当前位置： SCI文献检索 > Journal of Biopharmaceutical Statistics期刊下所有文献 > Balancing statistical and ethical considerations in planning clinical trials: recommendations for response-adaptive randomization urn designs.

Balancing statistical and ethical considerations in planning clinical trials: recommendations for response-adaptive randomization urn designs.

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 three response-adaptive randomization urn designs, Randomized Play-the-Winner, Modified Play-the-Winner, and Birth-and-Death Urn with Immigration, to the traditional equal allocation design with respect to power and allocation of patients to the better treatment. Because these designs have been described separately, our motivation was to systematically compare them and provide recommendations. With simulations, we varied sample size and combinations of treatment and control success probabilities. We also compared the response-adaptive randomization designs using exact distribution algorithms and applied them to past clinical trial data that used an equal allocation design. We conclude that Modified Play-the-Winner tends to be unpredictable and can result in allocation of all of the patients to the better treatment. Randomized Play-the-Winner allocates more patients to the better treatment than Birth-and-Death Urn with Immigration, but Birth-and-Death Urn with Immigration is more consistent in its allocations. Randomized Play-the-Winner and Birth-and-Death Urn with Immigration produce allocations that have comparable powers to equal allocation design.

journal_name

J Biopharm Stat

journal_title

Journal of biopharmaceutical statistics

authors

Piccorelli AV,Fraker SA

doi

10.1080/10543406.2018.1437172

subject

Has Abstract

pub_date

2018-01-01 00:00:00

pages

1105-1118

issue

6

eissn

1054-3406

issn

1520-5711

journal_volume

28

pub_type

杂志文章

相关文献

Journal of Biopharmaceutical Statistics文献大全
  • Two-stage optimal designs based on exact variance for a single-arm trial with survival endpoints.

    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

    pub_type： 杂志文章

    doi：10.1080/10543406.2020.1730869

    authors： Shan G

    更新日期：2020-09-02 00:00:00

  • Detection of activity centers in cellular pathways using transcript profiling.

    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

    pub_type： 杂志文章

    doi：10.1081/BIP-200025678

    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

  • Nonparametric tests for evaluation of biosimilarity in variability of follow-on biologics.

    abstract：:As more biologic products are going off patent protection, the development of follow-on biologic products (also known as biosimilars) has gained much attention from both the biotechnology industry and regulatory agencies. Unlike small molecules, the development of biologic products is not only more complicated but als...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2014.941991

    authors： Zhang N,Yang J,Chow SC,Chi E

    更新日期：2014-01-01 00:00:00

  • Dose finding with the sequential parallel comparison design.

    abstract：:The sequential parallel comparison design (SPCD) is a two-stage design recommended for trials with possibly high placebo response. A drug-placebo comparison in the first stage is followed in the second stage by placebo nonresponders being re-randomized between drug and placebo. We describe how SPCD can be used in tria...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2014.924960

    authors： Wang JJ,Ivanova A

    更新日期：2014-01-01 00:00:00

  • Mixture-based gatekeeping procedures in adaptive clinical trials.

    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

    pub_type： 杂志文章

    doi：10.1080/10543406.2017.1399901

    authors： Kordzakhia G,Dmitrienko A,Ishida E

    更新日期：2018-01-01 00:00:00

  • Further developments of the Fisher information matrix in nonlinear mixed effects models with evaluation in population pharmacokinetics.

    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

    pub_type： 杂志文章

    doi：10.1081/BIP-120019267

    authors： Retout S,Mentré F

    更新日期：2003-05-01 00:00:00

  • Test procedures for disease prevalence with partially validated data.

    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

    pub_type： 杂志文章

    doi：10.1080/10543406.2010.544527

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

    更新日期：2012-01-01 00:00:00

  • Group Sequential Design for Randomized Phase III Trials under the Weibull Model.

    abstract：:In this article, a parametric sequential test is proposed under the Weibull model. The proposed test is asymptotically normal with an independent increment structure. The sample size for a fixed sample test is derived for the purpose of group sequential trial design. In addition, a multi-stage group sequential procedu...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2014.971165

    authors： Wu J,Xiong X

    更新日期：2015-01-01 00:00:00

  • Comparison of concordance correlation coefficient and coefficient of individual agreement in assessing agreement.

