Related Experiment Video
Updated: Nov 18, 2025
06:46
Competing-Risk Nomogram for Predicting Cancer-Specific Survival in Multiple Primary Colorectal Cancer Patients after Surgery
Published on: September 27, 2024
502
Demystifying the estimand framework: a case study using patient-reported outcomes in oncology.
Mallorie H Fiero1, Madeline Pe2, Chana Weinstock3
1Office of Biostatistics, US Food and Drug Administration, Silver Spring, MD, USA.
The Lancet. Oncology
|October 1, 2020
Summary
The estimand framework clarifies patient-reported outcome (PRO) objectives in cancer drug trials. Applying this structured approach enhances the interpretation of patient experiences and drug benefit-risk assessments.
Area of Science:
- Clinical trial methodology
- Oncology drug development
- Patient-reported outcomes
Background:
- Patient-reported outcome (PRO) measures are crucial for assessing cancer drug benefits and risks.
- Ill-defined PRO research objectives in clinical trials can lead to inaccurate conclusions about patient experiences.
- The estimand framework offers a structured method to align trial objectives with design, endpoints, and analysis.
Purpose of the Study:
- To provide an overview of the estimand framework for a multistakeholder audience, based on ICH E9(R1).
- To demonstrate the application of the estimand framework to PRO research objectives using a hypothetical breast cancer trial.
- To improve the design, analysis, and interpretation of PRO results in oncology drug development.
Main Methods:
- Review of the estimand framework principles.
- Application of the framework to a hypothetical breast cancer clinical trial.
- Focus on physical function as a PRO to define specific research objectives.
Main Results:
- The estimand framework provides a structured approach to defining PRO research objectives.
- Application in a hypothetical trial illustrates improved clarity in study design and interpretation.
- The framework facilitates better alignment between trial objectives and PRO data.
Conclusions:
- Implementing the estimand framework can enhance the clarity and reliability of PRO data in cancer drug development.
- This structured approach improves communication among stakeholders regarding patient experiences.
- Clearer interpretation of PROs leads to more accurate benefit-risk assessments for oncological drugs.
More Related Videos
07:42Patient-Derived Tumor Explants As a "Live" Preclinical Platform for Predicting Drug Resistance in Patients
Published on: February 7, 2021
5.4K
06:28E-Patient Counseling Trial E-PACO: Computer Based Education versus Nurse Counseling for Patients to Prepare for Colonoscopy
Published on: August 1, 2019
8.6K
Related Concept Videos
01:23Dosage Regimen Designs: Nomograms and Tabulations
75
Nomograms and tabulations are vital tools used by clinicians to design accurate and individualized dosage regimens. These instruments provide a straightforward method for adjusting dosages based on individual patient characteristics, including age, weight, and physiological condition. The foundation of a drug's nomogram is population pharmacokinetic data collected and analyzed using specific models. This data simplifies complex equations, presenting them diagrammatically or tabularly for easy...
75
01:24Kaplan-Meier Approach
393
The Kaplan-Meier estimator is a non-parametric method used to estimate the survival function from time-to-event data. In medical research, it is frequently employed to measure the proportion of patients surviving for a certain period after treatment. This estimator is fundamental in analyzing time-to-event data, making it indispensable in clinical trials, epidemiological studies, and reliability engineering. By estimating survival probabilities, researchers can evaluate treatment effectiveness,...
393
01:11Guidelines for Writing Outcome
3.4K
When developing expected outcomes for a patient care plan, the nurse should adhere to the following recommendations:
Patient outcomes reflect the patient's response to the goal rather than what the nurse aims to achieve. Terminology should be observable and measurable to avoid the reader's interpretation. The desired outcome should be realistic and achievable in the designated care timeframe. Expected outcomes should align with adjunctive therapies. The outcome should enhance care...
Patient outcomes reflect the patient's response to the goal rather than what the nurse aims to achieve. Terminology should be observable and measurable to avoid the reader's interpretation. The desired outcome should be realistic and achievable in the designated care timeframe. Expected outcomes should align with adjunctive therapies. The outcome should enhance care...
3.4K
01:21Cancer Survival Analysis
525
Cancer survival analysis focuses on quantifying and interpreting the time from a key starting point, such as diagnosis or the initiation of treatment, to a specific endpoint, such as remission or death. This analysis provides critical insights into treatment effectiveness and factors that influence patient outcomes, helping to shape clinical decisions and guide prognostic evaluations. A cornerstone of oncology research, survival analysis tackles the challenges of skewed, non-normally...
525
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
265
Biopharmaceutical studies constitute a vital field aiming to enhance drug delivery methods and refine therapeutic approaches, drawing upon diverse interdisciplinary knowledge. In research methodologies, the choice between controlled and non-controlled studies significantly influences the study's reliability and accuracy.
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...
265
01:15Nursing Evaluation
4.0K
The evaluation stage signals the end of the nursing process. The nurse gathers evaluative data to assess whether or not the patient has attained the expected results. Whereas the nurse collects data in the nursing assessment to identify the patient's health concerns, the evaluation stage data determines if the indicated health issues are resolved. Evaluative data collection includes two sections: the data acquired to evaluate patient outcomes and the time criteria for data collection.
4.0K