OBJECTIVES

The Danish Medicines Council was established in 2017 in order to enable the timely diffusion of new medicines across hospitals in Denmark. The council in collaboration with Amgros evaluates hospital drugs based on a methodology that incorporates assessments of clinical added value using the Grading of Recommendations Assessment, Development and Evaluation system. Clinical added value may range from significant to no and non-documented categories. Due to accelerated regulatory approval, compounds with limited clinical trial scope but high unmet need may be assessed as of non-documented clinical added value yet still receive a positive recommendation. The aim of this study was to determine the role of clinical added value in council decision-making and the granting of recommendations.

METHODS

A review of Council assessment decisions between 2017-2019 was undertaken. Categorization of clinical added value, disease severity, incremental costs, budget impact, final recommendation and decision on reimbursement were analyzed.

RESULTS

The council assessed a total of 67 assessment reports covering different therapeutic areas. Hematology and oncology comprised 62% of reports followed by neurology and neuromuscular disorders (10.5%), dermatology (7.5%) and rheumatology (6%). Six applications were assessed as of non-documented clinical added value, whereof two were approved. 13 consisted of combined non-documented and low or no clinical value-added assessments, reflecting variable treatment line indications and patient subpopulations. Of these, eight were granted a partial recommendation and two were not recommended.

CONCLUSIONS

The Medicines Council process attempts to synthesize considerations of clinical efficacy using GRADE, cost-effectiveness and budgetary sustainability. While important in informing council decision-making, GRADE may not be fit for purpose in situations where clinical value is non-documented.