The Senate on Thursday passed by unanimous consent a measure designed to make it easier for terminally ill patients to get access to experimental treatments without oversight from the Food and Drug Administration.
The bill would provide drug companies some legal protection if a treatment results in harm.
“Patients with terminal diseases ought to have a right to access treatments that have demonstrated a level of safety and could potentially save their lives,” Sen. Ron Johnson (R-Wis.), who wrote the bill, said in a statement after the vote.
But critics say the legislation is unnecessary. Alison Bateman-House, an assistant professor of medical ethics at NYU Langone Medical Center, called the bill “inherently deceptive” because it simply says patients can ask drug companies for the treatments. Companies often are reluctant to provide unapproved products for a variety of reasons, and the FDA has an efficient system to handle requests for experimental drugs, she said.
A previous version of the measure barred the FDA from considering any information on safety problems as part of its approval process for a drug used under right to try. The latest version was modified to allow the agency to consider such information if it is critical to determining whether the drug meets the agency's safety standards.
The Senate's action on the right-to-try bill was part of a deal struck between Senate Health, Education, Labor and Pensions Committee Chairman Lamar Alexander (R-Tenn.) and Johnson, who had threatened to hold up a must-pass FDA funding reauthorization bill unless a vote on his legislation took place.
The reauthorization bill was approved 94 to 1. Cleared by the House, it now goes to President Trump for his signature. It authorizes the collection of billions of dollars in user fees from the drug and device industries to help pay for reviews for their products over the next five years.
The White House had asked for even higher user fees, and a sharp, offsetting reduction in federal funding for the FDA, but Congress rejected that approach.
The legislation directs the FDA to accelerate generic drug applications for products that have little or no competition. It also includes a provision designed to sharply increase the number of approved cancer treatments for children by giving the agency authority to direct drugmakers working on oncology therapies for adults to test them in children, as well.
“There are 900 drugs in the adult cancer pipeline and hardly any are being studied in children with cancer,” said Nancy Goodman, executive director of Kids v Cancer, which pushed the legislation. “And there are so many new and exciting innovations in cancer treatment, from precision medicine to immunotherapy to nanoparticles, and we hope children, as well as adults, will benefit.”
Goodman, whose son Jacob died of a brain tumor when he was 10, said most pharmaceutical companies focus on adults when they develop treatments because that population of cancer patients is so much larger. As a result, almost all cancer treatments for children are used off label, adapted for them by pediatric oncologists.
The legislation was praised by the American Cancer Society Cancer Action Network, the advocacy arm of the American Cancer Society. It said the bill would provide for increased incorporation of patients' perspectives in the FDA's drug approval process.