Bayesian design and conduct of phase II single-arm clinical trials with binary outcomes: a tutorial

Contemporary Clinical Trials
Sarah ZoharYinghui Zhou

Abstract

The aim of phase II single-arm clinical trials of a new drug is to determine whether it has sufficient promising activity to warrant its further development. For the last several years Bayesian statistical methods have been proposed and used. Bayesian approaches are ideal for earlier phase trials as they take into account information that accrues during a trial. Predictive probabilities are then updated and so become more accurate as the trial progresses. Suitable priors can act as pseudo samples, which make small sample clinical trials more informative. Thus patients have better chances to receive better treatments. The goal of this paper is to provide a tutorial for statisticians who use Bayesian methods for the first time or investigators who have some statistical background. In addition, real data from three clinical trials are presented as examples to illustrate how to conduct a Bayesian approach for phase II single-arm clinical trials with binary outcomes.

References

Dec 15, 1994·Statistics in Medicine·H C Brunier, J Whitehead
Aug 1, 1993·Statistics in Medicine·D A Berry
Jun 26, 2001·British Journal of Clinical Pharmacology·N StallardA Whitehead
Nov 20, 2002·British Journal of Haematology·Dominique FargeUNKNOWN Intensification et Autogreffe dans les Maladies Auto Immunes Resistantes (ISAMAIR) Study Group
Mar 17, 2004·Controlled Clinical Trials·Matthew S Mayo, Byron J Gajewski
Feb 18, 2006·Nature Reviews. Drug Discovery·Donald A Berry

❮ Previous
Next ❯

Citations

Sep 29, 2011·Pediatric Nephrology : Journal of the International Pediatric Nephrology Association·Véronique BaudouinChantal Loirat
Jan 1, 2016·Radiotherapy and Oncology : Journal of the European Society for Therapeutic Radiology and Oncology·Rick L M HaasBrian O'Sullivan
Jul 19, 2012·Statistics in Medicine·Satoshi TeramukaiSarah Zohar
Mar 24, 2016·Nature Reviews. Clinical Oncology·Agnès BuzynGilles Vassal
Oct 3, 2014·Statistics in Medicine·S BersimisT Papaioannou
Jul 9, 2013·Transfusion and Apheresis Science : Official Journal of the World Apheresis Association : Official Journal of the European Society for Haemapheresis·Fanny ChambonJustyna Kanold
Mar 31, 2012·Statistical Methods in Medical Research·Yiyi ChenMotomi Mori
Jul 29, 2020·Pharmaceutical Statistics·Liyun JiangXuelin Huang
Jan 27, 2021·Clinical Cancer Research : an Official Journal of the American Association for Cancer Research·Sophie CousinAntoine Italiano
Aug 28, 2021·International Journal of Environmental Research and Public Health·Valeria Sambucini

❮ Previous
Next ❯

Related Concepts

Trending Feeds

COVID-19

Coronaviruses encompass a large family of viruses that cause the common cold as well as more serious diseases, such as the ongoing outbreak of coronavirus disease 2019 (COVID-19; formally known as 2019-nCoV). Coronaviruses can spread from animals to humans; symptoms include fever, cough, shortness of breath, and breathing difficulties; in more severe cases, infection can lead to death. This feed covers recent research on COVID-19.

Blastomycosis

Blastomycosis fungal infections spread through inhaling Blastomyces dermatitidis spores. Discover the latest research on blastomycosis fungal infections here.

Nuclear Pore Complex in ALS/FTD

Alterations in nucleocytoplasmic transport, controlled by the nuclear pore complex, may be involved in the pathomechanism underlying multiple neurodegenerative diseases including Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Here is the latest research on the nuclear pore complex in ALS and FTD.

Applications of Molecular Barcoding

The concept of molecular barcoding is that each original DNA or RNA molecule is attached to a unique sequence barcode. Sequence reads having different barcodes represent different original molecules, while sequence reads having the same barcode are results of PCR duplication from one original molecule. Discover the latest research on molecular barcoding here.

Chronic Fatigue Syndrome

Chronic fatigue syndrome is a disease characterized by unexplained disabling fatigue; the pathology of which is incompletely understood. Discover the latest research on chronic fatigue syndrome here.

Evolution of Pluripotency

Pluripotency refers to the ability of a cell to develop into three primary germ cell layers of the embryo. This feed focuses on the mechanisms that underlie the evolution of pluripotency. Here is the latest research.

Position Effect Variegation

Position Effect Variagation occurs when a gene is inactivated due to its positioning near heterochromatic regions within a chromosome. Discover the latest research on Position Effect Variagation here.

STING Receptor Agonists

Stimulator of IFN genes (STING) are a group of transmembrane proteins that are involved in the induction of type I interferon that is important in the innate immune response. The stimulation of STING has been an active area of research in the treatment of cancer and infectious diseases. Here is the latest research on STING receptor agonists.

Microbicide

Microbicides are products that can be applied to vaginal or rectal mucosal surfaces with the goal of preventing, or at least significantly reducing, the transmission of sexually transmitted infections. Here is the latest research on microbicides.