Viewpoint: 4 ambiguities FDA should address about Right to Try

President Donald Trump signed the Right to Try Act into federal law May 30, giving patients with life-threatening conditions the opportunity to access investigational drugs without FDA authorization. To ensure this law provides the maximum benefits for patients, the FDA should provide clarity over several ambiguous requirements outlined in the law, three health experts argued in an op-ed published in The Journal of the American Medical Association.

Here are the op-ed's authors:

  • Holly Fernandez Lynch, PhD, assistant professor and assistant faculty director of online education within the department of medical ethics and health policy at Philadelphia based-University of Pennsylvania Perelman School of Medicine
  • Patricia Zettler, PhD, associate professor of law at Georgia State University in Atlanta
  • Ameet Sarpatwari, PhD, associate epidemiologist at Boston-based Brigham and Women’s Hospital and assistant director of the Program On Regulation, Therapeutics and Law, a research group affiliated with Boston-based Harvard Medical School and Brigham and Women's Hospital

Here are four ambiguities the FDA should address in the Right to Try Act, according to the authors:

1. What patients are eligible? For patients to be eligible for the Right to Try path, they must be unable to participate in a clinical trial involving the unapproved drug. "Defining such inability to include only patients who do not meet the eligibility criteria for a trial within a reasonable geographic vicinity will avoid interference with clinical trial enrollment," the authors wrote.

2. What drugs are eligible? For drugs to be eligible for Right to Try, they must have been assessed through a phase 1 study, and demonstrate active ongoing drug development. However, this requirement can be ambiguous, since trials can vary in design and many do not progress to phase 2 trials. The authors said the FDA should clarify these guidelines to ensure only drugs capable of progressing to further clinical trials are eligible for Right to Try.

3. What are the reporting requirements? Right to Try requires the drug manufacturer or sponsor to annually report the "number of doses supplied, the number of patients treated, the uses for which the drug was made available and any known serious adverse events," the authors wrote. The FDA should use this reporting requirement to also collect information on patient demographics, if they were charged for the drug, etc., to help assess the law's implementation and identify ways to further help patients, according to the authors.

4. How does the act interact with Expanded Access? The authors urged the FDA to facilitate Expanded Access as the preferred pathway for accessing experimental drugs outside clinical trials. To subdue drugmakers' concerns about Expanded Access, they argued the FDA should formalize its current practice of using clinical outcomes only when requested by a sponsor or when necessary for reviewing a drug's safety.

"[T]hese recommendations may help prevent the rise of a market in unproven therapies and preserve the ability to collect critical information about investigational drugs, while enabling access as the law intended (but did not guarantee)," the authors concluded.

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