A letter drafted by Alison Bateman-House, PhD, MPH, associate professor of medical ethics at NYU Langone Health, warns U.S. lawmakers of the risks of legislation allowing patients to access unapproved drugs.
The Senate already passed the so-called “right-to-try” bill, which allows patients with life-threatening conditions to access drugs that have not been approved by the U.S. Food and Drug Administration (FDA); the House Committee on Energy and Commerce is now considering it.
The FDA’s expanded-access or “compassionate-use” program acts as a pathway for patients to access experimental treatments outside of clinical trials. According to an FDA study, 99 percent of requests made from 2005 to 2014 for “compassionate use” were approved. A right-to-try bill would allow doctors and patients to sidestep the FDA.
“Patients with terminal conditions who access unapproved therapies outside of clinical trials may be at risk of hastened death or reduced quality of the life that they have left and deserve protections similar to patients taking part in clinical trials,” wrote the 40-plus signatories, which included doctors, medical ethicists, and lawyers.
Andrew Powaleny, a spokesperson for the pharmaceutical industry organization Pharmaceutical Research and Manufacturers of America, said, “It is crucial that any right-to-try policy proposals protect patient safety and the integrity of the clinical trial process along with FDA oversight.”
In his State of the Union address, President Trump expressed support for the bill. “Patients with terminal conditions should have access to experimental treatments that could potentially save their lives,” the president said. “People who are terminally ill should not have to go from country to country to seek a cure – I want to give them a chance right here at home. It is time for the Congress to give these wonderful Americans the ‘right to try.’”
Sources: CNBC News, January 30, 2018; STAT News, February 1, 2018.