A New Validated Approach for Identifying Childhood Immunizations in Electronic Health Records in the United Kingdom.

BACKGROUND: Routinely collected electronic health records (EHR) offer a valuable opportunity to carry out research on immunization uptake, effectiveness, and safety, using large and representative samples of the population. In contrast to other drugs, vaccines do not require electronic prescription in many settings, which may lead to ambiguous coding of vaccination status and timing. METHODOLOGY: We propose a comprehensive algorithm to identifying childhood immunizations in routinely collected EHR. In order to deal with ambiguous coding, over-recording, and backdating in EHR, we suggest an approach combining a wide range of medical codes in combination to identify vaccination events and using appropriate wash-out periods and quality checks. We illustrate this approach on a cohort of children born between 2006 and 2014 followed up to the age of five in the Clinical Practice Research Datalink (CPRD) Aurum, a UK primary care dataset of EHR, and validate the results against national estimates of vaccine coverage by NHS Digital and Public Health England. RESULTS: Our algorithm reproduced estimates of vaccination coverage, which are comparable to official national estimates and allows to approximate the age at vaccination. Electronic prescription data only do not cover vaccination events sufficiently. CONCLUSION: Our new proposed method could be used to provide a more accurate estimation of vaccination coverage and timing of vaccination for researchers and policymakers using EHR. As with all observational research using real-world data, it is important that researchers understand the context of the used dataset used and the clinical practice of recording.

Public value judgments about the criteria for reimbursement of medicines in South Korea.

OBJECTIVES: Although reimbursement decisions for new drugs involve value judgment, the value judgment of laypersons has not been thoroughly investigated. This study quantified the public value (PV) of the criteria and sub-criteria in the current drug reimbursement systems in South Korea and examined sociodemographic factors that associated with PV. METHODS: The Analytic Hierarchy Process (AHP) was used to quantify the PVs of criteria and sub-criteria. We developed a questionnaire to generate pairwise comparison matrices among criteria and sub-criteria. From 27 March to 1 April 2023, we recruited 1,000 study participants using a quota sampling method stratified by age, sex, and region based on Korean census data. We used eigenvalue methods to calculate the PV of the criteria and sub-criteria. The effects of sociodemographic factors on the PVs were analyzed using linear regression methods. RESULTS: The PVs for the criteria were highest for clinical usefulness (28.5%), followed by cost-effectiveness (27.1%), budget impact (24.3%), and reimbursement in other countries (20.1%). The sociodemographic characteristics of the participants had a significant impact on the PVs of the criteria. Willingness to pay additional premiums for national health insurance was negatively associated with PV for clinical usefulness and cost-effectiveness and positively associated with PV for reimbursement in other countries. CONCLUSIONS: The public prioritized clinical usefulness and cost-effectiveness as the main criteria. However, the PVs of the criteria were divergent and associated with sociodemographic factors. Divergent public interests require an evidence-informed deliberative process for reimbursement decisions.

A study on the effect of clinical intervention of evidence-based nursing measures on complications in patients after breast-conserving surgery.

BACKGROUND: Breast-conserving surgery is an important treatment for breast cancer, which not only eradicates the disease, but also protects the integrity of the breast, however, postoperative nausea and vomiting often bother patients. OBJECTIVE: This study examines the effects of evidence-based nursing practices on nausea and vomiting in patients after breast-conserving surgery, with the aim of providing new perspectives for clinical nursing practice. METHODS: One hundred and sixty patients who underwent breast-conserving surgery from January 2023 to December 2023 in Fudan University Shanghai Cancer Center were enrolled. The patients were divided into an intervention group (evidence-based nursing group) and a control group (conventional nursing group) using the random number table method, both groups comprised 80 patients. The control group used conventional nursing methods, and the intervention group added evidence-based nursing intervention on this basis. Comparative analysis focused on the incidence of nausea and vomiting, quality of life metrics, and postoperative satisfaction. RESULTS: In the intervention group, notably lower incidence rates of postoperative nausea and vomiting were observed compared to the control group within both the 0-24 hour and 24-48-hour postoperative periods (P< 0.05). Furthermore, the intervention group exhibited significantly higher scores across all five dimensions as well as the overall score of the FACT-B scale in comparison to the control group (P< 0.05), accompanied by heightened satisfaction with the nursing staff. CONCLUSION: This study demonstrated the positive clinical intervention effects of evidence-based nursing measures and emphasized their importance in improving postoperative nausea and vomiting and quality of life. Future studies are expected to incorporate evidence-based nursing practices into nursing care to improve patient recovery and overall quality of care.

