Patients as Stakeholders in Setting Kidney Disease Research Priorities

Related Article, p. 674 Related Article, p. 674 Chronic kidney diseases are a major public health problem.1Eckardt K.U. Coresh J. Devuyst O. et al.Evolving importance of kidney disease: from subspecialty to global health burden.Lancet. 2013; 382: 158-169Abstract Full Text Full Text PDF PubMed Scopus (813) Google Scholar Multiple elegant experimental studies have led to progress in our understanding of podocyte pathology; inflammatory, fibrotic, and cystic diseases of the kidney; and novel mechanisms leading to acute kidney injury.2O'Toole J.F. Sedor J.R. Kidney disease: new technologies translate mechanisms to cure.J Clin Invest. 2014; 124: 2294-2298Crossref PubMed Scopus (13) Google Scholar, 3LeBleu V.S. Taduri G. O'Connell J. et al.Origin and function of myofibroblasts in kidney fibrosis.Nat Med. 2013; 19: 1047-1053Crossref PubMed Scopus (934) Google Scholar, 4Harris P.C. Torres V.E. Genetic mechanisms and signaling pathways in autosomal dominant polycystic kidney disease.J Clin Invest. 2014; 124: 2315-2324Crossref PubMed Scopus (231) Google Scholar Some, but not all, of these studies have been translated successfully to the clinical arena, resulting in improved quality of care delivered to our patients. Relative to the cost of kidney diseases, the amount of available research funding is small. A recent analysis from the American Society of Nephrology (ASN) suggests that federal research investment corresponds to less than 1% of the yearly Medicare expenditure for patients with kidney disease.5American Society of Nephrology. Kidney research is underfunded compared to the cost of care. http://www.asn-online.org/policy/webdocs/KidneyResearch.pdf. Accessed January 12, 2015.Google Scholar Hence, in the era of limited funding and resources, efforts are needed to set research priorities that reflect the desires of kidney community stakeholders and to focus studies on the most important priorities. Early work to engage stakeholders in priority setting already has begun, yet methods for this process are not well defined. In this issue of AJKD, Tong et al6Tong A. Chando S. Crowe S. et al.Research priority setting in kidney disease: a systematic review.Am J Kidney Dis. 2015; 65: 674-683Abstract Full Text Full Text PDF PubMed Scopus (78) Google Scholar present a systematic review of approaches to research prioritization in kidney disease and summarize different research priorities of various stakeholders, including patients, providers, and policy makers. The authors identified 16 studies, predominantly conducted in developed countries. These studies adopted various approaches to identify key questions and reach consensus, including the Delphi technique, expert panels, consensus conferences, and focus groups. Research priorities identified by these studies were broad and included 3 main areas: classification and diagnosis options for acute kidney injury; diagnosis, prevention, and treatment of chronic kidney disease (CKD) and its complications; and treatment of various symptoms and complications relating to dialysis and transplantation. Notably, in most (75%) priority-setting efforts, patients were not included as stakeholders. This omission is not surprising because patient inclusion in defining research hypotheses can be challenging and does not reflect traditional principal investigator–driven research conventions. This systematic review is a valuable contribution in that it highlights the diversity of approaches for identifying priorities, the broad range of topics identified as research priorities, and the lack of patient voices in priority-setting efforts. How often do patients’ perspectives align with currently funded research? In another report, Tong et al7Tong A. Exploring research priorities in chronic kidney disease. http://www.cochrane-renal.org/docs/CKDprioritiesworkshop_summaryreport_FINAL_140414lowres.pdf. Accessed January 12, 2015.Google Scholar summarized findings from a national workshop conducted in Australia to identify the research priorities in kidney disease in which people with CKD, their family members, nephrologists, nurses, and allied health professionals participated. The authors listed the top 10 questions for each stage of kidney disease; for CKD, the top research question was: “How effective are lifestyle programs (diet, exercise, and smoking cessation) for preventing deterioration in kidney function in patients with early CKD?” Strikingly, only a very small proportion of trials registered at ClinicalTrials.gov focus on dietary and behavioral therapies in CKD.8Inrig J.K. Califf R.M. Tasneem A. et al.The landscape of clinical trials in nephrology: a systematic review of clinicaltrials.gov.Am J Kidney Dis. 2014; 63: 771-780Abstract Full Text Full Text PDF PubMed Scopus (105) Google Scholar This discrepancy between patient expectations and the hypotheses addressed by ongoing clinical studies in kidney diseases argues for broadening efforts to include patient input in