MAY 21, 2018
For the past two years, Sarah Benoit has been
getting around with the help of a walker, waiting for a medicine that’s out of
reach.
Benoit, a former congressional aide, has ALS, a
fatal neurological disease that gradually causes muscle weakness and paralysis.
As it progresses, she struggles to maintain a normal life, but holds out hope
that a drug called Radicava — the first new treatment in 22 years — will slow
her deterioration.
Unfortunately, she can’t get the medicine, even
though her doctor prescribed it last fall.
Like an untold number of ALS patients, Benoit
faces a conundrum. She can’t afford the $145,000 price of the drug
without insurance, but her insurance
provider has denied access to the drug, even though it was approved by the Food
and Drug Administration last fall for all ALS patients. And as time goes
by, Benoit knows that she is losing a chance to delay the inevitable.
“I’m
getting slower and weaker. It’s becoming harder to shampoo my hair or use a
pencil. My disease is progressing slowly, but it is progressing,” said Benoit,
36, who lives in San Bernardino, Calif., and was diagnosed with ALS nearly six
years ago. “I think the drug would make it possible to be with my family
longer. So, it’s frustrating.
“I do understand that it’s business. I’m realistic.
The drug company wants money to cover research, which we need and I support,”
she said. “And the insurance company wants to keep costs down. I get all that.
But I am shocked at the cost.
There’s no two ways around it. And the last
thing an ALS patient needs is hoops to jump through. Everyone is wasting
valuable time talking to insurance companies.”
Certainly, there is nothing new about a
high-priced medicine or insurers acting as impenetrable gatekeepers. In this
case, the manufacturer of Radicava, MT Pharma, maintains that
years of research expense must be recovered. And for their part, various
insurers argue that coverage decisions, while nuanced, reflect legitimate
criteria.
But the sort of battle facing patients like
Benoit serves as a cautionary tale. Costly medicines can give rise to misplaced
expectations for patients and doctors navigating America’s Byzantine
health care system. And the problem is compounded by well-intentioned
regulators trying to help desperate patients whose medical needs are unmet.
“The problem is the way the system creates
incentives for both drug makers and insurers to act as they do,” said I. Glenn
Cohen, a Harvard Law School professor who is an expert in bioethics and health
law policy. “When you allow private insurers to make decisions with only a
patchwork of state regulations, they will choose not to cover every drug
approved by the FDA. And drug companies are for-profit entities that set prices
based on market conditions. This is why you have debates over coverage.”
There is little debate, though, that Radicava
marks a turning point in treatment.
ALS, or amyotrophic lateral sclerosis, attacks
brain cells controlling muscle movement. Initial symptoms can vary. Some people
experience weakness in their legs, or have difficulty grasping things or
swallowing. There is no cure and the mean survival time is three to five years,
although some patients live 10 years or more. About 20,000 people in the U.S.
have the disease at any given time.
Radicava is far from a cure, though. The drug
was originally approved as a stroke treatment more than a decade ago in Japan,
where Mitsubishi Tanabe Pharma later tested it for ALS. The trial indicated the
medicine could slow the decline in function — by about 33 percent — although as
with many medications, the results can vary among patients.
The FDA, which has been under increasing
pressure to approve drugs for unmet medical needs, was convinced to greenlight
the intravenous medication in May 2017 as an effective treatment. Given the
dearth of new ALS medicines for nearly a quarter-century, the approval was a
big deal for patients and their families, who have been yearning for a salve.
“Understandably, this created a lot of
excitement,” said Calaneet Balas, who heads the ALS Association, a national
advocacy group, which receives funding from the
manufacturer and launched the celebrated Ice Bucket Challenge four years ago to
raise research money and awareness about the disease.
But for some ALS patients, the excitement has
given way to frustration and bitterness as they encounter roadblocks to
obtaining the drug. Over the past few months, a few patients have responded by
creating online petitions to
shame insurers. Others visit a Facebook page devoted to Radicava, where
questions about cost and insurance coverage are interspersed with advice about
usage and side effects.
Why is this happening? Let’s start with the
price.
In Japan, the drug costs $38,000. The drug maker
argued the $145,000 price tag in the U.S. reflects years of research and
development investment, including clinical work used to win FDA approval for a
small patient population, when compared with the number of stroke victims in
Japan. And a follow-up study required by the FDA as a condition of approval may
cost up to $125 million, which has to be recouped.
“We feel the price we came to was done very
carefully, very thoughtfully, and fairly reflects out-of-pockets costs spent on
this clinical journey, not opportunity costs,” explained Kevin O’Brien, vice
president of market access at MT Pharma, the U.S. unit.
