Pharmacoepidemiology and Drug Safety最新文献

{"title":"Using Quantitative Bias Analysis to Adjust for Misclassification of COVID-19 Outcomes: An Applied Example of Inhaled Corticosteroids and COVID-19 Outcomes.","authors":"Marleen Bokern, Christopher T Rentsch, Jennifer K Quint, Jacob Hunnicutt, Ian Douglas, Anna Schultze","doi":"10.1002/pds.70086","DOIUrl":"10.1002/pds.70086","url":null,"abstract":"<p><strong>Background: </strong>During the pandemic, there was concern that underascertainment of COVID-19 outcomes may impact treatment effect estimation in pharmacoepidemiologic studies. We assessed the impact of outcome misclassification on the association between inhaled corticosteroids (ICS) and COVID-19 hospitalisation and death in the United Kingdom during the first pandemic wave using probabilistic bias analysis (PBA).</p><p><strong>Methods: </strong>Using data from the Clinical Practice Research Datalink Aurum, we defined a cohort with chronic obstructive pulmonary disease (COPD) on 1 March 2020. We compared the risk of COVID-19 hospitalisation and death among users of ICS/long-acting β-agonist (LABA) and users of LABA/LAMA using inverse probability of treatment weighted (IPTW) logistic regression. We used PBA to assess the impact of non-differential outcome misclassification. We assigned beta distributions to sensitivity and specificity and sampled from these 100 000 times for summary-level and 10 000 times for record-level PBA. Using these values, we simulated outcomes and applied IPTW logistic regression to adjust for confounding and misclassification. Sensitivity analyses excluded ICS + LABA + LAMA (triple therapy) users.</p><p><strong>Results: </strong>Among 161 411 patients with COPD, ICS users had increased odds of COVID-19 hospitalisations and death compared with LABA/LAMA users (OR for COVID-19 hospitalisation 1.59 (95% CI 1.31-1.92); OR for COVID-19 death 1.63 (95% CI 1.26-2.11)). After IPTW and exclusion of people using triple therapy, ORs moved towards the null. All implementations of QBA, both record- and summary-level PBA, modestly shifted the ORs away from the null and increased uncertainty.</p><p><strong>Conclusions: </strong>We observed increased risks of COVID-19 hospitalisation and death among ICS users compared to LABA/LAMA users. Outcome misclassification was unlikely to change the conclusions of the study, but confounding by indication remains a concern.</p>","PeriodicalId":19782,"journal":{"name":"Pharmacoepidemiology and Drug Safety","volume":"34 1","pages":"e70086"},"PeriodicalIF":2.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11706700/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142952668","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Real-World Pharmacovigilance Study of FDA Adverse Event Reporting System Events for Obeticholic Acid.","authors":"Le Hai, Jiaojiao Wu, Xiaohong Pan, Weicheng Yin, Zhishan Wu","doi":"10.1002/pds.70084","DOIUrl":"https://doi.org/10.1002/pds.70084","url":null,"abstract":"<p><strong>Background and objectives: </strong>Based on the Adverse Event Reporting System (FAERS) data from the US FDA, this study mined the adverse drug reactions of obeticholic acid (OCA) in the real world and provided reference for clinical safe drug use.</p><p><strong>Methods: </strong>Adverse event reports for OCA from the second quarter of 2016 to the third quarter of 2023 were extracted. The analysis for adverse reaction signal detection was conducted using reporting odds ratio, proportional reporting ratio, Bayesian confidence propagation neural network, and multi-item gamma Poisson shrinker methods.</p><p><strong>Results: </strong>A total of 5661 OCA-related adverse event reports were collected, and 105 OCA-related adverse reaction signals were obtained, involving 14 systems, among which 46 new signals were not previously mentioned in the product labeling. Severe adverse event of OCA accounted for a relatively high proportion (1445 cases, 25.53%), among which the number of hospitalization reports was the largest (1042 cases, 18.41%). The top five adverse events were pruritus, fatigue, constipation, elevated blood alkaline phosphatase, and abdominal distention. The top five adverse reaction signals intensity were abnormal blood alkaline phosphatase, abnormal ratio of albumin globulin, spider nevus, combined with abnormal bilirubin, and γ-abnormal glutamyl transferase.</p><p><strong>Discussion: </strong>Based on the pharmacovigilance study of the FAERS database, it is necessary to strengthen the clinical medication monitoring of OCA, so as to provide reference for effective pharmaceutical monitoring and rational clinical medication.