STEVE INSKEEP, HOST:
Now it's not just patients with hepatitis C who might be prescribed a drug combination that has not been approved by the FDA. It's actually quite common for doctors to prescribe drugs off-label, as it's called. In fact, one of five drugs are prescribed this way.
NPR's Patti Neighmond has been asking why.
PATTI NEIGHMOND, BYLINE: There are many reasons why doctors prescribe drugs off-label - either alone or in combinations. It could be a last resort. A cancer drug that shows promise when all other options have failed. Or it could be anecdotal evidence from other doctors who say the medication works for certain conditions.
Dr. Caleb Alexander co-directs the Johns Hopkins Center for Drug Safety and Effectiveness. He says patients may not know it but their prescriptions are often made for conditions that haven't been scrutinized or approved by the FDA.
DR. CALEB ALEXANDER: For example, the use of anti seizure drugs to treat migraine headaches or depression or neuropathic pain.
NEIGHMOND: Or the use of certain cancer drugs to treat macular degeneration or blood pressure medication to treat heart failure or help prevent headaches, all considered off-label.
DR. RANDALL STAFFORD: Off-label drug prescribing is neither bad nor good, per se.
NEIGHMOND: Internist Randall Stafford with the Stanford Prevention Research Center.
STAFFORD: But really depends on the level of evidence available to support a particular use of a drug for a condition that may not have been specifically approved by the FDA.
NEIGHMOND: Evidence is the critical word here. For some off-label use research and studies show they're effective. But they're not FDA approved to treat these health problems. Researcher Alexander says that's because drug companies often don't have a financial incentive to spend the hundreds of millions of dollars it could cost to get FDA approval for an additional use.
ALEXANDER: If there's only a few years left in a drug's patent life, then pretty soon the drug will be going generic and pharmaceutical manufacturers face little incentive to seek an additional FDA approval.
NEIGHMOND: So that often leaves decisions up to the individual doctor.
ALEXANDER: Clinicians are between a rock and a hard place because there are thousands of drugs on the market and many clinicians use dozens of medicines at least to treat patients.
NEIGHMOND: In one survey of doctors, Alexander found in about half of all cases where medications were prescribed, doctors didn't know whether they were FDA approved for the specific condition.
Stanford's Randall Stafford says that can result in risky prescribing. For example, some doctors use anti-psychotic medication to treat patients with advanced dementia who have problems sleeping at night.
STAFFORD: They may have reversal of day and night and medications in the class of anti-psychotics often act as a mood stabilizer and have sedating properties.
NEIGHMOND: Which may make it easier to care for these patients but at the same time can increase their risk of heart attack.
Researchers agree it's complications like this that make it so important to have a comprehensive nationwide database for doctors to consult when considering medication that's not approved by the FDA.
Until that's available, it'll be up to patients and their doctors to discuss the pros and cons of using medication off-label.
Patti Neighmond, NPR News.
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