OBJECTIVES: HST submissions are often for ultra-orphan diseases with limited trial data available. Therefore, they may feature managed access agreements (MAA), which necessitate a reassessment of the original recommendation upon the publication of more evidence. The objective of this study was to explore the differences between timelines for submissions and resubmissions for NICE highly specialised technology (HST) assessments.

METHODS: Of the 29 NICE HST assessments, six were eligible, including one submission and one resubmission for three products. These were asfotase alfa, ataluren and onasemnogene abeparvovec. Original timelines, from invitation to submit to posting of the final appraisal document (FAD), were compared with the resubmission timelines. Reasons for resubmissions were investigated.

RESULTS: Resubmission timelines were shorter for asfotase alfa (722 days fewer) and onasemnogene abeparvovec (449 days fewer) than original submission timeframes. Fewer committee meetings were also required for these drugs: 5 versus 2 meetings for asfotase alfa and 3 versus 1 for onasemnogene abeparvovec. Ataluren had the same number of committee meetings and similar timelines for the original submission versus the resubmission, with the resubmission being only eight days shorter. Resubmissions were primarily linked to new data becoming available. Asfotase alfa had data collected as part of a MAA in the original submission. Ataluren also had a MAA with new data requirements, as well as real-world evidence collected outside clinical trials. For onasemnogene abeparvovec, clinical evidence for presymptomatic spinal muscular dystrophy led to a change in population: the original population only included Type 1 spinal muscular atrophy.

CONCLUSIONS: The introduction of MAAs require a resubmission upon the publication of more evidence. This was the case for the three resubmissions assessed; with increased data availability, all products achieved successful recommendations, with one submission targeting a wider patient population. Resubmission timelines were either comparable to or shorter than original submission timelines.