Rare Daily Staff
The National Organization for Rare Disorders along with 37 patient and provider organizations in a letter to leaders of the House of Representatives called on lawmakers to reject proposed legislation that would give people with life threatening diseases access to potentially lifesaving experimental therapies.
While the groups expressed support for providing such access, they said the proposed legislation would not increase access and could harm patients by exposing them to people seeking to capitalize off creating false hope for patients who are desperate. Instead, the organizations asked Speaker Paul Ryan (R-Wisconsin) and Minority Leader Nancy Pelosi (D-California) to consider alternative legislation that would safely increase access to experimental therapies.
Separate House and Senate bills under consideration both would remove the Food and Drug Administration from the initial approval process for accessing an investigational therapy outside of a clinical trial. The groups, however, argue that removing FDA from this process is unlikely to increase access to investigational therapies since the FDA currently approves 99.7 percent of all expanded access requests submitted by physicians and companies for patients with immediately life-threatening illnesses who cannot participate in clinical trials.
When a patient seeking access to an experimental therapy is refused, the groups note that it is generally the drug company that denies the request. In those cases, the letter says that it is for reasons “that appear to be reasonable.” This includes such things as a determination that the benefits do not outweigh the risks, an unavailability of sufficient product to offer outside of clinical trials, costs, or concerns about adversely affecting clinical trial enrollment.
The letter cited a recently released report from the Government Accountability Office that examined the current FDA expanded access program and found that substantial changes were not needed within the program, aside from greater clarity on the use of adverse event data.
While the groups acknowledge that there is room for improvement to existing expanded access policies, it argues that patients seeking expanded access to unapproved therapies outside of clinical trials should be afforded the same ethical standards and protections as patients taking part in clinical trials.
“Our organizations support patient access to unapproved therapies. However, the Right to Try bills currently under consideration in the House do not effectuate policy changes that would afford our patients greater access to promising investigational therapies,” the patients organizations said in their letter. “Instead, these bills would likely do more harm than good. We would welcome the opportunity to discuss alternative legislative proposals that would improve the ability of patients to genuinely and safely access unapproved therapies.”
February 6, 2018
Photo: Speaker Paul Ryan