Background Following approval of fremanezumab for the prevention of migraine in adults, health care decision makers are interested in understanding real-world clinical characteristics and treatment patterns among patients initiating fremanezumab therapy. Methods Data were obtained for this retrospective (pre-post) study from the Veradigm Health Insights database. The study period was January 1, 2014, to June 30, 2019. Patients were included if they were aged ≥ 18 years; had ≥ 1 migraine diagnosis during the study period; and had a medication record for fremanezumab on or after diagnosis during the identification period (September 1, 2018–December 31, 2018). Treatment patterns, including adherence, persistence, and utilization of acute and preventive migraine medication prescriptions, were evaluated. Results Of 987 patients initiating fremanezumab during the study period, 738 (74.8%) were adherent to fremanezumab by proportion of days covered (PDC; ≥ 80%) and 780 (79.0%) were adherent by medication possession ratio (MPR; ≥ 80%). A total of 746 (75.6%) patients were persistent for ≥ 6 months. Quarterly fremanezumab (n = 186) was associated with higher rates of adherence versus monthly fremanezumab (n = 801) by PDC (quarterly, 91.3%; monthly, 84.9%; P < 0.001) and MPR (quarterly, 92.2%; monthly, 87.9%; P = 0.006) and higher persistence at ≥ 6 months (quarterly, 82.8%; monthly, 73.9%; P = 0.011). After fremanezumab initiation, patients who were persistent for ≥ 6 months experienced significant reductions from baseline in the mean monthly number of acute and preventive migraine medication prescriptions (P < 0.001). Subgroup analyses in patients with comorbid depression and anxiety showed meaningful real-world benefits based on significant reductions in the number of patients who were prescribed antidepressants (baseline, 68.6%; follow-up, 56.4%; P = 0.0025) and anxiolytic medications (baseline, 55.0%; follow-up, 47.2%; P = 0.037), respectively. In a subgroup of patients with comorbid hypertension at baseline, fremanezumab treatment resulted in nonsignificant reductions in blood pressure. Conclusions Overall, adherence and persistence to fremanezumab in this real-world study was high in patients with migraine, with higher rates observed for quarterly fremanezumab. Patients who were persistent for ≥ 6 months experienced significant reductions in acute and preventive migraine medication use, while a subgroup of migraine patients with comorbid depression and anxiety at baseline showed significant reductions in antidepressant and anxiolytic medication use.

Background Perianal fistula (PAF), a complication of Crohn’s disease (CD), is associated with substantial economic costs and poor prognosis. We determined prevalence of PAF CD in the United States and compared costs and health care resource utilization (HRU) of PAF CD patients with matched non-PAF CD patients. Methods This was a retrospective cohort study of claims data from the IBM MarketScan Commercial Database from October 1, 2015, to September 30, 2018. Eligible patients were aged 18 to 89 years with ≥2 CD diagnoses. Patients with PAF CD had ≥1 PAF diagnosis or procedure code and were matched with non-PAF CD patients. Cumulative prevalence of PAF CD in the US population was calculated across total patients in MarketScan. All-cause and gastrointestinal (GI)-related costs and HRU were compared between groups using a generalized linear model (GLM). Results Cumulative 3-year prevalence of PAF was 7.70% of patients with CD (N = 81,862) and 0.01% of the US population. Among PAF CD (n = 1218) and matched non-PAF CD (n = 4095) patients, most all-cause costs and HRU were GI-related. Mean total all-cause and GI-related health care costs per patient and per year for PAF CD were $85,233 and $71,612, respectively, vs $40,526 and $29,458 for non-PAF CD (P &lt; .0001). Among PAF CD vs non-PAF CD patients, GLM-adjusted proportions of patients with GI-related inpatient, outpatient, or pharmacy visits, mean GI-related inpatient length of stay, and mean GI-related surgeries were higher (P &lt; .0001 for all comparisons). Conclusions Costs and HRU are significantly higher for patients with PAF CD vs non-PAF CD patients, highlighting the economic burden of the disease.

