Milana Krstin

Background: Medication adherence is one of the critical factors in optimizing treatment outcomes for chronic diseases such as atopic dermatitis (AD). Existing studies use aggregate data, but there is a need for assessment of medication adherence phases, such as the initiation and discontinuation of therapy. The aim of this study was to assess medication adherence across patients with moderate to severe AD, investigating the impact of drug treatment characteristics, particularly the route of administration, on adherence levels during treatment. Methods: A retrospective observational study on an Italian sample included 821 newly diagnosed AD patients from January 2021 to June 2022. Medication adherence was evaluated by EMERGE guidelines, focusing on initiation and discontinuation. Discontinuation was assessed at 6 and 12 months, comprising sensitivity analysis. Statistical analysis included chi-square tests and descriptive statistics on treatment duration. Results: Treatment initiation is significantly lower for tacrolimus ointment (38% non-initiation) than for dupilumab injection (12% non-initiation), due to initial healthcare support for dupilumab patients. After six months, 75.6% of dupilumab injection patients remained on therapy, while 24.4% of patients continued tacrolimus ointment treatment. After one year, therapy persistence was 68.7% among users of dupilumab, while only 22.5% of patients remained on tacrolimus therapy. Dupilumab demonstrated a significantly longer median treatment duration compared to tacrolimus (4.4 vs. 2.6 months; p < 0.01). Conclusions: The observed differences in adherence patterns between topical tacrolimus and subcutaneous dupilumab suggest that distinct contextual and behavioral factors influence patient adherence during therapy.

Background and aim: Advanced Therapy Medicinal Products (ATMPs) are innovative drugs based on genes, tissues, or cells that target rare and severe diseases. ATMPs have shown promising clinical outcomes but are associated with high costs, raising questions about cost-effectiveness. Hence, this systematic review aims to analyze the cost-effectiveness and cost-utility profiles of the European Medicines Agency-authorized ATMPs for treating rare diseases. Methods: A systematic review was conducted following PRISMA guidelines. Studies were identified by searching PubMed, Embase, Web of Science, and ProQuest scientific databases. Economic evaluations reporting incremental cost-effectiveness/utility ratios (ICERs/ICURs) for ATMPs were included. Costs were standardized to 2023 Euros, and a cost-effectiveness plane was constructed to evaluate the results against willingness-to-pay (WTP) thresholds of EUR 50,000, EUR 100,000, and EUR 150,000 per QALY, as part of a sensitivity analysis. Results: A total of 61 studies met the inclusion criteria. ATMPs for rare blood diseases, such as tisagenlecleucel and axicabtagene ciloleucel, were found to be cost-effective in a majority of studies, with incremental QALYs ranging from 1.5 to 10 per patient over lifetime horizon. Tisagenlecleucel demonstrated a positive cost-effectiveness profile in the treatment of acute lymphoblastic leukemia (58%), while axicabtagene ciloleucel showed a positive profile in the treatment of diffuse large B-cell lymphoma (85%). Onasemnogene abeparvovec for spinal muscular atrophy (SMA) showed uncertain cost-effectiveness results, and voretigene neparvovec for retinal diseases was not cost-effective in 40% of studies, with incremental QALYs around 1.3 and high costs exceeding the WTP threshold set. Conclusions: ATMPs in treating rare diseases show promising economic potential, but cost-effectiveness varies across indications. Policymakers must balance innovation with system sustainability, using refined models and the long-term impact on patient outcomes.