Status In progress
Process HST
ID number 943

Provisional Schedule

Evaluation consultation: 1 12 February 2018 - 05 March 2018
Committee meeting: 2 20 March 2018
Expected publication 27 June 2018

Project Team

Project lead Joanne Ekeledo

Email enquiries


Companies sponsors BioMarin Pharmaceuticals
Others Department of Health
  NHS England
  Birmingham Children’s Hospital NHS Foundation Trust
  Welsh Government
Patient carer groups Batten Disease Family Association
Professional groups Royal College of Physicians


Associated public health groups None
Comparator companies None
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Welsh Health Specialised Services Committee
Relevant research groups None


Key events during the development of the guidance:

Date Update
17 January 2018 Committee meeting: 1
07 August 2017 In progress, Evaluation in progress
07 August 2017 Invitation to participate
10 May 2017 Please note that following on from advice received from the company this evaluation has been rescheduled. Therefore, we now anticipate that the evaluation will begin during mid August 2017 when we will write to you about how you can get involved. The deadline for submissions is expected in approximately mid October 2017.
01 March 2017 Referral
09 August 2016 - 31 August 2016 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