Rare insights: examining the social values of treating rare diseases

Between 2019 and 2022, NICE conducted a large-scale review of the methods and processes it uses to carry out assessments of a treatment’s cost-effectiveness. During the review, the BIA argued that NICE should consider adopting specific measures to its methods to support improved patient access to innovative treatments for rare diseases. One such approach could have been the introduction of a modifier for rare diseases, however, NICE concluded that there was “no evidence that society values more highly health benefits in rare diseases” and that the information presented during the consultation did not provide sufficient evidence to support adding a modifier for rare diseases.

Following the conclusion of the review, the BIA decided to conduct primary research to support NICE’s requirement for more robust evidence on the social value associated with treating rare diseases. The aim of the research was to understand public opinion on how treatments for rare diseases should be funded and evaluated. The research was carried out using an established method of qualitative research to determine social value. Subsequent quantitative research was also used to test the findings among a broader sample size.

The research findings, set out in this report, demonstrate that the public believes that a distinctive and alternative approach should be adopted for making funding decisions about treatments for rare diseases, including the methods and processes in place to make these decisions. The findings also indicate a need for further research to determine the social value associated with rare diseases and how this should underpin its decision-making processes.


Based on the findings of this research, the BIA recommends the following actions:

  • NICE should utilise its NICE Listens programme to undertake primary research on the social value associated with treating rare diseases

  • NICE should undertake, as a priority, a modular update review with a specific focus on rarity

  • As part of a modular update, NICE should reconsider the case for a rarity modifier, or consider a sliding scale of incremental cost-effectiveness ratio (ICER) thresholds within its single technology appraisal (STA) process

  • NHS England, the Department of Health and Social Care, and HM Treasury should commit to working with NICE to support the implementation of any changes to its methods and processes that are necessary to reflect contemporary social value judgements on rare diseases.

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