The very fact that multiple sclerosis (MS) is incurable and necessitates life-long care makes it one of the most burdensome illnesses. The aim of this study was to compare the cost-effectiveness of orally administered medications (e.g., fingolimod, dimethyl fumarate, and teriflunomide), interferon (IFN)-based therapy, and monoclonal antibodies (MABs) (e.g., natalizumab and rituximab) in the management of relapsing-remitting multiple sclerosis (RRMS) in Saudi Arabia using real-world data. This was a retrospective cohort study in which patients with RRMS aged ≥18 years without any other chronic health conditions with non-missing data for at least 12 months were recruited from the electronic health records of a university-affiliated tertiary care center. Multiple logistic regressions controlling for age, sex, and duration of therapy were conducted to examine the odds of disability progression, clinical relapse, MRI lesions, and composite outcome (e.g., relapse, lesion development on MRI, disability progression). The number of patients who met the inclusion criteria and were included in the analysis was 146. Most of the patients were female (70.51%) and young (e.g., ≤35 years of age). There were 40 patients on the orally administered agents (e.g., dimethyl fumarate, teriflunomide, fingolimod), 66 patients were on IFN-based therapy (e.g., Rebif®), and 40 patients were on monoclonal antibodies (e.g., rituximab and natalizumab). Patients on MABs had lower odds of the composite outcome (OR = 0.17 (95% CI: 0.068–0.428)). The use of orally administered agents was dominant (e.g., more effective and less costly), with average annual cost savings of USD −4336.65 (95% CI: −5207.89–−3903.32) and 8.11% higher rate of effectiveness (95% CI: −14.81–18.07) when compared with Rebif®. With regard to the use of MABs in comparison to Rebif®, MABs were associated with higher cost but a better rate of effectiveness, with an average additional annual cost of USD 1381.54 (95% CI: 421.31–3621.06) and 43.11% higher rate of effectiveness (95% CI: 30.38–61.15) when compared with Rebif®. In addition, the use of MABs was associated with higher cost but a better rate of effectiveness, with an average additional annual cost of USD 5717.88 (95% CI: 4970.75–8272.66) and 35% higher rate of effectiveness (95% CI: 10.0–42.50) when compared with orally administered agents. The use of MABs in the management of RRMS among the young patient population has shown to be the most effective therapy in comparison to both IFN-based therapy (e.g., Rebif®) and orally administered agents, but with higher cost. Orally administered agents resulted in better outcomes and lower costs in comparison to IFN-based therapy. Future studies should further examine the cost-effectiveness of different disease-modifying therapies for the management of RRMS using more robust study designs.

Background Vaso–occlusive crises (VOCs) are acute and common painful complication of sickle cell disease (SCD), and are the main reason behind the frequent emergency department visits among SCD patients. Hydroxyurea (HU) is an old and commonly used medication that demonstrated its effectiveness in reducing the risk of VOCs and the incidence of hospitalization. Although multiple studies have examined the impact of HU on the rates of VOCs, few have explored its effectiveness among SCD patients in Saudi Arabia. Methods This was a single–center retrospective cohort study in which the electronic medical records of patients with SCD who have not had any previous exposure to HU prior to the initiation of HU treatment for ≥12 months were recruited. Paired t–test was conducted to examine the difference in the rates of VOCs, and levels of hemoglobin (Hgb), hematocrit (HCT), and platelet counts (PLT Ct) prior to the initiation of HU therapy and 12 months later. Multiple linear regression was conducted to examine whether age, gender, use of opioid analgesics, Hgb, HCT, and PLT Ct levels predict higher or lower rates of VOCs. Results One hundred and fifty–six patients met the inclusion criteria and were included in the analysis. About 51% of the patients were males, and their mean age was 12.69 years. The mean HU dosage was 16.52 mg/kg/day, and the mean reduction in the rate of VOCs was 1.36 events per patient per year (95% CI [1.03–1.70], p < 0.0001) after the initiation of HU. Females were more likely to have greater reduction in the rates of VOCs in comparison to their male counterparts (β–estimate = 12.85, 95% CI [0.759–24.93], p = 0.0374). Conclusion The use of HU results in a significant reduction in the rates of VOCs and emergency department visits. Future studies with robust research designs should be conducted to further examine the impact of HU on VOCs, hospitalization, and length of stay as well as compare HU to other newly approved medications for SCD, such as crizanlizumab.