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Randomized Behavioral Clinical Trials: 10 Lessons I Learned
Transcript of Randomized Behavioral Clinical Trials: 10 Lessons I Learned
10 Lessons I Learned from the
NIH Summer Institute
Lucy Popova, PhD
Center for Tobacco Control Research and Education
September 8, 2015
NIH summer institute Randomized Clinical Behavioral Trials
Deadline for applications: Early February
- Personal Statement
- Sponsor Letter (from mentor)
Lesson 2: See the big picture
Lesson 8: There is more than one way to randomize participants
In fact, more than two or three...
Lesson 6: Appropriate control group
The ethical basis for medical research that involves assigning patients randomly to different treatments in a clinical trial.
A state of general uncertainty on the part of the clinical investigator regarding the therapeutic merits of each arm in a trial.
An ethically necessary condition in all cases of clinical research.
If equipoise is disturbed during the course of the trial, the trial may need to be terminated, and all subjects previously enrolled offered the superior treatment.
Freedman, B: Equipoise and the ethics of clinical research.
NEJM 1987; 317:141-5.
Clinician and the investigator to be different individuals
Perfectly unbiased investigator.
Therapeutic uncertainty in the clinical community: “clinical equipoise.”
Community vs. Individual Equipoise
A clinician may be convinced that Treatment A is better than Treatment B, but is aware that no consensus exists among clinicians who care for patients that need these treatments.
Lack of agreement is sufficient justification for conducting and participating in a well-executed clinical trial that will provide the necessary evidence.
Lesson 7: Be honest with participants
Pilot study results should not be used for sample size determination due to the inherent imprecision in effect size estimates from small samples and high likelihood in Type I and Type II error.
Lesson 5: Effect size
Czajkowski, S. M., Powell, L. H., Adler, N., Naar-King, S., Reynolds, K. D., Hunter, C. M., ... & Charlson, M. E. (2015). From Ideas to Efficacy: The ORBIT Model for Developing Behavioral Treatments for Chronic Diseases.
Don't try to do it alone!
Scientific and/or clinical expertize
Administrative expertize and support
Data management expertize
Choosing a comparator group
Standard drug trial: easy
Behavioral trial: Challenging
1. The purpose of the trial
2. The purpose of the comparator
3. The purpose of the trial design
In the trial
Not in the trial/
On your own
Opposite of "hard sell"
Impossible to predict next assignment
No differences between groups
Easy to implement
Robust against changes in study
Lesson 4: Pick a single outcome
Closely tied to the target of the intervention
Less influenced by factors other than the intervention
Expect a robust impact from the intervention
Substantial public health significance (clinically meaningful)
Reduced events (death, disease)
Changes in intermediate outcomes that have been associated with more distal outcomes (e.g. 10% BMI reduction associated with reduced health risks)
And ONLY 1 primary outcome
More proximate or distal to intervention than primary
Mediators of change
Measures that will help explain a null effect
Don't get carried away with mediators and moderators
Register with clinicaltrials.gov and use CONSORT guidelines for reporting
Begin with an important problem.
Must provide a definitive, unbiased estimate of the effectiveness of an intervention, treatment, or strategy on health.
Selected outcome measure must be persuasive to clinicians and practitioners.
Minimally clinically important difference
Prospective study comparing the effect and value of intervention(s) against control in human beings
Friedman et al. 2010.
Fundamentals of Clinical Trials
, 4th ed.