Thank you for the opportunity to speak today. My name is Dr. Stephanie Fox-Rawlings. I am a Senior Fellow at the National Center for Health Research. Our research center analyzes scientific and medical data to provide objective health information to patients, providers and policy makers. We do not accept funding from the drug or medical device industry. I have no conflict of interest.
Before coming to the National Center for Health Research, I worked as a developmental neuroscientist at the Children’s National Medical Center. My project was to understand how cells respond to damage, and how neural stem cells respond to changes in their environment to promote recovery. If my work in the laboratory taught me anything, it’s that cells, and especially stem cells, are extremely dynamic. They continuously react to and are modified by their environment. Small changes can greatly affect the way that cells behave. For example, exposing cells to a different growth factor or signaling molecule or even varying the oxygen level can change the number of cells and what they become.
Cells and tissues are much more complicated than drugs and biologics. They are not a simple compound or single protein that can be easily characterized in a lab test. A cell is a living, changing organism. They can move throughout the body and can make other cells change their behavior. Stem cells can even transform into new cell types. Because of this, cells and tissues have an amazing and exciting potential to heal people and cure disease.
But just as these cells have the potential to heal, they also carry the potential to harm. That’s why cells and tissues should be properly tested and regulated before widespread use in patients.
The FDA’s guidances provide a scientifically logical distinction between which cell and tissue treatments need stricter regulation and which do not. The guidances require a cell or tissue product where cells are changed or used for a new function to be clinically tested to insure that they are safe and effective. This is reasonable, because we cannot assume that they will function in this new way in this environment or that they would not do something unexpected to cause harm.
This regulatory process is equivalent to the simpler drugs and biologics. The fact that cells and tissues are more complicated does not mean that they should be less regulated. To the contrary, their increased complexity should warrant an increased need for testing.
FDA proposes less stringent regulation for cell and tissue treatments for rare diseases or for diseases that currently lack approved treatment options. Fortunately, the FDA already has mechanisms in place for reviewing those types of urgently needed treatments. But those mechanisms must not be weakened.
We don’t know how many people are helped or harmed by many of the cell therapies currently being marketed. How many of the clinics providing treatments have studies to back up their success rates or side effects? In some cases, the harms are sensational enough to make the news. But when treatments are harmful, there is little incentive to report them to the FDA. And in some cases, neither patients nor physicians will realize that a complication was caused by the treatment.
Even if a treatment isn’t dangerous, an ineffective treatment harms patients because it is so expensive. And, of course, many of these treatments offer little beyond false hope.
At worst, serious clinical side effects can occur, such as tumors and vision loss.
That’s why clinical trials are absolutely necessary. Patients should be able to make an informed decision about a treatment with information based on data and good science, not just hype and hope.
Regulations will also insure that the cells that clinics claim to use are actually the cells that are put into a patient’s body. It can insure that the chemicals used to process cells are safe for this process.
Regulation and rigorous scientific testing benefits patients now and in the future. If there are too many cases of patients harmed or too many treatments fail because some clinicians used untested treatments, the whole field could be disregarded as snake oil. Not only will patients be harmed by bad treatments, but also the failure to develop real treatments.
In conclusion, we strongly support the FDA’s regulation of cell and tissue products. The guidances are reasonable. Through regulation the FDA can protect patients and encourage innovation in the development of new treatments based on sound science. However, enforcement will be critical to stop untested and potentially harmful ‘therapies’.
Thank you for your time and consideration of our views.