Health care transition models in spina bifida care: evidence-based lessons in support of neurosurgical practice.

OBJECTIVE: The purpose of this study was to conduct a literature review on transition programs from pediatric to adult care and the role of neurosurgery as individuals with spina bifida (SB) transition, and to provide a framework for neurosurgical providers to assist in the transition to adult-centered care. METHODS: A comprehensive literature review was conducted according to the PRISMA statement, with a search in Medline and Embase to identify US clinical programs reporting on their experiences establishing a transition program for adolescents and young adults with SB. Data were collected for authors, year, transition clinic location, model of care for transition clinic, ages served, and specialty clinical team. RESULTS: The literature search yielded 698 articles, 5 of which met the inclusion criteria. These 5 studies included 4 transition programs for which models of care and approach to transition, clinical services involved, establishment of goals, and age of initiation and transition were identified. All programs described setting transition goals, ranging from community services, to self-management, to health care navigation, to patient-driven goals, with 1 program reporting a quality-of-life measurement component to their model. CONCLUSIONS: Robust SB transition programs can be established by applying the expanded chronic care model, reviewing lessons learned by other programs, advocating at the institutional level, and seeking support via professional organizations. While the comprehensive role of neurosurgical providers in these programs is still being defined, a shared vision of enhancing the health and quality of life for individuals with SB and their families is needed by all subspecialists involved.

Retrospective, Real Life Study on the Effectiveness and Safety of a Depigmented-Polymerized Subcutaneous Vaccine Containing a Mixture of Grasses and Olea europaea.

INTRODUCTION: Allergen immunotherapy is the only modifying treatment of the natural course of respiratory allergic diseases; however, the lack of evidence leads to little inconclusive results. Real life studies are on the rise and are becoming a valuable tool to confirm and complement findings from clinical trials. The objective of this study was to evaluate the effectiveness and safety of a depigmented-polymerized undiluted subcutaneous extract of grass and olive pollen, under routine clinical practice conditions. METHODS: This was an observational, retrospective, longitudinal, single-center study on the use of a 2-pollen (grass mix and Olea europaea) undiluted subcutaneous extract over at least 3 consecutive years. Data were collected from 76 patients (n = 44 female; median age: 12.5 years old) diagnosed with allergic rhinoconjunctivitis with/without allergic asthma due to sensitization to both grasses and O. europaea. Primary and secondary effectiveness endpoints were symptom severity, concomitant medication, and immunological profile before and after completing the immunotherapy. A 2-year follow-up of patients' symptoms and medication history after completing the subcutaneous immunotherapy (SCIT) was performed. RESULTS: There was a significant improvement of symptoms and medication consumption after 3 years of SCIT treatment, and a significant decrease in specific IgE levels for grasses and O. europaea was observed after finishing the treatment. CONCLUSION: Three years treatment of allergic patients using an undiluted mixture of two allergen extracts was shown to be safe and effective for rhinitis and asthma, with efficacy maintained for at least 2 years after finishing SCIT. These results reinforce the importance of real life clinical data in addition to those from clinical trials, helping to individualize allergic treatments.

Effect of Providing Evidence-Based Mental Health Treatment on Retention in Care Among Medicaid-Enrolled Youths.

