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Four new rare disease treatments awaiting NICE review
https://pharmaphorum.com/views-and-analysis/four-new-rare-disease-treatments-awaiting-nice-review/
Highly Specialised Technology appraisals
Pharma collaborates to improve chances of NICE backing Duchenne drugs
Project will initially focus on DMD but could benefit other rare diseases
Alexion lambasts NICE over rare disease ruling
Watchdog accused of ignoring stakeholder consensus and call for negotiations