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  1. Advancing Regulatory Science

Adapting a Measure of Heart Failure to an Adolescent Population

CERSI Collaborators: Stanford University: Christopher Almond, MD, MPH; Chiu-Yu Chen, MD, PhD; Korey Hood, PhD; Molly Tanenbaum, PhD

FDA Collaborators: Center for Devices and Radiological Health: Fraser Bocell, PhD; Vasum Peiris, MD, MPH; Anindita Saha; Brittany Caldwell, PhD; Michelle Tarver, MD, PhD

CERSI Subcontractors: Mayo Clinic: Jonathan Johnson, MD; Jennifer Ridgeway, PhD; Emma Behnken

Project Start Date: December 2018

Regulatory Science Challenge

The evaluation and approval of medical devices in pediatric populations lags far behind adult populations. Many devices, including life-saving devices, are adapted and used in children without the benefit of first going through a well-controlled study to evaluate their safety and effectiveness. One way to encourage studies of devices in pediatric populations is to provide additional measures to facilitate efficient evidence generation, such as Patient-Reported Outcomes Measures (PROMs). PROMs are questionnaires about health status that are answered by the patient. PROMs have become an important method to include the patient voice in clinical studies and can help provide evidence of the safety and effectiveness of a device and evaluate the impact a treatment on a patient’s life. However, PROMs must be developed to fit the clinical research in which they will be used, and companies within the medical device industry do not always have the resources necessary to develop new PROMs for new areas of research. Thus, this project was conceived to provide an example of how to efficiently and cost-effectively adapt an existing PROM, making changes to the questions, to fit a new population; in this case, pediatric heart failure patients.

The adaptation of a PROM for appropriate use in this population will provide an additional method for collecting evidence to support the use of devices in pediatric heart failure. Additionally, it provides an example of methods other stakeholders can use to adapt other PROMs for use in device studies.

Project Description and Goals

The aim of this project is to understand how adolescents perceive the symptoms and impact of their heart failure, while adapting existing adult PROMs to capture and quantify this perception for use in clinical care and regulatory decision making. The development of a PROM is a multi-step undertaking where each subsequent step is informed by the prior. The major steps include a literature review of existing publications and evidence, a short survey to capture clinician input, focus groups and interviews to confirm the question selection and inform adaptation, the completion of the adapted PROM, and a study to evaluate the PROM’s properties in a larger sample. The final PROM will be available for further testing and potential use in pediatric cardiology device submissions.

Research Outcomes/Results

An analysis of interview transcripts found common and consistent themes from patient interviews, which included:

  1. Symptoms of fatigue, shortness of breath, and chest discomfort.
  2. Limitations in ability to perform ordinary tasks, participate in extracurricular activities, and keep up with peers.
  3. Social and emotional impacts from being treated differently by others.
  4. Burden of medical care.

Caregivers reported similar symptoms and impacts on patient function, and further described social exclusion by peers and caregiver anxiety about illness burden in the future such as college and career limitations, ability to have children, etc.

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