| 20/08/2024 Dear Families, We wanted to provide you with an update on the second NICE committee meeting to assess the use of Brineura on the NHS. Following a brief pause in the process due to NICE assessing new evidence from BioMarin, NICE have now confirmed that the second committee meeting will go ahead as planned. The meeting will be held on Thursday 5th September 2024 at 09:00am. This meeting will be virtual, and you can register to join if you wish to observe proceedings via this link: Evaluation of cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (review of HST12) [ID6145] Please note that the deadline for registration is 4:00pm on Wednesday 29th August 2024. The BDFA will be joining and will share an update with you at a debrief meeting on Monday 9th September at 12pm, details of which will follow soon. Meeting objectives The committee will consider the information provided by BioMarin after the first meeting. The committee requested to know further information and to see clinical and cost-effectiveness analyses: · Exploring clear starting and stopping rule(s) for patients. This was discussed during the first committee meeting, and the committee would like to understand in whom, and when, the treatment is most effective and cost-effective · Other modelling issues Meeting attendance The three patient experts, Liz Brownnutt, Lucy Carroll and Gail Rich will once again be attending to provide evidence at the meeting. They attend as individuals, not as representatives of the BDFA, but they are there to represent CLN2 patients and families. Their role at the meeting is to use their experience to make sure that: · The committee has heard what is important to the children and their families · The committee has been given an accurate description of key issues being discussed regarding the treatment · All of the evidence is presented and that there is nothing missing The committee, which makes the decision, is made up of the Chair and Vice Chair, 2 lay members, academics (such as health economists), NHS staff and representatives from the pharmaceutical and medical devices industry. The stakeholders in attendance include patient experts, clinical experts, BioMarin staff, members of NHS England, NICE staff and the Evidence Assessment Group. Meeting agenda The meeting will broadly follow the same format as the meeting in June. It will be divided into two parts. In the first part, the committee members and all stakeholders are present, and members of the public can watch. In the second part, only committee members can attend. During the first part of the meeting the agenda is assumed be to be as followed: · Introductions · A presentation which summarises the key issues for the committee to discuss and consider at the meeting following which there will be questions for patient experts and other stakeholders · A discussion including the committee members and stakeholders regarding the key issues After this, stakeholders are asked to leave, and the committee members continue their discussions to make their preliminary decision during the second part of the meeting. Any decision will not be immediately shared with us or any other stakeholders. We are not likely to be notified of any decision or have sight of the draft guidance for a few weeks. Please feel assured that we are your voice; we will share the draft guidance with you as soon as we are able to and will be keeping families informed every step of the way. As ever, please get in touch if you have any questions about the next NICE committee meeting. Kind regards, Liz |
