WORLDWIDE SALES OF
DRUGS WITH ORPHAN
INDICATIONS TO REACH
EXCEED $43 BILLION BY
2008
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
consequences.
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
$200 Million in 2002 as A Percentage of Overall Global Drug Sales, through 2008
($ Millions)
2002 2003 2008 AAGR %
2003-2008
Sales of drugs with orphan indications 25,181 28,190 43,625
9.1
Global sales of all drugs 365,640 401,610 585,300
7.8
% Sales of drugs with orphan
indications of total global drug sales 6.9 7.0 7.5
1.2
Source: BCC, Inc.
HOME
DRUGS WITH ORPHAN
INDICATIONS TO REACH
EXCEED $43 BILLION BY
2008
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
consequences.
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
$200 Million in 2002 as A Percentage of Overall Global Drug Sales, through 2008
($ Millions)
2002 2003 2008 AAGR %
2003-2008
Sales of drugs with orphan indications 25,181 28,190 43,625
9.1
Global sales of all drugs 365,640 401,610 585,300
7.8
% Sales of drugs with orphan
indications of total global drug sales 6.9 7.0 7.5
1.2
Source: BCC, Inc.
HOME
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MANUFACT.
Nutrition
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NEW DRUGS
Arranon
Actoplus Met
Alfuzosin
Ambien CR
Avastin
Asmanex(R)
Erbitux
(cetuximab)
Gardasil
GRN163
Herceptin
Increlex (rhIGF-1)
Lidoerm
(Lidocaine)
Liraglutide
Lyrica (pregabalin)
Rozerem
Sorafenib
Sutent
Tamsulosin
Torcetrapib
Zometa
OLD DRUGS
Lithium Carbonate
DRUG NOTES
[CONSTRUCT]
Clinical Research
Terminologies
FDA ALERTS
Protein Drug
Market
Niche Market
Orphan Drug
levothyroxine
Protein Drug 1
Protein Drug2
Double-blind
Study
INDUSTRY
Foundamental
Science
Colloidal
Stabilization
Drug Product
Development
Patent Application
Product
Development
Product
Ingredients
Colorant
Scale up a batch 1
Drug Delivery
Industry
Drug Delivery -
Smart Bomb
Lotion, Cream,
Preps
Enteric
Coating-Eudragit
Enteric
Coating-Nutrateric
SR
Coating-Aquacoat
Drug Industry
Drug
Competitions
Drug in
Development
Cholesterol-lowering
Drug-Terminologies
Packaging
Materials
Market
Drug Dev- 2004
Drug Dev -
2005Q1
Drug Dev- 2005Q2
Drug
Dev-2005Summer
Drug Dev-2005Q3
Orphan Drug
Protein Drug
Niche Market
Novo Norkdisk
R&D
Equipment
Blenders
CONTRACT
MANUFACT.
Nutrition
Supplements
NEW DRUGS
Arranon
Actoplus Met
Alfuzosin
Ambien CR
Avastin
Asmanex(R)
Erbitux
(cetuximab)
Gardasil
GRN163
Herceptin
Increlex (rhIGF-1)
Lidoerm
(Lidocaine)
Liraglutide
Lyrica (pregabalin)
Rozerem
Sorafenib
Sutent
Tamsulosin
Torcetrapib
Zometa
OLD DRUGS
Lithium Carbonate
DRUG NOTES
[CONSTRUCT]
Clinical Research
Terminologies
FDA ALERTS
Protein Drug
Market
Niche Market
Orphan Drug
levothyroxine
Protein Drug 1
Protein Drug2
Double-blind
Study