There is no right way to deal with the devastating news that you’ve run out of treatment options as a dying or desperately ill patient.
On the surface, the debate over Right to Try legislation appears to be about whether seriously ill people, for whom no effective treatment remains, are allowed to access experimental drugs that have not been approved by the Food and Drug Administration (FDA).
Framing the issue as “right to try” tugs at the heartstrings and suggests that medicine’s failures are not scientific but regulatory. But behind the appealing, how-could-anyone-not-support-it name, Right to Try is a libertarian effort by the Goldwater Institute and some Republicans to deregulate the drug market and weaken the FDA. It promises a quick fix for a problem that doesn’t exist.
The implication is that the cure is out there but it’s being kept from patients by government bureaucrats. In reality, medical advancement is painfully slow. But the FDA approves new treatments faster than any other developed country. For seriously ill patients, it already has a compassionate use program that offers a pathway to obtain experimental drugs. The FDA approves 99 percent of compassionate use requests, usually within a few days—or just 24 hours in emergency situations.
It’s almost always the drug companies, not the FDA, that don’t grant access to experimental treatments. Drug companies may deny requests for access outside a clinical trial because there isn’t enough of a limited-supply investigational drug or because they decide the benefits don’t outweigh the risks. Nothing about Right to Try legislation changes that. It may make companies more reluctant to provide experimental drugs without FDA oversight. And it could undermine the clinical trial model at a time when many trials are already struggling to enroll patients.
The heartbreaking truth is that even when patients get access to investigational treatments, they’re still not necessarily lifesaving. Experimental drugs are just that: experimental. We don’t yet know if they work or if they’re safe. These deregulatory efforts don’t include protections against unscrupulous doctors or drug companies charging as much as they want for unproven therapies.
I just completed treatment for an exceedingly rare cancer. I understand as well as anyone the urgent need for more effective, less toxic treatments. I believe we should all demand more from the billions spent on medical research, as well as a health system that prioritizes patient well-being. But Right to Try legislation attacks the one agency tasked with ensuring the safety and efficacy of medications, and its promises are based on nothing more than false hope. Taking the FDA out of the picture is not the way to promote and protect public health—and it won’t save lives.