Per patient costs for orphan drugs are 13.8 times higher than non-orphan drugs

Published: 6-Nov-2015

Annual EvaluatePharma orphan drug report 2015 sees continued market growth despite industry pricing pressures


With 2020 worldwide sales forecast at US$178bn, the orphan drug market is expected to grow by 11.7% per year (CAGR 2015–2020) nearly doubling the yearly growth of the overall prescription drug market.

Moreover, worldwide orphan drug sales will account for 20.2% of worldwide prescription sales by 2020 (excluding generics), according to the EvaluatePharma Orphan Drug Report 2015 from life science market intelligence firm, Evaluate.

‘For three years, Evaluate has tracked the trajectory of this speciality market, which has achieved almost 12% 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 concerning 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 to reduce the overall healthcare budget.’

Highlights include:

  • median cost per patient is 13.8 times higher for orphan drugs compared with non-orphan: Advate delivers highest revenue per patient orphan drug in the US
  • worldwide orphan drug sales are forecast to total $178bn (CAGR 2015–2020: +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-orphans, but Phase III drug development is no quicker for orphan than non-orphan drugs
  • return on investment (ROI) 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, whereas 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 here.

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