</p>","PeriodicalId":19782,"journal":{"name":"Pharmacoepidemiology and Drug Safety","volume":"34 1","pages":"e70084"},"PeriodicalIF":2.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142952832","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Masking in Active Comparator Designs in Pharmacovigilance: A Retrospective Bias Analysis on the Spontaneous Reporting of Thiazolidinediones and Cardiovascular Events.","authors":"William Bai, Antonios Douros, Christopher A Gravel","doi":"10.1002/pds.70102","DOIUrl":"https://doi.org/10.1002/pds.70102","url":null,"abstract":"<p><strong>Introduction: </strong>Masking is a reporting bias where drug safety signals are muffled by elevated reporting of other medications in spontaneous reporting databases. While the impact of masking is often limited, its effect when using restricted designs, such as active comparators, can be consequential.</p><p><strong>Methods: </strong>We used data from the US Food and Drugs Administration Adverse Event Reporting System (1999Q3-2013Q3) to study masking in a real-world example. Rosiglitazone, a thiazolidinedione with elevated reporting after safety concerns over cardiovascular risks, was the masking candidate. We hypothesized that stimulated reporting masked signals for another thiazolidinedione, pioglitazone. We computed estimates of proportional reporting ratios and information components, using the Bayesian confidence propagation neural network, for pioglitazone-myocardial infarction and pioglitazone-cardiac failure under unrestricted and active comparator designs, with and without the mask, before (1999Q3-2007Q1) and after (2007Q2-2013Q3) safety concerns. Relative change-in-estimates were computed to compare results with and without rosiglitazone.</p><p><strong>Results: </strong>From 1999Q3-2007Q1, relative change-in-estimates of proportional reporting ratio for pioglitazone-myocardial infarction was 0.00 in unrestricted design and 0.10 in active comparator, and for pioglitazone-cardiac failure, the change was 0.01 and 0.62, respectively. From 2007Q2-2013Q3, relative change-in-estimates for pioglitazone-myocardial infarction was 0.41 in unrestricted design and 18.00 in active comparator; the change for pioglitazone-cardiac failure was 0.04 and 1.03, respectively. Relative changes in estimates of information component mirrored these trends.</p><p><strong>Conclusions: </strong>Masking can influence signal detection in active comparator designs where external events impact reporting rates in reference sets. Evaluating masking in related contexts is essential for drug safety monitoring and resource allocation for follow-up studies.</p>","PeriodicalId":19782,"journal":{"name":"Pharmacoepidemiology and Drug Safety","volume":"34 1","pages":"e70102"},"PeriodicalIF":2.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142984575","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"INSIGHT: A Tool for Fit-for-Purpose Evaluation and Quality Assessment of Standardized Observational Data Sources for Real World Evidence on Medicine and Vaccine Safety.","authors":"Vjola Hoxhaj, Constanza L Andaur Navarro, Judit Riera-Arnau, Roel J H J Elbers, Ema Alsina, Caitlin Dodd, Miriam C J M Sturkenboom","doi":"10.1002/pds.70089","DOIUrl":"10.1002/pds.70089","url":null,"abstract":"<p><strong>Purpose: </strong>To describe the development of INSIGHT, a real-world data quality tool to assess completeness, consistency, and fitness-for-purpose of observational health data sources.</p><p><strong>Methods: </strong>We designed a three-level pipeline with data quality assessments (DQAs) to be performed in ConcePTION Common Data Model (CDM) instances. The pipeline has been coded using R.</p><p><strong>Results: </strong>INSIGHT is an open-source tool that identifies potential data quality issues in CDM-standardized instances through the systematic execution and summary of over 588 configurable DQAs. Level 1 focuses on conformance to the ConcePTION CDM specifications. Level 2 evaluates the temporal plausibility of events and uniqueness of records. Level 3 provides an overview of distributions, outliers, and trends over time to facilitate fit-for-purpose evaluation. Therefore, level 1 and 2 assure a proper data standardization, while level 3 provides information regarding the study population, and potential sub-populations. The DQAs are run locally and assessed centrally by a data quality revisor together with the data access provider's representatives.