Journal of comparative effectiveness research最新文献

In this update we cover the US-UK Economic Prosperity Deal announced in December 2025, which includes NICE's first major cost-effectiveness threshold increase in over two decades. We also analyze the latest developments in US pharmaceutical pricing policy, including the second cycle of Inflation Reduction Act drug price negotiations and the implementation of the GENEROUS, GUARD and GLOBE Models for Most-Favored-Nation pricing.

Aim: To study the effect of infertility support education on treatment outcomes in women with unexplained infertility. Materials & methods: This quasi-experimental study with a pretest-posttest comparison group was conducted on women aged 19-45 years who were admitted to EO University Health, Practice and Research Hospital Department of Reproductive Endocrinology and Private D Health Hospital IVF Clinic with the diagnosis of unexplained infertility and decided to undergo in vitro fertilization (IVF)/intracytoplasmic sperm injection (ICSI) and were included in the treatment plan. A study group and a control group were formed by randomization method with at least 25 infertile women for each group. Data were collected using the Introductory questionnaire, Fertility Readiness Scale for women receiving fertility support, Healthy Lifestyle Behaviors Scale II and post-IVF/ICSI success evaluation form. The pretest scales were administered to the education and control groups at the first interview. Infertility support training was given to the education group in three sessions at 15-day intervals. Post-test data were obtained at the interview on the day of ovum pickup. Embryo transfer was performed in 25 infertile women in both groups, and human chorionic gonadotropic hormone evaluation was performed on day 12. Approximately 4 weeks later the presence of a fetal heartbeat was analyzed by reviewing the medical records, and the post-IVF/ICSI success evaluation form was completed. Results: Fetal heartbeat was detected in 15 women in the training group and only in 10 women in the control group, although the pregnancy rate increased after training and this difference was not statistically significant. Conclusion: Infertility support education has been found to have positive effects on fertility as well as general health.

Aim: This study examined the clinical outcomes of patients in the US with low/intermediate-risk localized prostate cancer (LIR-LPC) and high-risk localized prostate cancer (HR-LPC) who received radical prostatectomy (RP) as initial treatment. Materials & methods: This is a retrospective analysis of the SEER-Medicare database. Patients newly diagnosed with LPC at age of ≥65 years during 2012-2019 who underwent RP as initial definitive treatment and had continuous Medicare Fee-For-Service for ≥12 months prior to RP were included. Eligible patients were stratified into LIR-LPC and HR-LPC cohorts. Overall survival, metastatic free survival and time to advanced prostate cancer treatment (TTAT) were described and compared using the Kaplan-Meier method and Cox proportional-hazards model. Results: The LIR-LPC cohort (n = 4120) and the HR-LPC cohort (n = 5359) had comparable socio-demographic characteristics, with a mean age of approximately 70 years. Survival analysis showed that HR-LPC was associated with significantly shorter overall survival, metastatic free survival and TTAT than LIR-LPC (log rank p < 0.001). After adjusting for comprehensive socio-demographic and baseline clinical characteristics, patients with HR-LPC had an approximately 70% increased risk for all-cause death (hazard ratio [HR]: 1.72; confidence interval [CI]:1.39-2.12), 2.5-fold increased risk for metastasis or death (HR: 2.57; CI: 2.14-3.09), and ninefold increased risk for initiating advanced treatments (HR: 9.06; CI: 6.22-13.18) compared with patients with LIR-LPC. Conclusion: In patients with LPC who received RP as initial definitive treatment, high risk is strongly associated with suboptimal clinical outcomes. Novel therapeutic approaches are needed to enhance the management and improve the outcomes for this patient population.

Aim: Complications of primary biliary cholangitis (PBC) are proposed to confer substantial economic burden to patients and healthcare systems. This retrospective observational study evaluated the cost of PBC-related hepatic decompensation and liver transplantation using a large administrative claims database. Materials & methods: Patients aged ≥18 years at the time of first claim with a diagnosis of PBC were identified using Optum's de-identified Clinformatics^® Data Mart Database. Two cohorts were established based on the type of event (hepatic decompensation or liver transplantation) that patients experienced on or after the date of their first claim with the PBC diagnosis. Costs for the hepatic decompensation hospitalization and 30-day post-discharge period were examined at the event level. Hospitalizations occurring within the 30-day post-discharge period after a hepatic decompensation event were considered readmissions, and costs from the initial event were combined with those from the ensuing readmissions. In the liver transplantation cohort, costs for the pretransplant evaluation, hospitalization for transplantation, and post-transplant care and complications were assessed per patient. Results: A total of 2118 and 163 patients met study inclusion criteria in the hepatic decompensation and liver transplantation cohorts, respectively. The overall mean cost per hepatic decompensation event (n = 3581) was $63,612.09. The mean cost per event with readmission within 30 days (n = 991, 27.7%) was $116,424.25; for events without readmission, the mean cost was $43,404.81. The mean total cost of liver transplantation per patient was $328,336.60. The mean cost per patient was highest for the hospitalization for transplantation ($226,908.70). Conclusion: This comprehensive cost analysis demonstrates the high-cost burden of PBC disease progression. Appropriate patient management may help to mitigate the economic burden of PBC-related hepatic decompensation and liver transplantation.

