Journal of comparative effectiveness research最新文献

Recent developments in digital infrastructure, advanced analytical approaches, and regulatory settings have facilitated the broadened use of real-world evidence (RWE) in population health management and evaluation of novel health technologies. RWE has uniquely contributed to improving human health by addressing unmet clinical needs, from assessing the external validity of clinical trial data to discovery of new disease phenotypes. In this perspective, we present exemplars across various health areas that have been impacted by real-world data and RWE, and we provide insights into further opportunities afforded by RWE. By deploying robust methodologies and transparently reporting caveats and limitations, real-world data accessed via secure data environments can support proactive healthcare management and accelerate access to novel interventions in England.

Aim: To evaluate the clinical characteristics and healthcare resource utilization for acute care and its costs for patients with primary biliary cholangitis (PBC) with or without cirrhosis. Materials & methods: This retrospective observational cohort study was conducted using two datasets (Komodo's Healthcare Map™ [Komodo Health] and Optum Clinformatics^® Data Mart [CDM] database) between 2015 and 2023. Patients (≥18 years) with PBC were identified based on ≥1 inpatient or ≥2 outpatient claims. Healthcare resource utilization for acute care (hospitalizations and emergency department [ED] visits [not leading to hospitalization]) were assessed in both datasets, and associated medical costs were evaluated in Optum CDM. Results: In Komodo Health, of the 29,758 patients with PBC (mean age: 59.2 years), 21.6% had cirrhosis and 50.4% of patients with cirrhosis had Medicaid or Medicare coverage. Of the total 8143 patients in Optum CDM (mean age: 67.0 years), 20.7% had cirrhosis, and most were enrolled in Medicare (69.7%). There was a larger proportion of men in the cirrhosis group compared with the no-cirrhosis group in Komodo Health (31.7 vs 16.3%) and Optum CDM (29.7 vs 16.5%). Annually, among patients with cirrhosis who had a hospitalization, 69.3% had additional hospitalizations, and among patients who had an ED visit, 52.9% had additional ED visits in Komodo Health; similar results were observed in Optum CDM. Among patients with at least one acute-care event, the mean annual acute-care costs with and without cirrhosis were $113,568 and $47,436, respectively. Conclusion: Data from two large healthcare claims databases showed that the majority of patients who had at least one acute-care event experienced additional acute-care events, particularly among those with cirrhosis. Timely treatment to avoid hospitalization and disease progression may help mitigate the clinical and economic burden for patients with PBC.

In this update, we discuss an article covering the implementation challenges of the upcoming European Union Health Technology Assessment regulation, particularly focusing on the complexity of population, intervention, comparator and outcomes requirements across member states; a user guide to applying generalized cost-effectiveness analysis for broader value assessment and finally highlight an ongoing debate surrounding National Institute for Health and Care Excellence's severity modifier implementation.

Aim: According to the results of the MASTRO I living systematic review and meta-analysis, use of the EmboTrap Revascularization^® Device in the treatment of acute ischemic stroke (AIS) results in higher rates of good functional outcomes (90-day modified Rankin Scale [mRS] 0-2) compared with use of the Trevo^® Retriever or the Solitaire™ Revascularization Device. The aim of this analysis was to assess the potential economic impact of achieving improved functional outcomes for three commonly used stent retrievers (SRs) in the treatment of AIS. Methods: An economic model with short-term and long-term costs, representing a healthcare system perspective was developed using a decision tree to simulate a cohort of 1000 hypothetical patients treated for AIS with mechanical thrombectomy (MT) using EmboTrap, Trevo or Solitaire SRs. Based on the proportion of patients who achieved a 90-day mRS score of 0-2 or 3-5 for each device reported in MASTRO I (excluding patients not surviving after 90 days), this model estimated per-patient costs and the associated incremental cost savings. Results are reported from the healthcare system perspective in the US, Canada, the UK, Sweden, Germany, France, Italy, Spain, Belgium and The Netherlands. Results: Across all ten countries, the use of EmboTrap during MT was associated with the lowest short-term (ranging from €8412 in Italy to $66,525 in the US), long-term (ranging from €5249 in Italy to $25,757 in the US) and total (ranging from €13,661 in Italy to $92,282 in the US) per-patient costs. The total per-patient cost was higher with Trevo (ranging from €14,601 in Italy to $97,487 in the US) and Solitaire (ranging from €14,840 in Italy to $98,814 in the US). Cost savings were highest when comparing EmboTrap versus Solitaire, followed by EmboTrap versus Trevo, with Trevo versus Solitaire having the smallest cost savings. Results of sensitivity and scenario analyses supported the robustness of the base-case results. Conclusion: Across the ten countries, treating patients with AIS with EmboTrap resulted in lower short-term, long-term and total costs to the payer. With rising healthcare costs and limited hospital budgets, these results suggest EmboTrap proves to be an evidence-based economical choice of SR for hospitals and healthcare systems.

