On May 30th President Trump signed the “Right to Try Act” into law. This legislation lets terminally ill patients try experimental medications that have completed Phase 1 clinical trial testing but have not yet been approved by the Food and Drug Administration (FDA). On the surface, this legislation seems like a victory for patients without other alternatives. However, the value of this legislation and its impact on patient safety has recently come under fire by bioethicists and medical experts.
The libertarian think-tank Goldwater Institute precipitated the original draft of the Right to Try Act in early 2014. As their political ideology suggests, they drafted this policy in an effort to return medical decisions “back to the local level” by hobbling the FDA. A valid ethical argument the Goldwater Institute and supporters cite is that if patients have the right to die through physician-assisted suicide or voluntary euthanasia, they should also be afforded the “right to try”. This argument was accepted by Colorado later in 2014, then in 39 other states by April 2018, before being signed into federal law a month later.
As the legislation begins to be used by terminal patients, there has been an outcry from legal and health scholars. These experts point out that the law does not require physicians to prescribe experimental therapies, does not require insurance companies to cover their cost, and does not require drug manufacturers to provide the therapies. Because the law does not actually provide a right to receive these experimental treatments, just permission, the toothless legislation can give false hope to desperate patients and their families.
Even if this legislation did grant terminally ill patients the right to receive the experimental treatment, there is no data the therapy would be effective. Phase 1 (of 4) clinical trials are used to evaluate the safety of a pharmaceutical in an uncompromised, fully-functioning immune system. The rationale behind this phase is that if the pharmaceutical elicits trauma in [mainly] healthy individuals, then it will certainly be detrimental in patients on their deathbed. A Phase 1 trial does not evaluate the efficacy of a drug in the patient population, nor even conclude the safety of the drug in sick, compromised patients.
As a result, these unproven therapies can accelerate death or lead to suffering and deterioration of the original condition (e.g. 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, etc). Additionally, the lack of safety data makes informed consent on the part of the patient more difficult, because informed consent entails 1) being provided with the data-driven pros and cons of a proposed treatment and then 2) the patients making an informed decision based on the provided pros and cons.
Typically, the FDA safeguards the public from prospective health hazards. The group even has a more comprehensive policy than Right To Try called Expanded Access/Compassionate Use, where a board of pharmaceutical, ethics, and medical experts assess a treatment’s “use of investigational drugs, biologics or medical devices outside the clinical trial setting for treatment purposes” for a wider array of patients than the Right to Try covers. In fact, 99.4% of Compassionate Use requests are approved within two weeks of submission. If the government is responding in a timely and sensitive manner, it comes into question what gaps Right to Try is bridging.
While the Right to Try Act is popular with the public, most do not fully appreciate the potential consequences of its passing. Authorities are concerned this neutering of the FDA will leave patients exposed, vulnerable and erroneously optimistic. With this shared view from experts of many different fields and political backgrounds, it is fair to wonder if this deregulation will cure anything.
Madelaine Wendzik is a Ph.D. student in the Neuroscience Program at the University of Georgia studying neuroinflammation and immune response in pediatric traumatic brain injury. She enjoys board games, downloading one too many podcasts, and anything to do with white chocolate macadamia nut cookies. You can email her at MWendzik@uga.edu or follow her on twitter @SciPolicyGirl. More from Madelaine Wendzik.