OBJECTIVE: Youths who start behavioral health treatment often stop before completing a therapeutic course of care. To increase treatment engagement and quality of care, the Evidence-Based Practice and Innovation Center in Philadelphia has incentivized use of evidence-based practices (EBPs) for mental health care of youths. The authors aimed to compare treatment outcomes between youths who received EBP care and those who did not. METHODS: Using EBP-specific billing codes and propensity score matching, the authors compared treatment retention among youths who received trauma-focused cognitive-behavioral therapy (TF-CBT; N=413) or parent-child interaction therapy (PCIT; N=90) relative to matched samples of youths in standard outpatient therapy (N=503). RESULTS: Youths with a minimum of one session of TF-CBT or PCIT attended a second session at higher rates than did youths in the matched control group (TF-CBT: 96% vs. 68%, p<0.01; PCIT: 94% vs. 69%, respectively, p<0.01). On average, these returning youths attended more sessions in the EBP groups than in the control group (TF-CBT: 15.9 vs. 11.5 sessions, p<0.01; PCIT: 11.2 vs. 6.9 sessions, p<0.01). CONCLUSIONS: These findings indicate that, in addition to improving quality of care, EBP implementation helps address the major challenge that most youths who engage with treatment are not retained long enough for care to have therapeutic effects. Future research should examine the mechanisms through which EBPs can improve treatment retention.

Evidence-based intrapartum care practice and associated factors among obstetrics workers in Ethiopia, systematic review and meta-analysis.

Background: Evidence-based practice is defined as using the best available research and clinical evidence by incorporating patients' values and preferences for their health needs. The use of evidence-based intrapartum care practices is an essential tool to improve the quality of obstetrics care. Objective: The primary objective of this study was to determine the prevalence of evidence-based intrapartum care and associated factors among obstetrics care providers in Ethiopia. Method: Important articles were retrieved from universally accepted and used databases, including Cochran, PubMed, HINARI, Google Scholar, Web of Science, African OnLine, and repositories of Ethiopian Universities. We extracted articles by using a standard JOANNA Briggs Institute data extraction sheet. To determine the existence of heterogeneity in studies, I 2 statistics and Cochran Q tests were used. The publication bias of the included studies was checked using Egger's test and a Funnel plot. Result: A total of 2035 obstetrics care providers were involved in this systematic review and meta-analysis. The estimated overall rate of evidence-based intrapartum care practice in Ethiopia was 54.45% (95% CI: 43.06, 65.83); I 2 = 96.6%, p < 0.001). The studies with a sample size greater than 300 count for 47.25% (95% CI: 36.14, 65.83). Whereas obstetrics care providers have a decent knowledge of intrapartum care evidence 3.31 times, a positive attitude toward evidence 3.34 times, training 2.21 times, and work experience ⩾5 years 3.31 times associated with the practice of evidence-based intrapartum care. Conclusion: The overall practice of evidence-based intrapartum care among obstetrics workers in Ethiopia is estimated to be low. Therefore, there should be a focused effort on training and disseminating protocols and guidelines to enhance knowledge and foster a positive attitude among obstetrics care providers. Additionally, the Ethiopian government should prioritize the implementation of the 2021 to 2025 National Health Equity Strategic Plan to achieve its objective of improving the quality of health services.

Long-term outcomes and predictors of vedolizumab persistence in ulcerative colitis.

