OBJECTIVES

: Health technology assessment (HTA) plays an important role in reimbursement decision-making in many countries, but recommendations vary widely throughout jurisdictions, even for the same drug. This variation may be due to differences in weighing of evidence or due to differences in processes or procedures, called HTA practices. This study aims to provide insight into the effects of differences in practices on interpretation of inter-country differences in HTA recommendations for conditionally approved drugs.

METHODS

HTA recommendations for conditionally approved drugs (N=27) up until June 2017 from England/Wales, France, Germany, Netherlands and Scotland were included. Recommendations and practice characteristics were extracted from these five jurisdictions and this data was validated. The effect of non-submissions, resubmissions and reassessments, cost-effectiveness assessments and price negotiations on changes in the percentage of negative recommendations and interpretation of inter-country differences in HTA outcomes were analyzed with Fisher exact tests.

RESULTS

: The inclusion of cost-effectiveness assessments led to significant increases in proportion of negative recommendations within England/Wales (from 4% to 50%, p<0.01) and Scotland (from 21% to 71%, p<0.01). The subsequent inclusion of price negotiations led to significant reductions in the proportion of negative recommendations in England/Wales (from 50% to 14%, p<0.01), France (from 31% to 3%, p=0.012), and Germany (from 34% to 0%, p<0.01). Results indicated that the inclusion of non- and resubmissions might impact Scottish negative HTA recommendations (from 7% to 21%), but this effect was not significant. No significant effects were observed in The Netherlands, possibly due to sample size.

CONCLUSIONS

: Variations in HTA practices between international jurisdictions can have a substantial and significant impact on conclusions about recommendations by HTA bodies, as exemplified in this cohort of conditionally approved products. Studies comparing international HTA recommendations should carefully consider possible practice variations between jurisdictions.