6. The essentials of running a clinical trial
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.