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

Predicted growth rate is double that of overall prescription drug market (Apr. 23)

The worldwide orphan drug market is set to reach $127 billion by 2018, accounting for nearly 16% of total prescription drug sales, according to a new report from EvaluatePharma, a London-based firm specializing in life-science sector analysis. The 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% between 2012 and 2018, nearly double that of the prescription drug market, excluding generics.

For pharmaceutical companies, the new 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 that are in phase III trials provide a 1.7-times greater return on investment than non-orphan drugs. Moreover, the analysis shows that phase III development costs for orphan drugs are half of those of non-orphan drugs.

The report’s key findings include:

  • In 2012, orphan drug sales increased 7.1% to $83 billion from the previous year. That compares with a 2.1% 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 (Onyx Pharmaceuticals), a multiple myeloma therapy, 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% in 2012, marking the first decline since 2007. Orphan designations in Europe increased 44%, reversing a decline in 2011.
  • Of the 43 new drugs approved by the FDA in 2012, 15 were orphan drugs, representing 35% of the industry’s new drug output.

Source: EvaluatePharma; April 23, 2013.

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