Abstract
OBJECTIVE: We evaluated the effectiveness of tofacitinib (oral Janus kinase inhibitor) additional risk minimisation measures (aRMM) in Europe via prescriber surveys. Operational challenges/methodological limitations of surveys and recommendations for future considerations were summarised. METHODS: In this post-authorisation safety study, multimodal surveys were conducted in eight European countries from 2021 to 2022 among prescribers of tofacitinib for rheumatoid arthritis and/or psoriatic arthritis (RA/PsA) or ulcerative colitis (UC) in the 12 months preceding survey administration. Prescribers' awareness (receipt) of aRMM materials, knowledge of key risk messages and adherence to risk minimisation practices were assessed. A priori effectiveness thresholds (≥ ~80%) were set across outcomes. RESULTS: Of 18,764 and 12,777 prescribers invited to take the RA/PsA or UC survey, 164 and 81 completed the surveys, respectively (response proportion: 326/18,764, 1.7%; 154/12,777, 1.2%, respectively). Among completers, self-reported receipt of all initial and/or 2019 updated aRMM materials was 33.5% and 32.7% in the RA/PsA and UC surveys, respectively. In the RA/PsA and UC surveys, 39.5% and 24.5%, respectively, answered ≥ ~80% of knowledge questions correctly; 51.2% and 69.7%, respectively, provided desirable responses to ≥ ~80% of adherence questions. CONCLUSIONS: Although the tofacitinib aRMM materials did not achieve the desired effectiveness for awareness (receipt), knowledge or adherence to risk minimisation practices among survey completers, the study was deemed inconclusive given the low response rate. This demonstrated that unless an acceptable response rate is achieved, surveys should not be the only method for aRMM evaluation. Secondary data may supplement future surveys to increase their robustness. HMA-EMA CATALOGUES REGISTRATION: EUPAS43143 (date of registration: 29/09/2021).