The Senate has voted in quick succession to pass bills reauthorizing the FDA to collect fees from the industry and expanding access to unapproved medicines. President Trump is now set to sign the user fee bill into law, while the right-to-try legislation will go to the House.
Passage of the user fee reauthorization bill by a vote of 94 to 1 clears the FDA to continue charging drugmakers to process applications until 2022. The current version of the law is due to expire at the end of next month. And, with the FDA having said the failure to reauthorize the bill would lead to layoffs, the Senate was under pressure to pass the law before it breaks for the summer recess.
Having done so, the Senate has set up latest iterations of PDUFA, MDUFA, GDUFA and BsUFA—the prescription, medical device, generic and biosimilar user fee acts—to pass into law following a signature from President Trump. The President has previously said he wants the FDA funded solely by user fees. But Regulatory Focus reported that his office said he will sign the bill passed by the Senate, despite it falling short of his extreme proposal.
The near-unanimous vote in favor of the user fee bill continued a rare day of bipartisan support for health legislation in the Senate. Hours earlier, the Senate passed right-to-try legislation by unanimous consent. The final version of the bill allows the FDA to use the experience of patients in the right-to-try context in approval decisions if the “clinical outcome is critical to determining the safety of the eligible investigational drug.”
That provision stops a situation arising in which the FDA knows of a safety problem from patients who received a drug on a right-to-try basis but is unable to factor the information into assessments of regulatory submissions.
The bill, and broader right-to-try movement, still have the potential to undermine clinical trials if patients shun studies that feature control arms in favor of getting the drug on a right-to-try basis. That potential will only be realized if the bill significantly increases the use of drugs on a right-to-try basis. Some doubt whether that will happen.
“What [patients] have a right to (and did long before this bill) is to ask drug companies for permission to use their experimental drugs outside of clinical trials. If the drug company says no, both before and after this legislation, that's the final word: neither the FDA nor the courts have to power to make companies provide access to their experimental drugs-in-development,” Alison Bateman-House, a medical ethicist at New York University who campaigned against the bill, told Politico.
The definite consequences of the bill, should it clear the House, will include more transparency about the uptake of right to try. The bill tasks companies with summarizing and submitting details of the right-to-try use of their drugs.