Median cost per patient for orphan drugs is 5.5 times higher than non-orphan drugs

The steady and inexorable growth of the orphan drug market remains one of the prominent themes in the fourth edition of the “EvaluatePharma Orphan Drug Report 2017”. What has changed in the last 12 months according to the report is the increased scrutiny of the price of these lifesaving products.

“The image of the plucky small biotech striving to develop treatments for the rare diseases largely ignored by big pharma is long gone. Instead, this year we again find big pharma dominating the sector. Seven of the top 10 companies by orphan drug sales are global industry players”, said Lisa Urquhart, EP Vantage editor and report contributor. “And the pricing incentives are substantial”, added Andreas Hadjivasiliou, report author and EvaluatePharma analyst, “Of the top 100 drugs in the US the average cost per patient per year for an orphan drug was $140,443 in 2016, compared with $27,756 for a non-orphan, putting pressure on the industry to continue to generate innovations that justify the huge costs of treatments”..

EvaluatePharma Orphan Drug Report 2017 Highlights:

  • Worldwide orphan drug sales forecast to total $209bn (CAGR 2017 to 2022:+11.1%); approximately double overall prescription market growth
  • Orphan drugs set to be 21.4% of worldwide prescription sales by 2022 (excluding generics)
  • Median cost per patient 5.5 times higher for orphan drugs compared to non-orphan
  • Celgene set to climb to number one position in orphan drug sales to 2022
  • Shire to be largest company by sales in the orphan non-oncology space in 2022
  • AstraZeneca, Abbvie and Johnson & Johnson set to march up the orphan drug sales ranking table
  • Revlimid (lenalidomide) No.1 orphan drug in 2022
  • Opdivo (nivolumab) No. 1 orphan drug in Europe in 2022
  • Orphan drugs forecast to account for 55% of the cumulative value of the European pipeline to 2022
  • Axicabtagene Ciloleucel (Kite Pharma)is most valuable R&D orphan drug
  • First decline since 2012 for FDA orphan designations in 2016 with 333; Record number of applications made to the FDA for orphan designation in 2016

The, “EvaluatePharma Orphan Drug Report 2017”, based on commercial intelligence and consensus forecasts from the EvaluatePharma service can be downloaded at

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