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An ‘alternative pathway’ to FDA’s existing expanded access programs?
S. 204, the Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act, was signed into law on May 30 in an Oval Office ceremony. The House had passed the bill by a 250-169 vote; last year the Senate passed it by unanimous consent. The passage follows years of back-and-forth legislative efforts in Congress, even while 39 states passed their own version of a right-to-try law. Trump considers the law a fulfillment of one of the proposals he cited in his first State of the Union address last January.
The terms of the law are fairly straightforward: any drug with an investigational new drug (IND) application and has passed a Phase I trial (which tests for toxicity) can be prescribed by a licensed physician for a patient with a “life threatening disease or condition” who has also exhausted existing therapies or was not accepted into any clinical trial. Drug sponsors are under no obligation to agree to provide the drug, and clinical outcomes are prohibited (with some exceptions) from being used as part of the regular drug approval process.
Critics of the legislation have contended all along that since FDA has approved some 99% of applications to the agency under the existing expanded access program (EAP), the bill is unnecessary. The law is also something of an end run around the existing—and elaborate—drug approval process, allowing drug companies and physicians to market investigational drugs. But libertarian-leaning policy groups, notably the Goldwater Institute, have argued that there’s no place for the federal government between a patient with a terminal illness and a physician who has a potential treatment. (This stance overlooks the fact that FDA was already involved in the Phase I trial.)
One positive outcome of the legislation might be to obtain some definitive information about how many patients seek and receive a drug therapy outside EAP; drug sponsors are supposed to report requests to FDA, and physicians are supposed to report outcomes. A recent Government Accountability Office report counted some 1,200 EAP approvals by FDA, but there appears to be no cumulative reporting on patient outcomes from those approvals. Except for one physician in Texas cited by the Goldwater Institute, there doesn’t appear to be any state-based totals from the states with right-to-try laws in place either.