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Dear Families,

Following the Nice Committee meeting on 12th June for the re-evaluation of Brineura, we wanted to make you aware that NICE have decided to pause the formal evaluation process while they assess new evidence from BioMarin.

NICE have now published the following statement on their website:

“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.”

The statement can be viewed on the NICE website:

We want to reassure you that a pause in the evaluation process, so that NICE can assess further evidence from the company, is not unusual.
We are hopeful that this will allow further consideration of all the evidence in full to allow the committee to evaluate the cost-effectiveness and value of Brineura.

What this means for timelines – In response to a query from the BDFA, NICE have also shared with us that recommendations in draft guidance will follow from the second committee meeting in September, before the end of the managed access period (November 2024). If more time is needed for the NICE process, NHS England will discuss an extension to the current managed access agreement with the manufacturer, BioMarin.

What we will continue to do – The BDFA will maintain regular communication with NICE, BioMarin and NHS England and  continue to make the strongest case possible for continued access to Brineura on the NHS. We will work collaboratively with all stakeholders through this process and we will not stop until long-term access to Brineura for patients is secured.

We are continuing to receive external professional advice on how to engage with the NICE appraisal process in the most constructive way possible and have a communications strategy in place as shared with families following the committee meeting.

Next steps – We would like to invite you to a call on Wednesday 17th July at 12pm where we can discuss next steps and answer any questions you may have.

We will continue to keep you informed every step of the way and are happy to answer any questions you may have.

Kind regards,

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