NEW YORK (GBI Research), 3 May 2012 - Patients suffering from orphan diseases can look forward to a wider range of drug options in the future, according to a new report by pharmaceuticals expert GBI Research.
The new report* shows that the relatively unsatisfied market for orphan disease drugs could prove to be a big hit for pharmaceutical companies able to improve upon the limited options currently available to patients.
GBI Research’s analysis of industry trends suggests that the orphan disease therapeutics market is likely to see a steady increase in value over the forecast period. There are more than 6,000 different orphan diseases recognized in the US, while in Europe there are approximately 5,000–8,000 orphan diseases affecting almost 29–36 million people. However, only a very small number of drugs are currently available to treat these diseases, indicating a high level of unmet need in terms of safety, efficacy and availability.
This need for an improved and expanded choice of drugs will boost research and development (R&D) for orphan diseases, and the market has seen a rapid increase in R&D investment from top pharmaceuticals companies in order to claim a slice of the potential profits. The therapeutic capabilities of biologic drugs, in regards to the control and treatment of orphan diseases, have also allowed pharmaceutical manufacturers to commence label extensions for multiple orphan diseases. These lucrative benefits, combined with the increasing number of approvals of new orphan drugs, will see the future market grow at an increasing rate. Improving patient and physician awareness of orphan diseases will also boost the market.
However, pharmaceutical development is not without its risks, and the production of challenging orphan disease treatments may risk complications and industry errors. Genzyme recently suffered a blow to its inventory when a virus at manufacturing-level caused a drug shortage, and the company may be considering out-licensing their non-core businesses to help cope with the setback. Meeting the needs of orphan disease patients is a demanding role and, as with other companies specializing in this challenging segment, dealing with non-core business could lead to further revenue losses.
The global orphan disease therapeutics market was worth $2.3 billion in 2010 and is estimated to reach $6 billion by 2018, demonstrating a compound annual growth rate (CAGR) of 13.1%.
*Orphan Disease Therapeutics Market to 2018 – Improved Understanding of Rare Diseases’ Heterogeneity and Novel New Clinical Trial Designs to Foster Innovation
This report provides in-depth analysis of unmet needs, drivers and barriers that impact the global orphan diseases therapeutics market. The report analyzes the markets for orphan diseases therapeutics in the US, the top five countries in Europe (the UK, Germany, France, Italy and Spain) and Japan. Treatment usage patterns, sales, and price are forecast until 2018 for the key geographies as well as selected therapeutic segments. Furthermore, the report provides competitive benchmarking for the leading companies, while analyzing the mergers, acquisitions and licensing agreements that shape the global markets.
This report is built using data and information sourced from proprietary databases, primary and secondary research and in-house analysis by GBI Research’s team of industry experts.
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