What use "off-label" of a drug really is, and why you need to know about it

When your doctor prescribes a medication for you, you assume probably - and rightly - which is an FDA approved treatment for your condition.

This may or may not be the case.On average, one in five prescriptions are written for "off-label" use of a drug. This means that your doctor thinks that the drug will help you, even if it has not been specifically approved for illness or symptoms.

Prescription medications off label is legal and common, and can be beneficial for patients. For example, beta blockers (drugs approved for lowering blood pressure and treating heart disease) may also help some people with migraines or anxiety. Tricyclic antidepressants are not FDA approved to treat nerve pain, but many doctors do a good drug of choice for this disease in question.

When the off-label use is supported by strong evidence presented to the FDA, which can lead to FDA approval for that use. Most people know of botulinum toxin type A (Botox or) as a way to remove wrinkles, but originally won approval from the FDA for the treatment of a number of different conditions, sweating Excessive use of muscle spasms.

While some off-label uses for drugs are well studied and have been a part of medical practice routine for years, many off-label uses not. When a drug has not been tested for an individual or a group of people in particular state, it could have unexpected and dangerous effects. For example, doctors prescribe antipsychotic drugs such as haloperidol, off-label to manage behavior problems in people with dementia. Now we have evidence that the administration of these drugs to people with dementia is associated with a higher mortality rate.

Advertising off-label and courtsThe application can not become particularly difficult when it comes to drug advertising. Historically, the FDA has prevented most pharmaceutical companies advertise proactive off-label uses for their products. But that could soon change, thanks to two recent judgments that federal courts have cleared the way for the promotion of unapproved uses for prescription drugs. These decisions and their implications are discussed in New England Journal of Medicine.

The first case occurred in 2012 and involved the drug Xyrem (FDA approved to treat a rare disorder called narcolepsy sleep). In this case, a representative of drug sales, Alfred Caronia, facing a lawsuit after suggesting to physicians who use the drug as a treatment for other conditions such as insomnia, fibromyalgia and Parkinson's disease, despite lack of evidence that the drug worked with these health problems.

As Dr. Ameet Sarpatwari, a lawyer and an epidemiologist at Brigham and Women's Hospital and Harvard Medical School, explains that "the government has not indicated that the off-label promotion of Caronia was false or misleading. Rather He argued that the restriction on off-label promotional moved directly its real interest in protecting patients from unsafe and ineffective drugs, and therefore the constitutional requirement to intervene in the protected commercial speech met. "A federal court It divided appeals disagreed, however, noting that the Government has failed to demonstrate that less restrictive measures of speech, that the exemptions would not have achieved the same goal.

A more recent case is that of a fish oil pill called Vascepa ordinance approved by the FDA to reduce high triglyceride levels. The manufacturer, Amarin, sought approval from the FDA to promote its use in patients with moderately elevated triglyceride levels - use of the indications that the FDA considered, but not adopted.  

When the FDA said no, Amarin sued, alleging that - as in the case Caronia - off-label promotion Vascepa was protected by the First Amendment. The court ruled in his favor. Dr. Aaron Kesselheim, director of the Division of Law Programme and the therapeutic Pharmacoepidemiology and Pharmacoeconomics settlement at Brigham and Women's Hospital and associate professor of medicine at Harvard Medical School professor, said the impact of the case could Amarin be substantial: "The decision on the case Amarin is a new setback for the FDA to try to maintain their level of off-label marketing If these cases become the law of the land, there would be no motivation for companies submit. its evidence to the FDA for approval of additional indications. "

The courts have decided that pharmaceutical companies have the right to provide, and consumers have the right to receive accurate and not misleading drugs about which can help them to information. However, Dr. Sarpatwari advises that patients should also be aware that the quality and objectivity of the information is at risk. "Off-label claims do not have full and impartial review of the promoted FDA has determined safe and effective use," he said. "However, companies that make these claims have financial incentives to encourage more prescription drugs."

What this means for you
You will need to work ever more closely with your doctor to assess the risks and benefits of any medication recommended for you to off-label use. When your doctor prescribes a new medication, ask if it is approved by the FDA for your condition. If not, ask what is already known (and unknown) to use for your health, and if there are drugs that are just as good for what approved. 
By: Gregory Curfman, MD,