On identifying effective and superior drug combinations via Holm's procedure based on the min tests.

abstract：:Multiple testing problems in regulatory applications are often more challenging than the problems of handling a set of mathematical symbols representing multiple null hypotheses under testing. In the union-intersection setting, it is important to define a family of null hypotheses relevant to the clinical questions at...

journal_title：Journal of biopharmaceutical statistics

authors： Hung HM,Wang SJ

更新日期：2009-01-01 00:00:00

abstract：:It is well known that there is strong relationship between HIV viral load and CD4 cell counts in AIDS studies. However, the relationship between them changes during the course of treatment and may vary among individuals. During treatments, some individuals may experience terminal events such as death. Because the term...

journal_title：Journal of biopharmaceutical statistics

authors： Lu T,Wang M,Liu G,Dong GH,Qian F

更新日期：2016-01-01 00:00:00

abstract：:Failure to recognize the serious implications of heterogeneous correlations and disregard of the multiple test problem in interpreting the results from repeated measurements ANOVA of any single primary outcome measure can produce false-positive error rates that are more than five times the alpha level that is reported...

journal_title：Journal of biopharmaceutical statistics

authors： Overall JE,Doyle SR

更新日期：1996-03-01 00:00:00

abstract：:For approval of generic drugs, the U.S. Food and Drug Administration (FDA) requires the evidence of bioequivalence in average bioavailability be provided. This is based on the Fundmental Bioequivalence Assumption from FDA that if two drug products are shown to be bioequivalent, it is assumed that they are therapeutica...

journal_title：Journal of biopharmaceutical statistics

authors： Lu Y,Zhang ZZ,Chow SC

更新日期：2014-01-01 00:00:00

abstract：:We study the statistical efficiency for rising-dose designs in the context of first-in-human studies. Specifically, we identify a class of crossover designs that are appealing in terms of both subject safety and statistical efficiency and, for a three-period, two-panel design in such a class, we compare its A-efficien...

journal_title：Journal of biopharmaceutical statistics

authors： Yan Z,Hosmane B,Locke C

更新日期：2013-01-01 00:00:00

abstract：:We extend the development of the expression of the Fisher information matrix in nonlinear mixed effects models for designs evaluation. We consider the dependence of the marginal variance of the observations with the mean parameters and assume an heteroscedastic variance error model. Complex models with interoccasions ...

journal_title：Journal of biopharmaceutical statistics

authors： Retout S,Mentré F

更新日期：2003-05-01 00:00:00

abstract：:Exact overall significance levels for selected sample sizes and response probabilities are graphically displayed when a dichotomous variable is compared between a placebo and two or more active treatments. A Z-statistic with a pooled variance estimator is used as the test statistic and critical values are based on the...

journal_title：Journal of biopharmaceutical statistics

authors： Whaley FS

更新日期：2003-11-01 00:00:00

abstract：:I review the designs available for Phase I dose-finding studies of chemotherapeutic agents in cancer patients. The designs are based on the assumption that both efficacy and toxicity increase with dose, and thus attempt to minimize the number of patients treated at low doses, and also to minimize the chance that patie...

journal_title：Journal of biopharmaceutical statistics

authors： Potter DM

更新日期：2006-01-01 00:00:00

abstract：:In method comparison and reliability studies, it is often important to assess agreement between multiple measurements made by different methods, devices, laboratories, observers, or instruments. For continuous data, the concordance correlation coefficient (CCC) is a popular index for assessing agreement between multip...

journal_title：Journal of biopharmaceutical statistics

authors： Barnhart HX,Lokhnygina Y,Kosinski AS,Haber M

更新日期：2007-01-01 00:00:00

abstract：:Due to the potential impact of ethnic factors on clinical outcomes, the global registration of a new treatment is challenging. China and Japan often require local trials in addition to a multiregional clinical trial (MRCT) to support the efficacy and safety claim of the treatment. The impact of ethnic factors on the t...

journal_title：Journal of biopharmaceutical statistics

authors： Huang Q,Chen G,Yuan Z,Lan KK

更新日期：2012-09-01 00:00:00

abstract：:Sample size calculation based on normal approximations is often associated with the loss of statistical power for a single-arm trial with a time-to-event endpoint. Recently, Wu (2015) derived the exact variance for the one-sample log-rank test under the alternative and showed that a single-arm one-stage study based on...

journal_title：Journal of biopharmaceutical statistics

authors： Shan G

更新日期：2020-09-02 00:00:00

abstract：:In a clinical dose finding study with active control a new drug with several dose levels is compared with an active comparator drug. The main focus of such studies often lies on the estimation of a target dose that leads to the same efficacy as the control. This article investigates the finite sample properties of the...

