Pharmaceutical Technology BIOLOGICS AND STERILE DRUG MANUFACTURING 2019 35
care. GlaxoSmithKline (GSK) made the decision to
offer Strimvelis, a treatment for severe combined
immunodeficiency due to adenosine deaminase
deficiency (ADA-SCID), at only a single treatment
center in Milan, assuming the need for a "special-
ized [treatment] environment."
To support commercialization
goals, manufacturers might
consider using predictive
analytics to inform strategic
decisions on [number and
location of] treatment sites.
The limited options for treatment meant higher
costs and challenges related to travel and cross-
border European reimbursement for many patients.
As a result, only four patients have been treated
with Strimvelis at the site since approval in 2016.
GSK has since announced its interest in divesting
its rare disease division, including Strimvelis (7,8).
To support commercialization goals, manufactur-
ers might consider using predictive analytics to in-
form strategic decisions on the appropriate number
of treatment sites, where they should be located, or
whether and how they might bring gene and cell
therapies directly to patients.
Conclusion
While factors including patient population, prod-
uct value and efficacy benefit, and pricing play
the major roles in successful commercialization
of gene and cell therapies, it is essential for drug
developers to recognize when and where decisions
related to production can also have an impact. The
application of technology is a critical consideration
in planning related to production time, scalability,
and product purity and safety, as well as in expan-
sion of target indications.
Without access to skilled expertise, many drug
developers risk making decisions related to pro-
duction that can limit or even jeopardize com-
mercial potential. Conversely, companies that can
access the talent and insight necessary to make
the right technology decisions at the right time at
every stage in a development program can build a
considerable competitive advantage.
References
1. G. Kolata, "New Gene-Therapy Treatments Will Carry Whop-
ping Price Tags," New York Times, September 11, 2017, www.
nytimes.com/2017/09/11/health/cost-gene-therapy-drugs.html
2. Gilead, "Kite's Yescarta (Axicabtagene Ciloleucel) Becomes
First CAR T Therapy Approved by the FDA for the Treatment
of Adult Patients With Relapsed or Refractory Large B-Cell
Lymphoma After Two or More Lines of Systemic Therapy,"
Press Release, October 18, 2017.
3. C. Challener, "Cell and Gene Therapies Face Manufacturing
Challenges," BioPharm International, January 1, 2017, www.bi-
opharminternational.com/cell-and-gene-therapies-face-manu-
facturing-challenges
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$40M for New CCRM-Led Centre to Solve Cell Therapy Manu-
facturing Challenges," Press Release, January 13, 2016.
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future/?set=609105.
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