Right to try legislation: Questions surround its substance

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A bill commonly known as “Right to Try,” or S.204, appears to be a step in the right direction for expanding access to treatment for terminally ill patients who have exhausted all other options. The new legislation is designed to allow these patients to seek treatment with experimental medications not yet available to the general public.

Indeed, terminally ill patients would seem to have little enough left to lose to warrant suspension of the rules that prevent ineffective or harmful drugs from entering the market. However, it’s unclear if the legislation can improve patients’ access to experimental treatments over the status quo, while it may remove important oversight of experimental treatment access by the FDA.

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Expedited access to experimental treatments not yet approved for use by the public has its roots in the scramble to provide treatment to individuals suffering from AIDS, the diagnosis of which was not known at the time. Azidothymidine (AZT) had been approved to treat AIDS patients, but the therapy had not yet undergone trials for patients whose HIV had not progressed. With the consequences of disease progression looming large, and without efficacious alternatives, HIV patients sought AZT outside of “traditional” channels.

As told by the James Baker Institute’s report on “The Impact of Right to Try Laws on Medical Access in the United States,” this prompted the FDA to create its “compassionate care” program, which exists today under the names “Expanded Access” or “Compassionate Use.”

This existing program within the FDA already provides terminally ill patients with access to experimental treatments that are not otherwise available, and does so at an impressive rate. The FDA reports that it approves more than 99% of the requests it receives for compassionate use.

Next: So what exactly does the Right to Try bill do that is distinct from the FDA’s current program?So what exactly does the Right to Try bill do that is distinct from the FDA’s current program? Primarily, the legislation opens a pathway for patients to access treatments that have only completed the first phase of trials, a stage at which the FDA does not grant access within its compassionate use program. It is unclear that this is meaningfully different from the status quo.

To date, over 30 states have passed laws mirroring S.204, and there has yet to be a single documented case of a patient receiving an experimental treatment outside of the FDA’s compassionate use program according to a report by Lisa Kearns and Alison Bateman-House (Therapeutic Innovation & Regulatory Science 2017; 51:170-6). Indeed, while the federal Right to Try legislation provides patients the ability to seek access to phase I experimental treatments, it does not provide any means to compel pharmaceutical companies to provide those treatments.

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Despite assurances present within existing Right to Try laws that pharmaceutical companies would be absolved of liability from resulting adverse consequences, they remain skeptical that these assurances will hold. Drugs in phase I trials pose significant risk of adverse events, and documented instances among Right to Try patients may deter patients from participating in actual clinical trials that serve as a prerequisite for use by the general public.

In allowing patients to seek experimental treatment outside the auspices of FDA oversight in its compassionate use program, the law may pose risks for patient safety. The federal Right to Try legislation allows patients to bypass certain safeguards, such as adjusting dosages and increasing safety monitoring. These safeguards are part of the compassionate care program using confidential information garnered from the ongoing trials process that would not be available to individual patients or their physicians.

The true impact of the law remains to be seen as patients, physicians, pharmaceutical companies, and lawmakers navigate its provisions.