Highly Specialised Technology appraisals Archives -

Following yesterday’s international Rare Disease Day, here is a snapshot of four new rare disease treatments currently under

Four new rare disease treatments awaiting NICE review

https://pharmaphorum.com/views-and-analysis/four-new-rare-disease-treatments-awaiting-nice-review/

Project will initially focus on DMD but could benefit other rare diseases

Pharma collaborates to improve chances of NICE backing Duchenne drugs

https://pharmaphorum.com/news/pharmas-join-improve-duchenne-muscular-dystrophy/

GlaxoSmithKline’s gene therapy Strimvelis for the treatment of the ultra-rare ‘bubble baby syndrome’ has been approved by NICE.

GSK’s ‘bubble baby’ gene therapy passes NICE’s new stricter test

https://pharmaphorum.com/news/strimvelis-approved-by-nice/

Watchdog accused of ignoring stakeholder consensus and call for negotiations

Alexion lambasts NICE over rare disease ruling

https://pharmaphorum.com/news/alexion-lambasts-nice-over-rare-disease-ruling/

The National Health Service should fund PTC Therapeutics’ Duchenne Muscular Dystrophy drug, Translarna (ataluren), NICE has said, after

NICE backs £220,000-a-year Duchenne drug following discount

https://pharmaphorum.com/news/nice-backs-ptc-muscular-dystrophy-drug-following-discount/

Highly Specialised Technology appraisals