Special considerations for conducting clinical trials

Course overview and trial designs

Learning outcomes:

  • Define randomized controlled trial
  • Identify three alternative study designs to randomized controlled trials
  • Identify five reasons for not conducting randomized controlled clinical trials
  • Identify four reasons for conducting randomized controlled clinical trials
  • Describe four randomized trial designs.
Selection of participants

Learning outcomes:

  • Explain why it is important to develop detailed and specific eligibility criteria in a clinical trial
  • Describe advantages and disadvantages of defining a broader versus narrower population in a trial
  • Describe at least three appropriate reasons for excluding participants from a clinical trial.
Recruitment

Learning outcomes:

  • Describe two goals of recruitment
  • Identify two study design issues
  • Identify three strategies to recruit appropriately
  • Identify four recruitment methods.
Choosing the interventions and controls

Learning outcomes:

  • Describe aspects of the experimental intervention that should be defined in the planning stages of a trial
  • Identify at least three important functions of a control or comparator intervention in a trial
  • Assess the strengths and weaknesses of common controls for pharmacologic, surgical, or behavioral interventions.
Randomization

Learning outcomes:

  • Describe the importance of randomization in clinical trials
  • Describe simple randomization
  • Describe randomized permuted blocks.
Blinding

Learning outcomes:

  • Define blinding and identify ways to blind many interventions
  • Identify three ways that blinding minimizes potential bias for
  • Identify four types of interventions that cannot be blinded
  • Describe strategies to implement if the study cannot be blinded.
Outcome measures

Learning outcomes:

  • Describe at least two reasons for using one primary outcome
  • Identify two types of data that support that a measure is a ‘valid’ surrogate marker for treatment
  • Identify the main criterion for determining whether a marker is a valid ‘surrogate’ endpoint
  • Describe two pros and cons for using composite outcomes.
Assessing safety

Learning outcomes:

  • Define a serious adverse event
  • Describe one pro and con of elicited vs volunteered adverse events
  • Describe reasons for using a formal adjudication process for clinical outcomes
  • Identify one disadvantage of adjudication.
Adherence and complete follow up

Learning outcomes:

  • Describe two important reasons for adherence to the protocol
  • Describe five ways that adherence can be measured
  • Identify two ways to maximize adherence to the protocol
  • Identify four ways to maximize follow up
  • Describe three analytic techniques to use for poor compliance during a trial
  • Describe two effects of non-adherence.
Ethical issues in clinical trials

Learning outcomes:

  • Identify ethical issues in clinical trials
  • Describe factors for acceptability of random assignment to a treatment
  • Define interim monitoring
  • Describe two basic goals of interim monitoring in a blinded trial
  • List four reasons to stop a trial early
  • List four components of a data monitoring plan
  • Describe conflict of interest issues in clinical trials
  • Describe three ways of performing scientific misconduct
  • Define contributions needed to qualify as an author on manuscript.
Regulatory issues

Learning outcomes:

  • Define regulations that apply to clinical trials
  • Describe good clinical practice.
How to write up industry-sponsored trials

Learning outcomes:

  • The evidence on misreporting of industry trials
  • Potential pitfalls of using composite end points in trials
  • Reporting of authorship for industry studies
  • How to report industry trials transparently
  • Good publication practice (GPP3) for industry studies.