Rare Daily Staff
The U.S. House of Representatives Wednesday night passed Right to Try legislation that would make it easier for patients with terminal illness to access to experimental therapies.
The passage of the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2018 passed by a vote of 267 to 149. The vote comes a week after the legislation failed to win a needed two-thirds majority to clear the House under an accelerated process.
“For those patients caught between the traditional drug approval delays, a clinical trial process for which they do not qualify, and limited time, this Right to Try simply establishes the freedom for patients to try therapies in situations where the benefits far outweigh the risks,” said Brian Fitzpatrick, R-Pennsylvania, who introduced the legislation. “With the House’s vote, both chambers have now showed that Right to Try can pass with bipartisan support.”
The legislation had the backing of President Donald Trump and conservative groups seeking limited government including the Goldwater Institute, Freedom Partners, and Americans for Prosperity. But more than 100 patient and provider groups opposed the legislation because they said it would weaken safety protections for patients.
Under the legislation, patients who are terminally ill and want to access an experimental therapy must have their physicians certify that other options are exhausted or not available. All products must have completed FDA phase 1 testing. Fitzpatrick said the legislation addresses concerns that could prevent its successful utilization by ensuring patients, doctors, and manufacturers do not assume any additional liability under this act.
“Right to Try will not help our patients access experimental treatments,” the National Organization for Rare Disorders said in a statement. “As this legislation heads to the Senate, we will continue to voice our concerns with a pathway that will diminish the ability of the Food and Drug Administration’s to protect patients, create confusion, and instill false hope.”
NORD also argued the legislation would do little to improve access to experimental medicines for terminally patients because the FDA already grants access in about 99 percent of the cases. Instead, they say the legislation fails to address the issues that lead pharmaceutical companies to deny access.
The rare disease patient organization called on the Senate to focus on legislation that reduces the financial disincentives companies encounter in offering their therapy through expanded access. It also said access to IRB review should be made more equitable, and FDA should clarify its use of adverse events in expanded access, as well as create a structure for accepting and reviewing supportive data from expanded access. And it said Congress should ensure liability laws do not act as an undue deterrent to expanded access.
March 22, 2018