Per Patient Costs for Orphan Drugs is 13.8 Times Higher Than Non-Orphan Drugs

December 3, 2015

With 2020 worldwide sales forecast at $178 billion, the orphan drug market is expected to grow by 11.7 percent per year, (CAGR 2015 to 2020) nearly doubling the yearly growth of the overall prescription drug market. Moreover, worldwide orphan drug sales will account for 20.2 percent of worldwide prescription sales by 2020 (excluding generics) according to the “EvaluatePharma Orphan Drug Report 2015” from life science market intelligence firm Evaluate Ltd.

“For three years, Evaluate has tracked the trajectory of this specialty market, which has achieved almost 12 percent annual growth, said Andreas Hadjivasiliou, report author and EvaluatePharma analyst. “Up until now, orphan drug developers have managed to defend the cost of these life-changing drugs but with the increasing scrutiny over drug costs and the growing number of designations awarded, orphan drug developers will have to continue to innovate and demonstrate how their products can help reduce the overall healthcare budget.”

EvaluatePharma Orphan Drug Report 2015 Highlights:

  • Median cost per patient is 13.8 times higher for orphan drugs compared to non-orphan; Advate delivers highest revenue per patient orphan drug in US.
  • Worldwide orphan drug sales are forecast to total $178bn (CAGR 2015 to 2020: +11.7%); nearly double the overall prescription market growth.
  • Celgene overtakes Novartis and climbs to the number one position in orphan drug sales by 2020 thanks to top selling Revlimid.
  • Phase III orphan drug development costs are half that of non-orphan but Phase III drug development is no quicker for orphan than non-orphan drugs.
  • Return on investment of Phase III/ filed orphan drugs is 1.14 times greater than the ROI for non-orphan drugs.
  • 2014 was a record year for orphan drug designations with the FDA awarding 291, Europe awarding 201 and Japan awarding 32 designations.
  • Opdivo ranks as the most promising new orphan drug approved in the US in 2014 while Obeticholic Acid (Intercept Pharmaceuticals) ranks as the most valuable R&D product.


The, “EvaluatePharma Orphan Drug Report 2015”, based on market intelligence and consensus forecasts from EvaluatePharma can be downloaded at


About Evaluate Ltd

Evaluate is the trusted source for life science market intelligence and analysis with exclusive consensus forecasts to 2020. Our services include EvaluatePharmaEvaluateClinical Trials and EvaluateMedTech. Our award-winning editorial team, EP Vantage, leverages our strategic analysis to cut through the noise, giving you daily opinion and insights. The Evaluate services enable the life science community to make sound business decisions about value and opportunity. For more information please visit  On Twitter: @evaluatepharma@evaluatemedtech@evaluateJP @epclinicaltrial@epvantage.

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