◼ TECHNOLOGY Bloomberg Businessweek March 30, 202024
JILL LEHMANN/GETTY IMAGES. DATA: U.S. FOOD AND DRUG ADMINISTRATIONTHE BOTTOM LINE Drugmakers count on an accelerated
regulatory process to get critical treatments to patients quicker.
The case of Makena is throwing speedy approvals into question.With the treatment widely available, the com-
pany says, doctors were hesitant to put prospec-
tive mothers in testing where they risked receiving
a placebo. It also says that demographic differences
among patients may have affected the results.
Until the FDA makes a decision about Makena—
it hasn’t indicated when this will happen—doctors
and hospital staff will find themselves weighing
an older favorable study against a newer damag-
ing one. The two major national professional orga-
nizations in this field—the American College of
Obstetricians and Gynecologists and the Society for
Maternal-Fetal Medicine—continue to say Makena is
an option for at-risk pregnant women. “The jury’s
still out on this,” says Mary Norton, a physician who
co-wrote the Society for Maternal-Fetal Medicine’sstatement about Makena, and noted that the drug
is considered safe.
An independent advisory committee that
included doctors, scientists, and statisticians recom-
mended in a close vote that the FDA pull Makena’s
approval. Adam Urato, chief of maternal-fetal med-
icine at MetroWest Medical Center in Framingham,
Mass., says he had concerns about the medication
even before the latest study and has been vocal in
urging the FDA to withdraw the drug. “You don’t
want to use medications in pregnancy unless you
know that they’re safe and effective,” he says.
The FDA acknowledges that accelerated
approval inherently creates uncertainty, but it
maintains that the risk is worth it. “We would
expect every once in a while that we would be
wrong,” says Janet Woodcock, director of the
Center for Drug Evaluation and Research at the
FDA. “Otherwise,” she says, “we’re not taking any
chances” and making patients wait too long for new
treatments. �Emma Court, with Cristin Flanaganconsider delisting Makena—a rare step for an
approved treatment. For the women in a follow-up
study required by the FDA, released last year,
Makena did not decrease repeated preterm births.
“To see these women and their families have to go
through this experience multiple times is heart-
breaking,” says Michal Elovitz, a physician and
director of the Maternal and Child Health Research
Center at the University of Pennsylvania’s Perelman
School of Medicine. “They want to know why and
why it didn’t work. And the only answer we have
for them is we have to figure that out.”
The controversy over Makena has intensified
the debate about the FDA’s use of its expedited pro-
cess, which accounted for 19% of approved drugs
in 2019, an increase from 13% from 2015. The FDA
has focused especially on stepping up the avail-
ability of new cancer drugs and has also approved
medicines to treat sickle cell disease and rare
inherited disorders like Duchenne muscular dys-
trophy and Fabry disease.
The drug industry is counting on the accelerated
approvals and other fast-track programs to clear
the way for new biomedical therapies. Biogen Inc.
may turn to the program to file for U.S. approval
of a controversial Alzheimer’s disease therapy that
millions of patients could be eligible for. Skeptics
say there is slim evidence that the drug, called adu-
canumab, is effective: Biogen initially declared it a
failure but later returned to the results and found
support that it worked at a higher dose.
Makena can cost around $16,000 per birth, and
cancer drugs can cost more than $10,000 a month.
“We as a society end up paying a lot more for drugs
that don’t end up working,” says Aaron Kesselheim,
a physician and professor of medicine at Harvard
Medical School who proposes price concessions for
drugs that receive speedy approval.
Among expedited approvals that went wrong
were those for the cancer drug Lartruvo, which
was pulled voluntarily by Eli Lilly & Co. last year
after research found it didn’t help patients live lon-
ger, and Roche Holding AG’s Avastin for treatment
of metastatic breast cancer, which the FDA with-
drew in 2011 over concerns about safety and effi-
cacy. For Makena—the first drug approved by the
FDA to reduce preterm birth risk—regulators relied
on just one research trial to make their decision.
Most therapies are studied in at least two trials
before they’re considered.
Amag Pharmaceuticals stands by the efficacy of
Makena and wants to keep it on the market while
more research is done. The recent trial, which took
a decade to complete and release, was complicated
by the drug’s U.S. approval, according to Amag.▼NewFDA-approved
drugsin 2019
Accelerated approval● Per birth, Makena
can cost around$16k
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prognosis