The Federal Right-to-Try: Is Removing FDA Authority Necessary?

By Kimberly Tuttle (CMC ’19)

On May 30, 2018, President Trump approved legislation that made the right-to-try federally legal. The right-to-try gives terminally ill patients the option to take non FDA-approved drugs for investigational treatment purposes. Before becoming federally legal, the right-to-try was permitted in 41 states. The program was first introduced in Colorado in 2014, though the law took years to gain traction federally due to stalls in the state legislature over concerns regarding patient safety and the genuinity of its investigational purposes. Today, terminally ill patients are utilizing their right-to-try. As concerns over public safety and the future of drug development remain at hand, it is important that citizens, doctors, and pharmaceutical companies alike are  aware of the law’s costs and benefits to ultimately make life better for patients.

To be eligible for the right-to-try, a patient must be diagnosed with a life-threatening disease or condition, have exhausted approved treatment options, be unable to participate in a clinical trial involving the eligible investigational drug, as certified by a doctor who is in good standing with their licensing organization and will not be compensated directly by the manufacturer, and given written informed consent regarding the risks associated with the investigational treatment.

The drugs and treatments that qualify for the right-to-try must also meet specific conditions. It must have completed an FDA-approved Phase 1 clinical trial, be in an active clinical trial that has been filed with the FDA or have intent to be filed with the FDA, and be in ongoing active development or production.

The FDA requires each drug to undergo three clinical trial phases before approving it for public use. A study by MIT in 2018 shows that roughly 14% of all drugs in clinical trials make it to the market, whereas approximately 70% of Phase 1 drugs move to the next phase. With the right-to-try, terminally ill patients have access to considerably more treatment options that do not require approval from the FDA.

A common misconception is  that expanded drug access is a byproduct of the right-to-try. Access to Phase 1 drugs has been available within the bounds of the FDA since the 1980s through its expanded access program, which is also called Compassionate Use. This program grants approval of investigational medicine to terminally ill patients. 99% of the applications submitted for Compassionate Use are granted. Emergency requests are typically granted over the phone instantly and non-emergency requests are typically approved in a few days.

The main distinction between the right to try and Compassionate Use is the involvement of the FDA. Supporters of the right-to-try argue that terminally ill patients should have complete autonomy over their medical choices and should be free to choose the outcome of their future without the constraints of the government. As a result of this rudimentary ideology surrounding free will and individual autonomy, the right to try is often classified as a libertarian movement.

Yet even the CATO Institute, a prominent libertarian think tank has criticized the feasibility of the right to try. The institute argues that even though the right to try is a step in a positive direction, the progress towards individual liberty will not last due to the public’s harsh criticism of procedural errors and the potential result of the inhibition of drug development. In other words, it is inevitable that some terminally ill patients will experience complications from the developmental drugs. As a result, clinical studies for developmental drugs could be jeopardized.

For instance, trial volunteers might reconsider their participation if there are reports of a devastating side effect with another patient. Similarly, investors may pull their support from a company if one of its medicines is tied to an unexpected complication, hindering future development. Thus, the right-to-try’s potential impediment on drug development limits individuals’ discretion in the future when making medical decisions.

Critics of this law do not appear to be questioning the individual autonomy of terminally ill patients, but rather the reliability of the safeguard to the public health system. FDA oversight through the Compassionate Use program requires drug experts to oversee treatment and disclose to formalized reporting requirements.  Whereas, the-right-try gives creates oversight of the FDA by giving doctors and pharmaceutical companies the power to administer developing drugs independently. This increases the risk factor of the drugs, since they are being administered without the strict standards of the FDA. Typically, the entities involved in the clinical trials are most knowledgeable and can advise how to administer the drug, though this safeguard is removed with the right-to-try.

Supporters tend to respond to critics by emphasizing the fundamental right of a person to choose for themself. While this right is deeply embedded in American society, it is still important to consider the long-term implications of removing the FDA from this medical process. How does one quantify the risks of stunting drug development and potentially taking lives against the potential to save lives of terminally ill patients?

The right-to-try presents a clear moral dilemma that is often debated in public health discourse. Is it worth it to tend the urgent needs of one person to slow the development of medicines for everyone else? As the right-to-try law matures, researchers will be able to quantify the answer to this question by measuring the exact impact on drug development. For now, it is critical that Americans stay engaged and consider the efficacy of both the right-to-try and Compassionate Use.

 

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