Global Markets for Orphan Drugs - Focus on Biologics

Jun 2013| PHM150A| BCC Publishing

Report Highlights

Report Includes

  • An overview of orphan drug criteria, the history of the Orphan Drug Act and its influence and advantages inherent in orphan drug designation and future growth trends; this report focuses on biologics
  • Analyses of global market trends, with data from 2011 and 2012, and projections of compound annual growth rates (CAGRs) through 2017.
  • Detailed discussion of successful orphan products.
  • Detailed analysis of blockbuster orphan drugs.
  • The rising influence of pharmacogenomics in the orphan drug market.
  • A discussion of specific rare diseases for which orphan drugs exist.
  • Analyses of the most promising therapeutic areas in the industry and leading orphan drugs in these areas.
  • An outline of legislation and regulation, including public and private agencies involved in support and development.
  • Technology trends analysis within the orphan drug market.
  • Comprehensive company profiles of major players in the industry.



The orphan drug market is one of the fastest-growing segments in the pharmaceutical and biotechnology industry. BCC’s goal in conducting this study is to provide an overview of the current and future global market for orphan drugs. The key objective is to present a comprehensive analysis of current orphan drugs and those under development as important therapies and treatments for rare diseases.

This report explores present and future strategies and regulations within the orphan drug market, advances in the market, the setbacks, the current situation and the needs of the market. The market for each drug listed by its generic name/active ingredient and approved indication(s) is also analyzed in this report.

A detailed analysis of the structure of the orphan drug industry has been conducted. Revenues are broken down by region drug type (i.e., biological and nonbiological), active ingredient and area of medical application. Sales figures are estimated for the five-year period from 2012 through 2017.

Applications for orphan drugs are also discussed in the report, with an emphasis on the usage of the drugs in various disease segments. The report also covers significant patents and their allotments in each category.


Extensive research in the field of orphan drugs has resulted in therapeutic treatments for a number of rare diseases. Most of the big pharmaceutical companies have now begun to explore the advantages of orphan drugs as a major source of profit and a steady market.

R&D spending, competition and new technologies are increasing. Advances, new product launches and assistance from regulatory bodies are positively impacting the growth of the market for orphan drugs in the near term. This study examines these factors.

This study focuses on the manufacturers and users of orphan drugs and its market growth. Pharmaceutical biotechnical companies, research institutes and physicians will find this study to be of interest.


The scope of this study includes orphan drugs in pharmaceutical markets worldwide. BCC analyzes each market and its applications, regulatory environment, technology, market projections and market shares. Technological issues include the latest trends and developments. The emerging market for orphan drugs includes countries such as India, China, Japan, Korea, Taiwan, Canada, Africa, Australia and New Zealand.


Both primary and secondary research methodologies were used in preparing this study. BCC conducted a comprehensive literature search, which included technical newsletters and journals and many other sources. Projections were based on estimates such as the current number of end users, potential end users, mergers and acquisitions and market trends.


Many companies were surveyed to obtain data for this study. Included were manufacturers and end users of orphan drugs within the therapeutic categories examined and various disease sector industries. Data were gathered from various industry sources. BCC spoke with officials within the industry, consulted newsletters, company literature, product literature and a host of technical articles, journals, indexes and abstracts. Exhaustive investigations of databases by key terminology were completed. In addition, data were compiled from current financial, trade and government sources.


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The information developed in this report is intended to be as reliable as possible at the time of publication and of a professional nature. This information does not constitute managerial, legal or accounting advice; nor should it serve as a corporate policy guide, laboratory manual or an endorsement of any product, as much of the information is speculative in nature. The author assumes no responsibility for any loss or damage that might result from reliance on the reported information or from its use.

Frequently Asked Questions (FAQs)

Analyst Credentials

Shalini Shahani Dewan focuses on pharmaceuticals and biotechnology and has been a BCC Research contributor since 2002 as both an analyst and project manager. She has explored a wide range of topics and companies, including working for Johnson & Johnson doing market surveillance. She has an undergraduate degree in pharmacy and master's degree in medicinal chemistry. She resides in the Bay Area.

Table of Contents & Pricing

All reports provided in PDF format. For shared licensing options (5+ Users), please call a representative at (+1) 781-489-7301 or contact us at
Title/Chapter NamePagesPrice Member Price
Full Report: Global Markets for Orphan Drugs - Focus on Biologics148 $2,685 Free
Chapter- 1: INTRODUCTION3Free
Chapter- 2: EXECUTIVE SUMMARY3$250Free
Chapter- 3: OVERVIEW23$417Free
Chapter- 7: PATENT ANALYSIS7$127Free
Chapter- 8: CURRENT SITUATION4$73Free
Chapter- 9: COMPANY PROFILES21$381Free
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