Aim: To compare oral corticosteroid (OCS) burden and healthcare resource utilization (HCRU) in patients with chronic rhinosinusitis with nasal polyps undergoing functional endoscopic sinus surgery (FESS; intervention) versus not undergoing FESS. Materials & methods: Retrospective cohort study using US claims data (Optum's de-identified Clinformatics^® Data Mart Database; 2011-2021). Groups were propensity score (PS) matched to adjust for confounding. OCS burden (cumulative dose in mg prednisone equivalents) and HCRU were assessed during baseline (365 days pre-index), intervention (days 0-44), and follow-up (days 45-365); costs during intervention and follow-up. Results: Before PS-matching, both groups had substantial comorbidity burden (>50% allergic rhinitis; >25% asthma) and over half of patients had used OCS (65% [FESS] vs 52% [non-FESS]; p < 0.01). After PS-matching (n = 8909 per group), OCS cumulative dose during follow-up was 18% lower among FESS versus non-FESS patients (mean difference: -40 mg per patient [95% CI: -57, -23; p < 0.01]). Similar proportions of patients filled OCS prescriptions during follow-up (35% [FESS], 36% [non-FESS]) and in these patients, OCS burden remained high (mean [SD] cumulative dose 521 [786] vs 612 [906] mg, respectively). Mean total healthcare costs per patient during the intervention period were $28,832 (FESS) and $2537 (non-FESS), but similar during follow-up ($15,659 and $15,926, respectively). HCRU was similar in follow-up, except more FESS patients visited an otolaryngologist (57% vs 32%, p < 0.01). Conclusion: In US clinical practice, OCS burden in patients with chronic rhinosinusitis with nasal polyps was significantly lower but remained substantial following FESS, and HCRU and costs during follow-up were similar to matched patients without FESS.

In this update, we review a new bias appraisal tool, explore lessons from a trial emulation study, and describe the development of real-world evidence guidance in the Philippines.

Aim: Proteinuria poses a significant challenge in focal segmental glomerulosclerosis (FSGS), particularly when resistant to standard treatments. Acthar^® Gel, a Food and Drug Administration (FDA)-approved treatment, may be a potential option for proteinuria in nephrotic syndrome (NS) due to FSGS, particularly given the limited alternative therapies. This study assessed the cost-per-response of Acthar Gel versus standard of care (SoC) for the treatment of refractory proteinuria in NS due to FSGS among adults from a US healthcare payer perspective over a 1- to 3-year horizon. Materials & methods: A probabilistic, cohort-based state-transition model tracked adults with nephrotic-range proteinuria due to FSGS through clinically relevant health states in 6-month cycles. All patients entered in relapse and received either Acthar Gel or SoC. At each cycle, individuals could transition to response or remain uncontrolled, progress to renal failure, or continue in relapse; death was permitted from any state. Responders were allowed to either sustain response or experience relapse in subsequent cycles. Model inputs for clinical event rates, healthcare utilization and medical costs were sourced from the published literature, and drug costs were valued using wholesale acquisition cost. Cost-per-response was defined as total healthcare costs (drug and nondrug medical costs) per patient divided by the response rate. Results: Acthar Gel showed a lower cost-per-response ($469,735) versus cyclophosphamide ($2,140,400) and rituximab ($1,272,477) over 1 year. This advantage for Acthar Gel was sustained for 2 and 3 years. Acthar Gel was potentially a dominant treatment option at 2 and 3 years, with a lower overall cost of care and higher response rates than SoC. Conclusion: From a US healthcare payer perspective, Acthar Gel appears to be a cost-effective, value-based treatment option for adults with proteinuria in NS due to FSGS over 1 to 3 years. These findings may aid providers and payers in making informed treatment decisions when conventional therapies are ineffective for these patients.

