OBJECTIVES : The Early Access to Medicines Scheme (EAMS) gives patients with life-threatening or seriously debilitating conditions early access to new medicines prior to marketing authorisation, with the potential for real world data collection to support market access. To date, NICE appraisals of EAMS medicines have not featured EAMS data. This analysis assessed the potential benefit of EAMS data to NICE Health Technology Assessment.

METHODS : Published NICE guidance on EAMS medicines was reviewed to assess whether data collected during the EAMS period would have made a difference to NICE decisions, where these were not recommended, optimised (restricted in a way not mandated by the marketing authorisation) or recommended in the Cancer Drugs Fund (CDF; because more clinical evidence is needed to prove cost-effectiveness).

RESULTS : Of the EAMS medicines appraised by NICE (n=16), 5 were recommended; 1 was not recommended because of structural issues with the model resulting in no robust cost-effectiveness estimate; 5 had optimised recommendations either to reflect how the medicine would be used in NHS clinical practice or to include a stopping rule because long-term data on treatment duration was lacking; and 5 were recommended in the CDF because of limited data on long-term survival and treatment duration. It is unlikely that EAMS data would have made a fundamental difference to the recommendations because treatment during EAMS usually reflects NHS clinical practice and it is not feasible for long-term data addressing uncertainties about treatment duration and survival to be collected during the EAMS period.

CONCLUSIONS : It appears that EAMS data collection is unlikely to have a direct impact on overall NICE committee recommendations. However, it could provide supplementary evidence to reduce uncertainty and enable more accurate estimates of cost-effectiveness for decision making, for example by validating trial findings and modelling assumptions, or providing new data to supplement trial results.