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A quantitative method for measuring the relationship between an objective endpoint and patient reported outcome
Chul Ahn1, Xin Fang1, Phyllis Silverman1
1Center for Devices and Radiological Health, Food and Drug Administration, Silver Spring, Maryland, United States of America.
Plos One
|October 26, 2018
Summary
This study introduces a new statistical method to assess the association between patient-reported outcomes and objective clinical endpoints. This approach enhances the validation of patient-reported outcome measures (PROMs) for drug and device evaluation.
Area of Science:
- Biostatistics
- Clinical Trial Methodology
- Health Outcomes Research
Background:
- Patient-reported outcome measures (PROMs) are crucial for evaluating drug and medical device effectiveness.
- Validating PROMs requires assessing their association with objective clinical endpoints.
- Existing methods for analyzing associations between continuous and discrete variables, especially with correlated data, are limited.
Purpose of the Study:
- To propose a novel statistical method for assessing the association between PROMs and objective clinical endpoints.
- To address limitations in current methods for analyzing correlated continuous and discrete variables.
- To provide a robust approach for validating PROMs in clinical research.
Main Methods:
- Developed a method to estimate the probability of a subject's true disease status based on their reported outcomes.
- Derived a consistent estimator for this probability, applicable to various data types and correlations.
- Utilized simulation studies to illustrate the operating characteristics of the proposed estimator.
Main Results:
- The proposed method effectively assesses the association between PROMs and objective endpoints, even with correlated measurements.
- A consistent estimator was derived and validated through simulations.
- The method's applicability was demonstrated using hypothetical clinical trial data for an ophthalmic device.
Conclusions:
- The developed statistical method offers a valuable tool for assessing PROM validity and reliability.
- This approach can improve the quality of evidence supporting PROMs in regulatory submissions.
- The method has broad applicability in clinical trials across various therapeutic areas.
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