In February, I was proud to join with my colleagues in health law, medicine, and ethics to sign a letter to Chairman Greg Walden and Ranking Member Frank Pallone in the U.S. House Committee on Energy and Commerce in opposition to S.204, the Right to Try Act of 2017 and similar bills being considered in Congress. The letter was subsequently reported by various media outlets.
The bill has been debated and amended since then, but it has not yet passed both houses of Congress. The House is set to vote on it in the coming days. Our message therefore deserves to be repeated below. To read the list of signatories, you can click here.
Dear Chairman Walden and Ranking Member Pallone,
The undersigned individuals write to express our strong opposition to S.204, the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, as well as to all “right to try” bills currently under consideration in the House Energy and Commerce Committee.
We thank the Committee for holding a hearing on this topic in October 2017, and we urge the Committee to recall that Food and Drug Administration (FDA) Commissioner Scott Gottlieb testified that he had concerns about the breadth of the language of S.204. He also testified that, in his experience, the primary factor that led to patients being denied access to investigational medicines was a lack of supply of these experimental products.
The FDA currently approves over 99 percent of the requests for expanded access that it receives, typically within a few days or even hours, and its review identifies changes necessary to adequately protect patients in 11 percent of expanded access programs. Because the FDA is not the obstacle to patient access to investigational drugs and plays a vital role in ensuring proper patient safeguards are in place, we implore the Committee to not pass legislation that would remove the FDA from the initial authorization process for accessing an investigational therapy outside of a clinical trial. The Government Accountability Office recently released a report examining the current FDA expanded access processes, and found that substantial changes were not needed within those processes, aside from greater clarity on the use of adverse event data.
It is important to remember that the current regulatory system for medical products and research in the United States was created as a result of serious patient harm. Birth defects from thalidomide are an example of what happens when drugs are given to humans without proper safety review and approval. While obtaining unapproved therapies outside of a clinical trial is not about research, the products themselves remain experimental and have not been shown to be safe and effective. Clinical research subject protections are in place when experimental products are being tested to ensure the safe and ethical treatment of research participants. Patients with terminal conditions who access unapproved therapies outside of clinical trials may be at risk of hastened death or reduced quality of the life that they have left, and deserve protections similar to patients taking part in clinical trials.
Our view on this matter is in alignment with that of numerous patient and provider groups, specifically the Alliance for Aging Research, the American Cancer Society Cancer Action Network, the American Lung Association, the American Society of Clinical Oncology, the Association of Pediatric Hematology/Oncology Nurses Cancer Support Community, the Children’s Brain Tumor Foundation, Children’s Cause for Cancer Advocacy, the Cystic Fibrosis Foundation, Fight Colorectal Cancer, the Friedreich’s Ataxia Research Alliance, Friends of Cancer Research, Grandparents in Action, the Leukemia & Lymphoma Society, Lung Cancer Alliance, LUNGevity Foundation, Max Cure Foundation, National Comprehensive Care Network, the National Health Council, the National Organization for Rare Disorders, the TargetCancer Foundation, and the United Mitochondrial Disease Foundation, as well as such groups as the Working Group on Compassionate Use and Pre-Approval Access and Public Citizen.
Expanded access can be improved, but the right to try approach is misguided and would likely do more harm than good. This legislation sells vulnerable patients and families false hope at the expense of weakening the FDA’s critical role in making sure that all Americans can have confidence in the safety and effectiveness of our medical products. We welcome the opportunity to answer any questions you have about our opposition to these pending bills.