U.S. watchdog lifts price estimate for Bluebird, Vertex/CRISPR gene therapies to $2.1 mln

Crispr Therapeutics AG

13/07/2023 13:00

By Bhanvi Satija
       July 13 (Reuters) - An influential U.S. drug pricing
watchdog raised the price estimate of two experimental gene
therapies from Vertex Pharmaceuticals  VRTX.O /CRISPR
Therapeutics  CRSPR.BN  and bluebird bio  BLUE.O  to as much as
$2.05 mln, saying the new price can be cost effective to treat
sickle cell disease.
    A price of up to $2.05 million a year, higher than the prior
estimate in April of up to $1.9 million would meet some commonly
used thresholds for cost effectiveness, the Institute for
Clinical and Economic Review (ICER) said.
    Sickle cell disease leads to a shortage of healthy blood
cells and restricts blood circulation, causing periodic episodes
of pain in patients.
    The higher estimated pricing reflects the ICER's assumption
of more pain attacks per patient, said David Rind, chief medical
officer of ICER. 
    "The treatment is preventing more attacks and it's worth
more," Rind said. 
    The ICER's draft report in April had assumed four attacks
per patient in a year, compared with 5.1 attacks in its final
report.
    It estimated a price range between $1.35 million and $2.05
million a year for the gene therapies, which suggests the
highest price a manufacturer in the United States should charge
for a treatment.
    Both the bluebird and Vertex/CRISPR gene therapies are yet
to be approved in the United States.
    Recently approved gene therapies in the U.S. carry a high
list price due to their potential to be one-time cures for rare
conditions. 
    CSL's  CSL.AX  Hemgenix for hemophilia B has a list price of
$3.5 million in the U.S., while BioMarin Pharmaceutical's
 BMRN.O  therapy, Roctavian, for severe hemophilia A is priced
at $2.9 million.
    Rind said the impact of high prices on healthcare budgets
could potentially be an issue, but it was too early to assess.
    "There's a fair amount of nervousness about the new gene
therapies," he said. 

 (Reporting by Bhanvi Satija in Bengaluru)
 ((Bhanvi.Satija@thomsonreuters.com; Outside U.S. +91
9873062788;))