Adjustment for unbalanced sample size for analytical biosimilar equivalence assessment.

abstract：:In randomized controlled trials, patients are recruited and randomly allocated to treatments. Patients are never randomly sampled from large population of patients on treatments under study. Therefore, it is important to consider the design and statistical analysis based on the randomization model. In this article, we...

journal_title：Journal of biopharmaceutical statistics

authors： Hasegawa T,Tango T

更新日期：2009-01-01 00:00:00

abstract：:A test and a reference analytical method are usually compared for agreement based on paired data obtained from several independent subjects. Bias between two methods can be classified as constant and proportional. In this article, we provide an approach for maximum likelihood estimation of total bias between two metho...

journal_title：Journal of biopharmaceutical statistics

authors： Magari RT

更新日期：2004-11-01 00:00:00

abstract：:Multiple comparisons are commonly seen in clinical trials and many other fields. An example, which is the focus of this paper, is the comparison of several test treatments (possibly different doses of a compound) with placebo (control). It is well known that steps must be taken to control the type I error rate when mu...

journal_title：Journal of biopharmaceutical statistics

authors： Chen M,Kianifard F

更新日期：1997-07-01 00:00:00

abstract：:Assessment of quality of life (QOL) in clinical trials becomes a challenging task from the viewpoint of clinical biostatistics. The responses of the items for measuring QOL indices usually vary widely from patient to patient and from time to time. Measurement errors might be present in the responses of the items, and ...

journal_title：Journal of biopharmaceutical statistics

authors： Siddiqui O,Ali MW

更新日期：1999-11-01 00:00:00

abstract：:Investigating the prevalence of a disease is an important topic in medical studies. Such investigations are usually based on the classification results of a group of subjects according to whether they have the disease. To classify subjects, screening tests that are inexpensive and nonintrusive to the test subjects are...

journal_title：Journal of biopharmaceutical statistics

authors： Tang ML,Qiu SF,Poon WY,Tang NS

更新日期：2012-01-01 00:00:00

abstract：:In the early stages of traditional drug development, the frequency of dosing (e.g., QD, BID, etc.) is typically determined by the pharmacokinetic properties of a compound. After an appropriate dose frequency is chosen, the magnitude of dose is then evaluated via parallel-group dose-response trials. For some drugs, how...

journal_title：Journal of biopharmaceutical statistics

authors： Hafner KB,Ruberg SJ

更新日期：1996-07-01 00:00:00

abstract：:The use of adaptive methods in clinical development has become very popular in recent years due to its flexibility in modifying trial procedures and/or statistical procedures of on-going clinical trials. Modifications to trial procedures are usually documented by protocol amendments. However, the actual patient popula...

journal_title：Journal of biopharmaceutical statistics

authors： Chow SC,Shao J

更新日期：2005-01-01 00:00:00

abstract：:An important application of confirmatory adaptive designs is the data-driven selection of treatment arms in multi-armed trials. A general methodology for adaptive designs is based on the combination testing principle. Using this principle, selection of treatment arms in multi-armed designs, recalculation of sample siz...

journal_title：Journal of biopharmaceutical statistics

authors： Wassmer G

更新日期：2011-07-01 00:00:00

abstract：:The small n, Sequential, Multiple Assignment, Randomized Trial (snSMART) is a two-stage clinical trial design for rare diseases motivated by the comparison of three active treatments for isolated skin vasculitis in the ongoing clinical trial ARAMIS (a randomized multicenter study for isolated skin vasculitis, NCT09239...

journal_title：Journal of biopharmaceutical statistics

authors： Wei B,Braun TM,Tamura RN,Kidwell K

更新日期：2020-09-06 00:00:00

abstract：:The study of drug combinations has become important in drug development due to its potential for efficacy at lower, less toxic doses and the need to move new therapies rapidly into clinical trials. The goal is to identify which combinations are additive, synergistic, or antagonistic. Although there exists statistical ...

