PSY96 - AN EARLY ASSESSMENT OF NICE HIGHLY SPECIALISED TECHNOLOGY EVALUATIONS

Abstract

To assess the progress of the highly specialised technology (HST) evaluation programme run by NICE in England. Qualitative comparative analysis of published HST evaluations to identify important features of evaluations having taken place followed by targeted secondary research to elucidate market opinions. Key themes were identified and explored in a consensus meeting, with implications drawn for pipeline products. Two HST evaluations have been published as of January 2016. HST1 concerns eculizumab for treating atypical haemolytic uraemic syndrome (aHUS) and HST2 considers elosulfase alfa for treating mucopolysaccharidosis type IVa (MPS IVa). Both evaluations recommended use, despite high cost of therapy (>£300,000/patient/year), which would not be acceptable in NICE’s conventional technology appraisal programme. Both treatments have orphan drug designation and are for very rare diseases, with MPS IVa affecting 88 people in England, while just 20-30 people in England are diagnosed with aHUS per year. Key to the recommendations were the limited availability of effective therapies and potential for substantial QALY gains delivered by the therapies. Recommendation was also conditional on considerable post-evaluation monitoring, with HST2 stipulating a managed entry agreement to assess clinical efficacy in the real world. The NICE HST programme provides a much needed route to market for highly expensive therapies that affect a very small number of patients and which would not meet NICE’s conventional cost-effectiveness criteria. Due to a high patient burden and unmet need, primarily driven by the lack of available treatments, therapies for rare conditions can gain national reimbursement in England, subject to evaluation. However, NICE is only currently anticipated to conduct approximately 3 HST evaluations per year, which will restrict the opportunity to a few products. Early engagement with NICE and horizon scanning centres is recommended to ensure suitable products are considered for HST evaluation and that clinical development is aligned with such criteria.