Background: Long-term vedolizumab (VDZ) outcomes in real-world cohorts have been largely limited to 1-year follow-up, with few bio-naïve patients or objective markers of inflammation assessed. Objectives: We aimed to assess factors affecting VDZ persistence including clinical, biochemical and faecal biomarker remission at 1, 3 and 5 years. Design: We performed a retrospective, observational, cohort study. Methods: All adult inflammatory bowel disease (IBD) patients who had received VDZ induction for ulcerative colitis (UC)/IBD-unclassified (IBDU) were included. Baseline phenotype and follow-up data were collected via a review of electronic medical records. Results: We included 290 patients [UC n = 271 (93.4%), IBDU n = 19 (6.6%)] with a median time on VDZ of 27.6 months (interquartile range: 14.4-43.2). At the end of follow-up, a total of 157/290 (54.1%) patients remained on VDZ. The median time to discontinuation was 14.1 months (7.0-23.3). Previous exposure to ⩾1 advanced therapy, steroid use at baseline and disease extension (E3 and E2 versus E1) were independent predictors for worse VDZ persistence. Clinical remission (partial Mayo < 2) was 75.7% (171/226), 72.4% (157/217) and 70.2% (127/181) at years 1, 3 and 5, respectively. Steroid use during maintenance VDZ therapy occurred in 31.7% (92/290), hospitalization in 15.5% (45/290) and surgery in 3.4% (10/291). The rate of serious adverse events was 1.2 per 100 patient-years of follow-up. Conclusion: VDZ effectiveness appears enduring with favourable long-term safety profile. VDZ persistence was influenced by previous exposure to biologics/small molecules, disease distribution and steroid use at baseline in our study.

Vedolizumab long-term use in ulcerative colitis What was this study done? • Vedolizumab efficacy and safety in ulcerative colitis have been firmly established by existing evidence. • Long-term data from the GEMINI trial further corroborate the favourable safety profile over an extended duration but there is little data on long-term vedolizumab use over 1 year. What did the researches do? • We performed a retrospective, observational, cohort study. All adult IBD patients who ever received vedolizumab induction from November 2014 to December 2021 for ulcerative colitis/IBDU were included. What did the researchers find? • This real-world study demonstrates that vedolizumab persistence exceeds 80% at 1 year and remains nearly 50% at 5 years with no new safety signals. • Worse vedolizumab persistence is associated with prior exposure to biologics/small molecules, more extensive disease involvement and steroid use at vedolizumab initiation. What do the findings mean? • These findings have important implications for drug positioning and sequencing, as well as for optimizing outcomes when vedolizumab is utilized as first-line therapy. Furthermore, it also emphasizes the long-term safety profile.

The impacts of housing conditions on physical and mental health - A mini-review informed by a rapid conversion of evidence from Alidoust and Huang (2021).

This article provides a summary and critical appraisal of the systematic review conducted by Alidoust et al. (1) regarding the various effects of housing on both physical and psychological well-being. We aim to discuss the review's findings against existing published evidence to draw out policy and practical implications. Our mini-review illuminates a wide range of housing-related factors which impact on health around which we draw evidence-based policy initiatives and implications, and outline avenues for future research. This mini-review is part of the wider Rapid Conversion of Evidence Summaries (RaCES) program which aims to critically appraise systematic reviews and highlight evidence-based policy and practice implications.

Comparison of renal prognosis between dipeptidyl peptidase-4 inhibitor users and non-users.

AIM: To evaluate the renal prognosis of dipeptidyl peptidase-4 inhibitor (DPP-4i) users and non-users using real-world Asian data. METHODS: Using databases from DeSC Healthcare, Inc., patients aged 30 years or older who used antidiabetic drugs from 2014 to 2021 were identified. Propensity score matching analyses were used to compare renal prognosis between DPP-4i users and non-users. The primary outcomes were estimated glomerular filtration rate (eGFR) decline and end-stage kidney disease (ESKD) development in the eGFR of 45 mL/min/1.73m2 or higher and eGFR of less than 45 mL/min/1.73m2 groups, respectively. RESULTS: In total, 65 375 and 9866 patients were identified in the eGFR of 45 mL/min/1.73m2 or higher and eGFR of less than 45 mL/min/1.73m2 groups, respectively. In the eGFR of 45 mL/min/1.73m2 or higher group, propensity score matching created 16 002 pairs. A significant difference was observed in the primary outcome of eGFR decline between DPP-4i users and non-users at 2 years (-2.31 vs. -2.56 mL/min/1.73m2: difference, 0.25 mL/min/1.73m2; 95% confidence interval [CI], 0.06-0.44) and 3 years (-2.75 vs. -3.41 mL/min/1.73m2: difference, 0.66 mL/min/1.73m2; 95% CI, 0.39-0.93). In the eGFR less than 45 mL/min/1.73m2 group, propensity score matching created 2086 pairs. After a mean of 2.2 years of observation, ESKD development was 1.15% and 2.30% in users and non-users, respectively, and Kaplan-Meier analysis revealed a significant difference (log rank P = .005). CONCLUSIONS: This retrospective real-world study revealed that patients using DPP-4is had a better renal prognosis than those not using DPP-4is.

