Rare Diseases

(asked on 8th December 2016) - View Source

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health, how many treatments for rare and ultra-rare diseases that have received orphan designation as part of their regulatory license from the European Medicines Agency have qualified for NICE's single technology appraisal.


This question was answered on 15th December 2016

The National Institute for Health and Care Excellence (NICE) has advised that, of the 46 products that have received a European Medicines Agency Marketing Authorisation and orphan designation since 2013, 44 have been considered through its topic selection process. Of these, 20 have been selected for appraisal through NICE’s technology appraisal programme, six have been selected for evaluation through its highly specialised technologies programme, seven were eliminated as they did not fall within NICE’s remit and 11 were not prioritised for referral.

Reticulating Splines