Two weeks ago, the Food and Drug Administration announced that it would remove the drug midodrine from the market because the drug’s maker never confirmed that the medicine — approved in 1996 under an abbreviated process — actually worked against dizzinessand fainting.
But 100,000 patients take midodrine for conditions many say would otherwise be disabling, and many flooded the agency with complaints. So on Friday morning, top F.D.A. officials announced that they had backtracked and would continue to allow midodrine to be sold. The announcement came after inquiries by The New York Times.
“In a different situation, we might act differently,” said Dr. Joshua Sharfstein, principal deputy commissioner. “But in this case, it does not make sense to pull access to the drug while we get better data.”
Dr. Janet Woodcock, director of the agency’s drug center agreed. “Patients are out there thinking doom is about to fall, and they can’t go about their normal business,” she said.
Lindsey Mills, a 20-year-old from Baltimore, was among those granted a reprieve. Five years ago, she came back from a trip to the mall and found that she could barely stand because of dizziness. After seeing a parade of doctors, a specialist finally prescribed midodrine, which “was the only thing that allowed me to keep some function and attend classes and not be completely bedridden,” Ms. Mills said.
The agency’s flip-flop demonstrates the difficult choices regulators face in policing the nation’s drug market. Cracking down on drug makers sometimes means stranding desperate patients. And now that Congress has given the Food and Drug Administration greater powers to insist on better information about life-saving medicines, such disputes may become more common.
In a similar case, the F.D.A. is now considering whether to withdraw its approval for the use of Avastin, a cancer medicine made by Genentech, for breast cancer. Recent clinical trials failed to prove that Avastin was helpful in treating breast cancer, but patients and some in Congress expressed outrage that the agency could withdraw approval for the use, an action that could lead insurers to stop paying for it.
Midodrine treats a condition known as postural orthostatic tachycardia syndrome, or Pots. Such patients suffer a severe drop in blood pressure when they stand because blood pools in their legs when vessels fail to constrict. Symptoms range from dizziness to fainting, and the condition is most common among teenage girls.
But midodrine’s maker got the drug approved in 1996 under a process known as accelerated approval, in which drugs for severe or life-threatening conditions are approved even though the evidence for their effectiveness is scant. Under the program, drug makers must promise to conduct more rigorous studies to confirm that the medicines work as hoped.
Accelerated approval had its beginnings in the AIDS crisis, when dying patients said they were willing to accept uncertainty in exchange for faster access to possible cures. But more than a third of the 90 drugs approved under the program since 1992 never had studies done proving efficacy. In some cases, drugmakers profited handsomely from sales, refused to invest in promised studies, and bet that the Food and Drug Administration would not risk angering patients by taking action.
The agency’s failure to crack down led to fierce criticism on Capitol Hill, and midodrine became a telling example of these failures. The drug was developed by Roberts Pharmaceutical and originally called ProAmatine. It has yielded more than $257 million in sales, according to government investigators. Roberts was bought in 2000 by Shire Pharmaceuticals. Removing midodrine from the market would have been the first time the F.D.A. took such action under the accelerated program.
Matthew Cabrey, a Shire spokesman, said he could not explain why Roberts never undertook the needed clinical trial. In 2004, Shire conducted a test in which 24 Pots patients were given either midodrine or a placebo, raised to a vertical position and asked to report when they felt like passing out. The trial failed because it was poorly designed, Mr. Cabrey said.
By then, generic versions of the medicine had cannibalized most of Shire’s sales, so the company did not conduct another trial. On Thursday, Mr. Cabrey said that Shire would not appeal the F.D.A.’s decision to remove midodrine from the market since the company now has less than 1 percent of midodrine’s sales. On Friday, Dr. Jeffrey Jonas, Shire’s senior vice president of research and development, said the company had changed its stance and would appeal.
“There is substantial evidence the drug does work,” Dr. Jonas said.
Parisa Emam, 21, said she was just starting her freshman year at Western Washington University in Bellingham when she began fainting and her vision became so blurry that she could no longer read. She had to use a wheelchair until starting on midodrine. Now she can cook and go grocery shopping, she said.
“This fall I’m going to attempt to take three classes, if I can still get hold of the medication,” she said.