Ozempic’s Popularity Spurs Renewed Interest in Off-Label Prescriptions

In case you haven’t noticed, we’re in the midst of an Ozempic boom.

Exploding demand for the weight-loss drug has sent the value of its manufacturer, Novo Nordisk, to the moon and the buzz shows no signs of diminishing.

Ozempic’s active ingredient, semaglutide—also found in the equally popular drug Wegovy—is a glucagon-like peptide-1 or GLP-1 receptor agonist approved by the Federal Drug Administration for the treatment of chronic weight issues.

But the benefits of semaglutide may extend far beyond its headline-grabbing attributes. In March, the FDA approved semaglutide injections “to reduce the risk of cardiovascular death, heart attack and stroke in adults with cardiovascular disease and either obesity or overweight,” while new studies have found that the ingredient may also be used to treat dementia, pancreatitis, and polycystic ovary syndrome.

These potential additional benefits of the hottest drug of the moment have revived discussions of an often-debated subject in medicine: the off-label use of prescription drugs.

Off-Label Prescribing Common Practice

The FDA does not regulate the practice of medicine; it regulates medical devices and prescription drugs. So doctors are free to prescribe medications for health conditions or at doses not approved by the FDA, although the practice can sometimes open practitioners to liability claims.

Still, the off-label use of medicine is widely accepted as a beneficial practice. About 21 percent of all prescriptions are for off-label uses.

While pharmaceutical companies are barred from labelling and marketing their drugs for off-label use, they can obtain FDA approval for doing so. Most companies, however, don’t pursue that path because of the cost and time involved.

So off-label uses exist in something of a gray area, employed by doctors on their accord, typically after reviewing studies documenting their effectiveness in treating conditions other than those the FDA has approved.

In a May 2023 article in The Geneva Papers on Risk and Insurance–Issues and Practices, Andrea Parziale of the Netherlands’s Maastricht University explained how vital that practice was during the COVID-19 pandemic, as experimentation was essential to managing the public health emergency.

“(O)ff-label research is key to reducing uncertainties and protecting patients and is recommended by international ethics,” Parziale wrote.

Risk of Off-Label Prescribing

A recent U.S. Supreme Court case illustrates the somewhat tenuous nature of off-label prescribing practices. In March, the Supreme Court rejected a challenge of the FDA’s approval of mifepristone, an abortion drug. The challenge was caught up in the nation’s on-going culture wars over abortion. But had a ban against the drug been upheld, it would have impacted the medicine’s availability for other uses, such as treating miscarriages and Cushing’s Syndrome, as well as pregnancy complications like ectopic pregnancy and preeclampsia.

The threat of liability also looms. In 2006, Maryland psychiatrist Dr. Peter Gleason was charged for promoting the use of Xyrem, a narcolepsy drug, to treat anxiety and depression. Gleason pleaded to a federal misdemeanor, was fined $25 and sentenced to one-year probation in 2010.

But the ordeal left him destitute, and faced with state medical board disciplinary proceedings, he committed suicide. In a November 2021 article in the Journal of the American Academy of Psychiatry and the Law, Shariful Syed of Stony Brook University Medical Center and several coauthors write that his off-label use of Xyrem was “entirely lawful.”

State Lawmakers Aim to Protect Off-Label Prescribing

To protect doctors’ use of off-label prescriptions in their state, Alabama lawmakers recently passed and Gov. Kay Ivey (R) signed SB 72, preventing the Alabama Board of Medical Examiners and the Medical Licensure Commission of Alabama from taking action against a physician for prescribing off-label treatments.

“This bill just allowed a doctor to do what they’re already doing without any fear of reprisal from the State Medical Board,” SB 72’s sponsor, Sen. Arthur Orr (R), said in an interview with the Alabama Reflector.

At least a dozen other states have also considered legislation dealing substantively with “off-label” use of prescription drugs, according to LexisNexis® State Net® legislative tracking system.

In New Jersey, for example, AB 1236 would grant terminally ill patients access to “investigational and off-label treatments.” That bill is pending.

Bills dealing directly with off-label practices also failed this year in Louisiana (HB 813), Maryland (HB 1132), Mississippi (HB 1601), South Carolina (HB 4684) and South Dakota (HB 1168).

Off-Label Drug Use Bills Considered in Over Dozen States

At least 13 states have considered bills dealing substantively with off-label use of prescription drugs in the current legislative biennium, according to the LexisNexis® State Net® legislative tracking system. Two of those states have enacted such bills.

Potential Problems Ahead for GLP-1 Drugs

Dr. Chun-Su Yuan, the Cyrus Tang Professor of Anesthesia and Critical Care at the University of Chicago, said the off-label use of GLP-1 drugs could potentially expand much more due to America’s “national obsession” with weight loss.

Yuan said the drugs are effective at reducing weight, but are still being studied, leading to uncertainty about their use under certain conditions—such as obesity without other medical concerns—and their long-term impacts.

But America’s obsession with weight loss could cause a run on GLP-1 drugs, he said, endangering their supply. He also noted insurance companies might not pay for them, and the increased interest in the drug could lead to more counterfeits flooding the market, which could be dangerous for patients.

“Off-label use could be a big problem,” Yuan said. “I don’t know.”

—By SNCJ Correspondent BRIAN JOSEPH

Visit our webpage to connect with a LexisNexis® State Net® representative and learn how the State Net legislative and regulatory tracking service can help you identify, track, analyze and report on relevant legislative and regulatory developments.