Risky business: Performing treatments not approved for use in U.S. jeopardizes medical license

Key Points

Three years ago, he attended a cosmetic meeting in Europe and learned about a wonderful new fat-dissolving substance called phosphatidylcholine. This material, he learned, can be injected in a variety of fatty deposits in the neck, eyelids and thighs, among other places. In fact, he learned it was even being used for cellulite.

Returning home, Dr. Fat learned that a whole group of physicians in the United States were already using materials containing phosphatidylcholine and deoxycholate (PC/DC) for fat-melting purposes.

Dr. Fat quickly became a leader in this new field, injecting hundreds of patients and greatly increasing his practice revenues.

Insurance woes

Unfortunately, a full-lid necrosis with significant scarring occurred in one of his lower-eyelid patients. A lawsuit followed.

When Dr. Fat called his malpractice carrier and notified them of the suit, they told him he was not covered. Dr. Fat informed his carrier of all the clinics doing this procedure and of the compounding pharmacies that readily make the material, and he asked why this "off-label" procedure would not be covered. Is Dr. Fat correct?

Dr. Fat is correct that PC/DC is made by many compounding pharmacies. He is also correct in stating that many clinics are now offering this cosmetic treatment. Additionally, he is correct in saying that, in general, off-label treatments are not only acceptable in medicine, but are encouraged by the Food and Drug Administration (FDA) and covered by most malpractice carriers.

Unapproved treatment

What is incorrect is Dr. Fat's assertion that using PC/DC injections for cosmetic purposes is an off-label treatment. This implies that the FDA has approved these substances for another purpose. In fact, the FDA has not.

Currently marketed as "Lipodissolve," PC/DC compounded in the United States is a copy or variant of Lipostabil (Aventis), an approved substance in Germany, Italy and the Czech Republic as an intravenous drug treatment for fat embolism. Subcutaneous injection - the method used by Dr. Fat - is specifically contraindicated in the European labeling of these products.

In the approved European product, phosphatidylcholine is the active product and deoxycholate is the excipient.

'Cease and desist'

Lipostabil has never been approved for any purpose in the United States. In fact, the FDA has already sent a warning letter to one Lipostabil distributor.

In that letter, the FDA specifically stated that there is "no substantial evidence that the product is generally safe and effective for its intended use." The FDA instructed the distributor to "immediately cease and desist marketing and distributing this product."

What is clear is that use of PC/DC in the United States is not off-label. Use of a drug off-label is the practice of a physician prescribing it for a purpose outside the scope of the drug's approved label. An obvious example of this is the use of Botox (botulinum toxin A, Allergan) for cosmetic purposes when, initially, it only had approval for medical purposes.

PC/DC is not approved for any indication in the United States. Although it is true that injectable DC for the purpose of melting fat is currently undergoing FDA trials in this country, it is not, as of this time, FDA-approved for any indication.

Dr. Fat has made a serious mistake. His malpractice carrier has the right to deny him coverage.

Furthermore, Dr. Fat may not only lose the malpractice case (he may be better off settling the case prior to trial) - he is also at risk for losing his medical license for performing treatments that are not FDA-approved for any purpose.

David Goldberg, M.D., J.D., is director of laser research, Mount Sinai School of Medicine, and adjunct professor of law, Fordham Law School.