Orphan Drugs: A Global Strategic Business Report
San Jose, California (PRWEB) September 04, 2013
Follow us on LinkedIn – Global demand for orphan drugs exhibits potential for healthy growth with rise in investments and interest of major pharmaceutical companies in orphan drug development. Factors such as rapidly aging population, increased incidence of orphan diseases among children, rise in diagnosis of rare cancers such as renal cell carcinoma, acute myeloid leukemia and thyroid cancer among others, are driving demand for novel therapies in the orphan drug market. Other factors driving market growth include legislative benefits such as extended market exclusivity period, tax credits, research assistance and partial funding of clinical trials. In addition, the growing number of orphan diseases and the lack of appropriate therapies resulting in significant unmet medical needs is a major factor generating interest in orphan drug development. Moreover, blockbuster sales of leading premium orphan drugs offers growth opportunities for companies involved in orphan drug development.
Despite the enormous potential offered, orphan drug development continues to face challenges in clinical trials such as the need for validated endpoints, patient recruitment and the lack of understanding of the progress of specific orphan conditions. Clinical trials involving therapies for rare diseases are impeded by the absence of adequate number of patients to study the efficacy of the drug. In addition, orphan drug development also suffers from lack of validated endpoints for a study and the unavailability of considerable clinical data for designing a study. In addition, the Eurozone crisis in Europe and the fiscal deficit in the United States and the resulting austerity measures adopted by governments to reduce spending in various areas including healthcare, also presents a challenging environment for drug development. With austerity emerging as the buzzword, orphan drugs which command high prices and also full reimbursement owing to lack of alternate therapies, are expected to come under pressure as reimbursement policies are reviewed and revised. Orphan drugs with some kind of alternatives, and/or prevalent off-label use are expected to be among the first to face the prospects of revision of reimbursement rates.
Breakthroughs in the field of biotechnology are expected to improve understanding of rare diseases and assist development of novel therapies. Further, the numerous collaborative research programs across the US and Europe, intended to improve patient access to appropriate medical care and support provide manufacturers, healthcare experts and patients with epidemiological research data on the disease. This trend will result in greater awareness among patients over available treatment options. Improved awareness of rare diseases and therapies available is expected to drive demand for orphan drugs. Biological Orphan Drugs represents the largest market segment with growth led by high prices of biological drugs and the ability of such drugs to target specific sites. Moreover, with about 80% orphan diseases being genetic in nature, these drugs are best suited for targeted therapies.
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 the largest markets worldwide. The rise in interest of major pharmaceutical companies driven by the incentives offered by the US Food and Drug Administration (FDA) as well as the European Medicines Evaluation Agency (EMA), and European Commission, including extended market exclusivity and grants for research are the major aspects driving growth in these markets.
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, 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, 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 details and types, company profiles, mergers and acquisitions and other strategic industry activities. The report provides market estimates and projections for all major geographic markets including the United States, Japan, Europe, and Rest of World.
For more details about this comprehensive market research report, please visit –
About Global Industry Analysts, Inc.
Global Industry Analysts, Inc., (GIA) is a leading publisher of off-the-shelf market research. Founded in 1987, the company currently employs over 800 people worldwide. Annually, GIA publishes more than 1300 full-scale research reports and analyzes 40,000+ market and technology trends while monitoring more than 126,000 Companies worldwide. Serving over 9500 clients in 27 countries, GIA is recognized today, as one of the world's largest and reputed market research firms.
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