Assessing the Impact of the NICE Methods Review on Rare Diseases

OBJECTIVES: NICE recently updated its methods and processes via the NICE methods review (2020-2022) with the aim of ensuring the HTA process remains cutting edge and future proof to support the needs of all parts of the healthcare and life sciences ecosystem. Pre-methods review, there were barriers to reimbursement in the rare disease space, particularly for medicines not deemed rare enough for HST - an issue recognised by NICE. The pipeline of complex, innovative technologies for rare diseases continues to grow and, as a result, the methods review was imperative to facilitate reimbursement and increase access to treatments for patients with rare diseases. The objective of this poster is to assess the likely impact of the NICE methods review outcomes on appraisals in rare diseases

METHODS: Updates in the methods review that have had a significant impact on rare disease technologies were identified as the inclusion of modifiers, increased acceptability of uncertainty, increased acceptability of real world evidence, updates to the HST eligibility criteria and updates to the discount rate. For each update, the potential impact on rare diseases was assessed.

RESULTS: Although the overall impact of the methods review is likely to be positive for rare diseases, it’s unlikely to be sufficient to overcome substantial access barriers in rare diseases. Most notably, the HST eligibility criteria are particularly restrictive. Creating barriers for access and, in combination with the application of an underwhelming ‘budget neutral’ approach to multipliers, did not address the perceived cliff edge for rare diseases not deemed rare enough for HST.

CONCLUSIONS: In conclusion, although changes were positive for rare diseases, they did not go far enough. This risks future access in rare diseases. Future modular updates should look to specifically address the issue of rare diseases to ensure that the UK remains a world-leader for rare disease technology patient access.