Friday, August 14, 2015

Off-label uses of drugs

From Wayne State:

An off-label treatment means that the drug has not been officially approved by the FDA for a particular disease, but there is evidence and experience to support the use of that drug for the treatment of that disease.Off-label treatments are very common in medical practice and usually are the result of years of experience and research carried out by doctors and scientists. However, since these drugs have not been formally tested in phase III trials with successful results presented to the FDA, they are considered “off-label”. Nonetheless, from small scale studies, there is reasonable evidence to support their use based on their ability to reduce the frequency of relapses or slow the disease progression or both. Examples of off-label treatments for multiple sclerosis include:

IVIG (intravenous immunoglobulins)
Plasmapheresis or plasma exchange
Autologous hematopoetic stem cell transplant therapy


Note that on January 22, 2010 the U.S. Food and Drug Administration approved Ampyra (dalfampridine) as a treatment for walking impairment in multiple sclerosis. The drug is not a disease-modifying drug but it does impact symptoms.

**The case of Neurontin had some publicity:

Gabapentin (Neurontin) was allegedly promoted for various off-label uses for treating a multiple number of neurological conditions. Amusingly, regardless of the very fact that the gabapentin was initially approved by US FDA in 1994 as adjunctive therapy in the treatment of partial seizures with and without secondary generalization in adults with epilepsy, its nominal market these days are epilepsy and seizures. A variety of off-label or unapproved uses of gabapentin have been reported, encompassing treatment of neuropathic pain, diabetic neuropathy, complex regional pain syndrome, bipolar disease, attention deficit disorder, migraine, restless legs syndrome, periodic limb movement disorders of sleep, etc., All these purported off-label uses have culminated in drug sells worth many billion dollars resulting in huge revenue

from S. Gupta, J Pharmacol Pharmacother. 2014 Apr-Jun; 5(2): 88–92

** From IPBiz in 2006,

The case of Dr. Gleason concerning the off-label promotion of Jazz Pharmaceutical's Xyrem has attracted some attention.

Doctors are free to prescribe drugs for uses which have not been approved by the FDA (off label uses). Drug companies are NOT free to advertise off label uses. Thus, there is some incentive for drug companies to "encourage" doctors to do what the drug companies can't do. A variant of this problem came up in the Hatch-Waxman context with the drug Neurontin, discussed by me in Intellectual Property Today.

Dr. Gleason got over $100,000 last year from Jazz. That alone probably is not the problem in the aggressive stance taken against Dr. Gleason. The active ingredient in Xyrem is gamma hydroxybutyrate, or GHB, the "date rape" drug. That reality sets this case a bit apart.

In June 2014,

Manufacturers of the drug Neurontin reached a $325 million class action lawsuit settlement Wednesday over allegations they fraudulently marketed the prescription drug, which is used to treat seizures, restless leg syndrome, and pain caused by shingles. Sommers Schwartz partner Jason Thompson was among the class action attorneys to first sue Pfizer over ten years ago for the abuses. The cases were filed on behalf of third-party payers – union funds, self-insured municipalities, and heath insurance companies – who had been overcharged for the drug.

The Neurontin settlement marks the end to a nearly 10-year-old class action lawsuit battle in which plaintiffs, who included both direct purchasers and third-party payers, accused Pfizer of delaying generic versions of Neurontin and promoted the drug for unapproved uses. Pfizer reached a $190 million Neurontin class action settlement with direct purchasers of the drug (consumers) in April.



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