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JAMAevidence JAMA Guide to Statistics and Methods

Estimands, Estimators, and Estimates With Dr Little

Aug 3, 2023
Dr. Roderick J. Little, PhD, discusses estimates, estimators, and their importance in capturing the benefit or risk of a treatment. They explore estimation in clinical practice, intention to treat vs per protocol estimates, benefits and challenges of randomization, and alternative methods for estimating trial products.
22:06

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Quick takeaways

  • Different estimates, such as the treatment policy estimate and the trial product estimate, are used to capture the benefit or risk of a treatment and inform decision-making based on the population assigned to treatment.
  • Valid and unbiased estimators are essential for accurately comparing outcomes, summarizing causal effects, and making informed decisions based on the findings of a clinical trial.

Deep dives

Definition and importance of estimates

An estimate is the value that we are trying to determine for a specific treatment in a target population. It captures the benefit or risk of the treatment and compares the summary effect of the treatment to a comparator. The estimate can be measured through different approaches, such as the treatment policy estimate (intention to treat estimate) that includes all individuals assigned to any treatment, regardless of compliance, and the trial product estimate (per-protocol estimate) that focuses on the outcome of individuals who actually comply with the assigned treatment. The use of these estimates depends on the specific context and the goals of the study.

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