Problem Statement: Sickle cell disease (SCD) is a rare, chronic, inherited blood disorder. People with SCD are at an increased risk of acute and chronic complications resulting in significantly reduced health-related quality of life, and reduced life expectancy. The treatment options available are very limited. The majority of people with SCD are from ethnic minorities (people of African, Caribbean, Middle-Eastern or South-Asian descent) and faces serious health inequalities.

Evidence generation and demonstrating comparative effectiveness against regular transfusion therapy (RTT) was challenging, as RTT was not included in the clinical trial. RTT is burdensome for patients, requires regular hospital appointments and associated with significant clinical risks, however it is the only option to improve haemolytic anaemia due to SCD.

Description: After two committee meetings NICE concluded voxelotor was not cost effective due to significant uncertainties in the evidence base and consequent unreliability of cost-effectiveness estimates. However, after a successful appeal, the Appeal Panel ruled that additional flexibility was needed to consider health inequalities in SCD. This ensured that in case of missing clinical trial data, NICE was able to consider a wider evidence base.

Multiple sources of evidence were submitted to fill the evidence gaps and demonstrate a reduction in the rate of RTT as a result of voxelotor treatment. These included quantitative real-world evidence (RWE) data sources (RETRO observational study, Sanius UK real-world data, Symphony RWE study and HES-CPRD data), as well as qualitative data sources (Delphi panel results and multiple clinician surveys).

Lessons Learned: The Appeal Panel’s ruling to apply additional flexibility when considering health inequalities in SCD ensured that NICE was able to consider a wider evidence base, including multiple sources of RWE. This example illustrates the importance of considering both quantitative and qualitative data sources when assessing technologies that help to address health inequalities.

Stakeholder Perspective: Industry.