Dear Vice President Pence,
Two weeks ago, you met with several families and encouraged them to lobby Congress for a federal “right-to-try” law, which would allow people with fatal illnesses to gain access to experimental medicines, even though they are not enrolled in a clinical trial.
Your goal is admirable. Unfortunately, though, you’re peddling false hopes.
Here’s why: From the start, the right-to-try movement has been misguided.
The effort grew out of mounting frustration with the Food and Drug Administration, which does have a program called expanded access that allows seriously ill people to obtain an experimental medicine. In fact, the agency has historically approved 99 percent of requests.
Yet critics complain the program is unwieldy and sometimes capricious. And they argue that, if only the FDA were sidelined, more dying patients could more readily gain access to potentially life-saving treatments. Not coincidentally, this argument mirrors general criticism of the FDA’s drug approval process.
Two years ago, as governor of Indiana, you signed a right-to-try bill into law. Now, 33 states have such laws. But there are outstanding questions about the extent to which the FDA has a legal right to preempt, or override, the state laws.
This explains why lawmakers introduced right-to-try bills in the House and Senate in recent weeks. The impulse to offer patients hope is understandable; after all, not everyone can get into a clinical trial. But these proposals have the same fundamental flaw as state laws — at the end of the day, requests must be granted by drug makers, not just the FDA.
And that’s always been the rub.
Drug makers may have several legitimate reasons for denying a request for access to an experimental drug they’ve been testing. They may not want to risk giving the drug to a patient outside of a controlled clinical trial, because if the patient has a bad reaction, that could undermine their effort to get the drug approved by the FDA.
And smaller companies, in particular, may lack sufficient supplies to grant a large number of requests.
“The legislation makes it seem there’s a single roadblock — the FDA,” said Ken Moch, who heads Cognition Therapeutics. He previously ran a biotech called Chimerix that created an outcry by initially denying an experimental drug to a dying boy. “In my personal experience, that’s untrue.”
Starlee Coleman, a spokeswoman for the Goldwater Institute, a conservative nonprofit that has drafted model right-to-try bills, acknowledged that some drug makers may avoid granting requests from patients.
“A drug company can still be an obstacle,” she told me. “But we think there will be scenarios where a drug company will be comfortable providing a drug outside the FDA process and we want to make sure they have a pathway to do this. We don’t have any expectation that every sick person would be helped if the law passed tomorrow. But we do want to create options quickly without bureaucratic delay.”
Hoping to alleviate some concerns, the federal bills say drug companies — as well as physicians — cannot be sued if something goes wrong. This provision may, perhaps, encourage a drug maker to provide its experimental medicine, but the complete lack of accountability is troubling.
Significantly, the bills also contain a provision that blocks the FDA from using information about any unintended consequences, such as a severe patient reaction, against the company when it comes time to consider the drug for approval.
This is a bad idea. The simple reason is that the FDA would be precluded from considering the impact of a serious side effect or later placing that information in the product labeling.
“Removing the FDA from one segment of the process is short-sighted, and puts a simple, but ineffective, solution on a much bigger problem, [which is] the long time frame [needed] … to gather sufficient safety and efficacy data,” said Beth Roxland, a law and health policy expert at the division of medical ethics at the NYU Langone Medical Center and a former bioethics and strategy leader at Johnson & Johnson.
She has a point. As public health goes, cutting the FDA out of the loop is simply bad policy.
In supporting such laws, you might mean well, but the federal legislation assumes there will never be any need for oversight. Beyond expanding access, right to try may wind up expanding uncertainty.