Advancing evidence-based practice through the Knowledge Translation Challenge: Nurses' important roles in research, implementation science and practice change.

AIM: To describe a knowledge translation capacity-building initiative and illustrate the roles of nurses in practice change using an exemplar case study. DESIGN: The report uses observational methods and reflection. METHODS: The Knowledge Translation Challenge program involves a multi-component intervention across several sites. The advisory committee invited eligible teams to attend capacity-building workshops. Implementation plans were developed, and successful teams receive funding for a 2 year period. Evaluation involved collecting data on program uptake and impact on practice change. Data has been collected from five cohorts. The exemplar case study employed an action-research framework. RESULTS: Four nurse-led teams have demonstrated successful implementation of their practice change. The case study on implementing a clinical toolkit for clozapine management further illustrates a thoughtful planning process, and implementation journey and learnings by a team of nurses. CONCLUSION: The Knowledge Translation Challenge program empowers nurses to use implementation science practices to enhance the quality and effectiveness of healthcare services. Success of this initiative serves as a model for addressing the persistent gap between knowledge and practice in clinical settings and the value of activating nurses to help close this gap. IMPLICATIONS: As the most trusted and numerous profession, it is vital that nurses contribute to efforts to translate research evidence into clinical practice. The Knowledge Translation Challenge program supports nurses to lead practice change. IMPACT: The Knowledge Translation Challenge program successfully equips nurses and other health care providers with the knowledge, skills and resources to implement practice improvements which enhance the quality and effectiveness of healthcare services and nursing practice. PATIENT OR PUBLIC CONTRIBUTION: The Knowledge Translation Challenge advisory committee has three patient-public partners that support teams to develop a patient-oriented approach for their projects by providing feedback on the implementation plans. Each team was also supported to include patient-public partners on their project.

Next-generation sequencing in oncology: challenges in economic evaluations.

INTRODUCTION: Next-generation sequencing (NGS) identifies genetic variants to inform personalized treatment plans. Insufficient evidence of cost-effectiveness impedes integration of NGS into routine cancer care. The complexity of personalized treatment challenges conventional economic evaluation. Clearly delineating challenges informs future cost-effectiveness analyses to better value and contextualize health, preference-, and equity-based outcomes. AREAS COVERED: We conducted a scoping review to characterize the applied methods and outcomes of economic evaluations of NGS in oncology and identify existing challenges. We included 27 articles published since 2016 from a search of PubMed, Embase, and Web of Science. Identified challenges included defining the evaluative scope, managing evidentiary limitations including lack of causal evidence, incorporating preference-based utility, and assessing distributional and equity-based impacts. These challenges reflect the difficulty of generating high-quality clinical effectiveness and real-world evidence (RWE) for NGS-guided interventions. EXPERT OPINION: Adapting methodological approaches and developing life-cycle health technology assessment (HTA) guidance using RWE is crucial for implementing NGS in oncology. Healthcare systems, decision-makers, and HTA organizations are facing a pivotal opportunity to adapt to an evolving clinical paradigm and create innovative regulatory and reimbursement processes that will enable more sustainable, equitable, and patient-oriented healthcare.

