Focus: Novartis wins Medicaid approval for new sickle cell drug in key U.S. states

Emmaus Life Sciences

20/12/2019 11:00

By Carl O'Donnell and Tamara Mathias
    Dec 20 (Reuters) - Novartis AG  NOVN.S  has secured Medicaid
coverage for a pricey new sickle cell disease therapy in two
U.S. states just weeks after winning U.S. approval, following an
early campaign to convince local officials of its value,
according to a company executive and a Reuters review of public
filings. 
    The approvals from the Florida and Alabama Medicaid health
programs for the poor and disabled mark exceptionally fast
acceptance for the treatment, which can cost up to $113,000
annually for an individual patient, excluding discounts, said
Ameet Mallik, who heads the Swiss drugmaker's oncology division.
    In November, the U.S. Food and Drug Administration approved
Adakveo and Global Blood Therapeutic Inc's  GBT.O  Oxbryta,
which carries a list price of $125,000 per year.
    They are among the first therapies that aim to provide 
significant relief from painful symptoms or address the cause of
sickle cell disease, a potentially lethal ailment in which
deformed blood cells limit the oxygen supply to parts of the
body. 
    Strong uptake by Medicaid programs is central to Novartis'
goal of making Adakveo a $1 billion drug, since a high
percentage of sickle cell patients rely on the state-run health
programs. Analysts at Cowen & Co forecast peak annual U.S.
Oxbryta sales reaching $1.7 billion. 
    GBT spokesman Steven Immergut said some health plans,
including private insurers as well as government-run Medicaid
and Medicare, have started to cover Oxbryta on a case-by-case
basis. The company said it has received positive feedback from
payers in early discussions. 
    Depending on the state, Medicaid can take as long as
12-to-18 months to approve new medications, particularly
expensive ones that put a strain on state budgets. GBT has said
it expects most Medicaid programs to take at least three-to-six
months to approve its drugs. 
    The majority of the nearly 100,000 people in the United
States with sickle cell anemia are African American. Around 85
percent of sickle cell patients live in 17 U.S. states,
including Florida and Alabama, and more than half are insured
either by Medicare or Medicaid, according to Novartis and GBT.
    
    'HISTORIC TIME FOR THE SICKLE CELL COMMUNITY'      
    Adakveo is a biologic drug injected into patients several
times each month to prevent the pain crises often associated
with sickle cell disease.
    Oxbryta is a once daily pill that treats the disease's
underlying cause by raising hemoglobin levels and preventing the
breakdown of red blood cells into a sickle - or crescent moon -
shape. 
    Their different impacts on the disease has raised the
prospect that both drugs would be prescribed simultaneously,
creating a greater cost burden to the U.S. healthcare systems.  
    Florida approved the Novartis drug with a requirement that
patients receive prior authorization from their physician,
Mallik said, meaning that the state would review any
prescriptions to make sure that patients meet certain criteria.
Alabama approved it without restrictions, he added. 
    Although state Medicaid programs cannot simply refuse to
cover FDA-approved drugs, they can attempt to save money by
moving slowly to place the drug on reimbursement formularies or
by requiring patients to go through extra steps to obtain
reimbursement. 
    A Novartis cancer therapy, Kymriah, which costs as much as
$475,000, is still not covered by Medicaid in every state
despite having been approved two years ago, Mallik said.        
    Both Novartis and GBT began meeting with state Medicaid
officials ahead of the FDA approvals to discuss the treatment
and have advocated for its inclusion on formularies, or official
reimbursement lists, both companies told Reuters.
    "We have been meeting with both government and commercial
payers for several months – initially educating about sickle
hemoglobin polymerization and, more recently, about Oxbryta,"
Immergut said.     
    Patient advocacy groups, such as the state-level chapters of
the Sickle Cell Disease Association of America (SCDAA), also are
meeting with policymakers to encourage them to make the drug
accessible to patients, said Biree Andemariam, the group's chief
medical officer. 
    "This is a really historic time for the sickle cell
community," she said. "Patients are ready to take advantage of
these new treatments." 

 (Reporting by Carl O'Donnell in New York and Tamara Matthias in
Bengaluru; Editing by Michele Gershberg and Bill Berkrot)
 ((Carl.ODonnell@thomsonreuters.com; 646-223-6629;))