Clinical Trials and Evidence-Based Oncology Practice Flashcards

Evidence-based practice (EBP) in oncology nursing requires integrating which three components?

Answer: Best research evidence, clinical expertise, and patient values/preferences

The Sackett model of EBP integrates three equally important elements: (1) best available research evidence (systematic reviews, RCTs, clinical guidelines), (2) clinician expertise and judgment, and (3) patient values, preferences, and individual circumstances. All three must be balanced in clinical decision-making.

An umbrella trial design in oncology differs from a basket trial in that an umbrella trial:

Answer: Tests multiple targeted therapies within one cancer type based on molecular subtype

Umbrella trials test multiple targeted drugs within a single cancer histology, with patients assigned to treatment arms based on their tumor's molecular profile (e.g., NCI-MATCH, LUNG-MAP). This is the opposite of basket trials (one drug, multiple cancer types). Umbrella trials efficiently match patients to therapies without running separate trials.

Statistical significance (p < 0.05) in an oncology trial means:

Answer: The probability that the observed result occurred by chance alone is less than 5%

A p-value <0.05 indicates that if the null hypothesis (no treatment difference) were true, the probability of observing results as extreme or more extreme is <5% — a conventional threshold for rejecting the null hypothesis. Statistical significance does NOT equate to clinical significance; effect size and clinical context must also be evaluated.

The Data Safety Monitoring Board (DSMB) in a clinical trial has authority to:

Answer: Recommend early trial termination for benefit, harm, or futility based on interim analyses

The DSMB (or DSMB/IDMC) is an independent committee that reviews unblinded accumulating data at pre-specified interim analyses. It can recommend early stopping for overwhelming efficacy, unexpected harm, or futility (low probability of showing benefit if continued), protecting trial integrity and participant safety.

Number needed to treat (NNT) in an oncology trial communicates which concept?

Answer: The number of patients who must receive the treatment for one additional patient to benefit compared to control

NNT is the reciprocal of the absolute risk reduction (NNT = 1/ARR). It represents the number of patients who need to be treated with the experimental therapy (versus control) to prevent one additional adverse event or achieve one additional positive outcome. Smaller NNT values indicate greater treatment efficiency.

Compassionate use (expanded access) allows patients outside a clinical trial to receive an investigational drug when:

Answer: The patient has a serious or life-threatening condition with no satisfactory alternatives and cannot enroll in the trial

FDA's expanded access (compassionate use) program allows patients with serious or immediately life-threatening conditions to access investigational drugs outside of clinical trials when: (1) no comparable alternatives exist, (2) the patient cannot participate in the clinical trial, (3) potential benefit justifies risk, and (4) access will not interfere with trial completion.