Delaney Szwed  

Associate Editor  

Loyola University Chicago School of Law, JD 2024 

When it comes to the regulation of a treatment option intended for individuals diagnosed with life threatening diseases, one may think that the Food and Drug Administration (FDA) is heavily involved. However, this is not the case under the Right to Try Act, which became effective in May 2018. Under this act, a patient with a serious or life threatening disease is allowed access to unapproved new drugs currently going through phase two of a clinical trial. Therefore, the FDA does not approve or review requests for Right to Try Act use, and treatment and distribution is handled exclusively between the treating physician and drug industry. While the lack of FDA oversight is intended to expedite the process of getting medicine in the hands of those who need it most, the trivial regulation might cause additional harm to patients exercising the Right to Try Act.   

Purpose of the Right to Try Act

Right to Try legislation was initially only at the state level. Consequently, only terminally ill patients living in states where the legislation passed had access to unapproved treatments. However, the Right to Try Act was recently passed on the federal level, granting all eligible patients and manufacturers permission to partake in the unapproved drug distribution. 

The initial purpose of the Right to Try Act was to permit terminally ill patients access to investigational drugs[SMK1] , which are those that have not yet been approved by the FDA. Eligible patients are those who have been diagnosed with a life threatening disease, have exhausted all other treatment options, and are unable to partake in a clinical trial due to their condition. Eligible drugs that may be distributed to eligible patients under the Act are those that have already completed phase one of a clinical trial and have an active investigational new drug application ongoing with the FDA. 

FDA involvement 

Because the Right to Try Act grants minimal authority to the FDA to regulate this investigational drug process, if a patient decides they would like pursue the treatment route, the arrangement and any treatment and distribution is completed solely between the patient, treating physician, and manufacturer. Under these circumstances, the patient is trusting the physician on their treatment recommendations, and the physician is relying on the manufacturer that the drug or treatment is safe for the patient. Nonetheless, the sponsor is required to annually submit to the FDA a summary regarding the number of doses supplied, number of patients treated, and any known adverse events. This information is then made public on the FDA website. 

Potential harms

The lack of FDA authority under the Right to Try Act has the potential to put already fragile individuals in a more vulnerable position for a number of reasons.

First, patients may possess limited understanding about the unapproved treatments they will have access to through the Right to Try Act. In this scenario where a patient agrees to receive treatment from an unapproved drug, the patient’s consent is replacing that of the FDA’s. However, the clinical trial process is extremely complex and necessary patient consent documents are complex and may be overwhelming to an individual in a critical state. Terminally ill patients might be easily persuaded by their physician to receive treatment from an unapproved drug with the hope it will improve their condition without completely understanding potential side effects. Furthermore, no entity exists beyond the treating physician and manufacturer to verify the consent form is accurate, so it is impliedly assumed those actors are complying with logistical aspects of the process. 

Second, the Right to Try Act eliminates any liability to manufacturers and physicians in the event a patient is harmed by an unapproved drug. So, in the event a patient experiences adverse side effects or death caused by the unapproved drug, the patient or the patient’s estate cannot sue. This is a large concern, because Furthermore, this part of the act eliminates any worries the manufacturer may have that a potential adverse effect would inhibit their ability to get their drug eventually approved by the FDA, as this data would not be considered in their approval process. This aspect of the RTA may appear favorable to manufacturers who are willing to take advantage of physicians and patients through foregoing compliance protocols in an attempt to expand their business. 

Finally, one of the main purposes of the FDA is to protect public health through adequate regulation of drugs. However, the Right to Try Act is arguably hypocritical to this FDA goal, since it assumes that a drug’s toxicity and adverse side effects will be discovered solely through phase one of a clinical trial. However, some manufacturers and physicians may only be able to provide an accurate assessment of a treatment or drug after it has passed through more clinical trial phases. This has the potential for consumers to not only lose trust in the FDA’s established clinical trial process, but also for them to question their protection under the FDA. 

Overall, the Right to Try Act gives hope to terminally ill patients who are desperate to try any treatment that may improve their health. However, compliance problems arise when manufacturers and physicians have little regulation over their actions and face zero to no liability. While the purpose of the act is to eliminate FDA oversight so terminally ill patients can get medicine quicker, further compliance oversight must be implemented.