hospital stay, readmission rate within 30 days), and sepsis-related mortality. To evaluate the significance of an accelerated start of sepsis management and its impact on the outcome, two different scenarios were compared: Scenario A represents the current status of diagnoses used in managing septic patients. Scenario B adopts an accelerated diagnostic approach used to detect suspected sepsis earlier. The information regarding cost for diagnostics, daily cost of intensive care unit (ICU) and General ward (GW), and potential impact of early vs delayed treatment start in patients on LOS (-38%), hospitalization costs (-40%), and mortality (-28%) were derived from published sources. Results: By using faster test methods and enabling an earlier detection of sepsis in patients who otherwise would have received delayed treatment, the number of patients with an appropriate treatment can be increased. In a setting where 1,000 patients are entering scenario B rather than scenario A, the LOS are shortened by 390 days (210 days in ICU, 180 days in GW) and the overall cost are reduced by USD 750,000. The highest cost reduction can be observed in ICU and GW related costs. Conclusions: The increasing cost pressure and the need for efficient bed utilization in hospitals will continue. The present analysis shows that scientific and technological development, the willingness to intervene in detrimental development and to invest in technologies can support an affordable health care.

Cost-effectiveness was dependent on assumptions made about survival, costs, and utilities; and whether the motor function milestones were sustained over lifetime. Given the relatively short-term effectiveness data available for these therapies, a registry to collect long-term data of infantile-onset SMA patients is recommended.

Background Perianal fistula (PAF), a complication of Crohn’s disease (CD), is indicative of high disease severity and poor prognosis. We estimated the cumulative prevalence and treatment patterns of PAF CD in the USA. Methods In this retrospective study of IBM® MarketScan® Commercial and Medicare databases (conducted 1 October 2015 to 30 September 2018), patients (pts) were 18 to 89 years of age with at least two diagnoses of CD at least 30 days apart, and had continuous health plan enrolment for at least 12 months pre- and post-index date (first PAF diagnosis or procedure [PAF pts]). Non-PAF CD pts were assigned the same index date as matched PAF pts based on birth year, sex, presence/lack of CD diagnosis before index date, CD disease location and follow-up duration. Descriptive analysis was used for all variables. Treatment patterns and costs related to opioid use were compared among PAF pts. We also assessed four PAF pt cohorts with PAF-related surgery treated with one (cohort 1) or more than one (cohort 2) opioid within 7 days of index date or one (cohort 3) or more than one (cohort 4) opioid more than 7 days after index date. Results Cumulative prevalence of PAF CD (n = 81 862) was 7.7% (0.01% of the US population) over 3 years. The economic impact and treatment patterns were assessed in PAF (n = 1218; mean age 42 years; 52.4% men; 56.5% preferred provider organization [PPO] health plan) and matched non-PAF CD pts (n = 4095; mean age 43 years; 50.9% men; 57.6% PPO health plan). During follow-up, 65.8% of PAF and 42.3% of non-PAF pts were treated with at least one biologic agent. In the 30 days post-index, 31.9% of PAF pts were treated with biologics, with this percentage increasing over time; steroid use also remained high (Figure 1). Opioid treatment was associated with higher mean per patient per year (PPPY) total gastrointestinal (GI)-related costs for PAF pts (p &lt; 0.0001). Mean PPPY total GI-related costs for pts with PAF-related surgery and opioid treatment were $50 605, $53 984, $82 973 and $92 375 for cohorts 1, 2, 3 and 4, respectively (Figure 2). Generalized linear model-adjusted mean PPPY PAF-related surgeries were 7.2 versus 0 for PAF pts and non-PAF pts (p &lt; 0.0001), respectively. In the 30 days post-index date, 22.5% of PAF pts had minor surgeries and 20.0% had definitive surgeries. Conclusion Based on treatment guidelines as well as the study population’s use of inflammatory bowel disease medications and opioids, and higher rates of PAF-related surgeries, a need for better disease state management of patients with PAF CD is warranted. Sponsor: Takeda Pharmaceuticals USA, Inc.