journal_title：Journal of biopharmaceutical statistics

authors： Helms HJ,Benda N,Friede T

更新日期：2015-01-01 00:00:00

abstract：:The adverse events data of randomized clinical trials are often analyzed based on either crude incidence rates or exposure-adjusted incidence rates. These rates do not adequately account for an individual patient's profile of adverse events over the study period when an individual may remain in the trial after experie...

journal_title：Journal of biopharmaceutical statistics

authors： Siddiqui O

更新日期：2009-09-01 00:00:00

abstract：:An adaptive enrichment design is a randomized trial that allows enrollment criteria to be modified at interim analyses, based on a preset decision rule. When there is prior uncertainty regarding treatment effect heterogeneity, these trial designs can provide improved power for detecting treatment effects in subpopulat...

journal_title：Journal of biopharmaceutical statistics

authors： Fisher A,Rosenblum M,Alzheimer’s Disease Neuroimaging Initiative.

更新日期：2018-01-01 00:00:00

abstract：:In various pharmaceutical applications, repeated measurements are taken from each subject, and model parameters are estimated from the collected data. Examples include dose response modeling and PK/PD studies with serial blood sampling, among others. The quality of the information in an experiment is reflected in the ...

journal_title：Journal of biopharmaceutical statistics

authors： Gagnon R,Leonov S

更新日期：2005-01-01 00:00:00

abstract：:In this paper, a propensity score-integrated composite likelihood (PSCL) approach is developed for cases in which the control arm of a two-arm randomized controlled trial (RCT) (treated vs control) is augmented with patients from real-world data (RWD) containing both clinical outcomes and covariates at the patient-lev...

journal_title：Journal of biopharmaceutical statistics

authors： Chen WC,Wang C,Li H,Lu N,Tiwari R,Xu Y,Yue LQ

更新日期：2020-05-03 00:00:00

abstract：:The potency of a batch of drug product needs to meet a release limits at the time of release so that the potency at the end of shelf life remains above the lower registration limit (LRL). This article discusses two methods which determine the release limits such that the chance to fail LRL at the end of shelf life of ...

journal_title：Journal of biopharmaceutical statistics

authors： Wei GC

更新日期：1998-03-01 00:00:00

abstract：:For the assessment of biosimilarity of biosimilar products, the United States (US) Food and Drug Administration (FDA) proposed a stepwise approach for providing the totality-of-the-evidence of similarity between a proposed biosimilar product and a US-licensed (reference) product. The stepwise approach starts with the ...

journal_title：Journal of biopharmaceutical statistics

authors： Wang T,Chow SC

更新日期：2017-01-01 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：: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：:We present a new computational method for identifying regulated pathway components in transcript profiling (TP) experiments by evaluating transcriptional activity in the context of known biological pathways. We construct a graph representing thousands of protein functional relationships by integrating knowledge from p...

journal_title：Journal of biopharmaceutical statistics

authors： Pradines J,Rudolph-Owen L,Hunter J,Leroy P,Cary M,Coopersmith R,Dancik V,Eltsefon Y,Farutin V,Leroy C,Rees J,Rose D,Rowley S,Ruttenberg A,Wieghardt P,Sander C,Reich C

更新日期：2004-08-01 00:00:00

abstract：:The Food and Drug Administration of the United States issued a draft guidance on adaptive design clinical trials in February 2010. This draft guidance has attracted a lot of attention because of the increasing interest in adaptive trials by the pharmaceutical industry in recent years. In this paper, we report on highl...

journal_title：Journal of biopharmaceutical statistics

authors： Chuang-Stein C,Beltangady M

更新日期：2010-11-01 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：:Receptor occupancy (RO) PET is a non-invasive way to determine drug on target. Given the complexity of procedures, long acquisition times, and high cost, ligand displacement imaging trials often have a limited size and produce sparse RO results over the time course of the blocking drug. To take the best advantage of t...

journal_title：Journal of biopharmaceutical statistics

authors： Vandenhende F,Renard D,Nie Y,Kumar A,Miller J,Tauscher J,Witcher J,Zhou Y,Wong DF

更新日期：2008-01-01 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 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：:A useful testing strategy is proposed for a confirmatory phase III clinical trial. It consists of a combined test of superiority and test of equivalence, and it is easy to apply. By introducing the strategy, we can perform a post hoc analysis in a confirmatory experiment. Thus a more flexible decision will be possible...

journal_title：Journal of biopharmaceutical statistics

authors： Morikawa T,Yoshida M

更新日期：1995-11-01 00:00:00

abstract：:In traditional dose-finding studies, dose-limiting toxicity (DLT) is determined within a fixed time observation window where DLT is often defined as a binary outcome. In the setting of oncology dose-finding trials, often patients in advanced stage of diseases are enrolled. Therefore, disease progression may occur with...

journal_title：Journal of biopharmaceutical statistics

authors： Biard L,Cheng B,Manji GA,Lee SM

更新日期：2020-09-15 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：: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