12 BARRON’S July 12, 2021
Biogen (ticker: BIIB) and other com-
panies developing Alzheimer’s drugs,
the long-term implications could un-
dermine the pillars of the biopharma
industry, some critics say.
The FDA’s reputation for predict-
able rigor is the very basis of U.S. drug
development. It’s what allows compa-
nies to charge high prices for new
drugs, and to get insurers to pay for
them. In the wake of the Aduhelm
decision, scientists are warning that
the agency may be showing signs of
de-emphasizing that role.
“It’s a Rorschach test,” says Dr. G.
Caleb Alexander, a professor of epide-
miology and medicine at the Johns
Hopkins Bloomberg School of Public
Health, and a member of a panel of
outside experts that advised the FDA
in November that the data supporting
Aduhelm’s efficacy was too weak. “It’s
too early to tell whether this marks
some fundamental inflection point
for the agency, but it certainly under-
scores concerns that have been pres-
ent for many years regarding how the
FDA navigates the perennial tension
between access and ensuring safety
and effectiveness.”
The FDA didn’t respond to ques-
tions submitted by Barron’s , but the
agency’s acting director, Dr. Janet
Woodcock, has elsewhere said that
the FDA didn’t lower its standards in
approving Aduhelm. “It isn’t lower-
ing the standard, but it’s using the
standard we use fairly frequently in
other circumstances in a new circum-
stance,” she told the health news
website STAT.
On Friday, however, Woodcock
acknowledged the concerns around
the approval, issuing a public letter
asking the acting inspector general of
the Department of Health and Hu-
man Services to investigate interac-
tions between Biogen and the FDA
during the process.
By the time the top FDA officials
met in April to discuss the plan for
approving Aduhelm, also known as
aducanumab, the problems with the
drug were well known. The panel on
which Alexander sat had rejected the
evidence that claimed to show that
Aduhelm helped patients, while the
agency’s own statistical reviewer
complained that the efficacy data
was inconsistent.
The officials in charge of the review
of Aduhelm, however, still wanted to
approve the drug, according to an in-
ternal memorandum written by those
officials and released by the FDA.
At the meeting, the officials backed
a strategy that would allow Biogen to
sell the drug without the FDA assert-
ing that it helps patients. The idea was
to give Aduhelm accelerated approval,
a designation usually used for cancer
drugs that have been shown to have
an effect on markers of disease, like
tumor size, but for which clinical ben-
efits like overall survival take too long
to measure.
Aduhelm was an unlikely choice
for the accelerated approval program.
While it has been shown to clear out
the junk brain proteins known as am-
yloid beta plaque, the link between
those proteins and Alzheimer’s dis-
ease is a matter of scientific dispute.
What’s more, it wasn’t as though there
had been no test of the drug’s clinical
benefit; rather, multiple trials had
been unable to definitely prove that
any clinical benefit existed.
Still, at that April meeting, the big-
gest names inside the agency signed
off on the idea. Dr. Peter Marks, the
leader of the division that reviewed
and authorized the Covid-19 vaccines,
supported it, according to the internal
memorandum. So did Dr. Richard
Pazdur, the oncologist responsible for
the agency’s oversight of cancer drugs,
and Dr. Patrizia Cavazzoni, who leads
the division that approves most other
pharmaceuticals.
A few weeks later, Cavazzoni an-
nounced the decision in a statement
that acknowledged that experts were
divided on Aduhelm, but called it
an “important and critical new treat-
ment.” Biogen set a price of $56,
a year, its shares jumped nearly 40%,
and analysts predicted annual sales
FDA Faces Scrutiny
Over Drug Approvals
The green light for Biogen’s drug to treat Alzheimer’s disease has
sparked fierce criticism of the agency. Why it matters to investors
“The FDA
is far less
predictable.”
Christopher
Raymond, an
analyst at Piper
Sandler
I
n April, top leaders at the U.S.
Food and Drug Administration
met to hear a novel plan to get
Biogen’s Alzheimer’s disease
therapy Aduhelm on the market.
The decision the agency
made set off a wave of resigna-
tions, denunciations, and public at-
tacks. So far, that fury is coming
largely from doctors and researchers.
But investors need to pay attention.
While the short-term effect of the ap-
proval has been to boost the shares of
By JOSH NATHAN-KAZIS
Illustration byEVA BEE