Aim: Screening and monitoring of diabetes or dyslipidemia frequently involves a multi-step process requiring patients to obtain test requisitions from their primary care physician (PCP), followed by a laboratory visit and re-consultation. Point-of-care testing (POCT) for hemoglobin A_1c (HbA_1c) and lipid panel can streamline the patient care pathway. This study assessed the budget impact of introducing Afinion™ 2 POCT (Abbott Rapid Diagnostics) from the Canadian and Italian societal perspectives. Methods: Budget impact models were developed for Canada and Italy over a 5-year time horizon (2025 to 2029). The analyses considered the screening and monitoring of diabetes or dyslipidemia for patients utilizing the public healthcare system and attending primary care, and included direct costs (testing, consultations) and indirect costs (productivity loss, transportation) based on published sources. The budget impact (BI) was calculated by comparing scenarios with and without POCT. All costs were adjusted to Canadian dollars ($) or 2024 Euros (€). Scenario analyses were conducted to explore the impact of alternative assumptions. Results: The 5-year cumulative BI was -$758,006,692 (-$50,709,964 direct, -$707,296,728 indirect) for HbA_1c POCT and -$726,452,755 ($2,684,011 direct, -$729,136,766 indirect) for lipid panel POCT in Canada and -€1,380,658,764 (-€6,391,954 direct, -€1,374,266,809 indirect) for HbA_1c POCT and -€851,792,115 (€55,962,879 direct, -€907,754,993 indirect) for lipid panel POCT in Italy. In both countries, cost savings for both the healthcare payer and patients were observed for HbA_1c POCT, while costs savings were derived from patient indirect costs for lipid panel POCT. The analyses estimated that 1,558,062 and 1,501,260 PCP consultations in Canada, 4,962,338 and 1,951,026 PCP consultations in Italy were avoided with implementation of POCT for HbA_1c and lipid panel, respectively. Scenario analyses demonstrated potential further cost savings with implementation of POCT in pharmacies. Conclusion: This study demonstrates that the adoption of Afinion 2 POCT for HbA_1c and lipid panel can provide efficiencies to different types of healthcare systems through reducing PCP consultations, saving time and money for patients and providing cost savings for payers.

Aim: This study assessed the economic impact of reducing one postoperative visit following inflatable penile prosthesis (IPP) implantation. Methods: Scenario analyses were used to model the effects of eliminating one 30-min IPP postoperative visit from the expected 2.5 visits accounted for by the American Medical Association resource-based relative value scale data. The reduction was attributed to simplified teaching with a modified device. The recaptured time was applied to: the most frequent in-office CPT codes utilized by IPP implanters; evaluation and management of new ED patients pursuing/receiving IPPs; and in-office vasectomy. Physician work time and reimbursement were conservatively estimated using the 2024 Medicare Physician Fee Schedule and an alternative scenario where Advanced Practice Providers conducted IPP teaching was also modeled. Results: Annually, reducing one 30-min IPP postoperative visit for practices performing 25/50/100 IPP implants recaptured 750/1500/3000 min, respectively. This recaptured time translates into as much as $18,325 additional annual Medicare reimbursement. At 25 implants yearly, urologists could help an additional 13-25 patients with office visits and observe an additional $2049-$2270 reimbursement. At 50 implants yearly, office evaluation and counseling for 7 ED patients who progress to IPP implantation results in an additional $4125 reimbursement, excluding any diagnostic procedures and/or downstream surgical cases. At 100 implants yearly, recaptured schedule capacity can facilitate 37 in-office vasectomies, which translates to a $12,563 reimbursement. Conclusion: Achieving fewer IPP postoperative visits can optimize postoperative care and open schedule capacity that improves access to care for patients with urological needs.