journal_title：Journal of biopharmaceutical statistics

authors： Fang HB,Tian GL,Li W,Tan M

更新日期：2009-07-01 00:00:00

abstract：:The widely used distinction of Little and Rubin (1) about types of randomness for missing data presents difficulties in its application to dropouts in longitudinal repeated measurement studies. In its place, a new typology of randomness for dropouts is proposed that relies on using a survival model for the dropout pro...

journal_title：Journal of biopharmaceutical statistics

authors： Lindsey JK

更新日期：2000-11-01 00:00:00

abstract：:During preclinical drug development, the immune system is specifically evaluated after prolonged treatment with drug candidates, because the immune system may be an important target system. The response of antibodies against a T-cell-dependent antigen is recommenced by the FDA and EMEA for the evaluation of immunosupp...

journal_title：Journal of biopharmaceutical statistics

authors： Shkedy Z,Straetemans R,Molenberghs G,Desmidt M,Vinken P,Goeminne N,Coussement W,Van Den Poel B,Bijnens L

更新日期：2005-01-01 00:00:00

abstract：:Hypoglycemia is a major safety concern for diabetic patients. Hypoglycemic events can be modeled based on time to recurrent events or count data. In this article, we evaluated a gamma frailty model with variance estimated by the inverse of observed Fisher information matrix, a gamma frailty model with the sandwich var...

journal_title：Journal of biopharmaceutical statistics

authors： Ma C,Qu Y,Fu H

更新日期：2020-05-18 00:00:00

abstract：:Pharmaceutical product development culminates in confirmatory trials whose evidence for the product's efficacy and safety supports regulatory approval for marketing. Regulatory agencies in countries whose patients were not included in the confirmatory trials often require confirmation of efficacy and safety in their p...

journal_title：Journal of biopharmaceutical statistics

authors： Gould AL,Jin T,Zhang LX,Wang WW

更新日期：2012-09-01 00:00:00

abstract：:Drug-related side effects are one of the leading causes of death and illness in the developed world. Finding genes that modify drug response has the potential to significantly improve drug delivery, by identifying both individuals that can benefit from therapy and those at increased risk of harm. We present a simple a...

journal_title：Journal of biopharmaceutical statistics

authors： He M,Allen A

更新日期：2010-03-01 00:00:00

abstract：:In confidence interval estimation of the difference between two proportions with overdispersion due to positive correlations, the usual asymptotic normality-based method generally has lower coverage rates than desired, especially when sample size is moderate. Applying the concept of effective sample size to existing m...

journal_title：Journal of biopharmaceutical statistics

authors： Chen C,Li J,Zhou Z

更新日期：2004-05-01 00:00:00

abstract：:The Youden index, a main summary index for the receiver operating characteristic (ROC) curve, is a comprehensive measurement for the effectiveness of a diagnostic test. For a continuous-scale diagnostic test, the optimal cut point for positive disease is the cut point leading to the maximization of the sum of sensitiv...

journal_title：Journal of biopharmaceutical statistics

authors： Zhou H,Qin G

更新日期：2012-01-01 00:00:00

abstract：:We introduce the idea of a design to detect signals of efficacy in early phase clinical trials. Such a design features three possible decisions: to kill the compound; to continue with staged development; or to continue with accelerated development of the compound. We describe how such studies improve the trade-off bet...

journal_title：Journal of biopharmaceutical statistics

authors： Brown MJ,Chuang-Stein C,Kirby S

更新日期：2012-01-01 00:00:00

abstract：:A framework is proposed for making quality predictions in situations for which only systematically inaccurate data are available. The predictions are based on the systematically inaccurate data, complete data from similar situations, and expert knowledge. The proposed predictive model is well suited to functional data...

journal_title：Journal of biopharmaceutical statistics

authors： Haaland B,Chiang AY

更新日期：2014-01-01 00:00:00

abstract：:Frequentist design for two-arm randomized Phase II clinical trials with outcomes from the exponential dispersion family was proposed previously, where the total sample sizes are minimized under multiple constraints on the standard errors of the estimated group means and their difference. This design was generalized fr...

