Prevalence and direct costs of patients with neuromyelitis optica: data from Campania region in southern Italy

Aim: The study aimed to estimate the prevalence and direct costs of neuromyelitis optica (NMO) patients in Campania, Italy. Materials & methods: We retrospectively evaluated 53 NMO patients (mean age: 50.9 ± 16.5 years; 34% men) from the Campania Region administrative databases identified through disease exemption codes in 2018 and analyzed the incidence of NMO among the Campania region population and the disease-related cost. Results: The prevalence of NMO was 0.91 per 100,000 individuals. The average regional cost per NMO patient was 10,836.2 euros. The highest cost was related to drugs (60.6%), followed by hospitalizations (32.7%), diagnostics (4.8%) and laboratory tests (1.0%). Conclusion: NMO is an extremely rare disease with an annual disease-related cost of 0.005% of public health expenditure.

Fluoroscopic and Endoscopic Gastrostomy Tube Placement: Regional Variation and Their Prevalence

Objective This study aims to assess the regional variation and overall longitudinal prevalence of approaches to gastrostomy tube placement in patients covered by Medicare or Medicaid. Background Gastrostomy tubes are most commonly inserted endoscopically given the approaches’ demonstrated safety, success, and patient outcomes as compared with laparoscopic approaches. Recently, the growth of interventional radiology services has provided patients with an alternative percutaneous approach. The safety and efficacy of this approach as opposed to endoscopic approaches has yet to be determined. Methods From 2005 to 2014, Medicare Standard Analytic Files derived from Medicare parts A and B, which contain 100% of inpatient and outpatient facility records billed to Medicare, were retrospectively analyzed. Age, sex, year of placement, region, comparative quarterly ratio, regional cost variation, and overall financial cost were compared between both cohorts. Results Our population included a total of 336,021 patients; of those, 30,327 patients underwent fluoroscopic guided procedures, and 305,694 patients underwent endoscopic procedures. Age (p < 0.001), region (p = 0.043), and year of placement (p < 0.001) varied significantly between these populations. Fluoroscopic-guided procedures were found to have a statistically significantly lower average cost of treatment compared with endoscopic gastrostomies ($2,018.62 vs. $2,471.33, respectively, p = 0.03). Conclusion This study demonstrates an increasing prevalence of fluoroscopically placed gastrostomy tubes as compared with those placed endoscopically.

Globalization of clinical trials: Variation in estimated regional costs of pivotal trials, 2015–2016

Background/Aims Despite the increasing globalization of clinical trials, little is known regarding how the trial site costs vary around the world. We quantified the geographical distribution and regional cost differences for the clinical trials that established the benefits for new therapeutic drugs approved by the US Food and Drug Administration in 2015 and 2016. Methods We included all pivotal clinical trials for 59 new molecular entities approved by the US Food and Drug Administration in 2015 and 2016 that included at least one site in North America. We derived cost estimates from IQVIA’s CostPro, a global clinical trial cost-estimating tool used by pharmaceutical sponsors. We assessed the patient and site allocation of these trials across eight geographic regions. To quantify the region-specific cost differences, we conducted a within-trial comparison by expressing the estimated regional costs associated with the sites in each global region as a percent of the same costs in North America. We also estimated the percentage breakdown of regional cost components (pass-through, site management, regulatory, and study conduct costs) for each trial and for all endpoints reported the median and interquartile range. Results Overall, 127 pivotal clinical trials enrolled 91,415 patients from 13,264 sites. Most patients (60.3%) and sites (57.3%) were outside North America. A median of 66% of the total estimated trial costs (interquartile range: 60%–72%) were spent on regional tasks, with the largest share (53.3%) going directly to trial sites and the remainder going to other regional trial management tasks. Differences were greatest in four lower cost regions: Africa, with an estimated regional cost per site of 49% of North America (interquartile range: 44%–56%), Central Europe 50% (interquartile range: 41%–63%), Middle East 53% (interquartile range: 42%–64%) and Latin America 59% (interquartile range: 50%–70%). Overall, 90 (71%) of the 127 pivotal trials had a total of 3160 sites in these lower cost regions. In contrast, savings were more limited in Western Europe, Oceania, and Asia, where estimated regional costs were 78% of North America (interquartile range: 67%–89%). One-quarter of the trials with sites in Asia and Oceana did not achieve cost savings in those regions relative to North America. Conclusion Among this sample of pivotal trials for recently approved US Food and Drug Administration products, most patients and sites enrolled were outside of North America, with selection of regional sites having a significant impact on total trial costs.