Status In progress
Process HST
ID number 943

Provisional Schedule

Committee meeting: 3 25 July 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
09 May 2018 Following the committee meeting on 25th April 2018, the committee has asked that NHS England engage with the company to formalise the details of the draft managed access agreement proposal, including data collection and confidential commercial proposals. The outcome from this engagement will be presented to committee for decision-making and therefore this topic will have a 3rd meeting on 25 July 2018. No formal documentation is being released in the interim.
25 April 2018 Committee meeting: 2
07 March 2018 The forthcoming committee meeting to discuss cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 on Tuesday 20 March 2018 has been rearranged to enable the company to prepare and submit additional information for the committee to consider . Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 will be now considered by committee on Wednesday 25th April 2018.
12 February 2018 - 05 March 2018 Evaluation consultation: 1
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