PGI16 A Structured Literature Review of Modelling Approaches for Steroid Use and Treatment Stopping Rules in Ulcerative Colitis for Economic Evaluation

Abstract

To review the evidence on systemic corticosteroid use and treatment stopping rules from literature and health technology assessment (HTA) critiques, and to assess the impact on health economic modelling in ulcerative colitis (UC). Electronic databases and citation searching were used to identify studies and critiques written in English, reporting steroid use and treatment stopping rules, without time constraints. Data extraction was performed by one reviewer and checked by a second reviewer. Methodological quality of included studies was assessed by both reviewers. This study identified and screened 66 studies reporting information on steroid use and treatment stopping rules, with 61 studies meeting the criteria for inclusion. Forty studies recognized patient quality of life implications associated with long-term steroid usage, however only 3 modelled the impact of steroid reduction or steroid-free remission on cost-effectiveness. One study modelled linear reduction in steroid use over 54-weeks in the intervention arm, with patients assumed as steroid free in future cycles if classified as steroid free at week 54. Stopping rules were more common in and primarily based on patient’s non-response to treatment during induction phases, loss of response during maintenance phases, and discontinuation due to adverse events, accounting for 53 studies. No studies modelled dynamic treatment posology despite NICE requesting functionality related to remission-based temporary discontinuations within economic models to better reflect real-world clinical practice. In addition to applying stopping rules for treatment effectiveness and associated adverse events in UC, it could be useful to include remission-based discontinuation within the models. There exists a need for modelling the steroid-sparing effects of therapies. HTAs may encourage such UC models to better reflect clinical practice.