Polycystic kidney disease (PKD) includes inherited diseases that cause chronic kidney disease (CKD). Autosomal dominant PKD, despite being a rare condition, is the most common genetic cause of CKD. The majority of individuals with PKD eventually require dialysis or transplant. PKD is the cause of kidney failure in approximately 5 percent of patients who initiate dialysis annually in the United States. These patients are systematically different from others with CKD. They have special characteristics that require attention when thinking about questions and designing comparative effectiveness research (CER) questions; for example, they are typically younger, have less co-morbidities, and are active in the workforce. Additionally, while there have been efforts to engage different stakeholders and establish outcome measures for CKD CER, debate continues among the scientific and regulatory communities in terms of what are the patient-centered outcomes of greatest importance that should be assessed in PKD studies. This lack of an explicit assessment of the different stakeholders’ views is a major gap in knowledge that hinders the development of meaningful CER.
The project’s objectives are (1) to create an infrastructure that allows our community to discuss and prioritize the research focus of PKD; (2) to better understand different stakeholders’ views on the important outcome measures that are patient centered and informed by the best available evidence; (3) to establish an advisory board to guide the process for partnership building, community engagement, and strategic planning; and (4) to develop a list of patient-centered CER questions for a follow-up PCORI research proposal designed by the partnership.