A Breakthrough Gene Therapy for Blindness Will Cost $425,000 Per Eye
A Breakthrough Blindness Treatment Will Cost $425,000 Per
Eye, If It Works
Spark Therapeutics proposes installment payments and
rebates
Novel approach will tie cost of medicine to patients’
benefit
By Michelle Cortez January 3, 2018, 5:30 AM PST Updated
on January 3, 2018, 6:34 AM PST
A transformative genetic treatment for a rare, inherited
form of blindness will come with a price tag of $425,000 per eye, or $850,000
for both, said Spark Therapeutics Inc., the tiny biotechnology company that is
bringing the therapy to market.
Since Spark’s Luxturna was approved by the U.S. Food and
Drug Administration last month, speculation over the price has grown as it
became clear the therapy would be one of the first in a wave of medicines that
yield remarkable results after a single treatment -- and would carry a
commensurate cost.
In a novel arrangement, Spark will offer discounts based
on whether or not the drug works initially and remains effective for the
estimated 1,000 to 2,000 patients in the U.S. with a type of inherited retinal
disease caused by a mutant gene.
“We believe that this price reflects not only the
breakthrough, life-altering value of one-time Luxturna, but it will enable us
to continue to invest and build on the revolutionary science that supports not
only Luxturna but the rest of our pipeline,” Chief Executive Officer Jeff
Marrazzo said in a phone interview.
Spark shares rose 1 percent to $53.85 at 9:33 a.m. in New
York.
New Way to Pay
The company’s effectiveness-based discount is sharply
different from how most drugs are currently sold. Health insurers are also used
to paying for medicine over the course of a disease or over a patient’s life,
in the case of some chronic conditions.
A one-time treatment presented a challenge, since the
cost would be paid for by one insurer or government, only to have others reap
the benefits when the patient changes coverage.
To help mitigate that dynamic, Spark is rolling out
several programs to spread out the cost over the years or give rebates to
payers if the benefits wane with time.
For example, the company said it’s discussing a program
with the U.S. Centers for Medicare & Medicaid Services that would spread
payments for Luxturna over several years, even though the therapy would be
given only once. It didn’t say how many installments would be made, or how long
it would take to pay the full cost of the drug.
In an agreement with the Boston-area insurer Harvard
Pilgrim Health Care, Spark will get the full price of treatment up front. If
patients don’t get an immediate benefit -- measured at 30 days, or a long term
one -- measured at 30 months, Spark will have to give some of the money back in
a rebate.
Spark has also proposed selling the gene therapy directly
to insurance companies or specialty pharmacies. That would sidestep the current
process that requires hospitals or health care providers to buy expensive
therapies upfront. Spark is working with Express Scripts Holding Co. on such an
arrangement, and said it’s talking with other drug plans.
‘Revolutionary Product’
Express Scripts has been a frequent critic of costly
drugs, yet said that the Spark treatment is an exception.
"Many people were anticipating this would be more
than a million dollars" said Steve Miller, the St. Louis-based company’s
chief medical officer. “In the end, this is a revolutionary product, and I
think in most plans this will be covered.”
Spark’s biggest challenge may be finding patients to
treat.
Of the few thousand people with the disease, only a few
have actually been tested and confirmed to have it, since there was no cure,
and thus little use in diagnosis. Many with more advanced forms of the disease
won’t qualify for treatment, according to the company.
For more on Spark Therapeutics, check out the Decrypted
podcast:
— With assistance by Robert Langreth
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