Better standards, validation needed for patient-reported outcome measures in CV research

September 20 2022

2 minutes to read

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50 . analysis Outcome measures reported by the patient The CV results used for trials show that most of them do not have the psychometric properties recommended by the FDA and lack differences of minimal significance, the researchers reported.

“Although the stated importance of patient-reported outcome measures is recognized in clinical and research settings, they remain unused in CVD trials,” Harriet Van Sall, MD, MPH, Associate Professor in the Department of Cardiology at McMaster University and a scientist at the Population Health Research Institute in Ontario, Canada, and colleagues write in Annals of Internal Medicine. “Both clinicians and researchers may lack familiarity with most general and disease-specific outcome measures available for use in CVD. To date, there has been no comprehensive review of the tools and their psychometric properties and validation process. Although the FDA has endorsed a course For approval of drugs and devices based on improved health conditions only, the concordance of patient-reported outcome measures with FDA recommendations for development and validation is unclear.”

A man after checking his heartbeat
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General vs. Disease Specific Measures

Van Spall and colleagues analyzed data from 83 studies that recruited participants with cardiovascular disease and described the development and validation of tools that assess health status and/or quality of life related to health. The researchers divided 50 patient-reported health status outcome measures (PROMs) by type of CVD. Of the 50 included PROMs, five were generic and 45 were disease-specific.

Of the public PROMs, three were validated for participants with CAD and three were validated for participants with HF. Only the short form 36 health survey reported the interpretability data and the method for creating a negligible difference.

The 45 disease-specific PROMs were developed for patients with a range of CV conditions, including cardiac arrhythmias (22%); Canadian dollar (18%); HF (36%); general cardiovascular disease (13%); and other conditions (11%), including implanted VADs or congenital heart disease. Only 20% of disease-specific PROMs had minimally significant difference data; Most (70%) of early disease-specific ROMs with minimally significant variance data used the distribution-based approach.

The researchers found that there are no generic PROMs and only eight disease-specific PROMs that adhere to all recommendations of the FDA framework for psychometric verification.

Public PROMs have not rigorously assessed content viability using the FDA-recommended process, which favors input required from patients with cardiovascular disease (via focus groups or individual interviews) to validate and confirm the importance of residual rupture of membranes elements. For the population of patients under study, the researchers wrote.

Of the 45 pre-disease-specific exercises, only six reported cross-cultural validation or variance of measurement, such as differences between subgroups by age, gender, or language. The authors also noted that 13% of disease-specific PROMs were supported by consistently medium or high-quality evidence across the remaining characteristics of content validity, structural validity, internal consistency, reliability, measurement error, criterion validity, construct validity and response.

Evaluation of psychometric properties

The researchers also applied cansensus-based standards for selection health measuring instruments (COSMIN) Criteria for PROMs to assess whether psychometric properties have been optimally measured. Among the 50 PROMs, only two disease-specific PROMs had all nine characteristics classified as ‘adequate’ or optimally quantified. The mean ratio of psychometric properties considered adequate was 38% for the five general PROMs and 47% for the disease-specific PROMs.

“Of the 50 validated PROMs in the CVD cohorts, only a few adhered to all FDA recommendations, had psychometric characteristics rated adequate by COSMIN, or very important differences were identified,” the researchers wrote. “As PROMs are used to guide FDA approvals for drugs and devices in CVD, there is a need for better adherence to quality standards in PROM validation studies.”