To appraise the clinical and cost effectiveness of voretigene neparvovec within its marketing authorisation for treating inherited retinal dystrophies caused by RPE65 gene mutations.
Status In progress
Process HST
ID number 1054

Provisional Schedule

Expected publication 08 January 2020

Project Team

Project lead Joanne Ekeledo

Email enquiries


Companies sponsors Novartis Pharmaceuticals (UK) (voretigene neparvovec)
Others Department of Health and Social Care
  NHS England
Patient carer groups Fight for Sight
  Retina UK
Professional groups Royal College of Ophthalmologists
  Royal College of Physicians


Associated public health groups Public Health England
  Public Health Wales
General commentators All Wales Therapeutic and Toxicology Centre
  British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups None


Key events during the development of the guidance:

Date Update
25 July 2019 Committee meeting
06 February 2019 The forthcoming discussion of this HST evaluation has been rescheduled. During the clinical expert selection process it was highlighted clinical experts would be unavailable to attend the NICE committee meeting as the Royal College of Ophthalmologists are holding their annual congress in Glasgow on 21 May 2019. NICE recognises the importance of clinical experts contribution to committee discussions. Therefore voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations will now be considered by the HST committee on Thursday 25 July 2019.
03 December 2018 Invitation to participate
03 December 2018 In progress, In progress
13 September 2017 (00:00) Scoping workshop
19 July 2017 - 16 August 2017 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

For further information on how we develop guidance, please see our page about NICE highly specialised technologies guidance