KHI Current Project

Patient-Reported Outcome Measures for Novel Renal Devices

Deliverables

  1. List of Current PROMs that have documentation of reliability and validity for use in individuals with advanced CKD approaching the need for dialysis or individuals with advanced CKD approaching the need for dialysis or individuals receiving dialysis.
  2. List of PROMs, which do not yet have published evidence documenting their reliability and validity when used with CKD or ESRD patients, but assess symptoms (fatigue, depression) and functioning (physical or social) that are known to be affected by dialysis devices and protocols for device use.
  3. Recommendations on which existing PROMs to consider for qualification (could involve adaptation of the existing PROMs or potential development of a new PROM or PROMs).
  4. Work plan to develop, validate, and qualify recommended PROMs in Phase 2 of the project.

Timeline

Completion goal June 2019

In Detail

Dialysis is a life-saving treatment for people with End Stage Renal Disease (ESRD) that can be administered by a range of modalities including hemodialysis (home and in-center), continuous renal replacement therapy (CRRT), hemodiafiltration, and peritoneal dialysis. Despite the proven efficacy of all the above modalities in preventing death due to uremia or metabolic/fluid complications, there has been little change in the basic construct of dialysis technology over the past 50 years.

In particular, there has been little focus on the development of next generation renal replacement therapy (RRT) devices that target the issues that are important to patients—time spent on dialysis, life intrusion and interruption, low energy and restrictions on travel and work. This complete lack of emphasis on patient concerns has resulted in a situation from the patient's perspective where, "Dialysis keeps you alive but does not allow you to live."

In order to address this stagnation in dialysis technologies, there are several synergistic and interconnected multi-stakeholder initiatives currently underway. The Kidney Health Initiative (KHI) issued the Technology Roadmap for Innovative Approaches to Renal Replacement Therapy in October 2018. This roadmap aims to stimulate the innovation needed to move toward advancing solutions that can improve the lives of millions of kidney patients with ESRD. As part of this the roadmap development, patients were surveyed to provide their perspective and/or prioritization of future device attributes. This online survey of 250 patients provided an initial insight into patients' preferences. More recently, the U.S. Department of Health and Human Services (DHSS) initiated an innovation accelerator (KidneyX) that aims to change the face of kidney disease (from diagnostics and medications to next generation dialysis and regenerative medicine).

These are unique and exciting opportunities, which could result in both iterative improvement and disruptive innovation, in the form of portable, wearable and implantable, bio-engineered renal replacement devices. However, for these initiatives to target the issues that are important to patients (and so realize their true impact and potential), we must incorporate the patient perspective into every aspect of the product development process for new technologies. Therefore, it is imperative that patient-reported outcome measures (PROMs) be included in clinical trials (as primary or secondary endpoints) for novel renal devices. These PROM data can then be used to inform regulatory and reimbursement decisions enabling such decisions to be based, in part, on direct patient experience.

PROMs must be the foundation for the development and approval of new RRT technologies. Such a foundation will ensure greater focus on the patient-stated priorities of well-being, function, and survival rather than the historical provider priorities of quantifiable biochemical targets.

The overarching objective of the research is to: 1) identify existing PROMs, and 2) assess their supporting evidence for use in the clinical development and regulatory science pathways for novel renal devices. We will incorporate the patient voice into all aspects of the project.

Role Name Organization
ChairJennifer E. Flythe, MD, MPH, FASNUniversity of North Carolina Kidney Center
MemberFraser BocellU.S. Food and Drug Administration
MemberMartin GerberMedtronic, Inc.
MemberDebbie S. Gipson, MD, MSUniversity of Michigan, Mott Children's Hospital
MemberAmanda GrandinettiKHI Patient and Family Partnership Council
MemberFrank P. Hurst, MD, FASNU.S. Food and Drug Administration
MemberRobert (Bob) Nordyke, PhD, MSEconStat Consulting
MemberRonald Perrone, MD, FASNTufts Medical Center
MemberPrabir Roy-Chaudhury, MD, PhD, FASNUniversity of Arizona
MemberMark L. Unruh, MD, MSUniversity of New Mexico