England Reimburses Ultra-Rare Disease Drug Joenja, While EU Regulatory Review Drags On

 
• By Neena Brizmohun

Pharming has convinced NICE to reverse its rejection of its treatment for APDS by providing the health technology assessment institute with more data. It has also dropped the price it was asking for the drug, which has a list price of £352,000 per year per patient.

Close-up shot of pills with stamped pound sterling GBP symbol on them
NICE believes that Joenja is cost-effective (Shutterstock)

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