The Future of Orphan Disease Therapeutics - Market Forecasts to 2015, Pipeline Analysis and Reimbursement

27 May 2010 • by Natalie Aster

GBI Research, the leading business intelligence provider, has released its latest research “The Future of Orphan Diseases Therapeutics - Market Forecasts to 2015, Pipeline Analysis and Reimbursement” The 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 2015 for the key geographies as well as select therapeutic segments. Further, the report provides competitive benchmarking for the leading companies and also analyzes 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.

The Global Orphan Disease Therapeutics Market is Forecast to Show Increased Growth Due to Increased Approval of Orphan Drugs

GBI Research examined Orphan diseases therapeutics markets in the US, the UK, France, Germany, Italy and Spain) and Japan. Of all the covered countries, the market size in the US was the largest as compared to other regions. The market is also growing the fastest in the US as compared to other regions. This is primarily due to increasing approvals of orphan designation and orphan approvals, higher volume of diseased population and improved disease awareness among physicians and the patients. Over the period November 1983 to December 2008, the FDA issued 1951 designations and 319 marketing authorizations (MA) for orphan medicinal products (OMP).

Orphan Drug Licensing is Growing at a Faster Rate Owing to High Market Potential

In 2008, orphan disease sector have witnessed numerous drug licensing agreements. With approximately 49 drugs licensing agreements in 2008 and it is evident that orphan disease therapeutics sector is expecting an enormous growth in the novel therapeutics area for a broad class of indications. The primary factor for this growth is because companies find the market potential to be attractive. In both the years the US and the EU market have dominated the sector. In a way these licensing agreements strengthen the financials of the new players in the market and enlarge pipeline portfolio of the major players involved in the deal. With respect to stages of drug development, most licensing deals took place for the drugs in the approval stage. This stage accounted for 44% of the total deals in 2008. This was followed by phase III and Phase II trials totaling to 39%.

High Unmet Needs in the Boosts the R&D Scenario in Orphan Disease Therapeutics Market

Taken together the industry and environmental trends suggest that orphan disease therapeutic area is likely to see a steady increase in market value within the next few years. GBI Research identified and quantified the unmet needs within the orphan disease therapeutics market. Currently, there are over about 6000 orphan diseases. Orphan drugs that are currently available in the market will treat only fewer orphan diseases. Even, the present therapeutic portfolio for the treatment of major orphan complications has shown to have high unmet needs in terms of safety as well as efficacy. Hence, the unmet need is very high for the existing orphan complications as well as for the availability of therapies for new orphan complications. This Boosts the R&D scenario in orphan diseases sector. GBI Research analyzed environmental and industry trends to assess the market evolution. Most trends observed in the pharmaceutical industry support the forecasting models, drivers and barriers outlined above. The top pharmaceuticals companies are seeing rapid increases in research and development costs accompanied by declining drug sales. The therapeutic capabilities of biologics in controlling and treating orphan complications are allowing pharmaceutical manufacturers to commence label extensions of their present portfolio of biologics for multiple orphan complications.