OBJECTIVES: After-market authorization, receiving positive recommendations from the respective Health Technology Assessment (HTA) bodies is necessary for achieving access to new drugs. Approximately, more than 40% of the new drugs approved by European Medicines Agency (EMA) are not recommended by National Institute for Health and Care Excellence (NICE). Understanding the factors influencing NICE's decisions is crucial for ensuring access to innovative and effective treatments. To understand the reasons for negative reimbursement decisions, we undertook a review of NICE decisions.

METHODS: Hand searching of NICE website was undertaken to retrieve the HTA submissions receiving negative recommendations in the last 5 years (2019-2023).

RESULTS: Search identified a total of 25 technology appraisals [24 Single Technology Appraisal (STA) and 1 Highly Specialized Technologies (HST)] that received negative recommendations from NICE. Among these, majority of the non-recommendations were in oncology (11/25) followed by blood and immune system conditions (4/25), (2/25) each in cardiovascular, respiratory, and neurological conditions and (1/25) each in infections, metabolic, neurodevelopmental, and digestive tract conditions. Reasons for negative recommendations were uncertain long-term benefits/efficacy measure (11/25), absence of direct comparison with relevant comparators (8/25), lack of meaningful improvement in health-related quality-of-life assessments (3/25), inadequate sample size (3/25), unclear method used for indirect comparisons (2/25), trial results not being generalizable to the National Health Service (NHS) (1/25) and failure to meet NICE's criteria for consideration as a life-extending treatment at the end of life (1/25). Additionally, all the 25 HTA reported that the cost-effectiveness estimates were uncertain and higher than what NICE normally considers an acceptable use of NHS resources.

CONCLUSIONS: Analysis of NICE's decisions indicated that unavailability of robust data establishing long-term efficacy, lack of direct comparisons, and high cost were the common reasons for receiving a negative decision. Addressing these factors is crucial for improving patient access to effective treatments and ensuring equitable healthcare outcomes.