topics for clinical studies. Does such patient involvement in setting health care priorities have an impact at the population level? In a randomized controlled trial, Boivin et al9Boivin A. Lehoux P. Lacombe R. Burgers J. Grol R. Involving patients in setting priorities for healthcare improvement: a cluster randomized trial.Implement Sci. 2014; 9: 24Crossref PubMed Scopus (153) Google Scholar analyzed the impact of patient involvement in setting priorities for improving chronic disease management in primary care. Patient participation increased agreement between patients and professionals on common priorities and did not adversely affect the intention of health care professionals to use quality indicators. In other disease states, various approaches for incorporating patient input have been proposed for establishing research priorities. These approaches include the global evidence-mapping priority-setting method and the James Lind Alliance partnerships.10Petit-Zeman S. Firkins L. Scadding J.W. The James Lind Alliance: tackling research mismatches.Lancet. 2010; 376: 667-669Abstract Full Text Full Text PDF PubMed Scopus (69) Google Scholar In the global evidence-mapping method, the first step is to inform the process by preparing a map of evidence based on existing systematic reviews. Then general priority topics are identified by holding 2 workshops (one with patients only; the other with clinicians, researchers, and patients). In the final step, the top 10 priority topics are determined and transformed into research questions in population, intervention, comparison, and outcome-measured format. The objective of the James Lind Alliance is to facilitate priority-setting partnerships by bringing patients, caregivers, and clinicians together to identify and prioritize research needs for addressing the most important treatment uncertainties. Detailed methods (outlined at www.jlaguidebook.org) have been adopted successfully by investigators in the kidney disease community.11Barnieh L, Jun M, Laupacis A, Manns B, Hemmelgarn B. Determining research priorities through partnership with patients: an overview [published online ahead of print November 30, 2014]. Semin Dial. http://dx.doi.org/10.1111/sdi.12325.Google Scholar, 12Manns B. Hemmelgarn B. Lillie E. et al.Setting research priorities for patients on or nearing dialysis.Clin J Am Soc Nephrol. 2014; 9: 1813-1821Crossref PubMed Scopus (221) Google Scholar Funding agencies in various developed countries have begun to mandate patient engagement for funding consideration. In the United States, the Patient-Centered Outcomes Research Institute (PCORI), an independent not-for-profit health research organization, was authorized by the Patient Protection and Affordable Care Act of 2010.13Selby J.V. Beal A.C. Frank L. The Patient-Centered Outcomes Research Institute (PCORI) national priorities for research and initial research agenda.JAMA. 2012; 307: 1583-1584Crossref PubMed Scopus (437) Google Scholar PCORI aims to enable better health care decision making and improve health care delivery and outcomes using evidence-based information obtained from patient-, caregiver-, and community-guided research. PCORI requires meaningful patient engagement in research proposal development (and execution, if the project is funded). Funding agencies in other countries, such as the United Kingdom (National Institute for Health Research) and Canada (Canadian Institutes for Health Research), have adopted similar strategies to enhance patient participation. Recently, in the United States, the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) developed a unique initiative to engage the investigative community in identifying key questions for kidney research: the Kidney Research National Dialogue (KRND). In its first phase, to promote open access and cost-effectively generate new research ideas, KRND created a website and invited more than 1,000 individuals to submit research topics relevant to various domains of kidney disease. The responses were summarized and published recently.14Rys-Sikora K.E. Ketchum C.J. Star R.A. Kidney Research National Dialogue (KRND) Editorial BoardKidney Research National Dialogue overview and commentary.Clin J Am Soc Nephrol. 2013; 8: 1599-1602Crossref PubMed Scopus (7) Google Scholar, 15Bonventre J.V. Boulware L.E. Dember L.M. et al.The Kidney Research National Dialogue: gearing up to move forward.Clin J Am Soc Nephrol. 2014; 9: 1806-1811Crossref PubMed Scopus (17) Google Scholar While this approach was novel and subsequently adopted by other institutes (it is important to note that 3 of the 16 studies included in the review by Tong et al6Tong A. Chando S. Crowe S. et al.Research priority setting in kidney disease: a systematic review.Am J Kidney Dis. 2015; 65: 674-683Abstract Full Text Full Text PDF PubMed Scopus (78) Google Scholar stemmed from the NIDDK-KRND efforts), patients were not explicitly involved. Thus, NIDDK is evaluating alternative approaches to solicit suggestions on research priorities from additional stakeholders. In the past few years, the ASN and the US Food and Drug Administration developed a public-private partnership, Kidney Health Initiative (KHI; www.asn-online.org/khi), to enhance patient safety and foster innovation in kidney diseases.16Archdeacon P. Shaffer R.N. Winkelmayer W.C. Falk R.J. Roy-Chaudhury P. Fostering innovation, advancing patient safety: the Kidney Health Initiative.Clin J Am Soc Nephrol. 