Clinical Trial Design Flashcards
7 cards from real CCDM practice questions. Tap to flip, then mark Knew It or Still Learning — missed cards come back until you master them.
Read the first 7 Clinical Trial Design flashcards as text
In a registry-based randomized clinical trial (RRCT), what is the primary source of outcome data?
Answer: Existing patient registry databases integrated with trial randomization
RRCTs leverage existing national or disease registries to collect outcome data, reducing costs and enabling pragmatic, large-scale evidence generation.
A 'platform trial' differs from a standard multi-arm trial in that it:
Answer: Allows new treatment arms to be added or dropped over time under a master protocol
Platform trials operate under a single master protocol and infrastructure, allowing flexible addition or removal of treatment arms based on pre-specified decision rules.
Which of the following endpoints would be classified as a 'composite endpoint'?
Answer: Time to first occurrence of myocardial infarction, stroke, or cardiovascular death
Composite endpoints combine multiple individual events into a single endpoint, which increases event rates and reduces required sample size, but can complicate interpretation.
In clinical trial design, 'multiplicity' refers to:
Answer: The inflation of Type I error risk when multiple statistical tests or endpoints are evaluated
Testing multiple hypotheses increases the chance of obtaining at least one false-positive result by chance, requiring pre-specified adjustment strategies like Bonferroni correction.
Which ICH guideline specifically provides guidance on statistical principles for clinical trials?
Answer: ICH E9 (R1)
ICH E9 (R1) provides guidance on statistical principles for clinical trials, and its addendum (R1) introduced the estimand framework for defining trial objectives precisely.
A 'hybrid decentralized clinical trial' (DCT) design incorporates which key feature compared to a fully site-based trial?
Answer: Some or all trial activities are conducted remotely at the patient's home or local facility
Hybrid DCTs blend traditional site visits with remote activities (telemedicine, home nursing, wearables), expanding access and potentially improving recruitment and retention.
The estimand framework introduced in ICH E9 (R1) requires specifying which of the following components for each trial objective?
Answer: Population, treatment, variable (endpoint), population-level summary, and intercurrent event handling strategy
The estimand framework requires precisely defining what is being estimated by specifying: the target population, the interventions, the endpoint, how intercurrent events are handled, and the population-level summary statistic.