Revised ‘Right-to-Try’ Bill Introduced in House

A revised “Right to Try” bill has been introduced in the House to improve access to experimental treatments for patients. “This updated ‘Right to Try’ bill is the direct result of conversations with our colleagues, the administration, and stakeholders on all sides of the issue,” said House Energy and Commerce Committee chairman Greg Walden (R-OR). “This is a complicated issue with passionate advocates on both sides and it was imperative we got the policy right. After months of thoughtful discussions, we believe this legislation is ready for a vote in the House.”

President Donald Trump has been a vocal supporter of advancing the “right-to-try” legislation. Speaking at a 2/1 Republican retreat in West Virginia, Trump echoed remarks he made during his State of the Union address 1/30 (see story). Legislation passed the Senate last year and it is currently being considered by the House Energy and Commerce Committee. Trump told the Republican lawmakers that FDA commissioner Scott Gottlieb is leading the right-to-try effort for the administration. However, during a 10/2017 House subcommittee hearing, one would not get an impression that Gottlieb was a strong proponent of the right-to-try legislation. His testimony focused on changes that the agency was making to ease patient access to experimental therapies under its expanded access program.

The hearing offered Gottlieb an opportunity to comment on the pending legislation. He said the agency recommends narrowing the eligibility from patients who face a “life-threatening disease or condition” to “terminal illness.” Many chronic conditions (e.g. diabetes) are life-threatening, but medical and behavioral interventions make them manageable without considering unapproved therapies. In addition, the term used in one bill for “terminal illness” would benefit from a clear definition. “We recommend defining it as ‘a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months,’” he said.

The revised bill appears to address Gottlieb’s concerns. It defines an “eligible illness” as “(a) a stage of a disease or condition in which there is reasonable likelihood that death will occur within a matter of months; or (b) a disease or condition that would result in significant irreversible morbidity that is likely to lead to severely premature death.” More specifically the revised bill will: 

• Establish a robust informed consent to access unapproved drugs
• Specify that any unapproved drug used in the new alternative pathway must have an active application and is not the subject of a clinical hold
• Include a sponsor and manufacturer notification to FDA after providing an unapproved drug to an eligible patient
• Guard patients from manufacturers purposefully misbranding or mislabeling drugs
• Provide liability protections for manufacturers, sponsors, physicians, clinical investigators, and hospitals that participate in the existing expanded access program and the new alternative pathway; unless there is reckless or willful misconduct, gross negligence, or an intentional tort
• Obligate sponsors and manufacturers to report adverse events in real time, through notification to the FDA – both within the existing expanded access program and through the new alternative pathway
• Provide certainty to manufacturers regarding how FDA will use patient outcomes when evaluating new drug applications

Advocacy group Public Citizen opposed the revised bill, calling it the “False Hope” legislation. “Although the legislation has several improvements over the U.S. Senate-passed version (S. 204), the bill still has fundamental flaws that would put vulnerable patients at risk and undermine their rights,” it said. “In particular, the bill still would offer false hope to patients by creating a dangerous pathway for access to experimental medications and biological products that bypasses the U.S. Food and Drug Administration’s (FDA) existing Expanded Access program. That program provides for FDA review of experimental therapies used outside the context of a clinical trial, thereby helping to ensure that proposed uses of the treatments do not pose unacceptable risk.”

Additionally, Public Citizen objected to the bill’s effect to “broadly immunize from liability manufacturers, sponsors, physicians, clinical investigators and hospitals for a wide range of conduct related to the provision of experimental medications to patients, including provision of such medications under the FDA’s existing Expanded Access program. In this way, the bill would bar suits in a variety of situations in which state law might reasonably impose liability and would eliminate a strong incentive for manufacturers and health care providers to act responsibly and in the best interests of patients.”