2013; 8: 1609-1617Crossref PubMed Scopus (45) Google Scholar KHI includes various stakeholders, and a Patient and Family Partnership Council works closely with the KHI team to advise and make recommendations for member project proposals and KHI projects, ensuring that patients’ voices, experiences, and involvement are meaningful and effective. Finally, it is important to note that involving patient organizations in ongoing trials and observational studies can lead to increased patient participation in related clinical studies, as shown by the experience of the PKD Foundation. Engaging patients in research priority setting, as well as the research itself, poses several potential challenges that warrant attention in future studies. Work is needed to identify whether there are specific types of research priorities for which patient input is maximally beneficial. For instance, patients may be suited best for describing the types and severity of symptoms or outcomes they experience, which can serve as important guides for efforts to drive new discovery. However, patients’ roles in laboratory-based research are less clear because patients often lack in-depth understanding of the mechanistic pathways, pathology, and molecular genetics involved and might not be aware of new technologies or animal models required for basic and translational studies. Similarly, patients might be able to describe the impact of specific health policies or health system processes (eg, insurance coverage or aspects of the clinical encounter) on their individual daily lives or health care experiences, but they may be unaware of specific policy or health system elements that need modification. Further, engaging patients in the process of research requires time and resources.17Ameling J.M. Ephraim P.L. Bone L.R. et al.Adapting hypertension self-management interventions to enhance their sustained effectiveness among urban African Americans.Fam Community Health. 2014; 37: 119-133Crossref PubMed Scopus (12) Google Scholar Though critically needed, the processes whereby funders will account for the resources required to engage patients, community members, and other stakeholders in research efforts are not yet consistently established. Despite the potential challenges, embracing greater patient involvement in research could have many indirect benefits beyond enhancing the relevance of study designs. For instance, patients with kidney disease often are unaware of kidney disease or the importance of identifying new therapies.18Tuot D.S. Plantinga L.C. Hsu C.Y. et al.Chronic kidney disease awareness among individuals with clinical markers of kidney dysfunction.Clin J Am Soc Nephrol. 2011; 6: 1838-1844Crossref PubMed Scopus (113) Google Scholar If they are engaged in the process of research, patients may be more aware of research efforts and better equipped to be informed end consumers of research. In turn, increased awareness and knowledge could enhance support for public funding of kidney research. However, as Tong et al6Tong A. Chando S. Crowe S. et al.Research priority setting in kidney disease: a systematic review.Am J Kidney Dis. 2015; 65: 674-683Abstract Full Text Full Text PDF PubMed Scopus (78) Google Scholar note, approaches to patient engagement in research are still in infancy. Additional studies are warranted to identify optimal methods for patient engagement, elucidate topics for which patient input is most valuable to the scientific research agenda, and develop metrics to assess other administrative and financial burdens. The resulting studies will help us determine whether patients can influence the quality and utility of precious kidney disease research investments, and if so, how these individuals may have the greatest possible impact. Support: None. Financial Disclosure: The authors report receipt of NIDDK grants (Dr Navaneethan: R01 DK101500; Dr Boulware: R34 DK094116, R01 DK098759; Dr Sedor: R01 DK097836, UM1 DK100846, U54 DK083912). Research Priority Setting in Kidney Disease: A Systematic ReviewAmerican Journal of Kidney DiseasesVol. 65Issue 5PreviewResources for research are insufficient to cover all unanswered questions, and therefore difficult choices about allocation must be made. Recently there has been a move toward more patient-centered research. This study aims to evaluate approaches to research prioritization in kidney disease and describe research priorities of patients with kidney disease, their caregivers, the health care providers involved in their care, and policy makers. Full-Text PDF

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