New report on European comparisons to the assessment of rare disease treatments

The SHCA has published a progress report on learnings from other European countries in the assessment of new rare diseases treatments. It builds on the findings from our 2024 report that found that many rare disease patients are still waiting too long to access new treatments, and in some cases are missing out altogether on potentially life-changing treatments available in other European countries.

2026 presents an important opportunity for NICE to review its approach to valuing rarity, building on the welcomed developments committed to in 2025:

• The MHRA will introduce a new set of regulations to speed up the time it takes to test, manufacture and approve rare disease treatments in the UK
• NICE will introduce the first increase to the cost-effectiveness thresholds used in the assessment of new treatments through its standard technology appraisal process since it was established in 1999
• As part of the changes to cost-effectiveness thresholds, NICE will introduce a new value set for use within EQ-5D-5L

To support NICE and the MHRA in reforming the way that rare disease treatments are assessed in England, this report sets out four case studies of changes introduced in other European countries, which provide examples of potential avenues that could be explored in England.

You can read the full report here.