Adaptive designs in clinical trials: why use them, and how to run and report them.

BMC Medicine
Philip PallmannThomas Jaki

Abstract

Adaptive designs can make clinical trials more flexible by utilising results accumulating in the trial to modify the trial's course in accordance with pre-specified rules. Trials with an adaptive design are often more efficient, informative and ethical than trials with a traditional fixed design since they often make better use of resources such as time and money, and might require fewer participants. Adaptive designs can be applied across all phases of clinical research, from early-phase dose escalation to confirmatory trials. The pace of the uptake of adaptive designs in clinical research, however, has remained well behind that of the statistical literature introducing new methods and highlighting their potential advantages. We speculate that one factor contributing to this is that the full range of adaptations available to trial designs, as well as their goals, advantages and limitations, remains unfamiliar to many parts of the clinical community. Additionally, the term adaptive design has been misleadingly used as an all-encompassing label to refer to certain methods that could be deemed controversial or that have been inadequately implemented.We believe that even if the planning and analysis of a trial is undertaken by an ...Continue Reading

References

Jul 7, 2001·BMJ : British Medical Journal·P JüniM Egger
Dec 4, 2003·Statistics in Medicine·Meinhard Kieser, Tim Friede
Dec 16, 2003·Statistics in Medicine·Caroline C Morgan
Jan 22, 2004·JAMA : the Journal of the American Medical Association·Bernard R ChaitmanUNKNOWN Combination Assessment of Ranolazine In Stable Angina (CARISA) Investigators
May 4, 2004·Statistics in Medicine·David L DeMets, Thomas R Fleming
Jul 19, 2005·Journal of Biopharmaceutical Statistics·Shein-Chung ChowAnnpey Pong
May 27, 2006·Journal of Biopharmaceutical Statistics·Paul GalloUNKNOWN PhRMA Working Group
Sep 16, 2006·Biometrical Journal. Biometrische Zeitschrift·P Bauer, J Einfalt
Sep 16, 2006·Biometrical Journal. Biometrische Zeitschrift·Helmut SchäferHans-Helge Müller
Sep 16, 2006·Biometrical Journal. Biometrische Zeitschrift·Weichung Joe Shih
Oct 27, 2006·Statistics in Medicine·Scott S EmersonDaniel L Gillen
Nov 4, 2006·Pharmaceutical Statistics·Paul Gallo
Feb 20, 2007·European Journal of Cancer : Official Journal for European Organization for Research and Treatment of Cancer (EORTC) [and] European Association for Cancer Research (EACR)·Peter F Thall, J Kyle Wathen
Mar 1, 2007·Clinical Trials : Journal of the Society for Clinical Trials·Roger J LewisDonald A Berry
Jun 19, 2007·Statistics in Medicine·Scott S EmersonDaniel L Gillen
Nov 21, 2007·Journal of Biopharmaceutical Statistics·Sarah Zohar, Sylvie Chevret
Apr 1, 2008·Clinical Trials : Journal of the Society for Clinical Trials·Thomas R FlemingRalph Stewart
Apr 3, 2008·Pharmaceutical Statistics·Tim Friede, Robin Henderson
May 6, 2008·Orphanet Journal of Rare Diseases·Shein-Chung Chow, Mark Chang
Jul 1, 2008·Drugs in R&D·Christopher S Coffey, John A Kairalla
Jul 22, 2008·Pharmaceutical Statistics·Paul Gallo, Christy Chuang-Stein
Jul 30, 2008·Biometrical Journal. Biometrische Zeitschrift·Jack Bowden, Ekkehard Glimm
Aug 30, 2008·Journal of the National Cancer Institute·Mahesh K B ParmarPatrick Royston
Feb 12, 2009·Statistics in Medicine·Frank BretzMartin Posch
May 14, 2009·Journal of the National Cancer Institute·Christophe Le TourneauLillian L Siu
Oct 15, 2009·Statistics in Medicine·Ekkehard GlimmFrank Bretz
Nov 13, 2009·Statistics in Medicine·Robert A Parker
Mar 25, 2010·BMJ : British Medical Journal·Kenneth F SchulzUNKNOWN CONSORT Group
Mar 27, 2010·Clinical Trials : Journal of the Society for Clinical Trials·Judith QuinlanMichael Krams
Jul 16, 2010·JAMA : the Journal of the American Medical Association·Susan S EllenbergThomas R Fleming
Aug 21, 2010·Statistical Methods in Medical Research·Nigel Stallard, Susan Todd
Dec 21, 2010·Statistics in Medicine·Gordana Jovic, John Whitehead
Apr 15, 2011·International Journal of Radiation Oncology, Biology, Physics·Edward ChowUNKNOWN International Bone Metastases Consensus Working Party
Apr 26, 2011·Journal of Biopharmaceutical Statistics·Sue-Jane WangRobert O'Neill
Sep 10, 2011·Statistics in Medicine·Sylvie Chevret
Nov 23, 2011·Statistics in Medicine·Cyrus R Mehta, Stuart J Pocock
Mar 15, 2012·Statistics in Medicine·Gaohong DongStephen Marcella
Mar 23, 2012·Statistics in Medicine·Peter K KimaniNigel Stallard
Jun 2, 2012·Journal of Biopharmaceutical Statistics·Shein-Chung ChowMin Lin
Jun 20, 2012·Cephalalgia : an International Journal of Headache·Tony W HoUNKNOWN Rizatriptan Protocol 082 Pediatric Migraine Study Group
Jun 30, 2012·Statistics in Medicine·Máximo Carreras, Werner Brannath

