Phase I studies of chemotherapeutic agents in cancer patients: a review of the designs.

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

abstract：:Multipopulation tailoring trials provide a trial design option that supports the realization of tailored therapeutics or personalized medicine. Several recent publications have focused on statistical and clinical considerations that arise in these trials that are designed to study the overall treatment effect in a pop...

journal_title：Journal of biopharmaceutical statistics

authors： Millen BA,Dmitrienko A,Song G

更新日期：2014-01-01 00:00:00

abstract：:There are minimal standards for the processing of whole blood components, and to apply those standards requires a system of quality assurances. Excessive indications of failures in compliance trigger inspections and other remedial actions, but the demarcation of what is excessive is a critical issue. Issues of low vol...

journal_title：Journal of biopharmaceutical statistics

authors： Lachenbruch PA,Foulkes MA,Williams AE,Epstein JS

更新日期：2005-01-01 00:00:00

abstract：:In clinical trials, it is important to set up a design to reach a decision on effectiveness of a drug in treating a disease with the loss of the minimum number of patients. Group sequential designs are very beneficial on this point. However, the proportional hazards assumption must hold to work under a group sequentia...

journal_title：Journal of biopharmaceutical statistics

authors： Demirhan H,Demirhan YP,Bacanli S

更新日期：2013-03-11 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：:After a group sequential test, the naive confidence interval (CI) is usually biased in the sense that it does not cover the true parameter at the correct nominal level. Furthermore, when the stopping time is taken into account, the actual conditional confidence coverage probability can be much less accurate. In this a...

journal_title：Journal of biopharmaceutical statistics

authors： Fan X,DeMets DL

更新日期：2006-01-01 00:00:00

abstract：:Rubinstein et al. presented a procedure for determining the required duration of accrual for a clinical trial comparing the survival distributions of two treatments using a classical hypothesis testing formulation. Here their testing procedure is modified in two ways. First, the asymptotic variances used in computatio...

journal_title：Journal of biopharmaceutical statistics

authors： Bristol DR

更新日期：1993-09-01 00:00:00

abstract：:A randomized, active-control clinical trial setting with the objective of testing noninferiority for a continuous response variable is considered. Noninferiority margin is based on the concept of preserving a certain fraction of the active control effect. Noninferiority is established if the ratio of the lower (upper)...

journal_title：Journal of biopharmaceutical statistics

authors： Chen M,Kianifard F,Dhar SK

更新日期：2006-05-01 00:00:00

abstract：:Clinical trials with data-driven decision rules often pursue multiple clinical objectives such as the evaluation of several endpoints or several doses of an experimental treatment. These complex analysis strategies give rise to "multivariate" multiplicity problems with several components or sources of multiplicity. A ...

journal_title：Journal of biopharmaceutical statistics

authors： Kordzakhia G,Dmitrienko A,Ishida E

更新日期：2018-01-01 00:00:00

abstract：:How long a dementia patient is cared for in the home before admission to a nursing home depends on the state of the patient and the state of the caregiver. Using 5-year follow-up data, the times until entry to nursing home and until death are modeled using a Cox survival model in which patient and caregiver variables ...

journal_title：Journal of biopharmaceutical statistics

authors： McGilchrist CA,Brodaty H,Peters KE,Harris L

更新日期：1994-03-01 00:00:00

abstract：:We consider the problem of estimating a biomarker-based subgroup and testing for treatment effect in the overall population and in the subgroup after the trial. We define the best subgroup as the subgroup that maximizes the power for comparing the experimental treatment with the control. In the case of continuous outc...

journal_title：Journal of biopharmaceutical statistics

authors： Joshi N,Fine J,Chu R,Ivanova A

更新日期：2019-01-01 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 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：: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：:According to ICH Q6A (1999), a specification is defined as a list of tests, references to analytical procedures, and appropriate acceptance criteria, which are numerical limits, ranges, or other criteria for the tests described. For drug products, specifications usually consist of test methods and acceptance criteria ...

journal_title：Journal of biopharmaceutical statistics

authors： Dong X,Tsong Y,Shen M

更新日期：2015-01-01 00:00:00

abstract：:A model-based approach is developed to estimate the distribution of time from seroconversion to diagnosis with acquired immunodeficiency syndrome (AIDS) as a function of selected time-dependent covariates. The approach is applied to longitudinal data collected over 4 years of follow-up from 450 men seropositive for th...

