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Peril or Promise: The Uncertainty of Off-label Prescriptions

by Jennifer Wider, MD

(Washington DC, 11/22/04):  The next time you have a prescription filled, make sure you're taking the medication for the right reasons. The use of "off-label" prescriptions is on the rise. "Off label" refers to the use of an FDA approved medication for any purposes other than what is specifically listed on the drug's labeling as the indication for that medicine.  Doctors frequently prescribe medications off label because there is evidence that the drug may effectively treat a condition, even if the drug isn't specifically FDA approved to treat that condition.  

For example, drugs tested and approved for treating seizures are being prescribed off-label for a wide range of conditions from depression to weight loss.  Anti-depressants are being used off-label to treat premature ejaculation, restless leg syndrome and chronic pain syndromes.

Although it is legal for doctors to prescribe FDA approved medication for any reason they deem appropriate, it is illegal for pharmaceutical companies to market drugs for off-label purposes.

"Under current regulations, the FDA can only allow a drug label to list the specific conditions or indications for which the pharmaceutical company sought and received approval, based on extensive testing," said Sherry Marts, Ph.D., vice president of scientific affairs for the Society for Women's Health Research in Washington, D.C.

There may be sufficient evidence to support the use of a drug for multiple purposes, but the company that owns the drug may not sponsor the necessary research trials required for FDA approval, according to Marts, who was previously involved in the ethical review of clinical research.  "In other circumstances, the clinical research may have been done," adds Donnica Moore MD, President of Sapphire Women's Health Group, "but the company may not choose to support the New Drug Application submission process and the subsequent marketing of the product for business reasons."

On the other hand, sometimes off-label usage does lead a company to go through all of the steps required to get a new indication approved for a medicine previously approved for another indication.  Prozac, for example, was originally designed to treat clinical depression but later it was discovered to be effective for premenstrual dysphoric disorder (PMDD). "Intermittent dosing worked and it worked rapidly," Marts said. "And the company was willing to do the clinical trials to get FDA approval." But that's not true for all drugs which may have other indications that aren't officially approved.

Sometimes, however, off-label usage can lead to unexpected and undesirable consequences, for consumers as well as for the pharmaceutical manufacturers.  Drugs prescribed for reasons other than what they were intended for can harm the patient and cost the manufacturing drug company millions of dollars.  For example, Fen-Phen, a combination of the drugs fenfluramine and phentermine, was widely used off label as appetite suppressants for the short-term (a few weeks) management of obesity.

"The drug was never recommended for people who were simply overweight," Marts said. "It was only indicated for people whose health was threatened by their obesity." It was later discovered that the combination of these two drugs could cause heart valve problems and fenfluramine was pulled off the market.  Costly lawsuits were quick to follow.

Another high profile case involves Pfizer, who recently agreed to pay over $400 million in fines and civil damages for illegally promoting Neurontin, an anti-seizure medication. The company falsely promoted it for the treatment of other medical conditions ranging from headaches to attention deficit disorder, despite the lack of scientific evidence.

There are cases when a patient's care requires the use of off-label treatments. Cancer care is a prime example, where treatments are rarely approved for general use against cancer. Instead, they are approved to treat specific stages of specific cancers.

A 1991 government study found that more than half of cancer patients received at least one drug for an off-label indication. Off-label use of drugs is widespread in cancer treatment for several reasons.

It is well known that many cancer drugs are effective against a variety of tumor types.

For example, Cisplatin, produced as Platinol® by Bristol Myers Squibb, works to stop the uncontrolled growth of cancer cells by interrupting the copying of DNA in growing cells. Because uncontrolled cell growth is part of most cancers, cisplatin is often used in the treatment of thyroid and lung cancers. It is only approved to treat bladder, testicular and ovarian cancers.

The use of multiple drugs, which is typical in cancer chemotherapy treatment, is usually an off-label treatment. The FDA generally does not approve multidrug regimens because the potential number of combinations is vast.

Medical care is always evolving. When published research shows that a new treatment approach produces better outcomes for patients, many doctors will incorporate the findings into their practice, ahead of formal approval processes which can take years. These timely changes to care can benefit patients.

Whatever the situation, patients need to protect themselves by asking doctors about their prescriptions.

"Patients should write their questions down before walking into the doctor's office," Marts cautioned.  Dr. Donnica also recommends writing down the reason you are taking each medication in black marker on the medicine bottle so that you don't forget.  "This way, if you see another doctor, it will be clear what medicines you're taking and why if you just bring all your medicine bottles with you."

Before a doctor hands you a medication, ask the following questions:

  1. What is the name of the medication and what is it for?
  2. What is your experience prescribing this?
  3. What kind of side effects can I expect?
  4. Is the medication safe to take with other drugs or dietary supplements that I may be taking?
  5. Is there anything I should avoid while taking it?
  6. What should I do if my symptoms do not go away?
  7. How long do I need to continue this medication?
  8. What side effects should I notify you about?

© October 28, 2004 Society for Women's Health Research

The Society for Women's Health Research is the nation's only not-for-profit organization whose sole mission is to improve the health of women through research. Founded in 1990, the Society brought to national attention the need for the appropriate inclusion of women in major medical research studies and the resulting need for more information about conditions affecting women. The Society advocates increased funding for research on women's health, encourages the study of sex differences that may affect the prevention, diagnosis and treatment of disease, and promotes the inclusion of women in medical research studies. Dr. Donnica Moore has been a member of the Society since 1990 and is a past member of its Board of Directors.

Created: 11/22/2004  -  Donnica Moore, M.D.

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