Ultra-rare, ultra-slow

NHS England and NICE have grappled for years with how to evaluate ultra-Orphan drugs, but neither has applied a cost-effectiveness analysis in funding decisions - until now. The proposed £100,000 threshold would not factor into NICE’s recommendations. But it would provide the NHS an out from automatic funding for expensive ultra-Orphan drugs, even if they have a positive recommendation from NICE.

In cases where NHS decided not to automatically fund one of these drugs, the joint NHS/NICE proposal could delay patient access to ultra-Orphan drugs by a year or more and could theoretically remove funding for some of the most expensive drugs. ...