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BioCryst & Celgene Suffer English Funding Setback For Rare Disease Drugs

NICE’s Recommendations Are Out For Consultation until 28 July

 
• By Neena Brizmohun

Orladeyo and Inrebic have been approved in Great Britain but they will not be available under the National Health Service unless their manufacturers can convince the health technology assessment body, NICE, that their products are cost-effective.

Financial management
NICE says that two new products are not cost-effective enough • Source: Alamy

Health technology assessment (HTA) body NICE has issued preliminary guidance recommending against making two new drugs for rare diseases available on England’s National Health Service – Orladeyo (berotralstat), from BioCryst Pharmaceuticals, for preventing hereditary angioedema, and Celgene’s myelofibrosis treatment, Inrebic (fedratinib).

Neither oral drug would be a cost-effective use of NHS resources, according to NICE, which also has concerns

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