US House passes bill giving patients right to try experimental drugs

The US House of Representatives passed a bill, by a vote of 267 to 149, that would give patients with terminal illnesses a right to try experimental treatments. The latest vote on the legislation  required only a simple majority for passage after being rejected by the House last week when it was being considered under a special procedure intended to speed the approval of bills, which needed a two-thirds majority for approval. The "right-to-try" legislation now needs approval from the Senate, which passed its own similar bill last year. 

Under the proposed legislation, patients with an illness deemed terminal who do not qualify for clinical trial participation and who have exhausted other treatment options, could potentially receive investigational therapies. However, the bill would not require drugmakers to provide experimental treatments if requested by patients. 

The legislation also protects drugmakers, physicians and medical institutions from certain legal risks associated with providing unapproved drugs to patients. Doctors and hospitals in particular would generally be protected unless they engaged in gross negligence or willful, reckless or criminal misconduct. 

Those in favour of the bill, including US President Donald Trump, claim it would provide patients with additional access to potentially effective treatments that could extend survival for terminally ill patients. However, opponents warn that the proposed legislation would weaken FDA oversight and provide patients with false hope, in addition to potentially allowing companies to promote the use of drugs whose safety and effectiveness has not yet been established. 

Meanwhile, a number of former FDA commissioners, as well as current Commissioner Scott Gottlieb, have argued that the bill is unnecessary given the existence of the agency's "expanded access" programme for critically ill patients. Gottlieb informed a House subcommittee last October that the FDA authorises 99 percent of applications received under the programme, but noted at the time that "the vast majority of people who will use a drug through expanded access are using a drug that doesn't work."

Gottlieb also argued that the biggest obstacle to the use of experimental therapies is drug availability, as some companies may not have enough resources to administer expanded access requests, and may worry that offering the drug would make it harder to recruit participants for the clinical trials needed for FDA approval.

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