</p><p><strong>Discussion: </strong>Data quality is the sum of several internal and external features of the data. While DQAs can provide reassurance about fitness-for-purpose for secondary-use data sources, improvements in data collection are essential to reduce errors and enhance overall data quality for Real World Evidence.</p><p><strong>Conclusion: </strong>INSIGHT aims to support clinical and regulatory decision-making for medicines and vaccines by evaluating the quality of observational health data sources to support fit for purpose assessment. Assessing and improving data quality will enhance the reliability and quality of the generated evidence.</p><p><strong>Study registration: </strong>This research was registered in EU PAS registration with number EU50142.</p>","PeriodicalId":19782,"journal":{"name":"Pharmacoepidemiology and Drug Safety","volume":"34 1","pages":"e70089"},"PeriodicalIF":2.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11730612/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142979423","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"In Memoriam-Helle Kieler (1956-2024).","authors":"Carolyn E Cesta, Gabriella Bröms, Kari Furu, Sonia Hernandez-Diaz, Krista F Huybrechts, Olof Stephansson, Helga Zoega","doi":"10.1002/pds.70073","DOIUrl":"https://doi.org/10.1002/pds.70073","url":null,"abstract":"","PeriodicalId":19782,"journal":{"name":"Pharmacoepidemiology and Drug Safety","volume":"34 1","pages":"e70073"},"PeriodicalIF":2.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142951488","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Predictors of Potentially Inappropriate Stimulant Prescribing Among Adults.","authors":"Pavan V Thakkar, Angelique E Boutzoukas, Scott N Compton, Ohviya Sivashankar, Kanecia O Zimmerman, Daniel K Benjamin, M Alan Brookhart","doi":"10.1002/pds.70079","DOIUrl":"10.1002/pds.70079","url":null,"abstract":"<p><strong>Purpose: </strong>Increases in adult stimulant prescribing pose a potential risk due to the higher prevalence of contraindicated conditions among this population. We sought to identify patient, provider, and visit characteristics predictive of potentially inappropriate adult stimulant prescriptions.</p><p><strong>Methods: </strong>We conducted a repeated cross-sectional study using the National Ambulatory Medical Care Survey, a nationally representative weighted sample of 5 453 702 723 ambulatory care visits from 2012 to 2019. Potentially inappropriate prescriptions were defined as prescriptions to patients with potentially contraindicated conditions, as determined by US Food and Drug Administration stimulant labels.</p><p><strong>Results: </strong>Of the 5 453 702 723 visits, stimulant use was prevalent at 121384694 (2.23%) visits and newly prescribed at 18880152 (0.34%) visits. Of these, 4 620 138 (24.47%) new stimulant prescriptions and 28 055 947 (23.11%) prevalent prescriptions were potentially inappropriate. Potentially inappropriate prescribing increased over time and with age. Visits to primary care providers (relative risk [RR] 1.65, 95% CI 1.05-2.59) were predictive of inappropriate prescribing. Non-Hispanic Black (RR 0.48, 95% CI 0.33-0.70) and Hispanic race/ethnicity (RR 0.46, 95% CI 0.35-0.60), coronary artery disease (RR 0.54, 95% CI 0.33-0.86), pregnancy (RR 0.05, 95% CI 0.03-0.11), hypertension (RR 0.69, 95% CI 0.56-0.84), and glaucoma (RR 0.07, 95% CI 0.02-0.24) were predictive of decreased prevalent stimulant prescriptions; substance abuse was predictive of new stimulant prescribing (RR 2.14, 95% CI 1.07-4.27).</p><p><strong>Conclusions: </strong>The proportion of potentially inappropriate adult stimulant prescriptions increased over time and with patient age. Visits to primary care providers were predictive of potentially inappropriate prescribing, and a history of substance abuse was predictive of new stimulant prescriptions; therefore, quality improvement interventions regarding safe stimulant prescribing practices may be warranted.</p>","PeriodicalId":19782,"journal":{"name":"Pharmacoepidemiology and Drug Safety","volume":"34 1","pages":"e70079"},"PeriodicalIF":2.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11731891/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142971818","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Post-Market Evidence for Cancer Medicines in Regulatory and Clinical Decision-Making: A Scoping Review.","authors":"Eliza J McEwin, Ashleigh Hooimeyer, Barbara J Mintzes","doi":"10.1002/pds.70093","DOIUrl":"https://doi.org/10.1002/pds.70093","url":null,"abstract":"<p><strong>Background: </strong>Cancer medicines usually have uncertain efficacy and safety profiles when they are first approved by medicines regulators because this evidence usually emerges post-market. Little is known about the extent to which post-market evidence is evaluated and integrated into evidence review processes in regulatory and clinical contexts.