PHCbi-Panasonic eBook - 29

Cell Therapy Today * New Developments in Immunotherapy * Cell Therapy Production Moves to Factory Floor

While the approval and commercial launch of the
first CAR-T therapies, Kymriah™ (Novartis) and
Yescarta™ (Gilead Sciences), marked an important milestone for the cell therapy industry,
the $475,000 price tag for a one-time dose of
Kymriah caused some sticker shock.
"Ensuring that patients have access to these
therapies once they are approved by regulatory
authorities is paramount," said Sanjaya Singh,
Ph.D., global head, Janssen BioTherapeutics,
Janssen Research & Development. "The healthcare community-industry, payers, providers-all
need to work together to figure out how best to
create a sustainable environment that supports
patients and further innovation."
In an effort to improve patient access, Novartis
has contracted an outcomes-based pricing
approach for Kymriah with the Centers of
Medicare and Medicaid. In the unprecedented
arrangement, Novartis will receive payment only

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if the patient responds to treatment within one
month, and since Novartis' price only applies
to its current indication for children and young
adults with r/r ALL, the company could initiate
indication-specific pricing in the future. These
pricing models could set the precedent for future
CAR-T therapies.

Increasing the market size for cell therapies
would allow manufacturers to implement more
efficient and cost-effective strategies. Novartis
estimates that only 600 patients per year will be
eligible to receive treatment for the current indication, but the company has studies underway
to assess Kymriah's effectiveness in diffuse large
B-cell lymphoma and r/r follicular lymphoma.
They also have several CAR-T therapies in development for glioblastoma, ovarian cancer, and
mesothelioma, and demonstrating clinical
success in solid tumors is considered the next big
milestone for the industry.

"I am 5 years cancer free." Emily's chalkboard is
emblematic of the hope inspired by CAR-T therapies. While the clinical success of cell therapies
has taken the world by storm, delivering these
therapies to the world will require a miraculous
story of manufacturing ingenuity. However, as
philosopher Bernard Williams once said, "There
was never a night or a problem that could defeat
sunrise or hope." n
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