Novartis, GBT sickle cell drugs too expensive, draft U.S. report says

Emmaus Life Sciences

24/01/2020 16:32

* U.S. group suggests SCD drugs too costly
    * Drugmakers criticize conclusions as "flawed"
    * Report may change after public comments

    By John Miller
    ZURICH, Jan 24 (Reuters) - Sickle cell disease drugs made by
Novartis  NOVN.S  and Global Blood Therapeutics (GBT)  GBT.O 
may not be cost effective at current prices, a draft report
published on Friday suggested, widening the debate over U.S.
healthcare affordability.
    Novartis and GBT disputed the preliminary conclusions by the
Boston-based Institute for Clinical and Economic Review (ICER).
The institute scrutinised Novartis's Adakveo and GBT's Oxbryta,
both approved last year, and Emmaus Medical's  EMMA.PK  Endari,
to treat the inherited disease that disproportionately affects
African Americans.  urn:newsml:reuters.com:*:nL1N28S14G  urn:newsml:reuters.com:*:nL4N2853ZH
    The finding that the costs of all three medicines would
likely have to be slashed to achieve acceptable
cost-effectiveness thresholds comes as Congress and U.S.
President Donald Trump weigh action over high healthcare costs.
 urn:newsml:reuters.com:*:nL1N28M1GL
    Insurers and governments use privately funded ICER's
conclusions to help set reimbursement rates.
    ICER said it concluded that Adakveo's annual cost should be
$25,410, down from an estimated $88,000 bill, and that Oxbryta
must be trimmed to $9,218 from about $84,000, to meet
cost-effectiveness thresholds. Endari must be cut to $3,859 from
$24,000 to be cost-effective, the group added.
    "Treatment costs were the main driver of the
cost-effectiveness results," said ICER, which is taking public
comment until Feb. 20 before final publication this year.
    ICER seeks to measure how many years of good health - a
quality-adjusted life year, or QALY - may be gained with a new
treatment, and the incremental cost for that health gain.
    At the current estimated costs for Adakveo, Oxbryta and
Endari, all the QALYs exceeded $1 million, ICER said, well above
the $150,000 the group ordinarily considers an appropriate
cost-effectiveness threshold.
    Novartis countered that ICER had not appropriately weighed
Adakveo's benefits, including reducing pain crises suffered by
sickle cell disease (SCD) patients or potentially helping cut
hospital stays. 
    "We look forward to gaining a better understanding of the
approach ICER used to make its assessment and to helping them
better understand the value of Adakveo," Novartis said.
    
    'FLAWED, PREMATURE'
    San Francisco-based GBT contended that ICER's review
conflicts with the U.S. Food and Drug Administration's goal of
speeding medicines to patients.
    "ICER's review is flawed, premature and risks adversely
impacting access to new, potentially transformative therapies,"
GBT said. 
    U.S.-based Emmaus could not immediately be reached for
comment.
    ICER said its draft report may change based on public
comments, including from drugmakers. It also acknowledged
setting a drug's price may go beyond traditional measures of
clinical effectiveness and cost-effectiveness, to include other,
non-financial considerations.
    Recent advances in treating long-neglected sickle cell
disease include the three medicines reviewed by ICER and an
experimental gene therapy from Bluebird Bio Inc.  BLUE.O .
 urn:newsml:reuters.com:*:nL3N21G391
    Still, costs are of particular interest. More than half of
the roughly 100,000 U.S. sickle cell disease patients are
covered by government health insurance.  urn:newsml:reuters.com:*:nL1N28S14G

 (Reporting by John Miller; Editing by Susan Fenton)
 ((J.Miller@thomsonreuters.com; +41 58 306 7734; Reuters
Messaging: j.miller.thomsonreuters.com@reuters.net))