OBJECTIVES: To determine the cost-utility of tofacitinib compared to adalimumab, in combination with methotrexate, for the treatment of patients with moderate to severe rheumatoid arthritis (RA) who had inadequate response or are intolerant to previous therapy with disease-modifying anti-rheumatic drugs in Portugal.

METHODS: A lifetime individual-patient simulation model was used to estimate costs, mortality, and quality of life based on patient’s disease severity (as measured by Health Assessment Questionnaire [HAQ]). Baseline characteristics and 6-months HAQ change were sourced from the tofacitinib clinical trial (ORAL Strategy). Medium-term response (6-36 months) was based on published literature. After 36 months it was assumed that the HAQ level was maintained. Patients discontinue treatment if there is inadequate response to therapy at 6 months, loss of response or serious adverse events (AE). AE rate was sourced from a meta-analysis of tofacitinib studies. Utility values by HAQ were estimated by a regression analysis based on data from the tofacitinib trials. Portuguese all-cause mortality was adjusted for the increased mortality associated with RA. Treatment sequence in clinical practice in Portugal was based on experts´ opinion. Resource consumption by HAQ level was based on an expert panel and on National DRG microdata. Unit costs were based on national legislation and an official drug cost database. A societal perspective was considered. Costs and consequences were discounted at 5%. Both deterministic and probabilistic sensitivity analyses were performed.

RESULTS: Compared to the adalimumab treatment sequence, the tofacitinib sequence was associated to average savings of € 15.881 per patient, and to an incremental 0.07 QALY. Cost differences were due mainly to the acquisition of drugs.

CONCLUSIONS: Tofacitinib in the treatment of RA is a less expensive and more effective option than adalimumab. It is a dominant treatment strategy in the Portuguese setting.