Stroke prevalence is highest in adults ≥65 years, the majority of whom are Medicare beneficiaries. Fee-for-Service Medicare (FFS) incentivizes utilization by paying for each service. Medicare Advantage (MA) uses capitated payments to reduce overutilization. It is not clear if stroke patients with FFS or MA receive different stroke preventive care and whether those differences are associated with differences in post-acute care utilization, cost and clinical outcomes. We performed an empirical narrative review of published peer-reviewed studies in the PubMed, EMBASE and Web of Science databases comparing stroke preventive care between FFS and MA using the American Heart Association's Life's Essential 8 and American Heart Association/American Stroke Association national guidelines. We added atrial fibrillation (AF), post-acute care utilization and outcomes, including mortality. 7/1356 studies met inclusion criteria. Studies were heterogenous in their design and settings. There was limited availability of clinical data. Within those limitations, published studies suggest that MA appears to allow for guideline-directed stroke preventive care for hyperlipidemia, smoking cessation and AF in specific study populations. Post-acute care utilization was generally lower in MA. Functional outcomes improvements were similar but occurred in fewer days in MA, though the absence of acute stroke treatment data is notable. Mortality data were mixed. Given the importance of stroke in Medicare and the growth in MA enrollment, comparing the effectiveness of MA and FFS warrants further study among appropriately matched MA and FFS beneficiaries with stroke.

External comparator (EC) studies can improve identification of compounds with the most promising performance in pre-pivotal single-arm trials (SAT). As SAT output lacks comparative insights, EC studies allow efficient contextualization of compound performance in early phases of drug development. Improving insights from pre-pivotal SATs allows more informed prioritization of compounds and investment choices and enables faster and more substantiated development of the most promising molecules to the benefit of patients. The extensive digitization of healthcare information routinely generated during patient treatment journeys makes EC studies using real-world data an attractive alternative to other approaches (such as systematic literature review and repurposed trial data). Using oncology as an illustration, this paper presents how real-world data EC studies can address some of the fundamental challenges the pharmaceutical sector faces in developing novel therapies, and especially those pertaining to emerging biopharma companies.

Aim: Pulsed field ablation (PFA) is a minimally thermal alternative to traditional thermal catheter ablation for atrial fibrillation (AF), with comparable efficacy and minimal risk of collateral tissue injury. Three-dimensional (3D) electroanatomical mapping systems have been adopted with nonintegrated PFA catheters to improve precision and procedural efficiency. This study compared arrhythmia-related hospital readmissions and procedure-related complications among patients with AF treated with PFA, using either the CARTO™ 3 or EnSite™ electroanatomical mapping system. Materials & methods: Patients who underwent PFA for AF using nonintegrated pulsed field catheters with either CARTO 3 or EnSite were identified from the 2023-2024 Premier Healthcare Database (PHD). The primary outcome was 30-day inpatient readmission and emergency room (ER) visit for AF/atrial flutter (AFL)/atrial tachycardia following the index PFA procedure. Secondary outcomes included 30-day cardiovascular-related readmissions and procedure-related complications. Cohorts were balanced using inverse probability of treatment weighting. Descriptive analyses and a weighted Generalized Estimating Equation (GEE) model were used to assess differences in outcomes between groups. Results: A total of 2894 patients were treated using CARTO 3 and 2015 using EnSite. After weighting, patient and hospital characteristics were well balanced across cohorts. The CARTO 3 group was significantly less likely to experience AF/AFL/atrial tachycardia-related readmissions (0.3% vs 0.9%, chi-square p = 0.023; odds ratio [OR], 0.39; 95% CI, 0.17-0.90, GEE p = 0.028) and a composite outcome of AF/AFL/atrial tachycardia-related inpatient readmission or ER visits (0.5% vs 1.1%, chi-square p = 0.020; OR: 0.44, 95% CI: 0.21-0.89, GEE p = 0.023) within 30 days of PFA procedure than the EnSite group. No differences were observed in cardiovascular-related readmission or complications. Conclusion: In this retrospective, real-world study, patients with AF who underwent PFA using CARTO 3 had lower 30-day AF/AFL/atrial tachycardia-related readmissions rates than those treated with EnSite suggesting that choice of mapping system may influence early AF outcomes and could translate into reduced downstream hospitalizations and resource use.