INTRODUCTION
STUDY GOAL AND OBJECTIVES
This report provides an in-depth look at the life stories of drugs with
orphan disease designation(s) that had sales in excess of $200 million in 2002.
The report examines in detail orphan drugs that have become multimillion-dollar
products, provides revenues for leading orphan products, presents current and
future strategies of companies developing orphan products as well as diseases
addressed by these products, describes current and possible future legislation
affecting the development and marketing of orphan drugs in the U.S. and
internationally, and examines the historical and current relationship between
the biotech industry and the Orphan Drug Act as well as the future relationship
of pharmacogenomics to the Act. Also included are profiles of companies with
blockbuster orphan products and companies specializing in orphan products, with
particular focus on their development and marketing strategies. Forecasts and
trends are gleaned from industry sources as well as from considered assessment
of emerging products and technologies.
REASONS FOR DOING THE STUDY
As of mid-2003, 221 orphan drugs had been developed that are indicated for
the treatment of rare diseases affecting approximately 11 million patients.
Between 1992 and 2002, orphan drugs accounted for 17% of all drugs and biologics
approved for sale in the U.S. Some orphan products have sales in excess of $1
billion annually.
A drug may receive orphan designation as its first
indication or it may be approved for an orphan disease after it has been
marketed for a nonorphan indication. This report describes in detail the special
advantages of orphan product designation, which include the following:
- assistance from the FDA in various stages of product development
- tax incentives for sponsors in the form of tax credits for clinical
research; the tax credit may be claimed whether or not the product is
approved by the FDA
- grant funding to defray costs of qualified clinical testing expenses
incurred in connection with the development of orphan products
- seven years of marketing exclusivity after approval of an orphan drug
product.
This report provides histories of drugs initially designated as orphan
products that became blockbusters by treating only orphan diseases as well as
those that subsequently received other approvals that turned them into
blockbusters. The overall purpose of the report is to provide a better
understanding of the characteristics of orphan drug designation and the
commercial advantages associated with the development of orphan drugs. To this
end, strategies of companies focusing on the development of orphan drugs as well
as those that incorporate orphan products into a broader development pipeline
are discussed.
CONTRIBUTIONS OF THE STUDY AND FOR WHOM
This report analyzes the advantages of orphan product development and traces
the development, commercialization and strategy for receiving initial and
subsequent orphan and nonorphan disease designations for products. The report
provides information needed to understand the impact of orphan product
development on the marketplace, both historically and in the future. This report
is intended to be a tool for business planners, acquisitions specialists,
licensing strategists, product managers, market research analysts, investor
consultants, and anyone interested in the pharmaceuticals and biopharmaceuticals
markets and the role of orphan drugs in these markets.
SCOPE AND FORMAT
This report focuses on marketed and in-development products with orphan
disease indications. The Overview chapter defines the criteria for orphan drug
designation, the history of the Orphan Drug Act and its influence on product
development, and the advantages inherent in orphan drug designation.
The
Products chapter provides detailed discussion of products with orphan drug
designation that had sales in excess of $200 million in 2002, recently
introduced orphan products with a sales potential of $200 million annually or
more, and selected orphan products in development. Sales projections are
provided for many of the 26 products analyzed in this section.
The
Applications chapter provides an overview of specific orphan diseases for which
products exist and the sales of leading orphan products for these diseases.
The
chapter on Legislation and the Regulatory Environment outlines public and
private agencies involved in the support and development of products for orphan
diseases, outlines orphan drug legislation in other countries and how this
legislation differs from the American Orphan Drug Act and discusses the
controversial topics of orphan drugs that have annual treatment costs of
hundreds of thousands of dollars a year and those orphan products that have
multibillion dollar annual sales.
The Industry chapter provides an analysis of
the relationship between the Orphan Drug Act and the biotechnology boom in the
1980s and the effect of pharmacogenomics on the development of orphan products
in the future, outlines leading companies in terms of number and sales of
marketed orphan drug products, provides strategies for success utilized by five
companies focusing on the development and marketing of orphan products and
assesses the impact of the Orphan Drug Act on public and private investment in
drugs for rare diseases.
The Company Profiles chapter provides a close look at
companies currently participating in the development and/or marketing of orphan
drugs, including companies that specialize in the development of products for
orphan diseases only.
METHODOLOGY
The base year for this study is 2003. Market figures are based on revenues at
the manufacturers' level and are projected at 2003 dollar value (i.e., inflation
is not computed into the projection figures).
Data to prepare this study was
derived from information obtained from product managers, marketing strategists,
research executives, government agency spokespersons and directors of
organizations focused on specific orphan diseases. In addition, information also
was obtained from others at pharmaceutical and biotechnology companies that have
products for orphan diseases on the market or in development.
INFORMATION SOURCES
The information and analysis presented in this report are based on an
extensive survey of the pharmaceutical industry as well as a detailed
examination of published literature and reports obtained from regulatory
authorities, medical research institutions, pharmaceutical trade associations,
and national and world health organizations. Key information from the business
literature was used as to obtain expert opinion on commercial potential and
market sizes from industry professionals.
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