OBJECTIVES:

Early scientific advice (ESA) can assist with the development of the evidence package to support health technology assessment (HTA) submissions. We conducted a study to understand if ESA might have improved outcomes in “cost-effectiveness” markets.

METHODS:

A review was conducted of negative appraisals in England (NICE), Scotland (SMC), Canada (CADTH), and Australia (PBAC) from 1^st January 2021 to 31^st May 2022. Technology appraisals for England, Scotland, Canada, and Australia were manually screened. For Australia, technologies that were “not recommended” and that were “new PBS listings” were selected for screening; outcomes from re-submissions overrode the respective original appraisals.

Appraisals were assessed to determine whether ESA could have improved the outcome. Candidates for ESA were based on published appraisal feedback on trial design, generalisability to clinical practice, comparator, and cost-effectiveness model structure.

RESULTS:

Across the four groupings, 50 negative appraisals were identified in separate technologies/ indications. Nine negative appraisals were identified in England, 10 in Canada and 10 in Scotland, and 27 in Australia, with 43 published reports. Issues concerning cost-effectiveness (42/43; 98%), comparative-effectiveness (28/43; 65%), and trial design (20/43; 47%) were most commonly cited as drivers for negative appraisals. Comparative effectiveness and trial design were cited as drivers in most (80%) of the published negative appraisals in Canada. Trial design was also commonly cited as a driver of negative appraisals in Scotland (70%).

Almost half (21/43; 49%) of published submissions were considered as candidates for potentially benefitting from ESA. Of these submissions, trial design (76%), comparative effectiveness (62%), comparator (48%), and clinical value (48%) were cited as reasons for the negative appraisals.

CONCLUSIONS:

Negative appraisals were not uncommon and were mostly driven by cost and comparative-effectiveness and trial design concerns. Almost half of submissions that resulted in published negative appraisals were identified as potential candidates for benefitting from ESA.