the pathway
enabled by RMAT. 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.

Ultimately, the agency’s goal is to make sure that the potential of regenerative medicine can
continue to advance to benefit the patients who need new and innovative options for their medical
problems. These technologies hold out the potential to significantly alter the course of a broad
range of diseases. We are committed to taking steps to make sure these opportunities advance as
quickly as possible. To do so, we must put in place the framework to separate the promising
treatments from those products that pose significant risks or offer patients little to no chance of
benefit. We will also continue to take steps to keep those who would exploit this promising area
from harming patients and abusing the public’s trust. We can’t let a small number of unscrupulous actors poison
the well for the good science that holds the promise of changing the contours of human illness and
altering the trajectory of medicine and science.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public
health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines
and other biological products for human use, and medical devices. The agency also is responsible
for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products
that give off electronic radiation, and for regulating tobacco products.



Lyndsay Meyer (