AOCNP Clinical Trials and Evidence-Based Oncology Practice 2 — Questions and Answers
Question 1: Evidence-based practice (EBP) in oncology nursing requires integrating which three components?
- Hospital policy, patient insurance, and cost-effectiveness data
- Best research evidence, clinical expertise, and patient values/preferences (Correct answer)
- FDA approval status, manufacturer data, and physician preference
- Clinical trial participation, peer consultation, and institutional guidelines
Correct 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.
Question 2: An umbrella trial design in oncology differs from a basket trial in that an umbrella trial:
- Tests one drug in multiple cancer types
- Tests multiple targeted therapies within one cancer type based on molecular subtype (Correct answer)
- Uses a sequential dose-escalation design
- Enrolls only pediatric oncology patients
Correct 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.
Question 3: Statistical significance (p < 0.05) in an oncology trial means:
- The treatment effect is clinically meaningful and important
- The probability that the observed result occurred by chance alone is less than 5% (Correct answer)
- The therapy is superior to all existing treatments
- The trial was adequately powered to detect any difference
Correct 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.
Question 4: The Data Safety Monitoring Board (DSMB) in a clinical trial has authority to:
- Enroll additional patients beyond the planned sample size without sponsor approval
- Recommend early trial termination for benefit, harm, or futility based on interim analyses (Correct answer)
- Change primary endpoints after enrollment begins
- Unblind individual patients to their treatment allocation
Correct 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.
Question 5: Number needed to treat (NNT) in an oncology trial communicates which concept?
- The average number of doses required to achieve response
- The number of patients who must receive the treatment for one additional patient to benefit compared to control (Correct answer)
- The number of adverse events per 100 patients treated
- The number of patients needed for statistical significance
Correct 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.
Question 6: Compassionate use (expanded access) allows patients outside a clinical trial to receive an investigational drug when:
- The drug has completed all Phase III trial requirements
- The patient has a serious or life-threatening condition with no satisfactory alternatives and cannot enroll in the trial (Correct answer)
- The patient specifically requests to pay out-of-pocket for the drug
- The drug has been approved in another country
Correct 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.
AOCNP Clinical Trials and Evidence-Based Oncology Practice 2
Evidence-based practice (EBP) in oncology nursing requires integrating which three components?