Is the United Kingdom (UK) medicines pricing policy failing patients? The impact of terminated National Institute for Health and Care Excellence (NICE) appraisals for multi-indication products on patients

BackgroundNational Institute for Health and Care Excellence (NICE) data regarding manufacturer-driven terminations indicate that some patients in the United Kingdom (UK) are unable to access treatments that are available in other European countries, which may result in reduced survival and quality of life (QoL). This study aims to quantify the health impact of NICE appraisals for multi-indication products terminated for reasons not related to clinical trial failure on the UK population. MethodsTerminated NICE appraisals (2014-2023) for multi-indication products were identified and a targeted literature search was conducted to identify data on the health impact of the interventions. The potential incremental quality-adjusted life year (QALY) loss and impact on overall survival (OS), progression-free survival (PFS), and QoL was calculated. ResultsOver 16,000 QALYs/year were potentially lost (with one QALY equal to one year of life in perfect health) across approximately 829,000 patients in the UK due to NICE appraisals for multi-indication products being terminated for reasons not related to clinical trial failure. Across oncology indications (approximately 18,900 patients), OS and PFS may have been reduced by over 9,400 years and 9,000 years, respectively. The potential impact of the treatments for non-oncology indications for which NICE appraisals were terminated on QoL was an incremental improvement of 13% (weighted average). ConclusionsDue to the increasing number of NICE terminations for multi-indication products, patients cannot access therapies that could lengthen their lives and increase their QoL. As the UK uniform pricing policy is likely to influence manufacturer-driven terminations, introducing alternative reimbursement arrangements such as indication-based pricing (IBP) agreements to ensure that prices remain commensurate with therapeutic value could improve access to therapies in the UK, thereby improving public health. HighlightsO_LINational Institute for Health and Care Excellence (NICE) termination data indicate that some patients in the United Kingdom (UK) are unable to access treatments available in other European countries, which could potentially prolong their lives and improve their quality of life (QoL) C_LIO_LIAcross approximately 829,000 patients in the UK, over 16,000 quality-adjusted life years (QALYs) per year (with one QALY equal to one year of life in perfect health) are potentially lost as a result of NICE appraisals for multi-indication products that have been terminated for reasons not related to clinical trial failure C_LIO_LIAssessing reimbursement options such as indication-based pricing (IBP) agreements for treatments that would typically not meet NICEs cost-effectiveness criteria at the current price provides an opportunity to improve access to therapies in the UK, thereby improving public health C_LI