Outcome_measures flashcards

What are some fundamental design aspects for major randomized controlled trials?

Choice of patients, treatments, outcomes, randomization, blinding, and trial size.

What should be considered when selecting patients for a clinical trial?

Precise eligibility criteria that are neither too specific nor too broad, with appropriate geographic representation.

What is the importance of specifying precise treatment regimens in a clinical trial?

It ensures clarity in treatment application and can include a placebo/sham control group or an active comparator.

What is a primary efficacy endpoint?

The primary outcome measure that the investigator considers most important to determine the intervention's effectiveness.

Define a 'hard' outcome in the context of clinical trials.

Hard outcomes are easily measured reliably and objectively, like death or tumor size.

What distinguishes 'soft' outcomes from 'hard' outcomes?

Soft outcomes require subjective assessments by investigators or patients, such as pain or quality of life.

What is the advantage of using surrogate outcomes in clinical trials?

They allow smaller and quicker trials, and may be necessary when it is not feasible to show an effect on clinical outcomes.

What is a composite outcome?

A measure that combines several events into a single outcome, such as various failure indicators in a clinical trial.

Why is it crucial to pre-specify primary outcomes in clinical trials?

To prevent 'outcome switching' and ensure the consistency and validity of the study findings.

What is the purpose of conducting sensitivity analyses in clinical trials?

To assess the robustness of conclusions and understand the impact of various assumptions or data handling methods.

What is the function of a Kaplan-Meier curve in survival analysis?

It presents survival data, showing the proportion of subjects surviving at different time points.

How do you interpret a hazard ratio (HR) of 1 in a clinical trial?

It indicates that the risk of an event in the experimental group is the same as in the control group.

What statistical method is commonly used for binary outcomes in clinical trials?

Calculating the odds ratio or risk ratio to compare treatment groups.

Why is it important to use validated surrogate outcomes?

To ensure that the surrogate reliably predicts clinical outcomes and is relevant to patient care.

What could be some consequences of using composite outcomes in clinical trials?

They can be difficult to interpret and may compromise the relevance of results if effects vary among components.

What is meant by missing data in clinical trials?

Observations that were intended to be made but were not, impacting valid inference from the data.

What are potential methods for handling missing data?

Imputation with the mean, analyzing only complete data, or carrying the last observation forward.