Studying treatment-effect heterogeneity in precision medicine through induced subgroups

Journal of Biopharmaceutical Statistics
Aniek SiesIven Van Mechelen

Abstract

Precision medicine, in the sense of tailoring the choice of medical treatment to patients' pretreatment characteristics, is nowadays gaining a lot of attention. Preferably, this tailoring should be realized in an evidence-based way, with key evidence in this regard pertaining to subgroups of patients that respond differentially to treatment (i.e., to subgroups involved in treatment-subgroup interactions). Often a-priori hypotheses on subgroups involved in treatment-subgroup interactions are lacking or are incomplete at best. Therefore, methods are needed that can induce such subgroups from empirical data on treatment effectiveness in a post hoc manner. Recently, quite a few such methods have been developed. So far, however, there is little empirical experience in their usage. This may be problematic for medical statisticians and statistically minded medical researchers, as many (nontrivial) choices have to be made during the data-analytic process. The main purpose of this paper is to discuss the major concepts and considerations when using these methods. This discussion will be based on a systematic, conceptual, and technical analysis of the type of research questions at play, and of the type of data that the methods can handle...Continue Reading

Associated Clinical Trials

References

Feb 1, 1983·Journal of Clinical Psychopharmacology·D V SheehanJ Van Wyck Fleet
Feb 1, 1994·Controlled Clinical Trials·K R Bailey
Apr 16, 1998·Journal of Clinical Epidemiology·A R Feinstein
Feb 13, 2001·Statistics in Medicine·R F PotthoffS L George
Nov 21, 2001·Current Controlled Trials in Cardiovascular Medicine·Peter Sleight
Nov 26, 2003·The Australian and New Zealand Journal of Psychiatry·UNKNOWN Royal Australian and New Zealand College of Psychiatrists Clinical Practice Guidelines Team for Panic Disorder and Agoraphob
Jan 13, 2006·Biometrics·Heejung Bang, James M Robins
Apr 21, 2006·The New England Journal of Medicine·Stephen W Lagakos
Jun 24, 2006·Neuropsychopharmacology : Official Publication of the American College of Neuropsychopharmacology·A John RushUNKNOWN ACNP Task Force
Aug 10, 2007·The Journal of Clinical Psychiatry·Daniel SoueryUNKNOWN Group for the Study of Resistant Depression
Jan 1, 2008·The International Journal of Biostatistics·Xiaogang SuSong Yang
Sep 28, 2010·The Journal of Clinical Psychiatry·Qijin ChengPaul S F Yip
Nov 26, 2010·The New England Journal of Medicine·Robert M GrantUNKNOWN iPrEx Study Team
Jun 15, 2011·Annals of Statistics·Min Qian, Susan A Murphy
Jun 17, 2011·The Journal of Neuropsychiatry and Clinical Neurosciences·Gianluca SerafiniRoberto Tatarelli
Aug 5, 2011·The American Journal of Psychiatry·Shu-Sen ChangDavid Gunnell
Nov 26, 2011·Statistical Methods in Medical Research·Wenbin LuDonglin Zeng
May 4, 2012·Biometrics·Baqun ZhangMarie Davidian
Mar 30, 2013·Journal of the American Statistical Association·Garritt PageDavid Dunson
May 3, 2013·PloS One·Anne-Laure BoulesteixManuel J A Eugster
Aug 8, 2013·Statistics in Medicine·Elise Dusseldorp, Iven Van Mechelen
Jan 1, 2012·Journal of the American Statistical Association·Sally PicciottoJames M Robins
Jan 8, 2014·Journal of Biopharmaceutical Statistics·James O BergerLei Shen
Jan 8, 2014·Journal of Biopharmaceutical Statistics·Ilya Lipkovich, Alex Dmitrienko
Jun 14, 2014·Clinical Trials : Journal of the Society for Clinical Trials·Bibhas ChakrabortyYing-Qi Zhao
Oct 31, 2014·Electronic Journal of Statistics·Eric B LaberSusan A Murphy
Jan 27, 2015·Statistical Science : a Review Journal of the Institute of Mathematical Statistics·Phillip J SchulteMarie Davidian
Mar 3, 2015·Journal of the American Statistical Association·Lu TianRobert Tibshirani
Sep 18, 2015·Journal of Biopharmaceutical Statistics·Thomas OndraMartin Posch
Jan 25, 2016·The Lancet. Psychiatry·Adam Mourad ChekroudPhilip Robert Corlett
Feb 20, 2016·Biometrika·E B Laber, Y Q Zhao
Apr 12, 2016·Biometrics·Xinchao LuoHongtu Zhu

❮ Previous
Next ❯

Software Mentioned

STIMA
R
rpart
dtrlist
package
QUalitative INteraction Trees ( QUINT

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.