www.biopharminternational.com May 2022 eBook BioPharm International 31
support expiration dates. The manu-
facturing formulation and processes
should be f lexible to allow developers
to respond to any changes in demand
for the product, as well as being glob-
ally acceptable. Oncology orphan drug
products can begin the commercial
lifecycle as low volume products, but
on occasion can experience increased
demand as the treatments are studied
in additional patient populations for
different indications.
THE BENEFITS OF
WORKING WITH A CDMO
BioPharm: How can a CDMO help
a sponsor company develop an orphan
drug? What specif ic services and/or
facilities can a contractor provide?
Jones: Typically, the manufacturing
of an orphan medicine is undertaken
at a considerably smaller scale than
traditional commercial manufacturing,
because of the very limited patient pop-
ulation. When orphan designated drugs
receive approval for expanded indi-
cations, this might affect the scale of
commercial manufacturing. Therefore,
a CDMO w ith the ex perience of
manufacturing at customized scales,
as well as the f lexibility to respond to
changes in the types of products being
sold, the volume of sales, and the facil-
ities and equipment needed for pro-
duction, may be better able to help a
sponsor company overcome barriers in
terms of cost and timeline during an
orphan drug development.
BioPharm: How can a CDMO
help a sponsor obta in reg u lator y
approval for an orphan drug? What
specific services and/or facilities can a
contractor provide?
Jones: When information is needed
on specific technologies, or processes
that require alteration, the CDMO's
regulatory team can respond quickly
with minimal risk of error. A CDMO
is also able to offer sponsors a global
network of regulatory affairs experts
that have local knowledge, to facili-
tate successful agency interactions. In
addition, CMC activities could be eas-
ily and quickly prioritized to ensure
that they do not present as bottlenecks
to the execution of the orphan devel-
opment program and submission of
application to regulatory agencies. A
CDMO should already be intimately
familiar with its dosage form(s), which
ca n be c ustomiz ed to potent ia l ly
address a specif ic orphan medicine's
need. They are then able to provide
complete advice and provide support
on the formulation, development, and
manufacturing intricacies. Leveraging
a CDMO to obtain regulatory approval
for an orphan drug may also result in
overall improvement of eff iciency in
orphan drug product development time
and accelerate product approval.
BioPharm: How can a CDMO
help a sponsor control the costs, spe-
cifically, of the development and man-
ufacture of an orphan drug?
Mollan: A major global CDMO
could provide access to an interna-
tional network of technical expertise
and state-of-the-art facilities. An inte-
grated CDMO can save costs by accel-
erating timelines, reducing program
risks, and ensuring a smooth transition
between early and late-phase manufac-
turing sites. These benefits are achieved
by harmonizing equipment, processes,
pharmaceutical excipient vendors, col-
laboration tools, and project manage-
ment structure between sites.
BioPharm: Can a CDMO help a
sponsor company determine which med-
ical needs might warrant the develop-
ment of an orphan drug?
Mollan: For the most part, CDMOs
do not determine which diseases to tar-
get, but can help refine the target prod-
uct prof ile and ensure that the f inal
product meets the needs of the target
patient population. Developing a for-
mulation and process that can accom-
modate a very wide range of potential
drug loading is a key decision that can
significantly de-risk changes in drug
loading requirements as more infor-
mation becomes known. Many factors
drive the development of the target
product profile, and these include the
physicochemical and pharmacokinetic
properties of the API, patient consid-
erations such as age and co-morbidi-
ties, and manufacturability of the drug
product used in the treatment.
REFERENCES
1. FDA, Developing Products for
Rare Diseases & Conditions, FDA.
gov, accessed Jan. 26, 2022.
2. M.E. Schneider, "Orphan Product
Designation Requests Climb in the
US, Remain Steady in the EU,"
Regulatory Affairs Professionals
Society, March 11, 2021.
3. D. Horgan et al., "Time for
Change? The Why, What and
How of Promoting Innovation
to Tackle Rare Diseases – Is It
Time to Update the EU's Orphan
Regulation? And if so, What
Should be Changed?" Biomed Hub
2020;5:1-11. doi: 10.1159/0005.
BP
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