Lawrenceville, NJ, USA—May15, 2023—Value in Health, the official journal of ISPOR—The Professional Society for Health Economics and Outcomes Research, announced today the publication of an ISPOR Good Practices Report providing recommendations for assessing the need to demonstrate comparability among ways to collect patient responses (eg, using a tablet or smartphone app versus paper) about their symptoms in research studies. The report, “Updated Recommendations on Evidence Needed to Support Measurement Comparability Among Modes of Data Collection for Patient-Reported Outcome Measures: An ISPOR Task Force Good Practice Report,” was published in the May 2023 issue of Value in Health.
With the rapid rise of electronic data collection in clinical trials, this report updates and simplifies recommendations for medical product companies, clinical researchers, and regulators. The new recommendations can make clinical trials for medical products more practical and accessible with better data collection and, potentially, make treatments available to patients sooner.
Patient-reported outcomes (PROs) are direct reports from patients—without interpretation by clinicians or others—on their experience of a disease and/or its treatment. Incorporating patient experience and the patient voice is a critical component of clinical trials for the US Food & Drug Administration. Historically, patients provided important feedback by filling out PRO questionnaires on paper. However, with advances in technology, electronic questionnaire formats have become commonly used in clinical trials. They can be easier for patients to use and improve the quality of data being collected about a patient’s experience with a disease and/or treatment.
The initial question for the Task Force was understanding whether paper and electronic questionnaires measure the same thing—are the results they provide comparable? Previously, to implement questionnaires on electronic systems there was often a need to demonstrate comparability prior to initiating a clinical trial. ISPOR’s earlier electronic data collection Good Practices Reports provided recommendations on how to do this to support a medical product labeling claim.
Advances in technology along with a substantial amount of outcomes research evidence regularly demonstrated measurement comparability between electronic and paper modes across various devices. This meant that values recorded for the same question using different types of data collection fell within an accepted range of each other. This was true for several questionnaires across a range of technologies. The robust evidence of comparability between modes of PRO data collection demonstrated that additional testing is no longer needed for many questionnaires.
“In a nutshell, a decision regarding the need to generate additional evidence is dependent on the amount of available supporting evidence demonstrating whether the change from paper to electronic has impacted the meaning of the questionnaire. For many commonly used questionnaires this evidence is available,” said author Paul O’Donohoe, MSc, Medidata, London, UK. The report’s recommendations apply broadly, from paper to electronic or when researchers are using a range of devices or data collection technologies within a trial, such as a bring your own device study in which patients can use their own smartphone or other device to respond to questions about their symptoms.
“This report helps advance clinical trials that incorporate the patient voice through patient-reported outcome data collection,” said author Sonya Eremenco, Executive Director of the PRO Consortium at the Critical Path Institute, Tucson, AZ.
The ISPOR Good Practices Report was accompanied by an editorial from Lori McLeod, PhD and Nicholas Rockwood, PhD, both of RTI Health Solutions. Their commentary, “Comparability Among Modes of Data Collection for Patient-Reported Outcome Measures: Opening the Gates for Faithful Migration,” also published in the May 2023 issue of Value in Health, provides important takeaways for those interested in data collection of patient-reported outcome measures. “The new ISPOR Good Practices Report on patient-reported outcome measures provides a wonderful summary of the immense quantity of published research in this area,” said McLeod and Rockwood. “Finalization of these recommendations is an important accomplishment by the diverse group of stakeholders with different backgrounds represented, including the US Food and Drug Administration, academia, research organizations, electronic clinical outcomes assessment service providers, and the life sciences industry.”
The editorial notes that while the report goes a long way toward meeting its goals, ISPOR could produce a valuable and practical companion to the report by providing case studies of the complete process. These case studies, say the authors, could serve as a detailed roadmap for considering measurement comparability and evaluating supportive evidence. McLeod and Rockwood also suggest that there will be “further debate” on a number of issues, including the use of the term “equivalence,” the measurement of evidence sufficiency, and security and privacy.
Background on ISPOR Patient Reported Outcome and Clinical Outcome Assessment Good Practices Reports
ISPOR has published nearly 70 expert consensus good practice standards for outcomes research (clinical, economic, and patient-reported outcomes) and on the use of this research in healthcare decision making. Of the nearly 70 reports, 13 are based on the US FDA’s 2009 Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. A 14th report on performance outcomes assessment will be published this summer. All of ISPOR’s Good Practice Reports, organized by research category, are available here.
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ABOUT ISPOR
ISPOR, the professional society for health economics and outcomes research (HEOR), is an international, multistakeholder, nonprofit dedicated to advancing HEOR excellence to improve decision making for health globally. The Society is the leading source for scientific conferences, peer-reviewed and MEDLINE®-indexed publications, good practices guidance, education, collaboration, and tools/resources in the field.
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ABOUT VALUE IN HEALTH
Value in Health (ISSN 1098-3015) is an international, indexed journal that publishes original research and health policy articles that advance the field of health economics and outcomes research to help healthcare leaders make evidence-based decisions. The journal’s 2021 impact factor score is 5.156 and its 5-year impact factor score is 6.779. Value in Health is ranked 9th of 88 journals in health policy and services, 18th of 109 journals in healthcare sciences and services, and 50th of 381 journals in economics. Value in Health is a monthly publication that circulates to more than 10,000 readers around the world.
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ABOUT ISPOR GOOD PRACTICES REPORTS
ISPOR has earned an international reputation for research excellence based, in part, on its Good Practices Reports. These highly cited reports are expert consensus recommendations on good practice standards for outcomes research (clinical, economic, and patient-reported outcomes) and on the use of this research in healthcare decision making. ISPOR Task Forces comprise subject matter experts representing different stakeholders from diverse work environments (ie, regulators, payers, manufacturers, technology assessors, etc from research, government, academic, and industry sectors around the world). All ISPOR Good Practices Reports are published in the Society’s scientific journal, Value in Health, and are made freely available as part of the Society’s mission. The Society’s Good Practices Reports have been recognized with an ASAE “Power of A” award that acknowledges innovative, effective, and broad-reaching programs that have a positive impact on the world.