Bayesian design and analysis of active control clinical trials

Biometrics
R Simon

Abstract

We consider the design and analysis of active control clinical trials, i.e., clinical trials comparing an experimental treatment E to a control treatment C considered to be effective. Direct comparison of E to placebo P, or no treatment, is sometimes ethically unacceptable. Much discussion of the design and analysis of such clinical trials has focused on whether the comparison of E to C should be based on a test of the null hypothesis of equivalence, on a test of a nonnull hypothesis that the difference is of some minimally medically important size delta, or on one or two-sided confidence intervals. These approaches are essentially the same for study planning. They all suffer from arbitrariness in specifying the size of the difference delta that must be excluded. We propose an alternative Bayesian approach to the design and analysis of active control trials. We derive the posterior probability that E is superior to P or that E is at least k% as good as C and that C is more effective than P. We also derive approximations for use with logistic and proportional hazard models. Selection of prior distributions is discussed, and results are illustrated using data from an active control trial of a drug for the treatment of unstable an...Continue Reading

References

Aug 1, 1991·Controlled Clinical Trials·A L Gould
Dec 1, 1982·Controlled Clinical Trials·W C Blackwelder

❮ Previous
Next ❯

Citations

Jan 10, 2003·Statistics in Medicine·H M James HungRobert T O'Neil
Jan 10, 2003·Statistics in Medicine·Sue-Jane Wang, H M James Hung
Jan 10, 2003·Statistics in Medicine·Mark RothmannHsiao-Hui Tsou
Apr 12, 2003·Controlled Clinical Trials·Sue-Jane Wang, H M James Hung
Jun 23, 2001·The New England Journal of Medicine·D C Throckmorton
Mar 17, 2007·Journal of Biopharmaceutical Statistics·H M James HungRobert O'Neill
Dec 29, 2007·Journal of Biopharmaceutical Statistics·Gene Pennello, Laura Thompson
Feb 26, 2010·Journal of Biopharmaceutical Statistics·Yi-Hau ChenMey Wang
Jun 19, 2008·Clinical Trials : Journal of the Society for Clinical Trials·Wendy Demark-WahnefriedRobin T Vollmer
Aug 23, 2011·Journal of Pharmacokinetics and Pharmacodynamics·Alberto RussuOscar Della Pasqua
Jan 16, 2010·Journal of Biopharmaceutical Statistics·Shein-Chung Chow, Jen-Pei Liu
Feb 20, 2002·Controlled Clinical Trials·Sue-Jane WangYi Tsong
Aug 8, 2015·Journal of Biopharmaceutical Statistics·Margaret Gamalo-SiebersGuochen Song
Dec 8, 2015·Pharmaceutical Statistics·Junjing LinRam Tiwari
Mar 7, 2007·Journal of Cardiac Failure·James D NeatonMarvin A Konstam
Jan 7, 2006·Biometrical Journal. Biometrische Zeitschrift·H M James HungRobert O'Neill
Mar 11, 2003·Statistics in Medicine·Sylvain Durrleman, Philip Chaikin
Apr 18, 2008·Statistics in Medicine·Jitendra GanjuAlessandra Anemona
Apr 10, 2009·Biometrical Journal. Biometrische Zeitschrift·H M James HungRobert O'Neill
Jul 20, 2006·Journal of Thrombosis and Haemostasis : JTH·A Iorio, G Agnelli
Nov 28, 2006·Pharmaceutical Statistics·Brian L Wiens
Aug 6, 2008·Statistics in Medicine·Tie-Hua Ng
May 11, 2006·Statistics in Medicine·Meinhard Kieser, Tim Friede
Sep 20, 2011·Pharmaceutical Statistics·Sue-Jane Wang, Jeffrey D Blume
Aug 31, 2012·Statistics in Medicine·Susan S Ellenberg
Sep 19, 2012·Statistics in Medicine·Guoxing SoonLei Nie
Aug 6, 2013·Pharmaceutical Statistics·Meg A GamaloLisa M LaVange
Mar 9, 2010·Statistics in Medicine·Lei Nie, Guoxing Soon
Dec 6, 2005·Journal of the American College of Cardiology·Sanjay KaulWilliam S Weintraub
Apr 18, 2001·Controlled Clinical Trials·D H Leung, Y Wang
Dec 10, 2016·Evidence-based Mental Health·Simon Wandel, Satrajit Roychoudhury
May 24, 2012·Statistical Methods in Medical Research·M Amper GamaloRam C Tiwari
Jan 6, 2012·Statistical Methods in Medical Research·Heinz SchmidliBeat Neuenschwander
Jun 17, 2009·Clinical Trials : Journal of the Society for Clinical Trials·Valen E Johnson, John D Cook
Nov 12, 2005·Clinical Trials : Journal of the Society for Clinical Trials·Gregory Campbell
Feb 24, 2018·Pharmaceutical Statistics·Santu GhoshSamiran Ghosh
Jun 23, 2004·Journal of Biopharmaceutical Statistics·H M James Hung, Sue-Jane Wang
Jan 10, 2003·Statistics in Medicine·Ralph B D'AgostinoLisa M Sullivan
Feb 13, 2019·Journal of Biopharmaceutical Statistics·Shrabanti ChowdhurySamiran Ghosh

❮ 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.