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.
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Estimands: Targets in Trials
- An estimand is the target value you want to estimate in a clinical trial's target population.
- It represents the treatment's benefit or risk, often a comparison between treatment and control groups.
Treatment Policy vs. Trial Product
- Treatment policy (intention-to-treat) assesses outcomes for all randomized participants, regardless of compliance.
- Trial product (per-protocol) assesses outcomes in a hypothetical perfect-compliance population.
Prescription Decisions: Use ITT
- For initial prescription decisions, the treatment policy (intention-to-treat) estimate is more relevant.
- It reflects real-world scenarios where patient compliance is uncertain.