Examining the Impact of Eams on NICE Recommendations over the Past Four Years

OBJECTIVES: Analysis of EAMS products and their NICE appraisal outcomes over the past four years.

METHODS: Analyze all products where the Early Access to Medicines Scheme (EAMS) scientific opinion (SO) expired within the period of January 2020 to December 2023 and the corresponding NICE recommendation, if available.

RESULTS: Of the 25 EAMS products, the average EAMS period was 8.7 months. This is twice as long as the average for EAMS products between 2015 to 2019 (average: 3.8 months). Among the 19 EAMS products that underwent a single technology (STA) or highly specialized technology (HST) appraisal by the National Institute for Health and Care Excellence (NICE), approximately 58% (11) received a positive recommendation, while 32% (6) were optimized, and 11% (2) were not recommended. In contrast, of the 232 non-EAMS products appraised by NICE from 2020 to 2023, 43% (99) were positive, 48% (111) were optimized, and 9% (22) were not recommended. Most of the evidence collected during EAMS and considered in the NICE appraisal was qualitative in nature, such as highlighting the unmet need and positioning of the product. The qualitative data collected during EAMS and considered in the NICE appraisal included safety data, infusion time, and shorter diagnosis periods.

CONCLUSIONS: This analysis reveals that EAMS products have a higher probability of receiving a positive NICE recommendation (58% vs 43%) than products appraised via standard pathways. Evidence collected during EAMS can support a product's value proposition, even if the data are only qualitative in nature. Consequently, when preparing for EAMS, it is crucial for manufacturers to thoroughly assess the data that can be collected to address uncertainties. While the duration of EAMS has doubled over the past four years, it remains uncertain whether this is a broader trend or if it was influenced by COVID-19-related delays.