Review of Health Technology Assessment Decisions Using Single-Arm Trial Data by the Danish Medicines Council

OBJECTIVES: For rare diseases or indications with no existing treatments, clinical efficacy in health technology assessment (HTA) may be based on single-arm trials (SAT). This study analyzed the Danish Medicine Council’s (DMC) HTA-assessments using SAT data as basis for clinical efficacy.

METHODS: HTA-decision reports in Denmark including SAT data as the main source of efficacy were identified through the NMAi database using a keyword search. A time period of nine years was reviewed (2015–2023) to capture any change connected to the introduction of quality-adjusted life years (QALYs) in 2021. Reports were excluded if the main source of efficacy was not a SAT. Data extracted were: decision date, indication assessed, orphan drug status, recommendation status and type of economic analysis.

RESULTS: 31 assessments were identified using SAT data as the main source of efficacy. The indications included oncology, coagulation disorders, genetic disease and ophthalmologic disease.

22 assessments were using an incremental cost per patient and added clinical value approach with a majority of the indications (15) being approved, with clinical added value ranging from important, small, non-estimable to no clinical added value. Incremental cost per patient ranging from cost savings to approximately 2.4M DKK. Negative decisions were associated with non-estimable clinical value and high incremental cost per patient (780,000 DKK to 13.5M DKK).

A cost-effectiveness approach was used in seven assessments, out of which two were cost minimization analyses. Only one indication, based on a cost minimization analysis was approved.

CONCLUSIONS: After the introduction of the QALY process, the DMC did not approve any indications with SATs using a cost-utility analysis. In the prior incremental cost per patient and added clinical value analysis DMC approved the majority of indications regardless of the estimated clinical value, basing the decision mainly on the incremental cost per patient.