By A. Rahman Ford, Columnist
For the second straight year, President Trump has endorsed making life-saving treatments like stem cell therapies more available to more Americans.
In his 2017 Joint Address to Congress, Trump highlighted the case of Megan Crowley, a young woman whose father had to launch his own drug company to help treat her Pompe Disease. Also in attendance that evening was Sarah hughes, who was forced to travel to Mexico to use her own stem cells to treat her systemic idiopathic juvenile arthritis.
In reference to both cases, the president lamented the pain and death caused by the “slow and burdensome approval process at the Food and Drug Administration” that “keeps too many advances … from reaching those in need.” He argued that regulatory restraints at the FDA should be “slashed” so that more Americans could benefit from life-saving therapies.
President Trump is keeping up the pressure. During this week's State of the Union address, he continued his theme of a patient-centered, less restrictive approach to medical treatment.
He did so by voicing his clear support for “Right to Try” legislation, which would increase the medical options of the critically ill by helping them avoid the unduly burdensome and bureaucratic spider’s web of the FDA.
In a seeming reference to Sarah Hughes and other stem cell medical tourists, Trump stated unequivocally that “patients with terminally conditions … should have access to experimental treatments immediately” and they “should not have to go from country to country to seek a cure.” He then urged Congress to pass the Right to Try Act, so that Americans can get help “right here at home.”
How Right to Try Works
The language of the Right to Try legislation is simple, straightforward and offers protections for patients and manufacturers. Under the Senate version, an “eligible patient” who has been diagnosed with a terminal illness may be prescribed an experimental drug or biological product to treat their illness, so long as the patient has a qualified physician certify that he or she has exhausted all other treatment options and is unable to participate in a clinical trial. The patient must also provide informed consent to the physician and the physician may not be compensated by the manufacturer of a treatment for certifying the patient. The patient, physician and manufacturer must all agree on the treatment.
Furthermore, the medical product in question must have successfully completed a Phase 1 clinical trial and must be enrolled in an FDA clinical trial. The treatment must be authorized by state law, which means that the state must have a Right to Try law – which 38 states currently have.
The manufacturers receive protection under Right to Try legislation, in that there can be no legal liability for injury that may result as a consequence of the medical product’s use, and adverse events that may occur during treatment will not negatively impact any eventual approval of the product by the FDA.
In an overwhelming and increasingly rare bipartisan display (94-1), the Senate has already passed the Right to Try Act. The House version is currently awaiting approval.
Critics Deny Democratic Choice
Critics of Right to Try make several claims to undermine the expansion of choices it would bring to critically ill patients. Some physicians and medical ethicists claim that the true goal of Right to Try is to weaken the FDA as the only objective and appropriate gatekeeper of drug approval and access. Some also claim that the legislation is redundant because the FDA already fills this need through its expanded access program.
Still other critics try to dissuade patients by surreptitiously noting that “scary” conservative and libertarian think tanks like Freedom Partners and Americans for Prosperity, which are partially funded by the Koch brothers, favor passage of Right to Try legislation. These criticisms warrant thoughtful consideration, but are not substantive enough to overcome overarching concerns of patients literally dying from their pain.
Ultimately, Right to Try and stem cell therapy are issues that embody the deepening and broadening of healthcare choice -- a choice that should be embraced by an informed American citizenry, a forward-thinking medical establishment and government agencies that must be by and for the people.
Carefully curated expansions of choice -- that privilege the humane while also giving due consideration to patient protection – serve as the foundation of all truly democratic institutions. The FDA should accept that it can better serve people by acceding some of its authority and become more lean and nimble in the process. Bigger is not always better.
Right to Try will not solve all the problems associated with stem cell therapy. There is no way to predict with any precision how the law will operate legally or logistically, whether for stem cell therapies or other drugs and medical products. Additionally, the Trump administration must revisit and revise the FDA’s stem cell guidance, specifically its limits on stem cells which are harvested, processed and administered to the same person to relieve conditions such as chronic pain.
However, for advocates of stem cell therapy and health choice in general, Right to Try is a step in the right direction.
A. Rahman Ford, PhD, is a lawyer and research professional. He is a graduate of Rutgers University and the Howard University School of Law, where he served as Editor-in-Chief of the Howard Law Journal. He earned his PhD at the University of Pennsylvania.
Rahman lives with chronic inflammation in his digestive tract and is unable to eat solid food. He has received stem cell treatment in China.
The information in this column should not be considered as professional medical advice, diagnosis or treatment. It is for informational purposes only and represent the author’s opinions alone. It does not inherently express or reflect the views, opinions and/or positions of Pain News Network.