National Brain Tumor Society Statement on Right to Try Act
“While the National Brain Tumor Society (NBTS) supports critically-ill patients seeking to explore all options for potentially life-saving treatments, we have significant concerns with the Right to Try Act.
Chief among our reservations is the potential for unintended consequences, including potential harm to patients and a disruption of the drug development system. Without adequate protections and information on proper dosing or potential major toxicities — information which would be more readily available through the FDA-regulated Expanded Access (“compassionate use”) program — there is the potential for harm to brain tumor patients from unproven and possibly unsafe treatments. Further, the Right to Try Act does not provide any guarantee of access – as patients may request a treatment from the manufacturer, but the company has no obligation to provide it. As such, these bills provide little, if any, additional benefit over current expanded access policies, while introducing undue risk.
NBTS is committed to helping patients who have exhausted all approved treatment options understand the current expanded access and clinical trials systems. We encourage those interested in accessing investigational treatments to learn more about these existing opportunities, including through resources like the Reagan Udall expanded access navigator (http://navigator.reaganudall.o
We will continue to advocate for improvements to the FDA’s Expanded Access program as well as urge biopharmaceutical companies to make their policies about compassionate use access to investigational drugs and devices even clearer, as we did as part of the recent 21st Century Cures law. In addition, we will continue to work aggressively to support and speed up innovative research headed toward the clinic and into drug and device development for brain tumor patients.
Ultimately, we are concerned that the Right to Try Act will constitute an unjustified and excessive risk to patients and the clinical research landscape, while providing no appreciable benefits over current policy. We encourage Congress to work with FDA, biopharmaceutical companies and patients to identify further steps to improve access to investigational drugs in a manner that would ensure the safety and efficacy of new treatments for all patients.”