Now that President Donald Trump has signed his long-sought “right to try” law, Americans have a dubious new freedom to bypass the drug quality-control system and experiment with medicines of questionable safety that may or may not work.
This solves no real problem.
The Food and Drug Administration has long granted access to medicines that are only partway through the U.S. approval process for patients in extreme circumstances who might benefit. But a myth had spread throughout the land, promulgated by conservative groups, that dying patients were being refused permission to try experimental drugs. Congress responded with a feel-good law.
From now on, these patients — in fact, practically anyone who has been diagnosed with any life-threatening disease or condition, whether or not he or she is near death — will be able to choose to trust doctors and drug companies alone to look out for their well-being.
That’s a questionable privilege — and not just because it’s risky. Before they exercise their right to try, patients will have to persuade the companies to provide the drugs. The new law doesn’t compel the companies to do so, and often they don’t want to.
Indeed, this, not FDA red tape, has been the main reason that people have had trouble getting ahold of experimental medicines. And it’s no wonder that the firms are reluctant, given that such drugs have passed only preliminary safety testing and are far from the point of being mass-produced or priced. Most of them never will be. Only a small fraction ever get approved.
Under the new law, without the FDA’s expert guidance, companies will have even more reason to turn down requests for experimental medicines. Those inclined to provide the drugs, if they’re wise, will continue to do so only through the FDA’s existing channel — as Janssen Pharmaceuticals has decided to do. The company says that the FDA may be “uniquely aware” of available safety data.
FDA Commissioner Scott Gottlieb has pledged to carry out the new law in a way that promotes access and protects patients. But he has no authority to do anything except issue guidance and receive annual summaries from companies providing investigational drugs.
A word of advice to doctors and patients who choose to sidestep the FDA: Proceed with care, and don’t count on a miracle.
FROM AN EDITORIAL ON BLOOMBERG VIEW