Worldwide Orphan Drug Sales Are Forecast To Grow At A Cagr Of 11.3% From 2018 To 2024, Double The Rate Forecast For The Non-Orphan Drug Market

LONDON, BOSTON, TOKYO (May 25, 2018) — Continuing unmet medical need is set to fuel the growth of the worldwide orphan drug market with sales predicted to grow at over 11% a year to $262bn by 2024, according to EvaluatePharma’s fifth Orphan Drug Report.

But while there is strong demand for new treatments, the price that orphan drug developers can command for their products could be coming under pressure. This year’s report shows that the year-on-year price increases orphan drugs can command have fallen. However, the mean cost per patient per year still managed to hit $147,308 in 2017, more than four times the $30,708 mean cost for non-orphan drugs.

“While the overall 2024 revenue forecast for orphan drugs shows growth at double the rate of non-orphan drugs, if you just look at the forecasts for orphan and non-orphan drugs currently in R&D, they show similar CAGRs of about +132%,” said Karen Pomeranz, PhD, managing analyst and report author. “It’s possible that increased pricing pressure is contributing to this equilibrium. However, there are likely other factors at play as well, such as the expected levels of penetration and prescribing, and differences in the number of products expected to launch in each segment.”

  • The mean price of the top 100 orphan drugs (ranked by sales) grew at an annual rate of 5.2% from 2013 to 2017 compared to 9.2% for the top 100 non-orphan drugs.
  • Revlimid (lenalidomide) forecasted to be the top selling orphan drug in the USA, in 2024.
  • Soliris (eculizumab) generated the highest USA revenue per patient for orphan drugs in 2017.
  • Expected strong sales of Revlimid and Pomalyst (pomalidomide) will secure Celgene’s position as 2024 worldwide leader in orphan drug sales.
  • Alexion Pharmaceuticals leads the worldwide orphan drug market for blood indications.
  • Oncology is the leading orphan drug therapy area in 2024, accounting for about 50% of the total worldwide market. Blood is the second leading therapy area, accounting for about 12% of the market in 2024.
  • Lanadelumab (Shire) is the most valuable orphan drug in R&D, with a Net Present Value of $7.48bn
  • Implementation of the FDA’s orphan drug modernization plan has led to an increase in the number of orphan designation granted in the USA. Overall, the FDA has granted more orphan drug designations than any other regulatory body.

The “EvaluatePharma Orphan Drug Report 2018”, based on commercial intelligence and consensus forecasts from the EvaluatePharma service can be downloaded at

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