OBJECTIVES: To investigate how ‘best treatment first’ prescribing policies instead of ‘cheapest treatment first’ policies impact patient outcomes and healthcare system sustainability through the Optimized Patient Treatment Initiative (OPT-In).

METHODS: A 3-year state-transition model was developed to estimate potential effectiveness improvements and budget reallocation possibilities associated with implementing optimal treatment sequences in 7 immune-mediated disorders: Crohn’s disease (CD), ulcerative colitis (UC), plaque psoriasis (PsO), psoriatic arthritis (PsA), rheumatoid arthritis (RA), ankylosing spondylitis (AS) and non-radiographic axial spondyloarthritis (NR-AxSpA). All treatments reimbursed in Italy with a European Medicines Agency marketing authorisation at the end of 2020 were included and used to identify all possible treatment sequences per disease; the efficacy of each sequence was estimated and ranked. Italian market share data from 2021 informed the current most prescribed sequences; these were compared against the sequence rankings to determine opportunities for improvement.

RESULTS: In PsO, 1,284 treatment sequences are possible with estimated average number of failures ranging from 0.58 to 2.44 over 3 years; the most prescribed sequences achieve 1.14 failures, suggesting possible improvements. If improvements were implemented, treatment failures could be reduced by up to 0.56 in PsO, 0.25 in UC, 0.16 in CD, 0.23 in PsA, 0.23 in RA, 0.12 in AS, and 0.10 in NR-AxSpA. If all patients in Italy were to receive at least the top 20% most efficacious sequences, budget changes vary between +12.9% in UC and -2.7% in RA.

CONCLUSIONS: The impact of a ‘best treatment first’ policy can result in improved patient outcomes across all diseases; if budget reallocation was possible, budget increases could be mitigated when considering all diseases. Results suggest that selecting the most efficacious treatment sequences provide the greatest opportunity to reduce treatment failures, maximise patient outcomes, and promote a sustainable healthcare system.