The Cystic Fibrosis Foundation joined more than 70 organizations to oppose the Right to Try Act. If passed into law, the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act would allow individuals to access investigational therapies outside of clinical trials without approval and consultation from the Food and Drug Administration (FDA).
The Cystic Fibrosis Foundation opposes this legislation because it creates additional and undue risks for those seeking access to investigational therapies. The legislation passed in the House would:
- Remove the FDA's consultation on dosing, route of administration, dosing schedule, and other important safety measures
- Allow a seven-day lag between access to investigational therapies and FDA notification, which could delay notice of and reaction to potential adverse events
- Limit the FDA's ability to halt access to these experimental therapies if significant safety concerns arise
In addition to increasing safety concerns, this proposal would not provide greater access to unapproved therapies in the CF drug development pipeline. Legitimate barriers to such access are often attributed to a limited supply of the investigational therapy or other concerns from the manufacturer. The Right to Try legislation does nothing to address these or other real hurdles to treatment access.
The FDA's current expanded access program, though imperfect, facilitates access to investigational therapies outside of clinical trials for more than a thousand patients facing serious and life-threatening conditions each year. This is an important way to provide therapies not yet approved by the FDA to those with limited treatment options. The current process requires approval from a physician and the manufacturer as well as the FDA.
The FDA approves more than 99 percent of expanded access requests while sometimes making important dosing and safety improvements to the proposals. We support continued use of this process for people to access investigational therapies.
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. 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. Read the full letter opposing the Right to Try Act.