</p><p><strong>Objectives: </strong>The objective of this scoping review is to examine the literature on how post-market evidence on benefits and harms is evaluated and integrated in regulatory decisions and guidance for clinical decision-making.</p><p><strong>Methods: </strong>This scoping review focussed on the organisations that review cancer medicines and post-market evidence for their benefits and harms. It examined all regulatory or clinical contexts in which this post-market evidence might be included in evidence review processes for evaluation then integration into regulatory or clinical contexts. Four electronic databases were searched. Titles and abstracts were screened for all retrieved references followed by full-text screening by two independent reviewers according to pre-specified inclusion criteria.</p><p><strong>Results: </strong>In total, 28 studies met inclusion criteria. These included 31 assessments by medicines regulators, four by clinical practice guideline developers and two by health technology assessment agencies. Half of the studies evaluated clinical outcomes for benefit or harms (e.g., overall survival, serious adverse events). We found that more published literature evaluated and integrated post-market evidence for benefits and harms of cancer medicines in regulatory than in clinical situations, such as treatment guidelines and health technology assessments. In these studies, post-market evidence for harms seemed to be integrated more often than for benefits. And the studies showed a gap: only some of the evaluated post-market evidence was subsequently integrated in both regulatory and clinical situations.</p><p><strong>Conclusion: </strong>Overall, these findings raise important questions around the availability, accessibility, and assessment of post-market evidence for benefits and harms of cancer medicines so that it can be used by health professionals working in cancer services and by people with cancer.</p>","PeriodicalId":19782,"journal":{"name":"Pharmacoepidemiology and Drug Safety","volume":"34 1","pages":"e70093"},"PeriodicalIF":2.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142979446","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"More Drugs and Fewer Strokes? Time Trends in CVD Medication and Incidence of Stroke With German Health Insurance Data.","authors":"Lieselotte Mond, Siegfried Geyer, Juliane Tetzlaff, Karin Weißenborn, Johanna Schneider, Jelena Epping","doi":"10.1002/pds.70077","DOIUrl":"10.1002/pds.70077","url":null,"abstract":"<p><strong>Background: </strong>Successful prevention of cardiovascular diseases (CVD) may reduce the burden of diseases. Preventive medication is an important measure to decrease the risks of cardiovascular events, in particular myocardial infarction and stroke. The aim of this study is to analyze the prevalence of CVD preventive medication in Germany over time with respect to sex and age and to compare it with the temporal development of strokes.</p><p><strong>Methods: </strong>The study is based on statutory health insurance claims data from the AOK Niedersachsen (AOKN) covering the years 2005-2018. The study population comprises all AOKN insured persons aged 18 years and older (N = 2 088 495). Age-standardized time trends of the prevalence of CVD preventive medication and incidence of stroke were calculated for men and women in different age groups. After that, the relationship of both measures was examined in an ecological correlation.</p><p><strong>Results: </strong>We found a clear increase in medication prevalence over time. In 2018, about 35% of the total population and about 85% of those over 85 years of age received CVD preventive medication. At the same time, age-standardized incidence rates of ischemic stroke were decreasing slightly. The ecological correlation showed a negative association between medication prevalence and stroke incidence especially in the higher age groups.</p><p><strong>Conclusion: </strong>High correlation coefficients indicate that higher medication prevalence could be linked to better population health. Further research is needed to draw conclusions about the effects of increasing medicalization, including adverse risks and side effects at the population level.