Advances in the treatment of kidney disease through better diagnosis and incentives for preventive care; increased access to home dialysis and kidney transplants; and greater investment in the development of breakthrough technologies like wearable or implantable artificial kidneys were recently launched by a 2019 Executive Order.
AIR recently supported these efforts by working with the American Society of Nephrology’s (ASN’s) Kidney Health Initiative (KHI) in executing a project funded by the Food and Drug Administration (FDA). The project sought to identify and assess relevant patient-reported outcome measures (PROMs) for potential application in clinical trials of novel renal devices. Accomplishments included:
- Conducting a comprehensive, targeted search of published and gray literature to identify and compile existing PROMs validated in the Chronic Kidney Disease/ End Stage Renal Disease patient population, or other conditions, with potential relevance for renal device development;
- Working with the KHI workgroup to iteratively develop a conceptual framework and criteria for evaluating measures that mapped to the framework’s domains;
- Holding an in-person meeting with the ASN Steering Committee and FDA to prioritize measures and develop recommendations for instruments with high relevance and strong measurement properties within each domain. Several measures were identified as helpful starting points for thoroughly capturing data about the impact of future devices on patients’ symptom and life experiences; and
- Meeting with ASN’s Patient and Family Partnership Council to obtain patient and family input, and conducted key informant interviews with industry stakeholders to gather perspectives on the developed conceptual framework and further elicit concepts within domains.
AIR also supported ASN in developing a framework to advance PROs specifically for cramping in dialysis. Tasks included: (1) supplemental targeted literature reviews to identify PROMs that assess patients’ muscle cramping experiences; (2) advising ASN on the development of a conceptual framework outlining the effects of cramping on patients’ health-related quality of life; and (3) conducting virtual focus groups to eliciting patient feedback on the conceptual model and patient experiences with skeletal muscle cramping.
Patients across all focus groups reported similar experiences with skeletal muscle cramping in dialysis including (but not limited to) incredible acute pain during muscle cramps; sore, achy muscles (lingering pain); sleep impacts (inability to stay asleep or get restful sleep); mood changes (frustration, aggravation, irritability); and anxiety due to muscle cramps often catching them off guard.
Patient feedback helped move the conceptual framework from one that was perceived by patients to be more applicable to the overall dialysis experience to one that better reflected patients’ priorities for muscle cramping in dialysis. The resultant conceptual framework is a tool to guide researchers in measuring the impact of new drugs or devices on muscle cramping, as reported by the patient.