The US Senate today approved a "right-to-try" bill allowing patients with life-threatening illnesses to use experimental drugs without any interference from the federal government.
The bill now goes to the House, which is considering a similar measure. More than 30 states have passed their own versions of right-to-try legislation, promoted by conservative and libertarian groups such as the Goldwater Institute.
Sen. Ron Johnson (R-WI), the bill's sponsor, told his colleagues on the Senate floor today that the legislation would help "real people facing their mortality with no hope." However, right-to-try critics, which include the American Society of Clinical Oncology (ASCO), contend that patients would miss out on protections under the US Food and Drug Administration (FDA), and not necessarily gain better access to experimental drugs.
Under Johnson's bill, patients with life-threatening illnesses who have exhausted approved treatments and cannot participate in a clinical trial can use a drug in the pipeline for FDA approval that has completed at least a phase 1 clinical trial. The drug's manufacturer or sponsor must file an annual report on the drug's use and any adverse events. However, manufacturers have the right not to make an experimental drug available to a patient.
The bill originally applied to terminally ill patients, but it was amended to encompass those with life-threatening illnesses, defined by law as having a high likelihood for death unless the course of the disease is interrupted, or with potentially fatal outcomes "where the end point of clinical trial analysis is survival."
Another amendment cracks open the door for malpractice suits against physicians. The original bill states that prescribers of experimental drugs, as well as manufacturers and pharmacies, can't be taken to court. In the amended bill, prescribers can face liability for "reckless or willful misconduct, gross negligence, or an intentional tort under any applicable state law."
Desperate patients already can obtain experimental drugs through the FDA's expanded-access or "compassionate use" program, which green-lights more than 1000 requests a year, or about 99% of those received. Supporters of national right-to-try legislation say that their approach can help far more Americans — millions, in fact — and that the FDA program works too slowly.
The agency counters that it approves nonemergencies in a median of 4 days, and emergencies immediately over the phone. It suggests that the real bottleneck lies with drug manufacturers, which turn down requests for experimental drugs at a far higher rate — 60% for one company — than the FDA does with expanded-access applications.
ASCO is on record saying that the FDA's expanded-access program, recently streamlined, makes right-to-try laws largely unnecessary. Such legislation, it stated in a recent position paper, potentially puts patients "at high risk for unclear benefit."
Yet another criticism of the Senate right-to-try bill is that, if a pharmaceutical company decides to make an investigational drug available, there are no limits on the price it can set. In contrast, patients participating in clinical trials pay nothing for their drugs, while the FDA's expanded-access program limits what a drug maker may charge.
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Cite this: Senate Passes Right-to-Try Bill - Medscape - Aug 03, 2017.