</p>","PeriodicalId":19782,"journal":{"name":"Pharmacoepidemiology and Drug Safety","volume":"34 1","pages":"e70077"},"PeriodicalIF":2.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11706669/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142951568","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety Assessment of Influenza Vaccination for Neurological Outcomes Among Older Adults in Japan: A Self-Controlled Case Series Study.","authors":"Mitsunori Ogawa, Yoshinori Takeuchi, Yukino Iida, Masao Iwagami, Kohei Uemura, Sachiko Ono, Nobuaki Michihata, Daisuke Koide, Yutaka Matsuyama, Hideo Yasunaga","doi":"10.1002/pds.70082","DOIUrl":"10.1002/pds.70082","url":null,"abstract":"<p><strong>Purpose: </strong>To assess adverse neurological risks following influenza vaccination in older adults.</p><p><strong>Methods: </strong>Using a linked database of healthcare administrative claims data and vaccination records from an urban city in Japan (April 1, 2014, to March 31, 2020), we conducted an observational study utilizing a self-controlled case series design. We identified individuals aged ≥ 65 years who experienced adverse neurological outcomes, defined as hospitalizations related to epilepsy, paralysis, facial paralysis, neuralgia, neuritis, optic neuritis, migraine, extrapyramidal disorders, Guillain-Barre syndrome, or narcolepsy. We used conditional Poisson regression to analyze within-subject incidence rate ratios, comparing the risk of these outcomes during risk periods following influenza vaccination (0-6 days and 7-29 days after each vaccination) with nonvaccination periods. Our analysis was adjusted for age and season groups as time-varying covariates.</p><p><strong>Results: </strong>We enrolled 3283 eligible individuals (men: 1643; mean [standard deviation] age: 76 [7.3] years). The incidence rate ratio for the outcome during the risk periods was 0.93 (95% confidence interval, 0.66-1.30) in risk period 1 (0-6 days after vaccination) and 1.14 (0.96-1.35) in risk period 2 (7-29 days after vaccination), respectively.</p><p><strong>Conclusions: </strong>We found no evidence that the risk of adverse neurological events was increased after influenza vaccination in older adults. These results may help reassure older adults who are hesitant to receive influenza vaccination because of concerns regarding adverse neurological outcomes.</p>","PeriodicalId":19782,"journal":{"name":"Pharmacoepidemiology and Drug Safety","volume":"34 1","pages":"e70082"},"PeriodicalIF":2.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11706699/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142951880","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Success of the German Cystic Fibrosis Registry.","authors":"Lutz Naehrlich, Manuel Burkhart","doi":"10.1002/pds.70076","DOIUrl":"https://doi.org/10.1002/pds.70076","url":null,"abstract":"<p><p>The German Cystic Fibrosis (CF) Registry (GCFR) is a national General Data Protection Regulation-compliant centralised database sponsored by the German Cystic Fibrosis Association (Mukoviszidose e.V.) and based on informed consent for each participating patient, ethical approval, and data protection votes. The aims of the GCFR are to optimise quality of care for CF at the centres, generate epidemiologic overviews, address research questions related to improved CF care, and inform caregivers, patients (aimed at patient empowerment), and health authorities and industry (aimed at care planning and pharmacovigilance). Established in 1995, the Registry has captured data on > 9600 individuals with a combined total of more than 140 000 annual assessments with an estimated coverage rate of > 90%. Patient data are collected after informed consent and confirmed diagnosis of CF, or a CFTR-related disorder, or a screening-positive inconclusive diagnosis of CF (i.e., CFSPID). The registry collects core, encounter, and annual health data. Data include demographics, anthropometrics, lung function, microbiology, CF-specific complications and chronic medications, hospitalisations, demand-oriented antibiotic therapies, and outcomes (death and transplants). Real world and pharmacovigilance studies have been published and additional research underway; there is a formal process for requesting access to the GCFR.</p>","PeriodicalId":19782,"journal":{"name":"Pharmacoepidemiology and Drug Safety","volume":"34 1","pages":"e70076"},"PeriodicalIF":2.4,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11706667/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142952123","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}