UK: NICE Wins Argument Over Appraisal Of BioMarin Rare Disease Drug
BioMarin To Re-Engage With Process But Is Doubtful Of Outcome
A decision by England’s High Court to reject claims that HTA body NICE acted unlawfully when evaluating BioMarin’s PKU treatment has quashed hopes that NICE’s Highly Specialized Technologies program will become more widely used to evaluate rare disease treatments. Lawyers representing a PKU patient are planning to appeal the judgement.
You may also be interested in...
bluebird is "shocked and disappointed" at the draft negative reimbursement decision on Zynteglo in UK but measures outlined in the NICE methods review could change fortunes of gene and cell therapies if taken forward.
A judicial review of how health technology appraisal body NICE decided which appraisal route BioMarin’s rare disease drug Kuvan would take could have lasting implications for future treatments.
The Scottish health technology appraisal body, the SMC, has announced its recommendations on Sanofi’s Dupixent, Ipsen’s Cabometyx, Sobi’s Zynlonta and Novartis’ Cosentyx.