OBJECTIVES

: Under conditional approval (CA) and approval under exceptional circumstances (AEC), European Medicines Agency (EMA) can grant marketing authorizations (MA) based on less comprehensive data than normally required. This study assessed whether CA and AEC are associated with differences in the UK’s NICE appraisal process in terms of appraisal duration, outcome, and recommendations with Patient Access Scheme (PAS) or within Cancer Drug Fund (CDF).

METHODS

: We reviewed all technology appraisals (TA) and highly specialized technologies evaluations of pharmaceutical products, completed between 01/01/2001 and 01/09/2017, excluding those that were later updated, withdrawn, or terminated. The start date (date of final scope), end date (date of publication), recommendation decision, and PAS/CDF status were extracted from the publicly available NICE documents. Medicines with CA/AEC and the relevant indication were identified through the EMA’s website until December 2016. The average duration of appraisal, the proportion of recommended medicines, and the proportion of PAS/CDF were compared for drugs with and without CA/AEC.

RESULTS

: Of the 473 appraisals conducted by NICE, 231 TAs reviewed 308 medicines with full marketing authorization and 15 TAs focused on medicines with CA/AEC. The average appraisal duration was 609 days for technologies with full MA and 659 days for those with CA/AEC (p=0.616). 87.5% of technologies with full MA and 80% of technologies with CA/AEC were recommended or recommended with optimized use (p=0.397). 34.4% of technologies with full MA were recommended with a PAS or within the CDF, compared to 80% of those that were conditionally approved (p=0.0003).

CONCLUSIONS

: This study suggests that the lower amount of evidence available for medicines with CA or AEC may not add undue difficulty on NICE’s appraisal process. During our study period, manufacturers were considerably more likely to negotiate and agree price discounts and implement risk-sharing schemes in the NHS, likely reflecting the uncertainty in the available evidence base.