The Global Market for Orphan Drugs is Forecast to Reach US$148.9 Billion by 2020

Increasing Incidence of Rare Diseases & Supportive Legislations Drive the Orphan Drugs Market, According to a New Report by Global Industry Analysts, Inc.

GIA announces the release of a comprehensive global report on Orphan Drugs. The global market for Orphan Drugs is forecast to reach US$148.9 billion by 2020, driven by the rising incidence of rare medical conditions, growing global population, and increasing focus of big pharmaceutical companies on orphan drug development.

The global market for orphan drugs is driven by the ever-increasing discovery of orphan or rare diseases, predominantly rare cancers, such as renal cell carcinoma, non-small cell lung cancer, acute myeloid leukemia, and thyroid cancer, among others. The lack of appropriate therapies for orphan diseases and the legislative benefits offered by the government for orphan drug development (tax credits, research assistance, partial funding of clinical trials, fast-track drug review and expedited approval, and extended market exclusivity period) are attracting major pharmaceutical companies to venture into this arena. Orphan drug development, previously the realm of smaller biotechnology enterprises, has now become one of the main focus areas of big players in the industry, a fact validated by the blockbuster sales of premium orphan drugs.  

Despite high market potential, orphan drug development is challenged by factors such as, reimbursement uncertainties, and high cost of drug development. The move to reduce healthcare costs and the trend towards value based reimbursement will bring under scrutiny orphan drugs which command high prices and full reimbursement. Payers may limit reimbursements for drugs approved for newer indications, or those without much difference in treatment efficiency from existing drugs. Despite their longer market exclusivity period than conventional drugs, the orphan drug market will continue to feel the competitive heat posed by the development of lower priced biosimilars and generics. In addition, clinical trials for investigational orphan drugs are challenged by the absence of adequate number of patients for enrollment. Orphan drug development also suffers from lack of validated endpoints for a study and the unavailability of considerable clinical data for designing a study.

Biotechnological and medical breakthroughs are expected to improve understanding of rare diseases and enable the development of novel biologic therapies. Further, numerous collaborative research programs across the United States and Europe, provide manufacturers, healthcare experts, and patients with epidemiological research data on the disease; and also enable early patient access to appropriate and timely medical care. Patient advocacy groups also result in greater awareness among patients about the efficacy of orphan drugs over available treatment options. Improved awareness of rare diseases and available therapies is expected to revolutionize the market for orphan drugs.

As stated by the new market research report on Orphan Drugs, the United States and Europe, which have well established regulatory as well as reimbursement frameworks, represent major markets worldwide. The United States is the largest as well as the fastest growing market, driven by incentives offered by the US Food and Drug Administration (FDA) such as extended market exclusivity for orphan drugs and research grants for drug development. The market for Biologics ranks as the fastest growing product segment supported their greater efficacy over Non-Biologics. With about 80% orphan diseases being genetic in nature, biologic orphan drugs are better suited for targeted therapies.

Key players in the market include AbbVie Inc., Actelion Pharmaceuticals Ltd., Amgen Inc., AstraZeneca Plc., Bayer HealthCare AG, Biogen Idec Inc., Bristol-Myers Squibb Company, Boehringer Ingelheim GmbH, Celgene Corporation, CEL-SCI Corporation, Concordia Healthcare Corporation, Eli Lilly and Company, F. Hoffmann-La Roche Ltd., GlaxoSmithKline Plc, Johnson & Johnson, Merck & Co. Inc., Merck Serono International S.A., Novartis AG, Pfizer Inc., Recordati S.p.A., and Sanofi SA (Genzyme), among others.

The research report titled "Orphan Drugs: A Global Strategic Business Report" announced by Global Industry Analysts Inc., provides a comprehensive review of market trends, growth drivers, product segments, company profiles, orphan drug designations and approvals, mergers and acquisitions and other strategic industry activity. The report provides market estimates and projections for major geographic markets, including the United States, Japan, Europe, and Rest of World.