National Institute of Health and Care Excellence (NICE) assessment of Highly Specialist Technologies (HST): what factors determine restricted reimbursement?

ISPOR Europe 2022 -- In 2013, NICE introduced the HST pathway, an alternative to the standard NICE evaluation aimed specifically at technologies for very rare conditions. The pathway considers a broader range of criteria about the benefits and costs than is the case with its appraisals of mainstream drugs and treatments. Since its introduction, the HST guidelines have been updated twice with the introduction of quality-adjusted life year (QALY) weighting in 2017 and updated routing criteria in 2022. All current guidance for technologies assessed under the HST pathway contains positive reimbursement recommendations. For some technologies, this is subject to population restrictions with reimbursement approved for a smaller population than market authorization and relatively complex commercial agreements, such as managed access agreements (MAA) or patient access schemes (PAS). This review was conducted to identify and understand factors that are associated with reimbursement restrictions.