EvaluatePharma Orphan Drug Report 2019

$242bn forecasted orphan drug sales by 2024, one-fifth of the total worldwide pharmaceutical market 

The EvaluatePharma Orphan Drug Report 2019 arrives at the start of a new era the orphan drug industry, following the FDA’s approval of the first gene therapy product. Coupled with other breakthrough cell therapy products, the orphan drug market is poised to capitalise on cutting-edge science to the tune of a 12% CAGR to 2024, double the 6% rate expected for non-orphan products.

The mean cost per patient per year of 2018’s top 100 orphan drugs is almost 4.5 times more than non-orphan drugs, which makes the sector an attractive proposition for big pharma. However, the explosion of new technologies, also means their very high price points are likely to attract more attention and pressure to reform the Orphan Drug Act.

Key Highlights

  • Blood, CNS and respiratory are the leading orphan drug therapeutic areas, accounting for more than 50% of the non-oncology orphan drug market.

  • Vertex’s triple combination for cystic fibrosis is the most valuable R&D orphan product, with an estimated NPV of $24bn.

  • In 2018, the number of products granted an orphan drug designation decreased by 27% in the USA, 16% in the EU and 27% in Japan, as compared to 2017.