Uptake of evidence-based practice and its predictors among nurses in Ethiopia: a systematic review and meta-analysis.

Background: While evidence-based practice has demonstrated its capacity to enhance healthcare quality and bolster clinical outcomes, the translation of research into clinical practice encounters persistent challenges. In Ethiopia, there remains a dearth of comprehensive and nationally representative data concerning the extent of Evidence-based practice adoption among nurses. Thus, this systematic review and meta-analysis endeavors to assess the overall prevalence of Evidence-based practice implementation and delve into its determinants among Ethiopian nurses. Methods: A systematic review and meta-analysis were conducted following the PRISMA guidelines. In order to identify pertinent studies, a search was conducted across PubMed, Scopus, Google Scholar, and EMBASE databases. A weighted inverse variance random-effects model was employed to estimate the pooled prevalence. Cochrane's Q-test and I2 statistics were calculated to assess heterogeneity among studies. Funnel plots and Egger's test were utilized to evaluate publication bias. Pooled implementation rates and meta-regression analysis were carried out using STATA 17. Results: Of the total 1,590 retrieved articles, twelve studies including 4,933 nurses were included in the final analysis. The pooled prevalence of Evidence-based practice uptake among nurses in Ethiopia is 53% (95% CI: 46%-60%). Having knowledge about Evidence-based practice (AOR = 2.29; 95% CI: 1.90, 2.69; I2 = 70.95%), holding a favorable attitude towards Evidence-based practice (AOR = 2.56; 95% CI: 1.63, 3.49; I2 = 88.39%), occupying a head nurse position (AOR = 3.15; 95% CI: 1.85, 4.46; I2 = 87.42%), possessing effective communication skills (AOR = 4.99; 95% CI: 1.47, 8.51; I2 = 99.86%), and having access to Evidence-based practice guidelines (AOR = 1.90; 95% CI: 1.55, 2.24; I2 = 57.24%) were identified as predictors of the uptake of Evidence-based practice. Conclusion: Only half of Ethiopia's nurses exhibit a strong embrace of Evidence-Based Practice within clinical settings, underscoring the urgent necessity for coordinated endeavors to cultivate this essential practice. Possessing knowledge, effective communication skills, access to updated guidelines, maintaining a positive attitude towards Evidence-Based Practice, and holding a position as head nurse emerged as predictors of successful implementation of Evidence-Based Practice. Hence, policymakers must prioritize capacity-building initiatives, disseminate the latest EBP guidelines widely, and strengthen mentorship roles for head nurses. Systematic Review Registration: https://www.crd.york.ac.uk/prospero/#searchadvanced, identifier CRD42023488943.

The 2024-2025 Commonwealth Budget for Mental Health: Funding unproven initiatives and stings in the tail.

We discuss the ramifications of the Commonwealth of Australia Budget allocations for mental healthcare for 2024-2025. There is funding for population-based mental health initiatives for milder anxiety and depression but no direct funding of services for the most severe and disabling forms of mental illness, other than pre-existing state/territory disbursements from the Commonwealth for state-based health services. There are substantial concerns that the Commonwealth funding has potentially been misallocated to ineffective interventions that are unlikely to reduce the population prevalence of mild anxiety and depression in Australia. Funds may have been better allocated to provide effective care for those with the most severe and disabling illnesses including schizophrenia, bipolar disorder and severe depression.

The use of weight-of-evidence approaches to characterize developmental toxicity risk for therapeutic monoclonal antibodies in humans without in vivo studies.