    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

    pub_type： 杂志文章

    doi：10.1080/10543400701329497

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

    更新日期：2007-01-01 00:00:00

  • An evaluation of increasing sample size based on conditional power.

    abstract：:We evaluate properties of sample size re-estimation (SSR) designs similar to the promising zone design considered by Mehta and Pocock (2011). We evaluate these designs under the assumption of a true effect size of 1.1 down to 0.4 of the protocol-specified effect size by six measures: 1. The probability of a sample siz...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2017.1289943

    authors： Gaffney M,Ware JH

    更新日期：2017-01-01 00:00:00

  • Sample size considerations for historical control studies with survival outcomes.

    abstract：:Historical control trials (HCTs) are frequently conducted to compare an experimental treatment with a control treatment from a previous study, when they are applicable and favored over a randomized clinical trial (RCT) due to feasibility, ethics and cost concerns. Makuch and Simon developed a sample size formula for h...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2015.1052495

    authors： Zhu H,Zhang S,Ahn C

    更新日期：2016-01-01 00:00:00

  • Dose-time-response modeling using negative binomial distribution.

    abstract：:People exposed to certain diseases are required to be treated with a safe and effective dose level of a drug. In epidemiological studies to find out an effective dose level, different dose levels are applied to the exposed and a certain number of cures is observed. Negative binomial distribution is considered to fit o...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2013.834916

    authors： Roy M,Choudhury K,Islam MM,Matin MA

    更新日期：2013-01-01 00:00:00

  • The analysis, interpretation, and presentation of quality of life data.

    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

    pub_type： 杂志文章

    doi：10.1081/BIP-120028506

    authors： Stephens R

    更新日期：2004-02-01 00:00:00

  • A quality by design approach for longitudinal quality attributes.

    abstract：:The concept of quality by design (QbD) as published in ICH-Q8 is currently one of the most recurrent topics in the pharmaceutical literature. This guideline recommends the use of information and prior knowledge gathered during pharmaceutical development studies to provide a scientific rationale for the manufacturing p...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2014.979197

    authors： Lebrun P,Giacoletti K,Scherder T,Rozet E,Boulanger B

    更新日期：2015-01-01 00:00:00

  • On Enrichment Strategies for Biomarker Stratified Clinical Trials.

    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

    pub_type： 杂志文章

    doi：10.1080/10543406.2017.1379532

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

    更新日期：2018-01-01 00:00:00

  • Statistical Properties of the Design for Simultaneous Global Drug Development Program.

    abstract：:Recently, a design was proposed for the Simultaneous Global Drug Development Program (SGDDP) to assess the impact of ethnic factors on the effect of a new treatment for a targeted ethnic (TE) population. It used weighted Z tests to combine the information collected from the TE and non-TE (NTE) subgroups in the SGDDP b...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2014.971166

    authors： Liu K,Yuan Z,Chen G,Huang Q,Wenrich J

    更新日期：2015-01-01 00:00:00

  • Interval estimation of the proportion ratio in repeated binary measurements under a stratified randomized clinical trial with noncompliance.

    abstract：:The proportion ratio (PR) of a positive response between an experimental treatment and a standard treatment (or placebo) is often used to measure the relative treatment efficacy in a randomized clinical trial (RCT). For ethical reasons, it is almost inevitable to encounter some patients not complying with their assign...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2010.508139

    authors： Lui KJ,Chang KC

    更新日期：2012-01-01 00:00:00

  • Potential use of the scan statistic for quality control in blood product manufacturing.

