Federal ‘right-to-try’ bill could undermine drug safety regulation

Jan. 12--Federal legislation that aims to give desperately ill patients greater access to unapproved drugs reflects a broader effort to weaken medical product regulation, two University of Pennsylvania bioethicists argue in a prominent medical journal.

The federal "right to try" bill, which passed the Senate in August, is being pushed by supporters in the House, and has the backing of President Trump and Vice President Pence. It entitles patients with life-threatening illnesses to try experimental drugs that have passed preliminary safety tests, without the U.S. Food and Drug Administration's traditional oversight of such "expanded access" requests.

Right-to-try laws have passed in 38 states, including Pennsylvania, championed by patient groups and politicians. But policy experts, physicians and the pharmaceutical industry say the laws are misleading because they imply that eliminating the FDA's review will guarantee access -- even though manufacturers aren't obligated to provide requested drugs. Historically, drug developers, not the FDA, have refused access for reasons ranging from a limited drug supply to fear of bad publicity if a patient is harmed.

In an opinion piece in the latest New England Journal of Medicine, Penn bioethicists Steven Joffe and Holly Fernandez Lynch, point out that the FDA promptly approves "virtually all requests" after manufacturers agree. The agency also has streamlined the application form that the patient's physician has to submit.

"By cutting the FDA out of the process, however, the bill would cause patients to lose the benefit of meaningful safety-oriented changes that the agency recommends in 10 percent of expanded access approvals," the bioethicists wrote. For example, the FDA may lower the planned dosage based on confidential drug-development information that isn't available to the patient's physician.

Overall, the Penn experts say, the impact of a federal law would probably be small because it does nothing to induce companies to be more compassionate.

Even so, they see the legislation as dangerous. It would do what state laws don't have the constitutional power to do: override the FDA's regulatory role. And it would bolster "broader efforts to weaken medical product regulation."

They noted that the Goldwater Institute, the libertarian think tank that is behind the right-to-try movement, has launched a new campaign that would let manufacturers promote approved drugs for unapproved, or "off-label," purposes. While the FDA gives doctors discretion to prescribe off label, companies are barred from marketing drugs for new uses -- say, a schizophrenia drug for dementia -- without first proving effectiveness. Violators of the rule have paid billions in fines.

The FDA's new commissioner, Scott Gottlieb, appears to be in favor of loosening off-label marketing restrictions, the bioethicists wrote.

"Are we prepared to abandon the FDA's gatekeeping role in favor of unfettered patient autonomy and market forces, risking precisely the problems that prompted Congress to grant the FDA its present authority?" they ask.

"We diminish the FDA's public health mission," they conclude, "at our peril."

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