WORLDWIDE SALES OF
DRUGS WITH ORPHAN
INDICATIONS TO REACH
EXCEED $43 BILLION BY
The U.S. FDA defines an orphan drug as a product that treats a disease
affecting 200,000 or fewer Americans. A disease that affects fewer than
this number is considered a rare disease and one for which there is not
a great deal of incentive to develop products because of the small
market size. However, there are an estimated 6,000 rare diseases that
collectively affect 25 million Americans. Approximately 70% of these
diseases are caused by genetic defects that have life-threatening
According to a soon-to-be-released report from Business
Communications Company, Inc. (www.bccresearch.com) RB-193
Orphan Drugs: Success Stories and Market Strategies, the total
worldwide market for orphan drugs, including sales in nonorphan
applications, exceeded $28 billion in 2003. This market is expected to
rise at an AAGR (average annual growth rate) of 9.1% to reach $43.6
billion in 2008. As a percentage of overall global drug sales, orphans
are expected to increase their share from 7.0% in 2003 to 7.5% in 2008,
as growth of the orphan market outpaces that of the total market.
The Orphan Drug Act was implemented in 1983 to provide incentives for
companies to develop products to treat diseases that annually affect
from less than a dozen to a few hundred thousand people in the U.S.
Because of the small patient numbers, these products were not
expected to generate sufficient revenues to pay for their development.
However, in 2003 some products that originally were approved as
orphan drugs each had sales in excess of $1 billion. These included
Avonex (interferon beta-1a), indicated for the treatment of multiple
sclerosis; Epogen? (epoetin alfa), indicated originally for the treatment
of anemia associated with human immunodeficiency virus (HIV) infection;
Intron A (interferon alfa-2b, recombinant), indicated originally for the
treatment of Kaposi's sarcoma; Neupogen (filgrastim), indicated
originally for the treatment of neutropenia associated with bone marrow
transplant; Remicade (infliximab), indicated originally for the treatment of
Crohn's disease; and Rituxan (rituximab), indicated originally for the
treatment of nonHodgkin's B-cell lymphoma.
The largest therapeutic markets for orphan drugs are cancer,
cardiovascular diseases and blood disorders (particularly anemia and
neutropenia), genetic disorders, immune disorders and neurological
disorders (primarily multiple sclerosis).
Sales of Drugs Approved for Orphan Diseases with Sales Exceeding
$200 Million in 2002 as A Percentage of Overall Global Drug Sales, through 2008
2002 2003 2008 AAGR %
Sales of drugs with orphan indications 25,181 28,190 43,625
Global sales of all drugs 365,640 401,610 585,300
% Sales of drugs with orphan
indications of total global drug sales 6.9 7.0 7.5
Source: BCC, Inc.