Have Questions?

Global Headquarters

Tel: +44 (0)20 7377 0800
Fax: +44 (0)20 7349 5157

North America

Tel: 1-617-573-9450
Fax: 1-617-573-9542


Tel: +81 (0)80-1164-4754

Request a Demo

What our customers are saying

When I think of the future, I think of EvaluatePharma

Senior Director,
Global pharmaceutical company

Press Release

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

Download PDF

The Bio Report Podcast: What's Driving the Booming Market for Orphan Drugs with Jonathan Gardner, Deputy News Editor at EP Vantage.

LONDON, BOSTON, TOKYO (October 29, 2014) — 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 market intelligence firm 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

The “Orphan Drug Report 2014” based on market intelligence and consensus forecasts from EvaluatePharma can be downloaded at www.evaluategroup.com/orphandrug2014.

About Evaluate Ltd.
Evaluate is the trusted source for life science market intelligence and analysis with exclusive consensus forecasts to 2020. We support life science and healthcare companies, financial institutions, consultancies and service providers in their strategic decision-making. Our services include EvaluatePharma, EvaluateClinical Trials and EvaluateMedTech. Our global team of dedicated healthcare analysts employs rigorous methodologies to deliver strategic commercial analysis. We make our services valuable by combining superior quality content, user-friendly reporting tools and outstanding customer service to solve client problems.

For more information visit: www.evaluategroup.com. On Twitter: @evaluatepharma, @evaluatemedtech, @epclinicaltrial, @epvantage.

Back to Press Releases