A prescription acne drug that has helped millions of Americans but has also been linked to serious birth defects soon may be much more difficult for consumers to obtain.
Federal regulators are considering placing extraordinary restrictions on the drug, Accutane, which would require a woman seeking a prescription to provide evidence of a negative pregnancy test each month. She also would have to agree to use two forms of birth control to get a prescription filled.
Also, patients using Accutane, pharmacy workers and prescribing doctors would be required to enroll in a federal registry.
A Food and Drug Administration scientific advisory panel recommended the stringent restrictions last month because previous, mostly voluntary safeguards have failed to sufficiently slow the number of birth defects among Accutane users.
But many dermatologists complained that the proposed registry and other safeguards would violate patients' privacy and make it difficult for some people to take the medication.
"It's going to be more work to take it," said Dr. Barbara Reed, a Denver dermatologist who represented the American Academy of Dermatology at a government hearing. "Patients may ask, 'How much trouble do I want to go through to get this drug?' "
Accutane can cause limb and organ deformities and mental retardation. More than 160 babies have been born with defects blamed on the drug since it went on the market in 1982; hundreds of other women who have become pregnant on Accutane have had abortions, according to the FDA.
Accutane also is available in a generic form called isotretinoin.
The drug's toxicity during pregnancy has long been known, but previous efforts to reduce birth defects -- including stricter product labeling and a limited pregnancy testing system -- have failed to resolve the problem, according to data presented at the hearing.
"The program that has been in place has not been entirely successful," said Arthur Levin, director of the Center for Medical Consumers, a nonprofit consumer advocacy organization in New York City that supports tighter restrictions. "We're not sure why."
Under the new regulations, initially proposed by the drug's manufacturer, Hoffmann-La Roche, women will have to agree to use two forms of birth control, one of which must be a "primary" form, such as the birth control pill. Abstinence, however, would be considered one form of birth control, said Gail Safian, a spokeswoman for Hoffmann-La Roche. Monthly pregnancy tests would have to be performed by a certified lab -- home tests won't do -- and the results would have to be verified.
But some speakers at the FDA panel hearing expressed concern that the restrictions might lead some patients to obtain Accutane on the Internet, without any medical supervision.
More than 1 million prescriptions for Accutane or its generic equivalent are written each year in the United States.
The drug, which is derived from vitamin A, is typically taken for at least five months. In most patients, it is highly successful in suppressing cystic acne -- a severe form of acne -- for long periods of time or even permanently. Most Accutane users have failed all other acne therapies and use the powerful drug as a last resort.
Despite its effectiveness, Accutane has been associated with other side effects, some serious. The drug is linked to an increased risk of depression and suicide.
Accutane also can cause elevated triglyceride levels, which can cause pancreatitis, and patients usually are required to undergo monthly blood tests to monitor triglyceride levels. Severe dryness of the lips, skin and mucous membranes is common when taking the medication.
Accutane prescriptions have fallen about 20% since 2001, and dermatologists say the proposed restrictions will cause more patients who could benefit from the drug to refuse a prescription.
"I think this will potentially have an impact in terms of making it a less desirable medication," said Dr. Steven Weiss, chief of dermatology at Cedars-Sinai Medical Center in Los Angeles. "But it's been a godsend to many patients."
If asked to enforce the stricter regulations, some doctors and pharmacists may decide to forgo offering the drug, said Reed, the Denver dermatologist.
Doctors already are required to register to prescribe Accutane, and more than 12,000 dermatologists have done so, according to Hoffmann-La Roche.
"I have heard from my acquaintances who say, 'I'm not going to do it; let someone else do it,' " Reed said. "Patients are going to have to find a doctor who will write the prescription and a pharmacy that will fill it."
That could become difficult in rural areas of the country, Reed adds.
How to enroll patients into the registry and honor new federal laws protecting patient privacy is another large hurdle, said Dr. Wilma Bergfeld, a dermatologist at the Cleveland Clinic and a member of the advisory panel. "How the FDA will work around that, I don't know," she said. "This is breaking new ground."
The FDA must decide whether to adopt the advisory panel's recommendation. It's unclear how the regulations would be enforced. Men also would be enrolled in the registry to receive counseling about the importance of not sharing their pills with women.