journal_title：Journal of biopharmaceutical statistics

authors： Jiang W,Wick JA,He J,Mahnken JD,Mayo MS

更新日期：2018-01-01 00:00:00

abstract：:In literature, there are a few unified approaches to test proof of concept and estimate a target dose, including the multiple comparison procedure using modeling approach, and the permutation approach proposed by Klingenberg. We discuss and compare the operating characteristics of these unified approaches and further ...

journal_title：Journal of biopharmaceutical statistics

authors： Tang Y,Cai C,Sun L,He J

更新日期：2017-01-01 00:00:00

abstract：:In this study, a Bayesian approach was suggested to estimate a change-point according to a covariate threshold when some patients never experienced the event of interest. Gibbs sampler algorithm with latent binary cure indicators was used to simplify the implementation of Markov chain Monte Carlo method. Then, the acc...

journal_title：Journal of biopharmaceutical statistics

authors： Shamsi F,Baghestani AR,Binesh F

更新日期：2020-03-01 00:00:00

abstract：:Recently, two CPMP Points to Consider, one on adjustment for baseline covariates and the other on multiplicity issues in clinical trials, have included recommendations on the use of subgroup analysis for regulatory purposes. However, despite their regular use and regulatory attention, the validity and nature of subgro...

journal_title：Journal of biopharmaceutical statistics

authors： Grouin JM,Coste M,Lewis J

更新日期：2005-01-01 00:00:00

abstract：:To confirm results obtained from local evaluation at investigational centers, many oncology studies utilize blinded independent central review (BICR) to make assessments of the primary endpoint, progression-free survival (PFS). The comparison of data often leads to large discordances between these observations, castin...

journal_title：Journal of biopharmaceutical statistics

authors： Mannino FV,Amit O,Lahiri S

更新日期：2013-01-01 00:00:00

abstract：:An ability to predict the metabolic fate of a drug is important to drug design. Programs for predicting drug metabolites are becoming available, as are databases that will facilitate the development of such programs. Objective analysis of the performance of these programs will require statistical methods. The developm...

journal_title：Journal of biopharmaceutical statistics

authors： Johnson M

更新日期：1991-01-01 00:00:00

abstract：:All too often in clinical trials the assessment of quality of life is seen as a bolt-on study. Consequently insufficient consideration is often given to its design, collection, analysis and presentation, and its impact on the trial results and on clinical practice is minimal. In many trials quality of life is a key en...

journal_title：Journal of biopharmaceutical statistics

authors： Stephens R

更新日期：2004-02-01 00:00:00

abstract：:We studied the sensitivity of the number of unique design points and their placement, in Bayesian optimal designs for pharmacokinetic models, with respect to the magnitude of prior uncertainty. We used two and three-parameter pharmacokinetic models with fixed and mixed effects and two Bayesian optimal design criteria,...

journal_title：Journal of biopharmaceutical statistics

authors： Dokoumetzidis A,Aarons L

更新日期：2007-01-01 00:00:00

abstract：:Rank-based test procedures in censored survival data differ considerably in their sensitivity to various alternatives to the hypothesis of equality of underlying distributions. Procedures based on the simultaneous use of multiple statistics provide an appealing approach to obtaining more global sensitivity while maint...

journal_title：Journal of biopharmaceutical statistics

authors： Yang P,Fleming TR

更新日期：2006-01-01 00:00:00

abstract：:A common question in clinical studies is how to use historical data from earlier studies, leveraging relevant information into the design and analysis of a new study. Bayesian approaches are particularly well-suited to this task, with their natural ability to borrow strength across data sources. In this paper, we prop...

journal_title：Journal of biopharmaceutical statistics

authors： Li JX,Chen WC,Scott JA

更新日期：2016-01-01 00:00:00

abstract：:The world of medical devices while highly diverse is extremely innovative, and this facilitates the adoption of innovative statistical techniques. Statisticians in the Center for Devices and Radiological Health (CDRH) at the Food and Drug Administration (FDA) have provided leadership in implementing statistical innova...

journal_title：Journal of biopharmaceutical statistics

authors： Campbell G,Yue LQ

更新日期：2016-01-01 00:00:00