The AAMC March 12 joined 85 organizations in a community letter that opposes H.R. 5247, the latest version of the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act. The legislation, authored by Rep. Brian Fitzpatrick (R-Penn.), would create a secondary pathway for accessing investigational therapies outside of clinical trials which would remove Food and Drug Administration (FDA) approval and consultation. The bill was brought to the House floor for a vote March 13 under a suspension rule, which permits non-controversial pieces of legislation to be acted upon expeditiously, provided they garner at least a two-thirds majority vote. However, the bill failed to advance by the necessary two-thirds majority. House Republicans have signaled they will bring the bill back to the floor for another vote next week.
The legislation’s alternative pathway would remove FDA approval and consultation. Currently, patients may apply for access to therapies not yet approved by the FDA through the FDA’s expanded access program. In the expanded access program, the FDA may make dosing and safety improvements to the proposed expanded use. According to the letter, the FDA repeatedly approves over 99 percent of all requests through the expanded access program. Conversely, the alternative pathway contemplated in the legislation would prohibit the FDA from halting access to the therapy in question short of placing a clinical hold on all clinical research on the therapy in question, thus keeping the FDA from advising on dosing, route of administration, dosing schedule, and other important safety measures.
While the letter's signers expressed appreciation that the legislation incorporates several key patient safety improvements, they noted that the legislation would not address the underlying issue of access to therapies.