The steady growth of the orphan drug market remains one of the prominent themes in the fourth edition of the annual EvaluatePharma Orphan Drug Report. In the last 12 months, there has been increased scrutiny of the price of these lifesaving products.
“The pricing incentives are substantial,” added Andreas Hadjivasiliou, report author and EvaluatePharma analyst. “Of the top 100 drugs in the US, the average cost per patient per year for an orphan drug was $140,443 in 2016, compared with $27,756 for a non-orphan.”
The pricing incentives are substantial, putting pressure on the industry to generate innovations that justify the huge costs.
“This puts pressure on the industry to continue to generate innovations that justify the huge costs of treatments.”
Despite a record number of applications made to the FDA for orphan designation in 2016, only 333 designations were made – the lowest since 2012.
The market
The market sees continued growth (CAGR 2017 to 2022:+11.1%) for orphan drugs, approximately double overall prescription market growth.
Lisa Urquhart, EP Vantage Editor and report contributor said:
“The image of the plucky small biotech striving to develop treatments for rare diseases largely ignored by big pharma is long gone.”
“Instead, this year we find big pharma dominating the sector. Seven of the top 10 companies by orphan drug sales are global industry players.”
The image of the plucky small biotech striving to develop treatments for rare diseases largely ignored by big pharma is long gone.
Orphan drugs are set to be 21.4% of worldwide prescription sales by 2022 (excluding generics) and Celgene is set to climb to number one position in orphan drug sales to 2022.
The report predicts that Shire will be the largest company by sales in the orphan non-oncology space in 2022; and AstraZeneca, Abbvie and Johnson & Johnson are set to march up the orphan drug sales ranking table.
Revlimid (lenalidomide) will be the No.1 orphan drug in 2022, while Opdivo (nivolumab) will be the No.1 orphan drug in Europe. Axicabtagene Ciloleucel (Kite Pharma) is the most valuable R&D orphan drug.
The report forecasts that orphan drugs will account for 55% of the cumulative value of the European pipeline by 2022.