He maintained internal estimates indicate about
90 percent of patients who are seeking Radicava have, so far, been able to
obtain the drug, although this could not be independently verified. O’Brien
also noted the drug maker offers a patient assistance program that provides up
to $20,000 to patients who cannot obtain the drug through private insurance.
The price of the drug, however, does not tell the
whole story.
Radicava must be infused 20 times a month
indefinitely, which adds to the cost for insurance coverage, especially if
infusion takes place at a clinic or infusion center. But one insurer maintained
that the price is not the deciding factor in whether to cover the drug or how
much coverage to provide in such circumstances.
“Are there drugs far more expensive than we
think they should be? Yes,” said Dr. Michael Sherman, chief medical officer at
Harvard Pilgrim Health Care, the second-largest health plan in New England.
“But where there is an unmet need and clinical efficacy, we won’t deny drugs
based on cost alone. Where you do see perceived inconsistent responses or
flat-out denials are in cases where evidence is weak.”
This brings us to the FDA approval.
The agency relied on that one small clinical trial involving
patients in Japan — not the U.S. — and then issued a blanket endorsement for
the drug. Whether a patient is in the earliest or latest stages of the disease,
the product labeling simply
indicates the medicine is for ALS patients — period. In other words, all comers
are welcome, or so it would seem.
But there are differing opinions about the value
of the drug because the clinical trial had only 137 patients and it took place
over just six months. Although the drug generated statistically significant
results when compared with a placebo, the findings relied on a functional
rating scale, which some health care providers argue is a largely subjective
metric that is too imprecise to be wholly reliable.
“Can it slow down the disease? For some people,
possibly,” said Dr. Jonathan Glass, an Emory University neurology professor.
“But initially, it didn’t work so [the company] had to do a post-hoc analysis
[of a smaller, secondary trial using a subset of patients]. I’m not arguing
that it’s bad data. It’s just minimal data. And the key question is whether the
data is good enough to approve the drug for everybody.”
Private insurers, however, are not always
obligated to cover a drug or provide a set level of coverage following FDA
approval. And in an age in which drug prices keep increasing — and price tags
for the newest medicines seem to reach ever-higher levels — some say insurers
are simply using well-honed gatekeeping skills in response to the cost for
Radicava.
“One
can’t assume it’s not part of their calculus,” argued Balas of the ALS
Association. “There are private payers that have not been following the label
[in determining coverage]. Payers have not said to us informally or formally
that cost is the issue. They point to the trial design. But if it were a third
of the price, might they cover it? I don’t know.”
Right now, she said, the clinical trial “does
give them cover.”
Nonetheless, one benefits expert suggested the
barriers to Radicava coverage are out of the ordinary.
“Typically, what’s going to happen is carriers
will follow guidelines associated with FDA approval, but they have the ability
— if patients don’t meet criteria — to deny coverage,” said David Dross, the
managed pharmacy practice leader at the Mercer benefits consulting firm. “But I
haven’t seen that to a great extent. It does seem unusual if FDA approval was
that broad.”
Many insurers are, in fact, approving Radicava — but only
for ALS patients whose functioning matches some or all of the characteristics
of the trial participants, whose disease is not as advanced. Their functioning
is measured using a scale that rates the ability to conduct 12 different activities,
including dressing, speech, swallowing, handwriting, walking, climbing stairs,
and turning over in bed.
There is another hurdle. One criteria cited by insurers
for determining coverage is when a diagnosis was made. Some insurers will pay
for Radicava only if a patient was diagnosed within two years of seeking the
medication. This automatically excludes patients, however slow or fast their
disease may be progressing.
Sarah Benoit, for instance, was twice denied
Radicava by Blue Cross California, which administers the Federal Employee
Program through which she receives coverage. The insurer, which requires prior approval before agreeing to cover
the drug, did not respond to requests for comment. The reason was that she did
not pass yet another functional rating scale that is used in Japan, which her
physician finds frustrating.
“It’s a piece of information that we can’t give
them, because we don’t use that scale in this country,” explained Dr. Jeffrey
Rosenfeld, a neurologist at Loma Linda University. “It was tested in another
country so we have no experience at all with this drug. So when I get a denial,
I will point out the FDA didn’t approve the drug with stipulations.
“I think they’re denying it because it’s costly
— they’re trying to be prudent and provide the drug to patients who might
benefit most. But we don’t have the experience to say it’s not worth the cost.
Unfortunately, this fits into a pattern, perhaps, of a non-sustainable expense
to the health care community.”