journal_title：Journal of biopharmaceutical statistics

authors： Dunlop DD,Tamhane AC,Chmiel JS,Phair JP

更新日期：1994-07-01 00:00:00

abstract：:In many clinical trials for chronic conditions a run-in period is used prior to randomization. Often, only those participants who meet certain criteria during the run-in phase go on to get randomized. The others, along with the information that they might have provided, are excluded from the study. This exclusion of t...

journal_title：Journal of biopharmaceutical statistics

authors： Berger VW,Vali B

更新日期：2011-03-01 00:00:00

abstract：:Statistical methodologies for human abuse potential studies are rarely evaluated. Human abuse potential studies assess whether test drugs produce positive and negative subjective responses on abuse-related measures using volunteers with histories of recreational drug use. These studies typically have a randomized, dou...

journal_title：Journal of biopharmaceutical statistics

authors： Chen L,Bonson KR

更新日期：2013-03-11 00:00:00

abstract：:In the era of precision medicine, drugs are increasingly developed to target subgroups of patients with certain biomarkers. In large all-comer trials using a biomarker stratified design, the cost of treating and following patients for clinical outcomes may be prohibitive. With a fixed number of randomized patients, th...

journal_title：Journal of biopharmaceutical statistics

authors： Wang X,Zhou J,Wang T,George SL

更新日期：2018-01-01 00:00:00

abstract：:The purpose of the research is to develop a statistical decision support algorithm for patients who may benefit from Adjuvant Cisplatin/Vinorelbine (ACT) and improve their survival rates. Genome-wide microarray data are used to identify feasible sets of genes and probe sets that constitute the gene signature. The data...

journal_title：Journal of biopharmaceutical statistics

authors： Moon H,Chao T,Ahn H

更新日期：2020-05-03 00:00:00

abstract：:During a clinical trial, balancing statistical and ethical considerations are important. Response-adaptive randomization methods use the information from past patients to increase the probability of the next patient receiving the better treatment while avoiding the statistical concern of selection bias. We compared th...

journal_title：Journal of biopharmaceutical statistics

authors： Piccorelli AV,Fraker SA

更新日期：2018-01-01 00:00:00

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：: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：:A main challenge in molecular diagnostic research is to accurately evaluate the performance of a new nucleic acid amplification test when the reference standard is imperfect. Several approaches, such as discrepant analysis, composite reference standard (CRS) method, or latent class analysis (LCA), are commonly applied...

journal_title：Journal of biopharmaceutical statistics

authors： Tang S,Hemyari P,Canchola JA,Duncan J

更新日期：2018-01-01 00:00:00

abstract：:One of the most critical decision points in clinical development is Go/No-Go decision-making after a proof-of-concept study. Traditional decision-making relies on a formal hypothesis testing with control of type I and type II error rates, which is limited by assessing the strength of efficacy evidence in a small isola...

journal_title：Journal of biopharmaceutical statistics

authors： Huang B,Talukder E,Han L,Kuan PF

更新日期：2019-01-01 00:00:00

abstract：:The Log-odds ratio for 2 × 2 contingency tables is often approximated by a normal distribution with an approximated variance. Hwang and Biswas (2008) illustrated that the standard expression for the variance should be modified in the presence of correlation. They also provided an adjustment to this variance expression...

journal_title：Journal of biopharmaceutical statistics

authors： Biswas A,Hwang JS

更新日期：2011-01-01 00:00:00

abstract：:In preclinical tumor xenograft experiments, the antitumor activity of the tested agents is often assessed by endpoints such as tumor doubling time, tumor growth delay (TGD), and log10 cell kill (LCK). In tumor xenograft literature, the values of these endpoints are presented without any statistical inference, which ig...

journal_title：Journal of biopharmaceutical statistics

authors： Wu J

更新日期：2011-05-01 00:00:00

abstract：:Nonparametric versions of normal theory step-down multiple-test procedures for inferring minimum effective dose (see Tamhane et al. (1)) were developed and studied by Monte Carlo simulation. Two types of step-down testing procedures were examined. For both procedures, pairwise, linear, or Helmert contrasts of mean ran...

journal_title：Journal of biopharmaceutical statistics

authors： Sidik K,Morris RW

更新日期：1999-05-01 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：:The aim of this work is to quantitatively assess the impact of structural model misspecifications on the estimates of mean and interindividual variability of clearance in the context of population approaches. This assessment is conducted from simulated datasets. Our results show that impact magnitude of model misspeci...

journal_title：Journal of biopharmaceutical statistics

authors： Merlé Y,Aouimer A,Tod M

更新日期：2004-02-01 00:00:00