The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing authoritative, evidence-based guidance for the National Health Service on whether drugs and other treatments represent an effective use of NHS resources.
NICE’s methods and processes for the assessment of drugs have been carefully developed over time and are internationally respected. NICE continues to keep its procedures under periodic review to ensure that they remain fit for purpose.
All topics, including drugs for rare diseases, are considered through a rigorous topic selection process before formal referral to NICE to ensure that NICE guidance is appropriate and will add value. NICE assesses most significant new drugs through its technology appraisal programme and has been able to recommend a number of drugs licensed for the treatment of rare diseases for routine use on the NHS.