❮ Previous
Next ❯

Citations

Mar 13, 2019·Tissue Engineering. Part a·Liesbet Geris, Ioannis Papantoniou
Jan 16, 2020·Cancer Metastasis Reviews·Teresa de RojasLucas Moreno
Sep 26, 2019·Journal of Clinical Pharmacology·Andreas KrauseJasper Dingemanse
Jan 5, 2020·Journal of Crohn's & Colitis·Ferdinando D'AmicoLaurent Peyrin-Biroulet
Nov 13, 2019·Hepatology : Official Journal of the American Association for the Study of Liver Diseases·Markus CornbergUNKNOWN 2019 EASL-AASLD HBV Treatment Endpoints Conference Faculty
May 3, 2020·The International Journal of Eating Disorders·Stephen A WonderlichHans W Hoek
Jun 22, 2019·BMC Medicine·Koen B PouwelsJulie V Robotham
Jul 7, 2020·JAMA Internal Medicine·Katherine R Courtright, Scott D Halpern
Sep 5, 2020·Journal of the American Medical Informatics Association : JAMIA·Nathan D SeligsonJames L Chen
Jul 16, 2019·NPJ Digital Medicine·Kumanan WilsonHolly Witteman
Aug 3, 2019·BMC Medicine·James M S WasonChristina Yap
Oct 30, 2019·International Journal of Stroke : Official Journal of the International Stroke Society·Julie BernhardtSteven C Cramer
Dec 17, 2019·Statistics in Biopharmaceutical Research·Byron J GajewskiRichard Barohn
Apr 16, 2020·Alzheimer's & Dementia : the Journal of the Alzheimer's Association·Esther S OhUNKNOWN NIDUS Writing Group
Sep 17, 2020·Brain Sciences·Skylar LuuDejan B Budimirovic
Jan 29, 2019·Therapeutic Innovation & Regulatory Science·Simon CoatesUlrike Lorch
Jul 13, 2019·Dementia and Neurocognitive Disorders·Seung Hyun KimUNKNOWN K-ARPI
May 12, 2020·Frontiers in Pharmacology·Rachel J TysonDaniel Gonzalez
Jun 3, 2020·Nature Communications·Martin WalkerMaria-Gloria Basáñez
Aug 25, 2020·Research and Practice in Thrombosis and Haemostasis·Hunter B MooreMichael B Yaffe
Nov 16, 2018·Intensive Care Medicine·M Gasparini, S Chevret
Sep 19, 2019·The New England Journal of Medicine·Leora I HorwitzSimon A Jones
Feb 18, 2020·Clinical Trials : Journal of the Society for Clinical Trials·Michael John Grayling, Graham Mark Wheeler
Aug 28, 2020·Journal of Medical Internet Research·Marcus Bendtsen
Jun 26, 2020·BMC Medical Research Methodology·Kim May Lee, James Wason
Jun 30, 2019·Journal of Pediatric Psychology·Aimee K HildenbrandTonya M Palermo

❮ Previous
Next ❯

Software Mentioned

RomiCar
UK
DEVELOP
CARISA
TRAFIC

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.

Related Papers

Thérapie
Raphaël Porcherparticipants of Round Table N° 2 de Giens XXVI
Annual Review of Public Health
C Hendricks BrownRobert D Gibbons
Special Care in Dentistry : Official Publication of the American Association of Hospital Dentists, the Academy of Dentistry for the Handicapped, and the American Society for Geriatric Dentistry
Mark LivingstonJohn Hyde
© 2022 Meta ULC. All rights reserved