WASHINGTON, DC - The Subcommittee on Health, chaired by Rep. Michael C. Burgess, M.D. (R-TX), today held a hearing examining patient access to investigational drugs and the U.S. Food and Drug Administration’s (FDA) expanded access program, which is designed to help patients who do not qualify for clinical trial access to unapproved therapies. #SubHealth also discussed legislation related to access to unapproved therapies.
Kicking off the hearing, Chairman Burgess stated, “Since 2014, 37 states, including my state of Texas, have passed a version of Right to Try laws, through a strong grassroots movement. With that in mind, it is my hope this hearing will start a constructive discussion on this important issue."
Dr. Scott Gottlieb, FDA Commissioner, highlighted his previous cancer battle and reliance on a variety of treatments, including a commercially-available therapy in an off-label manner, and spoke to the agency’s expanded access program.
“While the agency permits almost all expanded access applications to proceed, it makes meaningful changes in approximately 10 percent of these cases to enhance patient safety," stated Dr. Gottlieb. “…The changes are based on the scientific and medical expertise of our staff, and informed by confidential information provided to FDA by product sponsors during the course of development. This information is often unavailable to the treating physician - and the larger medical community - and becomes available only after a drug is approved. It is important to note that access to investigational products requires the active cooperation of the treating physician, industry, and FDA in order to be successful."
Mr. John Dicken, Director for Health Care, U.S. Government Accountability Office (GAO), summarized the findings from a July 2017 GAO report saying that “of the nearly 5,800 expanded access requests that were submitted to FDA from fiscal year 2012 through 2015, FDA allowed 99 percent to proceed." Additionally, Mr. Dicken highlighted that “FDA typically responded to emergency single-patient requests within hours, and responded to all other requests within 30 days."
Rep. Andy Biggs (R-AZ) testified in support of legislation he co-authored with fellow witness Rep. Brian Fitzpatrick (R-PA), saying, “Our legislation allows terminally ill patients who have no further options left - I repeat that: no further options left - the opportunity to try experimental drugs that could save their own lives."
Lieutenant Commander Matthew Bellina, who spoke in his capacity as an advocate for the ALS Community, shared his personal story his support for legislation, saying, “Often times the greater good is to put the trial over the rights of the individuals. This however is not a one-size-fits-all situation. The processes for all trials and drugs are unique and must be treated that way."
Dr. Alison Bateman-House, Assistant Professor of medical ethics at NYU Langone Health and co-chair of the Working Group on Compassionate use and Pre-Approval Access, spoke to some of the working group’s findings in this space.
Dr. Bateman-House testified, “After more than three years of studying all facets of compassionate use/pre-approval access, including Right to Try, the Working Group has found that the FDA’s expanded access program has been doing an excellent job of helping patients obtain access to experimental drugs."
“There is ongoing concern that some patients may not be allowed to access investigational drugs even after exhausting all other treatment options," said Energy and Commerce Committee Chairman Greg Walden (R-OR). “We must examine whether there are regulatory, legal, or commercial barriers to patient access for experimental drugs."
