Status In progress
Technology type Medicine
Decision Selected
Reason for decision Anticipate the topic will be of importance to patients, carers, professionals, commissioners and the health of the public to ensure clinical benefit is realised, inequalities in use addressed, and help them make the best use of NHS resources
Further information Awaiting development
Process HST Standard
ID number 6145

Project Team

Project lead Vonda Murray

Email enquiries

External Assessment Group Centre for Reviews and Dissemination and Centre for Health Economics, University of York

Stakeholders

Companies sponsors BioMarin (cerliponase alfa)
Others Birmingham Children’s Hospital NHS Foundation Trust Lysosomal Storage Disorders Unit
  Bristol Royal Hospital for Children
  Department of Health and Social Care
  Newcastle upon Tyne Hospitals NHS Foundation Trust, Great North Children’s Hospital
  NHS England
Patient carer groups Batten Disease Family Association
Professional groups Royal College of Physicians
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
  Medicines and Healthcare products Regulatory Agency
  Scottish Medicines Consortium
  Welsh Government
  Welsh Health Specialised Services Committee
Relevant research groups National Institute for Health Research
  Rare Disease Research Partners

Timeline

Key events during the development of the guidance:

Date Update
09 October 2024 Since 2019, cerliponase alfa (Brineura®) has been provided for eligible NHS patients under a Managed Access Agreement (MAA). With the support of NICE, NHS England and BioMarin have agreed a six-month extension of the current MAA. The extended MAA will now run until 27 May 2025. All parties will now use the extension period to try to reach an agreement that secures permanent access for current patients and allows new patients to start treatment. During this period, newly diagnosed patients can be assessed for treatment eligibility and start treatment under the terms of the current MAA. There are currently no arrangements to enable access to cerliponase alfa as part of standard NHS care following the expiry of the MAA. As part of the ongoing NICE evaluation (review of HST12) [ID6145] the next steps will aim to enable commercial discussions between NHS England and BioMarin to progress further and will not affect continuation of treatment under the now extended MAA. Discussions between NICE, NHS England and BioMarin so far have been constructive, leading to the agreement of the six-month extension, and will now continue. All parties are committed to providing updates to stakeholders, notwithstanding the necessity that discussions are undertaken in a confidential setting.
05 September 2024 Committee meeting
14 August 2024 The second committee meeting for this evaluation will take place on Thursday 5 September 2024.
04 July 2024 This evaluation was discussed by the HST evaluation committee on Wednesday 12 June 2024. Following the committee meeting, this evaluation will be paused while additional analyses are provided by the company. The reason the committee asked for this is because it wanted to know further information and see clinical and cost-effectiveness analyses: • exploring clear starting and stopping rule(s) for patients. This was discussed during the committee meeting, and the committee would like to understand in whom, and when, the treatment is most effective and cost-effective. • Other modelling issues. We expect to hold a second committee meeting on 5 September 2024 to consider the additional analyses from the company. The publication timelines will also change to align with this committee meeting date. We will confirm the timelines this on the website and let participating stakeholders know by August 2024.
12 June 2024 Committee meeting
31 October 2023 Invitation to participate
14 September 2023 - 12 October 2023 Consultation on suggested remit, draft scope and provisional stakeholder list of consultees and commentators
14 September 2023 In progress. Draft scope issued
11 August 2023 Awaiting development

For further information on our processes and methods, please see our CHTE processes and methods manual