October 12, 2020 BARRON’S 19
says Dr. Brenda Cooperstone, the
company’s rare-disease chief develop-
ment officer. If the prevalence turns
out to be significantly different than
expected, “we would of course revisit
the price,” Varma says.
He’s quick to add, however, that
“patients don’t actually pay the list
price.” What really matters to the
company, he says, “is whether patients
can access the therapy.” And that,
Pfizer says, is why it offers patient-
support programs—and why it’s suing
the federal government to expand
those programs.
Some researchers say there’s a sim-
pler fix: slash the price. But even if the
price is cut in half, “the cost to patients
would still be extraordinarily high”
under Medicare Part D, says Jonathan
Selib, Pfizer senior vice president for
global policy and public affairs.
Pfizer’s solution is to seek a legal
green light for two types of patient-
assistance programs: One would
directly cover tafamidis copays for
many Medicare Part D beneficiaries
prescribed the drug through copay
cards or coupons, and the other
would fund an independent charity
that would give copay assistance to
ATTR-CM patients. These programs
would help middle-income Medicare
patients who don’t qualify for other
types of assistance aimed at lower-in-
come patients, the company says,
such as an existing Pfizer program
that provides free drugs and a Medi-
care subsidy that helps cover pre-
scription costs.
The programs don’t constitute ille-
gal kickbacksand wouldn’t improp-
erly alter clinical decision-making,
the company says, in part because
tafamidis is the only FDA-approved
drug for ATTR-CM.
The defendants—the Department
of Health and Human Services and
its Office of Inspector General—have
not yet responded to the complaint in
court, and the inspector general’s
office declined to comment on the
case. In a late-September advisory
opinion, the inspector general said
that Pfizer’s proposed copay-assis-
tance program is “highly suspect”
under anti-kickback laws, but that it
couldn’t reach a definitive conclusion
on its legality because it hasn’t been
implemented.
The program would cover patients
with income up to 800% of the fed-
eral poverty level, which includes
more than 90% of all Medicare bene-
ficiaries, the inspector general wrote,
and would let “the Medicare program
and taxpayers bear the financial
brunt of an unchecked drug price.”
A central element of Pfizer’s argu-
ment might be easily adopted by other
drugmakers. Restrictions on drug
companies’ interactions with charities,
Pfizer claims, infringe on its First
Amendment rights. Regeneron
Pharmaceuticals (REGN) sounded a
similar note in a late-August effort to
dismiss government allegations that it
paid kickbacksthrough a copay foun-
dation, saying that preventing com-
munications with the charity restricts
its “First Amendment right to engage
in charitable speech.” The company
has said there is no merit to the
government’s complaint.
Pfizer’s Selib says the company isn’t
launching a “frontal assault on the
Anti-Kickback Statute” but asking for
a narrower interpretation of the law.
The U.S. attorney’s office in Boston,
which brought the recent cases involv-
ing drugmakers and copay charities,
declined to comment.
Pfizer and many patients and re-
searchers agree on one thing: Medicare
Part D is broken, exposing patients to
unreasonable drug costs. Pfizer would
like to see a cap on patients’ out-of-
pocket costs in Part D and is willing to
pay more into the program to help that
happen, Selib says.
David Mitchell, founder and presi-
dent of the nonprofit advocacy group
Patients for Affordable Drugs, priori-
tizes a broader solution. “Medicare
should be able to negotiate directly
with drug companies to lower drug
prices, like every other major country
does,” he says. “Those negotiated
prices should extend to all people, and
we should use our collective purchas-
ing power to get a better deal.”B
Medicare Part D
drug spending
grew nearly 10%
annually from
2009 to 2018,
reaching
$168 Bil
Pfizer posted a
billboard outside its
Manhattan head-
quarters last year
promoting aware-
ness of a debilitat-
ing heart condition.
bristle when they weigh the drug’s
cost against its medical benefits. Kazi
and his fellow researchers ran 10,
simulations, varying the assumptions
on rates of hospitalization, the drug’s
effectiveness, and other factors, and
found tafamidis to be cost-effective in
none of them. That’s an “extremely
unusual” result for a drug that has
real medical benefits, Kazi says. To
meet a traditional cost-effectiveness
threshold, the researchers found, the
drug’s price would have to fall 93%, to
about $16,500.
Pfizer says that traditional cost-
effectiveness analysis is flawed for
tafamidis and other rare-disease
drugs, in part because the effort to put
a price on additional years of life is
biased against older people with
shorter life expectancy.
Another factor in the drug’s price,
Varma says, is the rarity of ATTR-CM,
which Pfizer pegs as affecting about
100,000 people in the U.S. The FDA
agreed, giving tafamidis the “orphan
drug” designation, which provides
regulatory and financial incentives—
including seven years of market
exclusivity—to companies developing
rare-disease drugs.
But many doctors and researchers
question whether ATTR-CM is all that
rare. The 100,000 figure may be
a “substantial underestimate,” the
JAMA Cardiology article said. One
study, the researchers noted, sug-
gested that 13% of people hospitalized
with a common type of heart failure
have one form of ATTR-CM, and 1 in
25 Black Americans has a gene muta-
tion that can cause a hereditary form
of the disease.
Tafamidis is an example of broader
problems with the orphan-drug desig-
nation, which has become a “runaway
train” driving drug prices higher, says
Robin Feldman, a professor at the
University of California’s Hastings
College of the Law. The designation
was never intended to apply in cases
where companies can generate a large
profit, she says.
Pfizer highlighted growth in
Vyndaqel/Vyndamax sales at the top
of its second-quarter earnings release,
noting that the drug had global reve-
nue of $277 million. By the end of
2020, the company said in mid-
September, the drug should be a
nearly $1 billion global brand.
The drugmaker is conducting two
large-scale studies to better under-
John Roca Photography/MEGAstand the prevalence of ATTR-CM,