What can you do if you learn you have a life-threatening illness but there is no Food and Drug Administration-approved medicine to treat it? Sometimes, there is nothing to do but hope. Very often, though, your doctor will be able to prescribe a drug or medical device that has been approved by the Food and Drug Administration (FDA) for a different condition. This practice, called “off-label” pre...

We identify the rate of off-label use of prescription drugs in the United States during 1993-2008. We apply Detection Controlled Estimation to a comprehensive crosssection of prescriptions and find that rates of off-label use rise from 30.2% to 39.1% during this period. This coincides with a surge in settlements of Department of Justice lawsuits for off-label marketing. Additionally, physicians...

Tinnitus is the conscious perception of a phantom sound in the absence of an external source. For 1 in 100 of the general population, the condition severely affects quality of life. In spite of the fact that the market for a drug indicated for tinnitus relief is huge, there are still no FDA-approved drugs, and the quest for a tinnitus-targeted compound faces important challenges. A wide variety...

We have concerned ourselves at the gastroenterology unit of the VU University Medical Center with drug repositioning since 2001. Generic therapy, often successfully used off-label in daily practice and well described in literature in large observational studies, should be further developed for safe and economically viable use. To prevent off-label use legislative authoritiesshouldstimulate re-r...

drug into interstate commerce unless the FDA has approved the drug and its label.2 A second provision prohibits manufacturers from introducing misbrandeddrugs into interstate commerce.2 Misbranding is considered to occur if drug labeling contains information that describes unapproved uses or is misleading or insufficient to support safe use for approved indications.2 Materials are considered pa...

Between 35% and 90% of the drugs prescribed to hospitalised children are either not licensed for children’s use or are prescribed outside the terms of their product licence (off label prescribing). 2 Subsequent adverse reactions are more likely than with licensed products (6.0% v 3.9%). We analysed the extent of prescribing off labelled products in a representative cohort of children in primary...

Many children in the USA are prescribed psychotropic drugs that have not been fully investigated in paediatric clinical trials. The common practice of prescribing psychotropic drugs off-label poses unknown and potentially serious short- and long-term consequences for these children. This paper briefly reviews the factors associated with the lack of paediatric clinical trials. We advocate a shif...

Now six months old, ALSUntangled (1) has 85 followers on twitter, and receives weekly questions about alternative and off-label ALS therapies. Our NING has 40 ALS clinician-scientists from four countries examining these therapies, with 18 active discussions. New discussions underway since our last update (2) include: intravenous glutathione, the Lourdes ALS Miracle, and the Nepsis Institute. Re...