Presentation (CTI)

OBJECTIVES: Health Technology Assessment (HTA) of novel pharmaceuticals in the UK involves a comprehensive evaluation of clinical effectiveness and cost effectiveness, to ensure value for money to the NHS. HTA also considers broader issues including burden of disease, unmet need, and benefits beyond those captured by the QALY. The objective was to develop a framework, based on UK HTA evidence requirements, to assess the evidence generation needs and inform the future UK HTA strategy of two novel therapies to treat patients with sickle cell disease (SCD).
METHODS: To understand the treatment landscape for SCD, a review of evidence from prior and ongoing NICE and SMC technology appraisals of current treatments was conducted. Information from each appraisal was extracted according to four key ‘Buckets of Evidence’: (B1) disease burden and management, and unmet need; (B2) relative clinical effectiveness versus comparator studies; (B3) relative cost effectiveness and budget impact; (B4) non-health benefits including carer/ family burden and health inequalities.
RESULTS: A total of four (three NICE; one SMC) technology appraisals across three SCD treatments were reviewed and extracted into the four ‘Buckets’. Key information to ‘fill’ the ‘Buckets’ was obtained from published materials including the draft/ final scopes and guidance, committee papers, managed access agreement documentation, public committee slides, and detailed advice documents. Key uncertainties included positioning within the NICE pathway and changes in the comparator landscape (B1); use of surrogate endpoints (B2); economic model structure and approach; utilities and utility decrements (B3); and consideration of inequalities and a managed access agreement (B4).
CONCLUSIONS: The ‘Buckets of Evidence’ framework provided key considerations to guide evidence generation and early mitigation planning to inform future HTA strategy for two novel treatments in SCD. This framework can be easily adapted for use with other rare disease pipeline treatments, offering a versatile tool for pre-phase 3 evidence planning and generation.