FDA Faces Challenges With Experimental Drug Law

The House recently sent a bill to the president’s desk to give terminally ill patients the right to sidestep the Food and Drug Administration to get experimental drugs. President Donald Trump is expected to sign the bill into law. That’s where it starts to get tricky for the FDA.

Under the legislation, known as “right to try,” people with life-threatening illnesses who have exhausted other treatment options and can’t participate in a clinical trial would be allowed to take an unproven medicine without the FDA’s approval and with little oversight.

The agency has an expanded access program that already OKs nearly all requests for drugs that have only reached the first stage of approval, but it often also suggests ways doctors can simply adjust how they’re already treating their patients.

Importantly, the FDA has information about how drugs, even the experimental ones, are being used.

Under the federal right to try bill passed May 22, drugmakers must submit an annual report to the FDA about which unapproved medicines they’ve made available to those who need them and the agency would post summary reports on the information.

Researchers worry that the agency won't have a clear, real-time picture of how some medicines are being tested and used.

“The FDA has been very useful in simply checking to see if there's a clinical trial useful for you or suggesting ‘why don't you try something else,’ ” Christopher Robertson, professor and associate dean for research and innovation at the University of Arizona, told me. “FDA has proprietary data and a much-broader perspective than any one physician can have.”

Rep. Greg Walden (R-Ore.), the chairman of the House Energy and Commerce Committee who also led an ultimately failed effort to pass a revised version of the right to try bill that included stricter reporting requirements, told me he may prefer his version, but he’s confident the FDA can maintain its role overseeing drug approvals.

“I know Dr. Gottlieb can implement it in a way that protects patient safety as necessary and follows through on the president's commitment to right to try,” Walden said, referring to Scott Gottlieb, the FDA commissioner.

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