Federal right-to-try law aims to broaden access to investigational drugs

A federal law that was enacted in May creates a new mechanism for U.S. patients to obtain investigational drugs, but the extent to which people will be able to use this option is unclear. The right-to-try legislation—officially known as the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017—was signed into law on May 30. The act is named for 3 Americans with amyotrophic lateral sclerosis (ALS) and 1 with Duchenne muscular dystrophy who reportedly lacked access to investigational drugs through FDA’s expanded access programs. The new law amends the Federal Food, Drug, and Cosmetic Act to permit patients to obtain investigational drugs outside of clinical trials or expanded access programs. The law also shields physicians, dispensers, and drug companies from liability related to both the use of the drugs and the refusal to provide them. To be eligible, patients must have a diagnosis of a life-threatening disease or condition and must have exhausted FDA-approved treatment options. Patients must also be unable to participate in a clinical trial involving the investigational drug. The medication must have cleared Phase I development and be under FDA review for marketing approval or under investigation in a clinical trial that is intended to support FDA approval or licensure.