The Senate approved a bill Thursday that would allow seriously ill patients in all 50 states to request access to experimental medicines without FDA approval.
Sen. Ron Johnson’s measure, S. 204 (115), would authorize the use of unapproved medicines by patients diagnosed with a life-threatening illness as long as the drugs in question have already gone through preliminary testing on humans and continue to be evaluated in research the FDA oversees. Eligible patients would have to exhaust other treatment options and be unable to participate in ongoing clinical trials.
The Senate’s unanimous approval follows a nationwide lobbying effort by the libertarian Goldwater Institute, which got similar right-to-try laws passed in 37 states. Vice President Mike Pence, who signed a right-to-try law while governor of Indiana, also has endorsed the federal effort. Reps. Andy Biggs and Brian Fitzpatrick have a companion bill pending in the House.
Johnson’s Trickett Wendler, Frank Mongiello and Jordan McLinn Right to Try Act of 2017 aroused concern from the drug industry, public health advocates and some at the FDA, who said it could undermine patient safety and drug development efforts. Drug companies wouldn’t be required to give patients medicine under the law, but it would remove a requirement that companies and physicians obtain the FDA’s sign off to treat patients with experimental medicines.
The measure is broader than some state laws, because it covers patients with serious but not imminently deadly conditions, such as muscular dystrophy.
Johnson (R-Wis.) threatened to hold up a five-year reauthorization of the FDA user fee programs if he didn’t get a vote on the bill. Senate leaders and the Senate HELP Committee struck a compromise under which Johnson got a vote on a revised version of his bill, separate from the FDA user fee bill, which was expected to be approved Thursday.
"We tried to run it by everybody who was affected," HELP Chairman Lamar Alexander said. He added that Johnson did "a skillful job of legislating" to get a result.
The compromise included major changes to Johnson’s original proposal to satisfy skeptics’ concerns. Among other tweaks, the bill now requires FDA to receive reports of safety events that occur in right-to-try situations. It also forbids patients from being charged more than the cost of production for the medicines.