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Off Label Drugs: Risk Vs. Benefit

WASHINGTON , DC (Ivanhoe Newswire) - Meant for a specific condition, but prescribed to treat another. It's called off-label drug use and doctors around the country are doing it for their patients.

To hear her now, you'd never know Marquita Lister almost lost it all.

"I've had the kind of life that people only dream about," Marquita Lister, an opera singer, said.

The international opera star was diagnosed with Polymyositis; it attacks the lungs and muscles.

"I almost died," Marquita said.

A medical team at national rehabilitation hospital came up with an innovative form of therapy, including a drug normally used to treat lymphoma patients. Doctors took a chance with the off-label drug use and it paid off.  Marquita pulled through.

Experts said as many as one in five prescriptions in the U.S. is used for something it's not approved for by the FDA, and it's perfectly legal. Antipsychotic drugs are often prescribed for dementia, anxiety drugs are used as sleep aids and some cancer drugs for eye problems.

Off-label drugs worked for Marquita, but they aren't safe in all cases. Studies show some anti-psychotics used to treat elderly patients with dementia did show benefits, but also increased risk of death. Other studies found a drug used to help patients from bleeding uncontrollably during surgery, increased the odds of blood clots when used off-label. While studies and results may differ on the practice, off-label drugs got Marquita back on stage, for her the risk was worth it.

Off-label drug use is possible because once the FDA approves drugs, physicians are allowed to use their own judgment on what they prescribe it for. However, the FDA does bar drug makers from promoting their products for off-label use.  Because of that, manufacturers are fighting the off-label rule in court right now, claiming the ban violates their first amendment rights.

RESEARCH SUMMARY

BACKGROUND: In the United States new drugs are tested in clinical trials before they are approved for use in the general public. The clinical trials are done to prove that the drug: works to treat a certain medical condition, works the way it is expected to, is safe when used as directed. When the US Food and Drug Administration, FDA, is satisfied that the drug works and is safe, it and the maker of the drug create the drug label. This is not an actual label that sticks onto a bottle, but a report of very specific information about the drug. The FDA must approve this report, which is made available to all health professionals who prescribe or sell the drug. The drug label gives information about the drug, including the approved doses and how it is to be given to treat the medical condition for which it was approved. When a drug is used in a way that is different from that described in the FDA-approved drug label, it is said to be an off-label use. This can mean that the drug is: used for a different disease or medical condition; given in a different way, given in a different dose than in the approved label. For example, when a chemotherapy drug is approved for treating one type of cancer, but it is used to treat a different cancer, it is off-label use. (www.cancer.org)

BENEFITS: Off label drugs are not all necessarily bad. According to the American Cancer Society, cancer treatment often involves using certain chemotherapy drugs off-label, because a chemotherapy drug approved for one type of cancer may actually target many different types of tumors. Off-label use of a drug or combination of drugs often represents the standard of care. Beta-blockers are another example of beneficial off-label prescribing. Such medications are FDA-approved for the treatment of high blood pressure, but are widely recognized by cardiologists as a standard of care for patients with heart failure. And in fact, some beta blockers are now formally approved to treat heart failure. It's not uncommon for off-label uses to eventually get approved by the FDA. (www.webmd.com)

RISKS: There is a debate about off-label drug use. Doctors emphasize that off-label drug prescribing has its place in medical practice, but they also admit that using a drug off-label can raise the risks of lawsuits should a patient have unwanted or bad side effects. Two FDA approved medications for individual short- term treatment for obesity had negative outcomes when used together. Doctor's began prescribing the two after the results of use showed dramatic weight loss. However, along with weight loss, patients experienced severe, and potentially deadly, heart valve damage, an outcome that triggered a multi-billion dollar lawsuit. Although off-label drugs can be beneficial, there is little or no scientific evidence to prove that they work. (www.webmd.com)

FOR MORE INFORMATION, PLEASE CONTACT:

American Cancer Society
www.Cancer.org

U.S Food and Drug Administration
www.FDA.gov