By the time Brittany Bonds gave birth to her third son in the back of an ambulance 10 weeks before he was due, she no longer trusted the drug Makena.
The drug was supposed to prevent premature birth and improve the health of a baby. Mrs. Bonds' son Phoenix was in the NICU for 83 days. He has a lot of health problems.
Makena is an example of a medication that was fast-tracked by the FDA to the market, even though there was considerable doubt about whether it worked.
The debate over Aduhelm's approval has renewed attention to the expedited pathway for drugs to reach the market. A bill sponsored by a Republican in Congress would make it easier for a company to get a drug approved. The F.D.A. would be given more authority to get definitive answers about fast-tracked drugs under a proposal by a leading Democrat.
The pharmaceutical industry spent a lot of money on political campaigns last year and is likely to object to any limits on the fast-track process.
There will be tremendous resistance from the pharmaceutical industry to tighten the rules on accelerated approvals, said Dr. Michael Carome, a director at Public Citizen.
A large study showed that Makena had the same effect as a placebo. A year ago, the F.D.A. proposed taking the drug off the market. It plans to hold a hearing on Makena's fate that will focus scrutiny on what some critics claim is a speed-over-science drug approval process.
Mrs. Bonds wants the drug taken off the market because it didn't work for her.
African American women, who face some of the highest preterm birthrates in the developed world, will be the focus of Covis Pharma's argument at the upcoming hearing.
Premature babies are more likely to be born dead or disabled. He was paid $1,200 in consulting fees when he testified in favor of the drug.
The F.D.A. has had to weigh passionate, sometimes desperate pleas for access to drugs against the available science-based evidence.
As of December, the agency had issued 278 approvals under the program. The approvals don't prove that a drug prolongs survival or improves quality of life. If a follow-up study proves a benefit, drugs can be put on the market and kept on the market.
This pathway has given patients earlier access to life-saving drugs, a point of pride for industry groups like BIO. A BIO representative told lawmakers last week that he supported a pending plan for drugmakers to use real-world evidence to prove that an accelerated approval drug works. The program is in its current form, according to the drugmakers' association.
Critics and watchdog groups contend that Medicare has spent billions on accelerated approval drugs, even as drugmakers drag their feet to complete follow-up studies which can lead to withdrawal of the drug. Fast-tracked drugs that showed little benefit stayed on the market.
The F.D.A. was heavily criticized for its actions on the pain drug Vioxx, which was withdrawn in 2004 over findings that it increased heart attacks and strokes. The 21st Century Cures Act gave more avenues for expedited reviews.
The F.D.A. faces another round of scrutiny next week when its advisory panel reviews a new drug, Amylyx, for a fatal neurological disorder. The maker of the drug is seeking traditional approval, even though it meets one bar for accelerated approval.
April Grant, a spokeswoman for the F.D.A., said that the agency was working to ensure that drugmakers completed follow-up studies of the drugs in a timely manner. The agency will work with Congress to close gaps if it finds gaps in its authority.
The program's history goes back to 1988, when AIDS activists protested at the agency headquarters, angry that it had done so little to help young men dying of AIDS.
They drew each other's bodies with chalk. The police dragged them away after cuffing them.
The Treatment Action Group is led by Mark Harrington, who helped organize the 1988 demonstration. He said that the protest led to discussions about access to new drugs.
By the mid-90s, drugs that initially seemed promising fell short, but by the end of the 90s, they delivered a medical miracle.
The regulations helped draw more companies into the space. They led to the discovery of effective treatments.
As researchers have identified drugs that deliver minimal, if any, gains for patients, Mr. Harrington and others have watched with concern.
According to an investigation published in The BMJ last year, nearly half of the drugs authorized under accelerated approval have not been proven to extend survival or improve quality of life. There were two dozen drugs that had been on the market for five years or more.
20 percent of 93 cancer drug treatments cleared since 1992 were proven to extend overall survival, while others remained on the market after follow-up studies showed more modest gains, like delaying tumor growth. The F.D.A. said it can be difficult to assess survival improvement.
One drug, Avastin, got accelerated approval to treat a brain cancer. A follow-up study didn't show extended survival or improved quality of life, but that didn't stop the FDA from giving full approval for that use.
The F.D.A. revoked accelerated approval for one use of a drug despite emotional pleas to allow it for cancer patients in 2011.
In 2010, the F.D.A. backed off its decision to withdraw Midodrine for patients with dangerously low blood pressure, just a month after telling the drugmaker it had not been able to provide evidence of the drug's benefit.
The agency's retreat was called dangerous by researchers.
The makers of Makena argued that their drug should remain available. The maker of Makena reminded the F.D.A. that the agency altered course on the blood pressure drug because of the concern over losing access.
The Preterm Birth Prevention Alliance, funded by Covis, may testify at the hearing over the fate of the drug.
Scientists don't know what causes the process of giving birth in humans, and that's the beginning of the story of Makena. The results of a study on the drug's active ingredient, a form of the hormone progesterone, appeared in 2003 and seemed to indicate a way to prevent it.
The study showed that those who took the medication had a 34 percent reduction in their risk of having a premature birth.
The data does not provide convincing evidence of effectiveness according to an F.D.A. statistical review. The report said that the drug seemed most effective when started before 18 weeks of the pregnancy, when the fetal or newborn death rates were the most pronounced.
The only approved drug meant to reduce the risk of recurrent preterm birth is the one granted accelerated approval by the F.D.A. Makena became so frequently prescribed that it was difficult to study in the United States, because top medical societies accepted funding from the drug's maker.
The results of a large study were in by 2019. The percentage of women who gave birth while on the drug was the same as those who took a placebo.
The F.D.A. looked at the data to see if there was a subgroup of patients in the United States. It couldn't find one. The F.D.A. wanted to stop using the drug.
The drug's maker argued that the studies left open the question of whether the drug benefited high-risk Black women.
It is a concern shared by others who have no financial stake. The second study was underpowered because it did not include many Black women.
It's unfair to yank the labeled indication for a drug that is helpful to a minority just because it's not helpful to the majority, said Dr. Greene, who is an associate editor of the New.
The debate over the drug has divided the maternal-fetal medical care community. He wants the drug to remain approved, but is unsure if that will happen.
Olivette Bennett, a pregnant Baltimore woman who is black, stopped taking the drug because she didn't think it was worth it.
The company leaders did periodic reviews of the study but continued to market the drug as something that could help women, according to the lawsuit. AMAG argued that the lawsuit was an attack on a drugmaker's right to advertise a drug approved by the FDA.
Mrs. Bonds said she began taking Makena after a stillbirth in 2011. Her first two sons were born several weeks short of full-term.
She took the drug while pregnant with her third child. She was disappointed that his birth was the earliest. Mrs. Bonds said the drug should have been studied more.
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