A5033784083- Statistical Methods in Clinical Trials
- Health Systems, Economic Evaluations, Quality of Life
- Pharmaceutical studies and practices
- Advanced Causal Inference Techniques
- Dermatology and Skin Diseases
- Statistical Methods and Bayesian Inference
- Allergic Rhinitis and Sensitization
- Statistical Methods and Inference
- Pharmaceutical Economics and Policy
- Asthma and respiratory diseases
- Optimal Experimental Design Methods
- Computational Drug Discovery Methods
- Biosimilars and Bioanalytical Methods
- Genetic Associations and Epidemiology
- Food Allergy and Anaphylaxis Research
- Bayesian Methods and Mixture Models
- COVID-19 epidemiological studies
- SARS-CoV-2 and COVID-19 Research
- Adolescent and Pediatric Healthcare
- Aging and Gerontology Research
- Child and Adolescent Health
- Autoimmune Bullous Skin Diseases
- Statistical Distribution Estimation and Applications
- Childhood Cancer Survivors' Quality of Life
- Urticaria and Related Conditions
Pfizer (United States)
2021-2024
Eli Lilly (United States)
2016-2021
Center for Food Safety and Applied Nutrition
2015-2016
United States Food and Drug Administration
2016
Background Baricitinib, an oral selective Janus kinase 1 and 2 inhibitor, effectively reduced atopic dermatitis (AD) severity in a phase II study with concomitant topical corticosteroids. Objectives To evaluate the efficacy safety of baricitinib patients moderate-to-severe AD who had inadequate response to therapies. Methods In two independent, multicentre, double-blind, III monotherapy trials, BREEZE-AD1 BREEZE-AD2, adults were randomized : once-daily placebo, mg, or 4 mg for 16 weeks....
Baricitinib, an oral selective Janus kinase 1 and 2 inhibitor, effectively reduced disease severity in moderate to severe atopic dermatitis (AD) phase 3 monotherapy studies.
Abstract Background Janus kinase (JAK) inhibition is a new mode of action in atopic dermatitis (AD); clarity about drug class safety considerations the context AD important. Baricitinib, an oral, reversible, selective inhibitor JAK1/JAK2, late‐stage development for adult patients with moderate‐to‐severe AD. Objective To report pooled data baricitinib clinical program including long‐term extension (LTE) studies. Methods This analysis included patient‐level from six double‐blinded, randomized,...
BackgroundThere are no treatments approved by the Food and Drug Administration for alopecia areata.ObjectiveTo evaluate efficacy safety of baricitinib in patients with ≥50% scalp hair loss a phase 2 study adults areata (BRAVE-AA1).MethodsPatients were randomized 1:1:1:1 to receive placebo or 1 mg, 4 mg once daily. Two consecutive interim analyses performed after all completed weeks 12 36 had discontinued treatment prior these time points. The primary endpoint was proportion achieving...
Children represent a large underserved population of "therapeutic orphans," as an estimated 80% children are treated off-label. However, pediatric drug development often faces substantial challenges, including economic, logistical, technical, and ethical barriers, among others. Among many efforts trying to remove these increased recent attention has been paid extrapolation; that is, the leveraging available data from adults or older age groups draw conclusions for population. The Bayesian...
Conventional analyses of a composite multiple time-to-event outcomes use the time to first event. However, event may not be most important outcome. To address this limitation, generalized pairwise comparisons and win statistics (win ratio, odds, net benefit) have become popular been applied clinical trial practice. benefit typically used separately. In article, we examine these three jointly for outcomes. First, explain relation point estimates variances among statistics, between...
Existing statutes in the United States and Europe require manufacturers to demonstrate evidence of effectiveness through conduct adequate well‐controlled studies obtain marketing approval a therapeutic product. What constitutes is usually interpreted as randomized controlled trials (RCTs). However, these are sometimes unfeasible because their size, duration, cost, patient preference, or some cases, ethical concerns. For example, RCTs may not be fully powered rare diseases infections caused...
The win ratio method has received much attention in methodological research, ad hoc analyses, and designs of prospective studies. As the primary analysis, it supported approval tafamidis for treatment cardiomyopathy to reduce cardiovascular mortality cardiovascular-related hospitalization. However, its dependence on censoring is a potential shortcoming. In this article, we propose inverse-probability-of-censoring weighting (IPCW) adjusted statistic (i.e., IPCW-adjusted statistic) overcome...
Drug developers are required to demonstrate substantial evidence of effectiveness through the conduct adequate and well‐controlled (A&WC) studies obtain marketing approval their medicine. What constitutes A&WC is interpreted as randomized controlled trials (RCTs). However, these sometimes unfeasible because size, duration, cost. One way reduce sample size leverage information on control a prior. consideration when forming data‐driven prior consistency external current data. It...
The cost and time of pharmaceutical drug development continue to grow at rates that many say are unsustainable. These trends have enormous impact on what treatments get patients, when they them how used. statistical framework for supporting decisions in regulated clinical new medicines has followed a traditional path frequentist methodology. Trials using hypothesis tests "no treatment effect" done routinely, the p-value < 0.05 is often determinant constitutes "successful" trial. Many drugs...
Abstract The win odds and the net benefit are related directly to each other indirectly, through ties, ratio. These three statistics test same null hypothesis of equal probabilities between two groups. They provide similar p ‐values powers, because Z‐values their statistical tests approximately equal. Thus, they can complement one another show strength a treatment effect. In this article, we that estimated variances also regardless ties or indirectly ties. Since its introduction in 2018,...
In many orphan diseases and pediatric indications, the randomized controlled trials may be infeasible because of their size, duration, cost. Leveraging information on control through a prior can potentially reduce sample size. However, unless an objective is used to impose complete ignorance for parameter being estimated, it results in biased estimates inflated type-I error. Hence, essential assess both confirmatory supplementary knowledge available during construction avoid "cherry-picking"...