Catalyzing Patient Centered Device Innovation

By Zach Cahill

April 13, 2020

cjasn_KHI_toward_patient_centered_innovation.PNGThe COVID-19 pandemic has brought new urgency to the need for alternatives to in center dialysis for people with kidney failure. Historically, integrating patient preferences into device development has been limited. Developments in the kidney community in recent years, including the KidneyX and the Advancing American Kidney Health initiative, have accelerated development of transformative devices for kidney replacement therapy.

As device development in the kidney space accelerates, patient perspective must inform these advances. The US Food and Drug Administration (FDA) has recognized that the patient perspective is essential in device development. Using patient reported outcome measures presents an opportunity to encourage more patient-centered innovation in the kidney community.

To help ensure that patients drive needed change, the Kidney Health Initiative (KHI), a public-private partnership between the FDA and the American Society of Nephrology committed to catalyzing innovation and the development of safe and effective patient centered therapies for people with kidney diseases, convened a workgroup chaired by Jennifer E. Flythe, MD, MPH, FASN of the University of North Carolina School of Medicine.

The workgroup’s objectives, as published in the Clinical Journal of the American Society of Nephrology were to develop a conceptual framework for a health related quality of life patient reported outcome measure (PROM), identify and map existing PROMs to the framework, and outline next steps for identifying PROMs appropriate for use in evaluating transformative kidney replacement therapy devices.

For PROs, a conceptual framework defines concepts measured by a PRO instrument and relationships between measures, domains, and scores. The workgroup’s framework included three domains likely to be impacted by all transformative kidney devices:

  • Fatigue and energy
  • Intrusion on family and social life
  • Ability to be active
     

A fourth domain was included to capture any device-specific symptoms that the transformative device might impact.

The workgroup then evaluated dozens of different instruments relevant to the framework domains and prioritized instruments most applicable to the clinical trial and regulatory context. This exercise was necessary because many PROM’s used in clinical practice may not be suitable for regulatory trials. The workgroup acknowledged that additional work to modify existing or develop new PROMs is needed.

The conceptual framework is device agnostic, intended to provide a guide to the priorities of people with kidney failure and show how these priorities might be measured in the context of a regulatory trial. It is also intended to stimulate the development of more rigorous and meaningful PROMs capturing patient-prioritized outcomes that may be alleviated by transformative kidney replacement devices. Developers and clinicians should be motived by the framework to prioritize outcomes that are important to people with kidney failure throughout the complete product lifecycle.

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Author:
Zach Cahill
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cjasn_KHI_toward_patient_centered_innovation.PNGThe COVID-19 pandemic has brought new urgency to the need for alternatives to in center dialysis for people with kidney failure. Historically, integrating patient preferences into device development has been limited. Developments in the kidney community in recent years, including the KidneyX and the Advancing American Kidney Health initiative, have accelerated development of transformative devices for kidney replacement therapy.

As device development in the kidney space accelerates, patient perspective must inform these advances. The US Food and Drug Administration (FDA) has recognized that the patient perspective is essential in device development. Using patient reported outcome measures presents an opportunity to encourage more patient-centered innovation in the kidney community.

To help ensure that patients drive needed change, the Kidney Health Initiative (KHI), a public-private partnership between the FDA and the American Society of Nephrology committed to catalyzing innovation and the development of safe and effective patient centered therapies for people with kidney diseases, convened a workgroup chaired by Jennifer E. Flythe, MD, MPH, FASN of the University of North Carolina School of Medicine.

The workgroup’s objectives, as published in the Clinical Journal of the American Society of Nephrology were to develop a conceptual framework for a health related quality of life patient reported outcome measure (PROM), identify and map existing PROMs to the framework, and outline next steps for identifying PROMs appropriate for use in evaluating transformative kidney replacement therapy devices.

For PROs, a conceptual framework defines concepts measured by a PRO instrument and relationships between measures, domains, and scores. The workgroup’s framework included three domains likely to be impacted by all transformative kidney devices:

  • Fatigue and energy
  • Intrusion on family and social life
  • Ability to be active
     

A fourth domain was included to capture any device-specific symptoms that the transformative device might impact.

The workgroup then evaluated dozens of different instruments relevant to the framework domains and prioritized instruments most applicable to the clinical trial and regulatory context. This exercise was necessary because many PROM’s used in clinical practice may not be suitable for regulatory trials. The workgroup acknowledged that additional work to modify existing or develop new PROMs is needed.

The conceptual framework is device agnostic, intended to provide a guide to the priorities of people with kidney failure and show how these priorities might be measured in the context of a regulatory trial. It is also intended to stimulate the development of more rigorous and meaningful PROMs capturing patient-prioritized outcomes that may be alleviated by transformative kidney replacement devices. Developers and clinicians should be motived by the framework to prioritize outcomes that are important to people with kidney failure throughout the complete product lifecycle.

Date:
Monday, April 13, 2020