The prescription drug market is one part Big Brother and one part Wild West. To get a product into pharmacies, drugmakers must supply every data point the Food & Drug Administration requests to demonstrate the medicine is safe and effective, a process that can take eight years or more. Once the agency has approved a drug for its specified treatment, though, doctors may prescribe it for any ailment, as long as there is reason to believe that the drug will do some good.
These so-called off-label uses can mean a ton of money to drug producers. For example, Lidoderm, the pain patch from Endo Pharmaceuticals Holdings Inc. (ENDP), is projected to generate $300 million in sales this year as a treatment for all kinds of aches and pains. But it was approved for just one thing: alleviating pain from shingles, which is caused by a virus related to chicken pox.
Drug companies often seek FDA approval only for narrow applications because it is easier to prove effectiveness for one illness than for a broad range of diseases. This was the case with Celebrex, Pfizer Inc.'s (PFE) versatile painkiller, which initially was O.K.'ed for just arthritis pain. Anti-psychotics are also widely prescribed off-label, as are drugs for cancer and heart disease. A 2006 study by Dr. Randall Stafford, a Stanford University medical professor, found that 21% of all prescriptions are for unapproved treatments. For cardiac medications, the share is as high as 46%.
The FDA formally bars drug company representatives from talking up off-label treatments. But the fact is, doctors are often open to testing an approved drug in novel settings when their patients don't respond to any standard treatment. Drug reps encourage this behavior by handing out studies showing how their drugs performed in various clinical trials when doctors request such data. They also give away samples as a favor to patients. Doctors and patients then pass stories of successful off-label treatments to one another, boosting demand for the medications.
Sometimes the drug reps cross the line. In the late 1990s, Warner-Lambert Co. illegally promoted off-label uses of Neurontin, an anti-seizure drug for epilepsy, hiring doctors to plug the product at physician conferences in resort settings. Although these practices occurred before Pfizer acquired Warner-Lambert in 2000, Pfizer paid a $240 million fine when the acquired unit pleaded guilty to charges of off-label marketing in 2004.
Cephalon Inc. (CEPH) is currently under investigation by the Connecticut attorney general for off-label marketing of Actiq, a narcotic lollipop approved for pain spikes that cancer patients suffer. The company says its reps promote products only for their labeled uses, and that it is cooperating with the Connecticut authorities.
By Michael Arndt