NICE, the key decision-maker for accessing medicines in England, Wales and Northern Ireland has two pathways for medicines for rare conditions. The criteria to decide which one is used is being updated.
The Highly Specialised Technology (HST) programme at NICE has been operating since 2015 and has approved 28 medicines for access in the NHS. The HST committee is specialised in making decisions around rare conditions and has extra tools to handle uncertainty. Access to this decision-making pathway is therefore highly prized by communities who want the best chance to access an innovative treatment for their condition.
NICE consulted on proposals to make the criteria for entry more consistent, predictable and improve the transparency of entry decisions. Genetic Alliance UK hosted a meeting of its member groups to discuss the likely impact on our community and identify key messages for our consultation response.
We are grateful that NICE invited us to discuss our community’s perspectives with Jonathan Benger, Deputy CEO and Medical Director of NICE in advance of the consultation.
Earlier this month, the Board of NICE approved the revised proposals following the consultation. We are pleased to see NICE is clearly listening to its stakeholder perspectives and have changed their proposed criteria following consultation. NICE have removed their proposed restriction on relapsing-remitting conditions which we argued against in our response; and have removed a restriction on there being no ongoing clinical trials, which we opposed too.
NICE acknowledged concerns raised in our response about specificity on prevalence and the risks of subjectivity on the definition of ‘exceptional negative impact’. We’re pleased to see scope for some of these to be addressed through future adaptations to processes and methods.This is a step forward for clarity, but HST will remain a pathway that is used infrequently. The new criteria are best found here in the Board papers and are not yet on their website beyond this announcement.
Addressing some of the bigger picture issues in funding decisions for rare condition medicines in the UK was never in scope of this consultation. Issues like the gap between this process, and the ‘standard’ Single Technology Assessment approach, which is used for all other medicines, still stand. Genetic Alliance UK is working with LifeArc on a report which will cover some elements of this challenge in more detail, and which is due to be published in the coming months.
Please keep sharing your experience of these decision-making processes with us! It’s only with this information that we can continue to hold NICE to account on these key decisions.
