Off Label Drug Use

Originally published in the North Lake Tahoe Bonanza (link), September 16, 2010. Medical News recently reported that Botox manufacturer, Allergan Inc., settled for a fine of $600 million for alleged promotion of off-label Botox use. Eli Lilly paid $1.41 billion and Pfizer paid $2.3 billion in fines for similar off-label drug promotion. This raises questions: What is off-label drug use? Is it good or bad?

Before a drug gains FDA approval, the manufacturer undertakes a lengthy application process during which they demonstrate their medication is safe in the studied dose and manner of administration. They also must show the medication is effective against the exact condition specified. For example, a pharmaceutical company may invent a new chemotherapy agent and show it is safe and effective in treatment of pancreatic cancer. When the FDA approves the drug, the approval will be for pancreatic cancer and pancreatic cancer only.

Once approved, the drug is available for physicians to use in any manner they feel best for their patients. They can prescribe the drug for pancreatic cancer, or may opt to use it for a related cancer. They might also use it in a different population — for example, a child or elderly patient with pancreatic cancer. These groups are rarely included in initial applications. Off-label prescribing in this manner is quite common; estimates suggest 50-75 percent of pediatric and oncology prescribing is off-label.

So why wouldn't drug companies get the drug approved for other cancers, so doctors don't have to use them off-label? The answer is simple: Money. On average, it costs $200 million and takes several years for a drug to gain approval. To complete supplemental applications for alternate uses of the same drug, pharmaceutical companies would have to spend many additional millions on studies for each additional use and it simply wouldn't be profitable — especially for less common medical problems, where the market is small.

Drugs used off-label are often older drugs, now off-patent, for which companies have little financial motivation to pursue a costly supplemental application. For years the FDA did not permit aspirin makers to tell doctors or patients about studies demonstrating aspirin's potent effect in preventing heart attacks, since no single company had completed a supplemental FDA application.

Off-label drugs are also commonly used in rapidly evolving fields. New discoveries are made by researchers every day. They report their findings in medical journals. I don't think physicians should withhold potentially beneficial treatments from patients awaiting FDA approval. Instead, doctors need to read the literature, study the risks and benefits, and then decide if they feel it appropriate to recommend the drug's use.

As both a patient, and a physician, I think this information should be freely available. I expect my doctor to offer me off-label drugs if evidence suggests they may benefit me. However, as in all things, it is imperative for patients to educate themselves about the risks and benefits of any medication they are considering. Do you need the medication at all? Are there lifestyle changes you could make instead? Are there alternative treatment options? Particular caution should be taken when considering a new medication — the FDA has approved many drugs later found to cause problems. Only after you consider all this, can you best decide to take any drug — off-label or not.

—Dr. Rebecca Gelber is an Incline resident and graduate of The Johns Hopkins School of Medicine. She practices locally at Tahoe Aesthetic and Integrative Medicine, 775-298-1750, or