Harvard Pilgrim, meanwhile, denied Radicava to
John Welch, a former elementary school teacher from Medford, Mass., three times
between September and March. The insurer said it initially did not have a
coverage policy in place when it issued the first two denials. But the reason
appeared to be his vital lung capacity, according to staff at
Massachusetts General Hospital, where he is treated. At the time his physician
first prescribed the drug, his reading was 78 percent, just a smidgen below the
80 percent cutoff.
Numerous appeals were filed. Ironically, by the
time he was approved in March, his lung capacity had fallen to 63 percent.
This perplexing approach to coverage infuriates
not only patients, but also health care providers.
In a letter to Anthem Blue Cross of California
concerning one of her patients who was denied Radicava, Dr. Abirami
Muthukumaran of the Cedars-Sinai Medical Center in Los Angeles wrote in vain
that “there is no evidence that Radicava will not slow the progression of her
disease in people who would not meet the inclusion criteria of the clinical
trial.
“The
reason the trial was only able to see an effect in the rapidly progressing,
recently diagnosed patients is because [the drug maker] only used a six-month
follow up time, which would not be long enough to detect slowing of disease
progression in patients who were declining more slowly either because that is
the nature of the disease, or they were more advanced,” she wrote.
“The criteria cited by insurers, in some ways,
do make sense — but for research reasons,” said Jennifer Lynne Scalia,
associate site director at the Neurological Clinical Research Institute at
Massachusetts General Hospital. “The company needed to study people who could
live longer, breathe better, and function better to see how the drug performed.
“But for clinical practice, it does not make
sense. John Welch is an example. Yes, there are some changes for the worse in
his [lung] capacity, but he remains high functioning. Clinically, he might have
benefited sooner if Radicava does slow the progression and his loss of
breathing function. Theoretically, he could have remained high-functioning for
a longer period of time. That’s why the FDA approved it.”
For Welch, the approval was bittersweet.
“I’m excited to finally get the drug, but my
body is getting weaker and it won’t make a comeback,” he told us.
“Hopefully, it will stabilize. But I felt much
better last September. I had strength in my legs. I think I would have had a
better quality of life if I’d gotten it then. And I blame the companies. The
drug company is going in for the kill. And the insurer looks at this like
actuaries.”
His physician, Dr. Haatem Reda, a Mass. General
neurologist, explains the frustration this way: “I don’t know that the drug has
made a big impact in patient lives, but it’s a bit of hope. Unfortunately, the
[insurance] process is difficult. He’s not unique. It’s been a struggle for a
lot of my patients to get Radicava. Even for those who are eventually approved,
they go through a grueling process.”
Others simply give up.
Jan Daily, 66, a former nursing professor at
Capital University in Columbus, Ohio, was approved for treatment last fall, but
could not find an infusion center to treat her for very long. Why? Some centers
are unwilling to accept reimbursement levels from Medicare Part B, which covers
specialized injectable and infused medicines, according to Peggy Clary, who
heads patient care services at the ALS Association chapter in central and
southern Ohio.
So Daily, who was already experiencing paralysis
in her legs, considered home infusion and turned to Medicare Part D, but this
meant she would be responsible for more of the expense — both the nursing
services and the drug. And in this instance, Radicava is at a higher tier on
the plan formulary administered by SilverScript, a unit of CVS Health. As a
result, she despaired at the out-of-pocket costs.
“I thought, what did I have to lose? I was
approved and I hoped the drug would slow the progress” of the disease, Daily
explained. “But the copay would have been $7,000 per month. It was too much. I
can’t afford that. It was a huge disappointment. Now, I’m kind of worn down. I
worked hours and hours on this, and finally had to accept it wasn’t going to
happen. Meanwhile, I’m progressing so quickly.
“The drug company drew a line in the sand, which
then means my insurance company draws their line.”
For its part, MT Pharma maintains things are
changing. As of this month, more than 2,500 patients have been treated with
Radicava, according to a company spokeswoman. “We are making progress with
these insurers,” said O’Brien.
One physician, meanwhile, describes the
situation this way.
“In our system, I would contend that it’s
justifiable to deny funding the drug for people who don’t fit into the
categories that were studied by the manufacturer,” said Dr. Kenneth Patric, who
sits on the medical policy committee at Blue Cross Blue Shield Tennessee, which
covers Radicava but if only certain criteria are met.
“Or if the FDA uses a broader scope for
approval, we may still disagree and say we’re not going to pay for it that way.
FDA approval is a first step, but only one step.
“That said, I think our approach to the whole
health care system in the U.S. sucks. In a different setting that’s not so
profit driven, the rationale would be different and you’d have a different
answer” for Radicava, he continued. “But it’s a money-driven system.”
This
story was updated to note that MT Pharma provides funding to the ALS
Association.
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