Orphan Drug 2022 Report 2022: NICHE NO LONGER

2022年 希少疾病用医薬品動向レポート 英語版

Treatments for orphan indications and rare diseases are no longer a niche area of focus for developers. Orphan products have become one of the fastest growing areas of drug development—outpacing the growth of the wider pharmaceuticals landscape, and accounting for an increasingly large proportion of novel drugs hitting the market. By 2026, orphans will make up a fifth of all prescription drug sales, and almost a third of the global drug pipeline’s value. 


Our Orphan Drug Report 2022 draws on Evaluate’s deep knowledge of the sector to highlight the big issues in this increasingly competitive – and lucrative space.


Our Orphan Drug Report 2022: Niche No Longer is now available for download. 



Big Pharma will book sales for nine of the top ten orphan drugs in 2026.
The rise of orphan blockbusters: Each of the top 10 orphans in 2026 will be worth between $3 billion and $13 billion.
Orphans’ strong growth is leading to crowding in some corners of the market.
The net present value of forecast 2026 sales of the top ten pipeline orphan drugs is more than $42 billion.
Calls to reform orphan drug legislation are starting gain traction in key markets.