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

Worldwide Orphan Drug Market to Grow to $127 Billion by 2018

Predicted growth rate is double that of the overall prescription drug market, according to Evaluate’s 2013 Orphan Drug Report

2013 BIO International Convention

Download PDF

LONDON and BOSTON (April 23, 2013) — The worldwide orphan drug market is set to reach $127 billion by 2018, accounting for nearly 16 percent of total prescription drug sales, according to the new 2013 Orphan Drug Report from Evaluate. The first-edition report sheds light on the market dynamics of orphan drugs — pharmaceutical products aimed at rare diseases or disorders — projecting that sales will experience a compound annual growth rate of 7.4 percent between 2012 and 2018, nearly double that of the prescription drug market, excluding generics.

For pharmaceutical companies, the findings confirm hypotheses that orphan drugs offer a greater return on investment than non-orphan drugs. Orphan drugs that have been filed for regulatory review or are in phase III trials provide a 1.7 times greater return on investment than non-orphan drugs. Moreover, EvaluatePharma’s analysis shows that phase III development costs for orphan drugs are half of those of non-orphan drugs.

“Pharma has traditionally been focused on developing drugs for large disease populations,” said Anthony Raeside, Evaluate’s Head of Research. “However, with R&D costs rising into the billions, pharma has shifted efforts to more niche indications which, as our analysis demonstrates, offer a greater return on investment.”

Here’s a look at some of the report’s key findings:

  • In 2012, orphan drug sales increased 7.1 percent to $83 billion from the previous year. That compares with a 2.1 percent decline in overall prescription drug sales (excluding generics), which fell to $645 billion.
  • Novartis will maintain its position as the world's No. 1 orphan drug company in 2018, with expected sales of $11.8 billion.
  • Kyprolis, a drug from Onyx Pharmaceuticals for multiple myeloma, was the most promising new orphan drug in 2012, with U.S. sales expected to reach $897 million in 2017.
  • The number of orphan drug designations in the U.S. fell 7 percent in 2012, marking the first decline since 2007. Orphan designations in Europe increased 44 percent, reversing a decline in 2011.
  • Of the 43 new drugs approved by the FDA in 2012, 15 were orphan drugs, representing 35 percent of the industry's new drug output.

The 2013 Orphan Drug Report, launched today at the 2013 BIO International Conference, is based on EvaluatePharma® data and complemented by commentary from the EP Vantage editorial team. For an in-depth look at why orphan drugs offer a greater return on investment, read EP Vantage’s commentary, “Orphan diseases’ appeal lies in return on investment.” For additional commentary on Novartis’ reigning position in orphan drugs, please read “Orphan drug lead cements Novartis ascendency.”

A hard-copy Executive Summary of the 2013 Orphan Drug Report is available at Evaluate’s Booth 3840. Anthony Raeside and EP Vantage Editor, Lisa Urquhart, will discuss the findings of the report during a press conference today, April 23, at 11 a.m. CST in room S106A at the 2013 BIO International Convention. A call-in number is available for media not attending BIO who are interested in participating in the press conference. Please refer to the media contacts for more details.

The complimentary report is available for download at http://www.evaluategroup.com/orphandrug2013.

Report Infographic - for quick overview of report findings 

About Evaluate Ltd.
Established in 1996, Evaluate Ltd. is the leader in high quality life science sector analysis. EvaluatePharma delivers exclusive consensus forecasts and trusted commercial insight into biotech and pharmaceutical performance. EvaluateMedTech sets a new standard in integrated analysis and consensus forecasts of the global medical device and diagnostic industry. EvaluateClinical Trials delivers unique intelligence to efficiently and accurately analyze the global clinical trial landscape. A team of more than 85 dedicated healthcare analysts employ rigorous methodologies to collate, organize and deliver the most-up-to-date commercial performance data available. An award-winning editorial team of journalists writing under the EP Vantage name support EvaluatePharma’s analysis. The Evaluate services enable the life science community to make sound business decisions about value and opportunity. 

Twitter: @evaluatepharma, @evaluatemedtech, @epclinicaltrial, @epvantage.

Back to press releases