Terminal patients who have exhausted all approved drug options would be able to seek out investigational treatments – even if they do not qualify for clinical trials – under a bill passed in the U.S. House, despite opposition from more than 100 patient and physician groups.
The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right To Try Act of 2018 (H.R. 5247) passed by a 267-149 vote, promising improved access to experimental treatments.
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H.R. 5247 creates a new, alternative pathway for patients who do not qualify for clinical trials to access experimental medications and requires informed consent to access these treatments.
For an unapproved drug to be made available to patients, it must have an active application that is not subject to any kind of clinical hold. Sponsors and manufacturers must notify the Food and Drug Administration when an unapproved drug is made available to the patient.
The bill also includes safeguards to prevent manufacturers from purposefully misbranding or mislabeling drugs.
H.R. 5247 provides liability protections to manufacturers, sponsors, physicians, clinical investigators, and hospitals that participate in providing experimental drugs to terminal patients through this new alternative pathway, although it does not shield them from liability stemming from reckless misconduct, gross negligence, or any other intentional violations. It requires sponsors and manufacturers to report all adverse events to the FDA.
It also provides certainty to manufacturers as to how the FDA will use patient outcomes from the use of treatments outside of clinical trials when it is evaluating the applications on these new drugs.
Rep. Michael Burgess (R-Tex.), the House Energy & Commerce Health Subcommittee chairman and a physician, spoke in support of the bill during a debate on the House floor.