By Angela Maas
This past quarter saw two new gene therapies: Novartis AG
subsidiary AveXis, Inc.'s Zolgensma (onasemnogene abeparvovec-xioi) received
FDA approval May 24 for the treatment of spinal muscular atrophy, and bluebird
bio's Zynteglo (autologous CD34+ cells encoding βA-T87Q- globin
gene) received conditional marketing authorization from the European Commission
for transfusion-dependent beta thalassemia.
While only a handful of therapies in the broader regenerative
medicine/advanced therapy (RM/AT) space are available globally, a new report
shows that is likely to change, as there are more than 1,000 products in the
pipeline.
The Alliance for Regenerative Medicine published the report,
titled Quarterly Global
Regenerative Medicine Sector Report: Q2 2019, on Aug. 1. It shows
there are 1,069 clinical trials using specific RM/AT technologies, which
include gene therapy, gene-modified cell therapy, cell therapy and tissue
engineering. Ninety-four of those products are in Phase III trials.
While Zolgensma's $2.125 million price for a one-time infusion
makes it the costliest drug on the planet, many other newer RM/AT products are
certainly not cheap. Though many manufacturers are offering various
reimbursement schemes, including rebates if a therapy doesn't work, other
outcomes-based deals and multiyear pay-over-time payment options, experts note
that many barriers exist in the execution of these strategies.
"In principle, spreading the cost over a five-year period
and putting the cost installments at risk based upon efficacy is a good
approach," says Winston Wong, Pharm.D., president of W-Squared Group,
about Zolgensma. "However, the devil is in the details. Do we have the
systems in place that have the capability to administer a five-year
contract?"
According to Wong, "From the manufacturer perspective, a
value-based contract implies that no payment would be made if the patient
relapses or passes on. Systems are not in place to have the ability to track
the patient once therapy is administered."
From
RADAR on Specialty Pharmacy
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