Jan 21 2010
The number of orphan product designations in the
U.S. more than doubled during the last decade, reflecting growing interest
by pharmaceutical and biotech companies in developing products to treat
orphan diseases, according to a study recently completed by the Tufts
Center for the Study of Drug Development.
According to Tufts CSDD, orphan product designations increased from 208 in
the 2000-02 period to 425 in 2006-08.
Orphan drugs are products developed for a rare disease or condition, which
affects fewer than 200,000 people in the U.S. In the European Union, orphan
products are defined as those for diseases or conditions affecting five
people out of every 10,000, or fewer.
"The Orphan Drug Act, without question, can be considered a success. It has
been emulated throughout the world, and today Japan and Europe have similar
programs, while Singapore and Australia give special allowance for the
importation and marketing approval of orphan products approved in the
U.S.," said Tufts CSDD Associate Director Christopher-Paul Milne, who
conducted the study.
Since the Orphan Drug Act of 1983 was signed into law in the U.S. more than
2,000 products in development have been designated as orphan drugs, while
the Food and Drug Administration has granted market approval to 350 drugs
and biologicals.
The new analysis, reported in the January/February Tufts CSDD Impact
Report, released today, also found that:
-- During the 2000s, orphan products comprised 22% of all new molecular
entities (NMEs) and 31% of all significant biologics (SBs) receiving
U.S. marketing approval.
-- Orphan products receiving priority review status rose from 35% of
all orphan NMEs in 2000-02 to 50% in 2006-08; during the same time
the share of orphan SBs receiving priority review status rose from
17% to 67%.
-- While biotech firms during the 2000s garnered, on average, about
one-third of all orphan drug approvals, they received just over 50%
of orphan drug designations.
-- Sponsors engaged in clinical development funded through orphan
grants reported that 22% of their programs led to approvals, which
compares with a clinical approval success rate of 16% among
mainstream drug developers.
SOURCE: Tufts Center for the Study of Drug Development