Regulatory guidance for global drug development relies on animal studies to evaluate safety risks for humans, including risk of reproductive toxicity. Weight-of-evidence approaches (WoE) are increasingly becoming acceptable to evaluate risk. A WoE for developmental risk of monoclonal antibodies (mAbs) was evaluated for its ability to retrospectively characterize risk and to determine the need for further in vivo testing based on the remaining uncertainty. Reproductive toxicity studies of 65 mAbs were reviewed and compared to the WoE. Developmental toxicities were absent in 52/65 (80%) mAbs. Lack of toxicity was correctly predicted in 29/52 (56%) cases. False positive and equivocal predictions were made in 9/52 (17%) and 14/52 (27%) cases. For 3/65 (5%) mAbs, the findings were equivocal. Of mAbs with developmental toxicity findings (10/65, 15%), the WoE correctly anticipated pharmacology based reproductive toxicity without any false negative predictions in 9/10 (90%) cases, and in the remaining case ( 1/10, 10%) an in vivo study was recommended due to equivocal WoE outcome. Therefore, this WoE approach could characterize presence and absence of developmental risk without animal studies. The current WoE could have reduced the need for developmental toxicity studies by 42% without loss of important patient information in the label.

Palbociclib in HR-Positive, HER2-Negative Advanced/Metastatic Breast Cancer: A Systematic Scoping Review of Real-World Evidence from Countries Outside of Western Regions that Are Underrepresented in Clinical Trials.

INTRODUCTION: Limited awareness exists regarding real-world data (RWD) for palbociclib in patients with hormone receptor-positive, human epidermal growth factor receptor 2-negative advanced/metastatic breast cancer in populations from certain countries outside of Western regions. METHODS: A systematic scoping review was conducted using PubMed and Embase to evaluate RWD for palbociclib from countries outside of Western regions that are underrepresented in clinical trials. Search criteria were aligned with our research question for relevant English-language publications, without restrictions on publication date, followed by Phase 1 (title and abstract) and Phase 2 (full-text) screening of retrieved citations as per Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. Data analyses of eligible studies were done separately for abstracts and full-text publications to enhance the precision and reliability of the results. RESULTS: Database search yielded 1485 non-duplicate records, 46 qualified for inclusion, of which 47.8% were published as full text. The analysis of outcomes, based exclusively on full-text publications that collectively included 2048 patients treated with palbociclib, revealed the median progression-free survival (PFS) of 20.2-36.7 months, overall survival (OS) of 39.9 months (reported in one publication) and objective response rate (ORR) of 45.3-80.0% with first-line treatment. In ≥ second line, the median PFS, OS and ORR ranged from 7.0 to 24.2 months, 11 to 19.6 months, and 13.9% to 47.9%, respectively. The safety profile of palbociclib was similar to that reported in pivotal clinical studies, and no new safety concerns were identified. CONCLUSIONS: A comprehensive volume of evidence demonstrates that palbociclib's effectiveness and safety profile in real-world settings align with those observed in clinical trials, offering valuable insights for clinical decision-making in countries outside of Western regions underrepresented in clinical trials.

Methodological Considerations in Evaluating Breast Cancer Screening Studies.

In evidence-based medicine frameworks, the highest level of evidence is derived from quantitative synthesis of double-masked, high-quality, randomly assigned controlled trials. Meta-analyses of randomly assigned controlled trials have demonstrated that screening mammography reduces breast cancer deaths. In the United States, every major guideline-producing organization has recommended screening mammography in average-risk women; however, there are controversies about age and frequency. Carefully controlled observational research studies and statistical modeling studies can address evidence gaps and inform evidence-based, contemporary screening practices. As breast imaging radiologists develop and evaluate existing and new screening tests and technologies, they will need to understand the key methodological considerations and scientific criteria used by policy makers and health service researchers to support dissemination and implementation of evidence-based screening tests. The Wilson and Jungner principles and the U.S. Preventive Services Task Force general analytic framework provide structured evaluations of the effectiveness of screening tests. Key considerations in both frameworks include public health significance, natural history of disease, cost-effectiveness, and characteristics of screening tests and treatments. Rigorous evaluation of screening tests using analytic frameworks can maximize the benefits of screening tests while reducing potential harms. The purpose of this article is to review key methodological considerations and analytic frameworks used to evaluate screening studies and develop evidence-based recommendations.

