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The Case for Practical Clinical Trials in Psychiatry John S. March, MD, MPH Duke University Medical Center March JS, et al. (2004), The Child and Adolescent Psychiatry Trials Network (CAPTN). J Am Acad Child Adolesc Psychiatry

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the case for practical clinical trials in psychiatry

The Case forPractical Clinical Trials in Psychiatry

John S. March, MD, MPH

Duke University Medical Center

slide2
March JS, et al. (2004), The Child and Adolescent Psychiatry Trials Network (CAPTN). J Am Acad Child Adolesc Psychiatry

March JS (2005), Using and Teaching Evidence-Based Medicine (EBM): The Duke University Child and Adolescent Psychiatry Model. Ch Psychiatr Clin North Am

March JS et al. (2005), The Case for Practical Clinical Trials in Psychiatry, J Amer Psychiatr Assoc

slide3
“Evidence-Based Medicine is the conscientious, explicit and judicious use of current best evidence in making decisions about the care of individual patients.”
  • David Sackett, MD
ebm requires best evidence
EBM Requires “Best Evidence”
  • Clinically meaningful
  • Generalizable (Few filters)
    • Real world patients
    • Real world doctors
    • Real world treatment(s)
    • Real world outcomes
  • Replicable (reliable estimator of the pop mean)
trial types
Trial Types
  • We need trials for decision makers
    • Public health value
    • Real effect sizes
    • Subgrouping variables (moderators)
  • We have mostly explanatory trials
what have we learned
What Have We Learned?
  • Not nearly enough
    • Too few mostly acute studies
    • Lack generalizability
    • Don’t adequately assess safety or moderators
    • Very expensive and take a long time to complete
    • Performed long after drugs are in widespread clinical use
measurement error
Measurement Error

R = T / T +e

R = Intraclass Correlation Coefficient

T = True score

e = Error

Fleiss, J. (1986). The Design and Analysis of Clinical Experiments. New York: Wiley.

minimizing error
Minimizing Error

e

Alpha reliability Measures

Stability Measures

Inter-rater QA

Site variability QA

sem and power
SEM and Power

CI population mean = +/- 2 [SEM]

a modest proposal
A Modest Proposal
  • Abandon “large efficacy/effectiveness trials”
  • Iteratively develop treatments and examine mechanisms (mediators) in small data intensive trials
  • Switch to large practical clinical trials to study real world outcomes, including subgrouping variables (moderators), and to disseminate treatments
practical clinical trials
Practical Clinical Trials

Practical clinical trials, which are almost always larger and always simpler than typical RCTs in psychiatry, “provide reliable estimates of health outcomes applicable to a broad patient base treated under usual clinical conditions without bias.”

Richard Peto

characteristics of pcts 1
Characteristics of PCTs 1
  • A straightforward clinically relevant question
  • Representative sample of patients and practice settings
  • Sufficient power to identify modest clinically relevant effects
  • Randomization to protect against bias
  • Clinical uncertainty regarding the outcome of treatment at the patient level

March, J., Silva, S., Compton, S., Shapiro, M., Califf, R., & Krishnan, K. R. (in press). The case for practical clinical trials in psychiatry. Amer J Psychiatry.

characteristics of pcts 2
Characteristics of PCTs 2
  • Best clinical practice diagnostic assessment
  • Best clinical practice treatments
  • Simple and clinically relevant outcomes
  • Limited subject and investigator burden
  • High value per dollar spent

March, J., Silva, S., Compton, S., Shapiro, M., Califf, R., & Krishnan, K. R. (in press). The case for practical clinical trials in psychiatry. Amer J Psychiatry.

typical pcts
Typical PCTs
  • Active comparator trials
  • Treatment addition (adjunctive or augmentation) trials
  • Population PK studies
  • Subgroup analyses, e.g age, pharmacogenetics
  • Expand to traditionally excluded or rare populations
  • Distal outcomes (5 year survivals), e.g. course of illness, not just episode, so can evaluate prognosis
slide18
Americans’ confidence in our academic research enterprise will unravel if researchers are not extremely cautious in their private interests and absolutely truthful in their research results, especially adverse events, in situations where their research activities and personal interests converge.

- Donna Shalala,

Secretary of the Dept. of Health & Human Services

May 7, 2000

slide20
UNIVERSITY

INDUSTRY

Knowledge for Knowledge’s Sake

Management ofKnowledge for Profit

Teaching

Profits

Research

Commercializationof New and UsefulTechnologies

Service

Product R&D

EconomicDevelopment

Academic FreedomOpen Discourse

ConfidentialityLimited Public Disclosure

Conflicting Values - Common Interest

Conflicting Values - Common Interest

slide21
A man who carries a cat by the tail learns something he can learn in no other way.

Mark Twain

summary
Summary
  • To maximize their public health value, RCTs should tilt toward either:
    • Explanatory trials that focus on treatment development and mechanisms
    • Practical clinical trials for decision makers that focus on producing generalizable knowledge
  • PCTs will require stable networks funded and governed by a consortium of stakeholders
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