Aim: To examine contributions of a patient advisory board (PAB) to the design and conduct of The Pulmonary Embolism Prevention after Hip and Knee Replacement (PEPPER) Trial (NCT02810704) and compare perceptions of PAB members and researchers on the Trial. Materials & methods This evaluation of the PAB was conducted by Clinical Coordinating Center (CCC) members who first discussed PAB contributions, leading to the design of a semi-structured WebEx interview individually querying PAB members on their experience. Two study team members analyzed transcriptions of the interviews for common themes, which were discussed and affirmed at an in-person meeting with PAB members. Results: The contribution most frequently cited as meaningful by PAB members was the creation of a recruitment video. In contrast, the research team considered the most impactful PAB recommendation to be omission of pneumatic compression boots as a study variable. PAB members spoke highly of their involvement in the trial and emphasized shared decision-making in the patient-physician relationship. Conclusion: Researchers and PAB members had different opinions about which PAB contributions were most impactful to the study. This likely derives from differences in perspective; PAB members focused on patient experience and the patient-surgeon relationship while researchers focused primarily on trial outcomes. PAB contributions led to two major protocol changes that had a substantial positive effect on trial design, recruitment and enrollment. This evaluation adds to the engagement literature, which contains little on what patients think of their involvement in the design and conduct of clinical research studies and will aid in encouraging treatment preference discussions between patient and surgeon, thereby supporting the goal of improved patient outcomes.

Aim: Anti-CD20 monoclonal antibodies and fumarates are common multiple sclerosis (MS) disease-modifying therapies (DMTs). Data on switching from anti-CD20s to other DMTs are limited. This retrospective, observational study of the US Komodo Health Sentinel claims database aimed to evaluate a de-escalation strategy in a real-world cohort, comparing clinical characteristics, relapses, healthcare encounters (HCEs) and healthcare costs (HCCs) between patients aged ≥18 years with stable MS who switched from anti-CD20s to fumarates ('Switchers') versus patients who stayed on anti-CD20s ('Stayers'). Materials & methods: Patients with MS (diagnosed 1 January 2015-31 August 2022) were propensity score matched 5:1 (Stayers:Switchers) and followed from index to end of study; end of insurance eligibility; >45-day gap in index DMT; or DMT switch. Primary outcomes were clinical characteristics and claims-based annualized relapse rate (ARR). Rates of HCEs and HCCs were estimated. Results: Baseline characteristics were well balanced between cohorts (Stayers, n = 540; Switchers, n = 108). Mean (SD) duration of post-index follow-up was 341.4 (250.0) days for both cohorts. Mean (SD) ARR was 0.08 (0.41; Stayers) versus 0.14 (0.5; Switchers; p = 0.3). Twenty-one Stayers (3.9%) and 1 Switcher (0.9%) were hospitalized for infections, with mean stays of 9.9 and 1 day, respectively. Mean annualized all-cause HCEs were similar between cohorts; annualized inpatient infection-related HCEs were higher for Stayers versus Switchers (mean difference: -0.05; p = 0.005). Annualized all-cause HCCs were similar between cohorts; Switchers had lower annualized infection-related HCCs overall (mean difference: -$2412; p = 0.002) and in the inpatient setting (mean difference: -$2325; p = 0.002). Conclusion: After 1 year, no significant differences in ARR emerged between cohorts. Switchers experienced lower inpatient infection-related HCEs, shorter inpatient infection-related hospital stays and lower overall infection-related HCCs.