The Cystic Fibrosis Foundation (CFF) has joined 70 other organizations in signing a letter opposing the latest version of the Right to Try Act, which the House passed recently.
It would allow terminally ill Americans to try medicines that have passed Phase 1 of the U.S. Food and Drug Administration approval process. This means the therapies would continue to be tested in clinical trials, and not yet be on pharmacy shelves.
The legislation was created to expand access to potentially life-saving treatments years before patients would normally have access to them. If passed into law, it would let patients obtain investigational therapies outside of clinical trials without FDA approval.
Those who support the legislation say the latest version would do a better job of safeguarding patients, compared with previous versions.
The CFF opposes the act. The foundation said it would create additional and undue risks for those wanting to try investigational therapies, including patients with cystic fibrosis (CF).
CFF leaders are concerned that such legislation: 1) would eliminate the need for FDA approval and consultation on dosing schemes, route of administration, and other safety measures; 2) would limit the FDA’s ability to halt access to investigational therapies if significant safety issues arose; and 3) would allow a seven-day interval between access to the experimental therapies and FDA notification, which could potentially delay the report of adverse events.
Although the act would appear to be a way to help some patients, the CFF fears it could do more harm than good.
The CFF and the others organizations that signed the letter contend that the FDA’s Expanded Access Program (EAP), although imperfect, already provides access to investigational therapies to over a thousand patients facing life-threatening conditions each year.
Almost all those who request access to the program receive FDA approval, with the agency sometimes making important dosing and safety improvements to the proposals. In fact, it is usually the pharmaceutical company that denies access to an investigational therapy outside of clinical trials.
While the latest version of the Right to Try Act includes improvements — such as a more limited eligibility criteria, more robust informed consent requirements, more frequent and thorough reporting to the FDA, and a requirement that manufacturers provide additional public reporting on its use — the proposal is still less safe for CF patients than the FDA’s current EAP program, the CFF maintains.
The organizations concluded their letter by thanking the House members who made changes to the initial proposal, but added that the revisions were insufficient for them to support the legislation.
All those who signed the letter welcome “the opportunity to continue constructive dialogue on ways to improve the ability of patients to genuinely and safely access both approved and unapproved lifesaving therapies,” it reads.
“We recognize and share the sense of urgency around developing new and effective treatments for cystic fibrosis and getting them into the hands of people with CF as quickly as possible,” the CFF said in a news release. “We look forward to working with Congress and other stakeholders to improve access to care and treatment for people with CF and other chronic, life-threatening conditions.”