(Adds comment in paragraphs 6, background throughout)
Dec 4 (Reuters) - The Centers for Medicare & Medicaid
Services on Wednesday said it had entered into agreements with
Vertex Pharmaceuticals VRTX.O and bluebird bio BLUE.O to
help increase patient access to their gene therapies.
The so-called "outcomes-based agreements" will tie payments
to whether the therapy improves health outcomes for patients who
receive these drugs and are enrolled in government-backed
Medicaid insurance plans.
WHY THIS MATTERS
The two one-time treatments called Lyfgenia and Casgevy
were approved in the U.S. last year for treatment of sickle cell
disease, an inherited blood disorder in which the body makes
flawed, sickle-shaped hemoglobin.
The Vertex-CRISPR therapy has a U.S. list price of $2.2
million, while bluebird's is $3.1 million.
The treatments' high prices have contributed to
restricted access and resistance from insurers to cover the
therapies.
KEY QUOTES
"Due to the high costs, these therapies can pose
challenges to state budgets. This (CMS) model will afford state
Medicaid agencies more budget predictability while helping
improve access to these innovative therapies," said Liz Fowler,
CMS deputy administrator.
BY THE NUMBERS
Reuters
reported in September
that the treatments had been used on around 100 people
globally at the time, including in clinical trials.
In the U.S., over 100,000 people live with sickle cell
disease, with about 50% to 60% enrolled in Medicaid, a state and
federally funded insurance program for low-income people.
Sickle cell disease disproportionately impacts Black and
Hispanic Americans.
Last month, bluebird said that 17 patients had started
treatment on Lyfgenia this year.
WHAT'S NEXT
CMS, the federal agency that looks over Medicare and
Medicaid insurance plans, said it will now move forward with
engaging states and U.S. territories that are part of the a drug
rebate program to help them decide whether to participate in the
model.
(Reporting by Bhanvi Satija in Bengaluru; Editing by Maju
Samuel)
((Bhanvi.Satija@thomsonreuters.com; Outside U.S. +91
9873062788;))