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EvaluatePharma Orphan Drug Report 2014

In 2020 orphan drugs are expected to account for 19% of the total share of prescription drug sales excluding generics, reaching $176 billion in annual sales, according to the new EvaluatePharma Orphan Drug Report 2014. 

At 11%, growth in the orphan drug market is set to be more than double the 5% growth rate of the overall prescription market excluding generics - view infographic hereThis year’s report also includes pricing estimates based on EvaluatePharma's new USA Sales, Volume and Pricing Analysis. In 2014, the average orphan drug cost per patient per year was $137,782 compared to an average of $20,875 for a non-orphan drug.

Key highlights include:

  • A record 260 orphan drug designations were granted in the US in 2013

  • Orphan drug sales to grow 11% per year through 2020, compared to 5% for drugs (excluding generics) treating larger patient populations

  • Vertex’ VX-809 in combination with Kalydeco was 2013’s most valuable R&D orphan drug based on NPV, while Pharmacyclics’ Imbruvica ranked as the most promising new orphan drug approved by the FDA in 2013

  • Bristol-Myers Squibb is set to leapfrog Novartis as the leader in orphan drug sales in 2020 thanks to cancer immunotherapeutic Opdivo

  • Phase III orphan drug development cost is half that of non-orphans but development time for orphans is not shorter than for non-orphan drugs

  • Anticipated return on investment for a Phase III/filed orphan drug is 1.89 times greater than for a non-orphan drug

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EvaluatePharma Orphan Drug Report 2014
Sales to Reach $176 Billion by 2020