1. The Pragmatic Trial at 50 ICTMC 2017 May 9th
Chair: Sandra Eldridge
Jeremy Grimshaw
Spencer Phillips Hey
Cory Goldstein
Charles Weijer

2. The Pragmatic Trial at 50:
Introduction
Sandra Eldridge
Title
Authors
Centre for Primary Care & Public Health
Blizard Institute
Pragmatic Clinical Trials Unit

3. What is a pragmatic trial?
Schwartz and Lellouch (J. Chron. Dis.1967;20:637-48)
Pragmatic trials = to help choose between care options
Explanatory trials = to test causal research hypotheses
Roland and Torgerson (BMJ 1998;316:285)
Pragmatic trials measure effectiveness—the benefit the treatment produces in routine clinical practice
Explanatory trials generally measure efficacy—the benefit a treatment produces under ideal conditions

4. How pragmatic/how explanatory?
EXPLANATORY PRAGMATIC
How pragmatic/how explanatory?

6. Pragmatic
Explanatory
Representative participants
Highly selected participants
Recruited in usual care, many centres
Recruited in another way, few centres
Setting where results apply
Different setting
Intervention slotted into usual care
Intervention requires additional resources
Flexibility in delivery
Very standardised delivery
Flexibility in adherence
Highly controlled adherence
Follow-up exactly as would be in usual care
More extensive follow up
Primary outcome relevant to participants
Primary outcome not relevant to participants
Primary analysis intention to treat
Primary analysis per protocol

7. When? (drug trials) Translational pathway EXPLANATORY PRAGMATIC
BASIC SCIENCE
EXPLANATORY PRAGMATIC
“Ideal” conditions
In routine practice
Can this treatment work?
Does this treatment work?

8. WAIT – Montelukast for pre-school wheeze
Population
Intervention
Outcome
Unscheduled medical attendances for wheezing
Control
Placebo

10. STOP – Smoking cessation through pharmacies
Population (pharmacies)
Intervention
Training and support for pharmacists
Outcome (customers)
Control
No training and support

11. How often? Zwarenstein & Treweek 2009
“Most trials done hitherto have adopted the explanatory approach without question; the pragmatic approach would often have been more justifiable.”
Schwartz & Lellouch 1967

12. Vichithranie Madurasinghe
Acknowledgements
Gordon Forbes
Kirsty Loudon
Shaun Treweek
Megan Clinch
Steph Taylor
Robert Walton
Wai Yee James
Sandra Jumbe
Vichithranie Madurasinghe
Ratna Sohanpal
Carol Rivas
Liz Steed
Chris Griffiths
Clare Rutterford
Jonathan Grigg
Chinedu Nwokoro
Title
Authors
Centre for Primary Care & Public Health
Blizard Institute

13. Pragmatic trials at 50 – Back to the future?
Pragmatic RCTs and the Learning Healthcare System
Precision Medicine and Pragmatic
Trials: Friends or Foes?
Towards ethical guidance for pragmatic randomized controlled trials