New Report Shows Orphan Drug Market to Reach $176 Billion by 2020

Growth set to be nearly double that of overall prescription market excluding generics

31-Oct-2014 - United Kingdom

In 2020 orphan drugs are expected to account for 19 percent 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” from Evaluate Ltd.

At 11 percent, growth in the orphan drug market is set to be more than double the 5 percent growth rate of the overall prescription market excluding generics. This year’s report also includes pricing estimates based on Evaluate’s new USA Sales, Volume and Pricing content set. 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.

“Orphan drugs are no longer a niche segment of the market and are projected to account for 19 percent of the prescription drug market by 2020,” said Anthony Raeside, Evaluate’s head of research.  “Although orphan populations are by definition the smallest, they represent big per-patient outlays, and insurers will be looking carefully at new tools to arrest cost growth as more and more orphan drugs launch.”

Some of the report’s key findings include:
  • A record 260 orphan drug designations were granted in the US in 2013
  • Orphan drug sales to grow 11 percent per year through 2020, compared to 5 percent 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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