Doctors often fail to treat their patients with the most effective medicines — but it’s not their fault. Is an outdated FDA regulation to blame?
The FDA prohibits pharmaceutical companies from sharing information about “off-label” uses of medicines with physicians. Many drugs received FDA approval for one disease, but also effectively treat other illnesses that aren’t listed on the official label. Off-label prescribing is actually quite common. Physicians prescribe one in five medicines off-label.
Currently, doctors have to make these off-label prescribing decisions based on anecdotal evidence, since they have no access to drug companies’ clinical trial data. If the FDA were to permit greater information sharing, it would enable doctors to treat patients more effectively.
Doctors have good reason to prescribe drugs off-label. Many drugs can treat a wide variety of diseases. In fact, the average drug can treat 18 different illnesses, according to researchers from the University of Illinois at Chicago, Stanford University, and the University of Chicago. For instance, the diabetes drug Metformin can treat breast cancer. And the cancer drug Bexarotene has the potential to treat Alzheimer’s disease.
Doctors also prescribe drugs off-label to treat critically-ill patients after all approved medicines have failed. For instance, many patients with certain autoimmune diseases don’t respond to approved therapies. As a last-resort, doctors will prescribe these patients Prograf, a drug approved to help prevent people’s immune systems from rejecting organ transplants.
When doctors prescribe drugs off-label, they gather valuable information about how well patients respond.
But current FDA policy restricts doctors from sharing this information with each other and with drug manufacturers. Drug makers are even subject to criminal prosecution and civil liability if they discuss unapproved uses of existing drugs.
Companies could seek to get their medicines approved for the off-label uses, but the FDA approval process is time-intensive and costly. In fact, the cost of reapproving a drug is greater than the profits an approval would generate, according to an article in the Mayo Clinic Proceedings. So drug companies often choose not to go through the process.
Policy makers increasingly recognize the senselessness of preventing doctors and drug companies from sharing data. As the new FDA commission Scott Gottlieb said, “Patients and physicians make the best decisions when they have access to as much truthful, non-misleading, scientifically based information as possible.” In late March, Rep. Morgan Griffith (R-Va.) introduced legislation that would make it easier for manufacturers to share information with medical professionals on alternative uses for existing medications.
Despite support for reforms, the FDA hasn’t yet relaxed its prohibitions on data sharing.
The FDA should, of course, impose requirements on this information sharing. For instance, if the information comes from a clinical trial, drug companies should detail the trial’s sample size, limitations, and methodology. If it comes from physicians, they should describe patients’ conditions and detail how the drug affected them.
These requirements would ensure that the information is clear and accurate. But it also would ensure that doctors have the most up-to-date information on a drug and the illnesses it can treat.
Off-label prescribing has already has saved thousands of lives. The FDA ought to make it easier for doctors and patients to make the most informed treatment decisions possible.
Peter J. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.
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