A new paper published on JAMA by Peter W. Marks and Stephen Hahn of FDA
The products administered by clinics under the broad rubric of regenerative medicine include those derived from individuals’ own bone marrow or fat, those derived from birthing tissues such as placenta or cord blood obtained from a donor unrelated to the recipient, as well as products that are secreted or derived from unrelated donor cells.
Despite assertions by some individuals to the contrary, these products, whether autologous or allogeneic, are not inherently safe and may be associated with serious adverse consequences. This is particularly true for those products that are not manufactured consistent with current good manufacturing practice. Lapses in good manufacturing practice are likely responsible for numerous serious bacterial infections requiring hospitalization. Such lapses may also be responsible for noninfectious complications due to substances introduced during the manufacturing process, as potentially was the case for 3 individuals who developed blindness following treatment with unproven and unapproved stem cell products.
The increasing number of adverse events being reported following the widespread use of unapproved regenerative medicine therapies at hundreds of clinics across the country make it necessary for the FDA to act to prevent harm to individuals receiving them.
in addition to consulting with their primary health care clinician, prior to considering cellular therapies, patients should ask if the therapy is approved by the FDA. If it is not, they should ensure that an active Investigational New Drug application is on file with the FDA, and they should expect to review and sign an informed consent.
Patients and their families also should not expect to be charged for investigational products they receive. An exception is cost recovery, whereby the sponsor provides evidence from a clinical trial to the FDA that a product may provide clinical benefit and the sponsor is then permitted to charge for manufacturing of the product but may not make a profit. Patients participating in clinical research should also expect to receive information regarding the results of the trial in which they have taken part. If questions remain, clinicians should consider contacting the FDA directly with their questions or concerns.
Appropriate Practices for the Investigation of Unproven Regenerative Medicine Therapies
- Active Investigational New Drug (IND) application for the specific product in development is on file with the Food and Drug Administration
- Requirement for the provision of written informed consent in an institutional review board–approved clinical trial under an IND
- No charge is requested from the patient for the unapproved product or for participation in the clinical trial
- Reporting of potential adverse events is encouraged and clear mechanisms are provided on how to do so
- A summary of results is reported back to those enrolled in the clinical trial
It is time for unproven and unapproved regenerative medicine products to be identified and recognized for what they frequently are: uncontrolled experimental procedures at a cost to patients, both financially and physically. Patients and their caregivers should feel empowered to report adverse events to help make sure that purveyors of unproved products are identified, and the FDA can take appropriate action to bring them into compliance and thereby help protect more patients from harm. This goes to the core of the mission to which the FDA is committed: promoting and protecting the public health.