sector a vibrant and interesting
one, it also invites more attention
from regulators and payers, increasing the pressure to produce
truly innovative therapies that
can help reduce overall healthcare
It’s important to understand what
defines an orphan drug, a pharmaceutical product aimed at rare dis-eases;or;disorders.;In;the;U.S.,;rare
disease populations are defined
by the FDA as having less than
200,000;patients;(< 6. 37;in;10,000).
Evaluate defines a clean “orphan”
subset of products within their
data as following certain criteria.
For instance, the first indication
approved has to be for an orphan
condition. Also, the product has
sales from orphan indications—
which excludes drugs like Avastin,
Enbrel, Humira and Remicade,
because the orphan designations
for those products don’t reach the
[See Orphan Drugs Report, pg. 42]
Orphan Market Drivers
Why is this upsurge occurring?
Mainly because “orphan” is not
such a diminutive term anymore.
The National Organization for
This enormous population of
potential patients presents great
opportunity for the pharmaceutical industry to develop appropriate treatments for unmet medical
needs. Treatments that generate
high levels of reimbursement.
Also, the Orphan Drug Act of
development of orphan drugs.
Companies developing these
indication designations covering
And there are significant financial
receives seven years of marketing exclusivity from approval. The
market exclusivity blocks “same
drug” recombinant products.
Indeed, large pharma groups finding orphan indications for some
of their biggest sellers mean that
by orphan indications are global
majors, and the likes of AbbVie are
trying to ensure their seat at the
big table through acquisition. The
Chicago-based group is now in the
thanks to its acquisition of Phar-macyclics.
The enduring appeal of orphan
drugs remains grounded in a variety of factors, including the lack
of alternatives for patients, lower
R&D costs, easily defined patient
populations, and the prices that
the drugs are able to command. So
far orphan drug developers have
managed to defend the cost of
these life-changing drugs, due to
the relatively small patient populations they serve and the continued
paucity of options for sufferers.
The one big change this year is
that Celgene replaces Novartis as
the world’s biggest orphan drug
is set to achieve this solely on the
back of the performance of its biggest drug Revlimid, which is used
The Orphan Drug Report also re-
veals that worldwide orphan drug
The proportion of orphan drugs
in relation to the rest of the indus-
try is also growing, with orphans
reported last year.
Other highlights from the report
drugs compared to non-orphan
march up the orphan drug sales
•;Phase;III;orphan;drug;develop-ment costs are half that of non-orphan, and potentially a quarter of
for orphan drugs is no faster than
of Phase III/ Filed orphan drugs
R&D orphan drug
European designations too with
new orphan drug approved by
[See more figures, next pages]