    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

    pub_type： 杂志文章

    doi：10.1081/BIP-200048790

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

    更新日期：2005-01-01 00:00:00

  • A summary statistic for measuring change from baseline.

    abstract：:A statistic, W, for measuring change from baseline is developed. Its distribution is found. Simulations using W and analysis of covariance (ANCOVA) are run and the results are compared. W is found to be less powerful than ANCOVA, yet is not seen to suffer some of the ill effects to which ANCOVA can fall prey, namely b...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543409708835187

    authors： Donahue RM

    更新日期：1997-05-01 00:00:00

  • Ratio of means vs. difference of means as measures of superiority, noninferiority, and average bioequivalence.

    abstract：:Ratio of means (ROM) and difference of means (DOM) are often used in a superiority, noninferiority (NI), or average bioequivalence (ABE) test to evaluate whether the test mean is superior, NI, or equivalent to the reference (placebo or active control) mean. The literature provides recommendations regarding how to choo...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2016.1265536

    authors： Sun W,Grosser S,Tsong Y

    更新日期：2017-01-01 00:00:00

  • Analytical expressions for combining population pharmacokinetic parameters from different studies.

    abstract：:We provide a set of formulas that allow the combination of separately performed analyses of population pharmacokinetic (PK) studies, without any further computational effort. More specifically, given the point estimates and uncertainties of two population PK analyses, the formulas provide the point estimates and uncer...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543400802071360

    authors： Dokoumetzidis A,Aarons L

    更新日期：2008-01-01 00:00:00

  • On the establishment of equivalence acceptance criterion in analytical similarity assessment.

    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

    pub_type： 杂志文章

    doi：10.1080/10543406.2016.1265539

    authors： Wang T,Chow SC

    更新日期：2017-01-01 00:00:00

  • Three-arm noninferiority trials with a prespecified margin for inference of the difference in the proportions of binary endpoints.

    abstract：:The design of a three-arm trial including the experimental treatment, an active reference treatment, and a placebo is recommended as a useful approach to the assessment of noninferiority of the experimental treatment. The inclusion of the placebo arm enables the assessment of assay sensitivity and internal validation,...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2013.789893

    authors： Hida E,Tango T

    更新日期：2013-01-01 00:00:00

  • A likelihood and resampling based approach to dichotomizing a continuous biomarker in medical research.

    abstract：:Dichotomizing a continuous biomarker is a common practice in medical research. Various methods exist in the literature for dichotomizing continuous biomarkers. The most widely adopted minimum p-value approach uses a sequence of test statistics for all possible dichotomizations of a continuous biomarker, and it chooses...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543406.2012.756503

    authors： Su M,Fang L,Su Z

    更新日期：2013-05-01 00:00:00

  • Confidence interval of the difference between two proportions with overdispersion.

    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

    pub_type： 杂志文章

    doi：10.1081/BIP-120037193

    authors： Chen C,Li J,Zhou Z

    更新日期：2004-05-01 00:00:00

  • Dual composite reference standards (dCRS) in molecular diagnostic research: A new approach to reduce bias in the presence of Imperfect reference.

    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

    pub_type： 杂志文章

    doi：10.1080/10543406.2018.1428613

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

    更新日期：2018-01-01 00:00:00

  • Bayesian assessment of the influence and interaction conditions in multipopulation tailoring clinical trials.

    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

    pub_type： 杂志文章,评审

    doi：10.1080/10543406.2013.856025

    authors： Millen BA,Dmitrienko A,Song G

    更新日期：2014-01-01 00:00:00

  • Identification of Risk Factors and Likelihood of Benefit from Adjuvant Chemotherapy for Early Stage Lung Cancer Patients.

    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

    pub_type： 杂志文章

    doi：10.1080/10543406.2019.1684310

    authors： Moon H,Chao T,Ahn H

    更新日期：2020-05-03 00:00:00

  • Impact of model misspecification at design (and/or) estimation step in population pharmacokinetic studies.

    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

    pub_type： 杂志文章

    doi：10.1081/BIP-120028516

    authors： Merlé Y,Aouimer A,Tod M

    更新日期：2004-02-01 00:00:00

  • A global model to define the behavior of partial agonists (bell-shaped dose-response inducers) in pharmacological evaluation of activity in the presence of the full agonist.

    abstract：:The dose-response models for full agonists and for a particular type of partial agonist can be described by sigmoidal curves and bell-shaped curves, respectively. The methods currently used to evaluate the interaction of a full agonist and a partial agonist require a large number of experimental units and base their a...

    journal_title：Journal of biopharmaceutical statistics

    pub_type： 杂志文章

    doi：10.1080/10543409808835266

    authors： Ghosh K,Shen ES,Arey BJ,López FJ

    更新日期：1998-11-01 00:00:00