Despite Right-to-Try Laws, Most Drug Makers Still Require FDA Reviews

The “Right-to-Try” law, which President Donald Trump signed in May 2018, allows more direct access to experimental drugs for terminally ill patients who have exhausted available treatment options and who do not qualify for a clinical trial, and does not require drug makers to seek endorsement from the U.S. Food and Drug Administration (FDA).

However, according to a report from the Government Accountability Office, 13 of 29 drug makers would prefer that regulators review requests to experimental treatments before they provide the medications to patients. Six of the 29 companies indicated they would ask the FDA to review, and five said they would require input from an institutional review board or research ethics committee.

The drug companies queried in the GAO report would prefer to go through the FDA’s expanded access program. Under this mandate, physicians submit requests to pharmaceutical companies and the FDA signs off on providing medications. Right-to-Try removes the endorsement step with the intention to expedite the process, but companies are hesitant to provide treatments without their sign-off for fear that unexpected adverse reactions to the investigational medications might endanger future FDA approvals.

“Right-to-Try continues to get a lot of attention, but hasn’t really changed anything,” said Arthur Caplan, PhD, head of the division of medical ethics at the New York University School of Medicine. “It still remains up to a company to make drugs available, not a doctor’s request or patient lobbying or advocacy. And companies still want to work with the FDA. They think the FDA has perspective or inside information about a dose or when to use a drug.”

Source: STAT, September 12, 2019.