The so-called right-to-try legislation winding through the US Congress would not help patients and would undermine the US Food and Drug Administration (FDA)'s authority, according to the proposed law's critics—a diverse group that includes many patient advocacy groups, the biotech industry and FDA officials. The right-to-try bill passed by the US Senate in 2017 is designed to ensure terminally ill patients can test investigational treatments. It proposes to achieve this by relaxing the FDA's oversight and remove liability for the doctors and companies involved. As Nature Biotechnology went to press, on March 21, the US House of Representatives voted 267-149 to approve a right-to-try bill, which is expected to become law.
The philosophy behind the right to try is to increase patients' access to promising investigational therapies when they have a serious and life-threatening disease, are unable to access a drug through a clinical trial and don't have any other options, says Paul Melmeyer, director of federal policy at the National Organization for Rare Disorders (NORD), which has come out alongside dozens of other mainstream patient advocacy organizations in opposing the right to try. But the bill before Congress is not going to fulfill that intention, he says. As written, it “removes FDA for the most part from the initial approval process for accessing these therapies. There's nothing in the bill that would be particularly positively impactful for our patients,” Melmeyer says. Nevertheless, the Trump administration has begun advocating for right-to-try legislation, with the president arguing during his January State of the Union speech that “patients with terminal conditions should have access to experimental treatments that could potentially save their lives,” and “It is time for the Congress to give these wonderful Americans the 'right to try'.”
This is a preview of subscription content, access via your institution