Supplementing Systematic Review Findings with Healthcare System Data: Pilot Projects from the Agency for Healthcare Research and Quality Evidence-based Practice Center Program.

OBJECTIVES: The US Agency for Healthcare Research and Quality (AHRQ), through the Evidence-based Practice Center (EPC) Program, aims to provide health system decision makers with the highest-quality evidence to inform clinical decisions. However, limitations in the literature may lead to inconclusive findings in EPC systematic reviews (SRs). The EPC Program conducted pilot projects to understand the feasibility, benefits, and challenges of utilizing health system data to augment SR findings to support confidence in healthcare decision-making based on real-world experiences. STUDY DESIGN AND SETTING: Three contractors (each an EPC located at a different health system) selected a recently completed systematic review conducted by their center and identified an evidence gap that electronic health record (EHR) data might address. All pilot project topics addressed clinical questions as opposed to care delivery, care organization, or care disparities topics that are common in EPC reports. Topic areas addressed by each EPC included infantile epilepsy, migraine, and hip fracture. EPCs also tracked additional resources needed to conduct supplemental analyses. The workgroup met monthly in 2022-2023 to discuss challenges and lessons learned from the pilot projects. RESULTS: Two supplemental data analyses filled an evidence gap identified in the systematic reviews (raised certainty of evidence, improved applicability) and the third filled a health system knowledge gap. Project challenges fell under three themes: regulatory and logistical issues, data collection and analysis, and interpretation and presentation of findings. Limited ability to capture key clinical variables given inconsistent or missing data within the EHR was a major limitation. The workgroup found that conducting supplemental data analysis alongside an SR was feasible but adds considerable time and resources to the review process (estimated total hours to complete pilot projects ranged from 283-595 across EPCs), and that the increased effort and resources added limited incremental value. CONCLUSION: Supplementing existing systematic reviews with analyses of EHR data is resource intensive and requires specialized skillsets throughout the process. While using EHR data for research has immense potential to generate real-world evidence and fill knowledge gaps, these data may not yet be ready for routine use alongside systematic reviews.

Patient-reported symptoms and burden of eosinophilic esophagitis: evidence from real-world clinical practice.

BACKGROUND: Eosinophilic esophagitis is a chronic inflammatory disorder of the esophagus. This real-world study used patient and physician surveys to describe the clinical characteristics and disease burden of eosinophilic esophagitis-overall and in a subgroup of patients with dysphagia despite treatment. METHODS: Data analyzed in this study were collected in 2020 from US and EU patients with eosinophilic esophagitis. Eligible patients were aged ≥ 12 years with a diagnosis of eosinophilic esophagitis, had an esophageal count of ≥ 15 eosinophils/high-power field at diagnosis, and were currently prescribed treatment for eosinophilic esophagitis. RESULTS: Overall, 1001 patients were included, of whom 356 (36%) had dysphagia despite treatment. Demographics and clinical characteristics were similar in both populations. The severity of eosinophilic esophagitis was mild in more patients overall (69%) versus those with dysphagia despite treatment (48%). Patient disease history was similar in both populations, with some exceptions: common patient-reported symptoms were dysphagia (70% and 86%) and heartburn/acid reflux (55% and 49%), and common physician-reported symptoms were dysphagia (75% and 91%) and food impaction (46% and 52%). Treatment history was similar in both populations; overall, the most common treatments were proton pump inhibitors (83%) and topical corticosteroids (51%). Patients reported slightly more days with symptoms, higher impacts on activities of daily living, and slightly higher anxiety or depression in the dysphagia-despite-treatment population versus the overall population. CONCLUSIONS: Eosinophilic esophagitis presents severe symptoms and comorbidities that substantially impact patients' well-being and quality of life. Greater awareness of and novel treatments for eosinophilic esophagitis are needed.