Clinimetrics corner: choosing appropriate study designs for particular questions about treatment subgroups

Peter Kent, Mark Hancock, Ditte H.D Petersen, Hanne L. Mjøsund

Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

Resumé

Background:
Many clinicians and researchers believe that there are subgroups of people with spinal pain who respond differently to treatment and have different prognoses. There has been considerable interest in this topic recently. However, problems occur when conclusions about subgroups are made that are inappropriate given the randomized controlled trial design used. The research design to choose, when developing a study protocol that investigates the effect of treatment subgroups, depends on the particular research question. Similarly, the inferences that can be drawn from an existing study will vary, depending on the design of the trial.
Objectives:
This paper discusses the randomized controlled trial designs that are suitable to answer particular questions about treatment subgroups. It focuses on trial designs that are suitable to answer four questions: (1) 'Is the treatment effective in a pre-specified group of patients?'; (2) 'Are outcomes of treatment applied using a subgrouping clinical reasoning process, better than a control treatment?'; (3) 'Are the outcomes for a patient subgroup receiving a particular treatment (compared to a control treatment) better than for patients not in the subgroup who receive the same treatment?'; and (4) 'Are outcomes for a number of treatments better if those treatments are matched to patients in specific subgroups, than if the SAME treatments are randomly given to patients?'. Illustrative examples of these studies are provided.
Conclusion:
If the clinical usefulness of targeting treatments to subgroups of people is to be determined, an important step is a shared understanding of what different RCT designs can tell us about subgroups.
OriginalsprogEngelsk
TidsskriftJournal of Manual and Manipulative Therapy
Vol/bind18
Udgave nummer3
Sider (fra-til)147-152
StatusUdgivet - 2010

Fingeraftryk

Randomized Controlled Trials
Research Design
Research Personnel
Research

Citer dette

@article{8495278cac0941058f87bde66b2c8b29,
title = "Clinimetrics corner: choosing appropriate study designs for particular questions about treatment subgroups",
abstract = "Background: Many clinicians and researchers believe that there are subgroups of people with spinal pain who respond differently to treatment and have different prognoses. There has been considerable interest in this topic recently. However, problems occur when conclusions about subgroups are made that are inappropriate given the randomized controlled trial design used. The research design to choose, when developing a study protocol that investigates the effect of treatment subgroups, depends on the particular research question. Similarly, the inferences that can be drawn from an existing study will vary, depending on the design of the trial. Objectives: This paper discusses the randomized controlled trial designs that are suitable to answer particular questions about treatment subgroups. It focuses on trial designs that are suitable to answer four questions: (1) 'Is the treatment effective in a pre-specified group of patients?'; (2) 'Are outcomes of treatment applied using a subgrouping clinical reasoning process, better than a control treatment?'; (3) 'Are the outcomes for a patient subgroup receiving a particular treatment (compared to a control treatment) better than for patients not in the subgroup who receive the same treatment?'; and (4) 'Are outcomes for a number of treatments better if those treatments are matched to patients in specific subgroups, than if the SAME treatments are randomly given to patients?'. Illustrative examples of these studies are provided. Conclusion: If the clinical usefulness of targeting treatments to subgroups of people is to be determined, an important step is a shared understanding of what different RCT designs can tell us about subgroups.",
author = "Peter Kent and Mark Hancock and Petersen, {Ditte H.D} and Mj{\o}sund, {Hanne L.}",
year = "2010",
language = "English",
volume = "18",
pages = "147--152",
journal = "Journal of Manual & Manipulative Therapy",
issn = "1066-9817",
publisher = "Taylor & Francis",
number = "3",

}

Clinimetrics corner: choosing appropriate study designs for particular questions about treatment subgroups. / Kent, Peter; Hancock, Mark; Petersen, Ditte H.D; Mjøsund, Hanne L.

I: Journal of Manual and Manipulative Therapy, Bind 18, Nr. 3, 2010, s. 147-152.

Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

TY - JOUR

T1 - Clinimetrics corner: choosing appropriate study designs for particular questions about treatment subgroups

AU - Kent, Peter

AU - Hancock, Mark

AU - Petersen, Ditte H.D

AU - Mjøsund, Hanne L.

PY - 2010

Y1 - 2010

N2 - Background: Many clinicians and researchers believe that there are subgroups of people with spinal pain who respond differently to treatment and have different prognoses. There has been considerable interest in this topic recently. However, problems occur when conclusions about subgroups are made that are inappropriate given the randomized controlled trial design used. The research design to choose, when developing a study protocol that investigates the effect of treatment subgroups, depends on the particular research question. Similarly, the inferences that can be drawn from an existing study will vary, depending on the design of the trial. Objectives: This paper discusses the randomized controlled trial designs that are suitable to answer particular questions about treatment subgroups. It focuses on trial designs that are suitable to answer four questions: (1) 'Is the treatment effective in a pre-specified group of patients?'; (2) 'Are outcomes of treatment applied using a subgrouping clinical reasoning process, better than a control treatment?'; (3) 'Are the outcomes for a patient subgroup receiving a particular treatment (compared to a control treatment) better than for patients not in the subgroup who receive the same treatment?'; and (4) 'Are outcomes for a number of treatments better if those treatments are matched to patients in specific subgroups, than if the SAME treatments are randomly given to patients?'. Illustrative examples of these studies are provided. Conclusion: If the clinical usefulness of targeting treatments to subgroups of people is to be determined, an important step is a shared understanding of what different RCT designs can tell us about subgroups.

AB - Background: Many clinicians and researchers believe that there are subgroups of people with spinal pain who respond differently to treatment and have different prognoses. There has been considerable interest in this topic recently. However, problems occur when conclusions about subgroups are made that are inappropriate given the randomized controlled trial design used. The research design to choose, when developing a study protocol that investigates the effect of treatment subgroups, depends on the particular research question. Similarly, the inferences that can be drawn from an existing study will vary, depending on the design of the trial. Objectives: This paper discusses the randomized controlled trial designs that are suitable to answer particular questions about treatment subgroups. It focuses on trial designs that are suitable to answer four questions: (1) 'Is the treatment effective in a pre-specified group of patients?'; (2) 'Are outcomes of treatment applied using a subgrouping clinical reasoning process, better than a control treatment?'; (3) 'Are the outcomes for a patient subgroup receiving a particular treatment (compared to a control treatment) better than for patients not in the subgroup who receive the same treatment?'; and (4) 'Are outcomes for a number of treatments better if those treatments are matched to patients in specific subgroups, than if the SAME treatments are randomly given to patients?'. Illustrative examples of these studies are provided. Conclusion: If the clinical usefulness of targeting treatments to subgroups of people is to be determined, an important step is a shared understanding of what different RCT designs can tell us about subgroups.

M3 - Journal article

VL - 18

SP - 147

EP - 152

JO - Journal of Manual & Manipulative Therapy

JF - Journal of Manual & Manipulative Therapy

SN - 1066-9817

IS - 3

ER -