The Food and Drug Administration determines which drugs can be prescribed in the United States. However, the FDA does not tell physicians how to prescribe drugs. When physicians prescribe drugs for uses other than their FDA-approved use, they engage in off-label drug prescription.
Off-label drug prescription is extremely common. By some estimates, 25 percent of all prescriptions are for off-label uses. These rates can be exceptionally higher for conditions with few available treatments. Cancer, HIV/Aids, many mental illnesses, and large sections of pediatrics heavily depend on off-label drug prescriptions.
Although how to prescribe drugs is left to the physician, the FDA heavily regulates information distribution about off-label uses. Unfortunately for physicians and drug providers, restrictions on what information is allowed to be distributed, as well as how it is distributed, are mind-bogglingly complex.
For instance, if physicians want to learn more about off-label use research, they must often contact drug companies themselves. Physicians can also attend continuous medical education programs, but only if the program is accredited. Drug providers are prohibited from promoting off-label uses of their drugs. However, providers can distribute information on off-label uses if done solely for communication purposes. The differences can be fuzzy. Information distributed for communication purposes must also follow a series of complicated restrictions.
The FDA can fine drug providers for noncompliance, and it levies a lot of fines. According to an article in Health Affairs, the agency collected more than $15 billion in fines from pharmaceutical companies from 2006 to 2016. The same article notes, “the revenues generated from off-label prescribing are substantial and can far outweigh the penalties.” Whether the noncompliance violations were intentional or not, the FDA appears to be doing a poor job regulating how of off-label drug use information is distributed.
But change is on the way.
Recent changes to off-label information policy work to broaden what information drug providers can distribute to payers, formulary committees, and other medical professionals, “even if the information is outside of the FDA approved labeling and was not submitted to the agency in the course of product approval.” This change makes it easier for drug providers to disclose innovative alternative uses. As a result, physicians gain access to medical discoveries faster, allowing them to exercise their medical expertise with up-to-date information.
But many are critical of these changes. Some allege that loosening the restrictions will expose physicians and patients to dubious evidence. Others allege these changes were heavily influenced by political pressures from Congress, which is working to dilute FDA’s authority. As John Osborn, a senior advisor to international law firm Hogan Lovell, critically wrote in a piece for STAT, “Apparently, the political imperative of maximizing value for prescription drugs is now so great that it has the power to override decades of intransigent FDA policy on off-label discussions.” Osborn continued to write:
This area of the law remains convoluted, but it has important implications for the practice of medicine. … The legal and regulatory framework needed to allow companies to communicate truthful and non-misleading speech must be reconciled by the FDA, lest it finds that Congress or the courts have done it for them.
Historically, the FDA held significant autonomy in regulating pharmaceuticals. But this has changed in the last decade. In United States vs. Caronia, the U.S. Supreme Court ruled that a drug provider marketing “truthful, non-misleading” off-label uses for drugs is protected under commercial free speech. The ruling contested the FDA’s authority to determine when drug providers violated agency rules. More recently, national right-to-try legislation granted terminally ill patients the ability to bypass the FDA approval process and gain access to experimental medication.
These, among other events, suggest the FDA’s regulatory power is waning.
But “Congress or the courts” are not assuming the FDA’s authority when “overriding” it. Instead, they are allowing interactions between medical providers and medical professionals to be governed by the market. Thus, it is crucial to ask whether these parties can find effective alternative uses of drugs with less government oversight.
Previous economic research in off-label drug prescription finds the answer is yes.
In an article for The Independent Review, economist Alex Tabarrok notes that physicians assess various sources of information (of higher and lower quality) to make medically sound decisions. Thus, dictating what information physicians can have likely makes their decisions less informed. Providing numerous historical examples of medical professionals reaching a medical consensus without the FDA’s input, Tabarrok concludes, “Off-label prescribing speeds medical innovations to patients, increases the number of drugs available to doctors, and lowers the costs of medical innovation.”
An article I published in the Journal of Private Enterprise argues that drug providers and physicians exercise medical entrepreneurship when prescribing drugs for off-label uses. For example, private-practice physicians understood aspirin could help prevent heart attacks as early as 1945. This discovery circulated in medical journals and reached widespread consensus all without FDA oversight. Alarmingly, the FDA to this day does not recognize this widely accepted use (nearly 70 years later).
Off-label drug use is a critically important part of the U.S. pharmaceutical market. For these medical discoveries to be utilized best, easy access to information is critical. Recent efforts to widen communication channels are a step in the right direction. Let’s hope it’s the first of many.
