We hope that by the point that readers see this CJASN article, they will have already read the three accompanyingnyingCJASN articles on patient preference information and patient-centered innovation in KRT (1–3). These four articles represent a combined effort to chronicle the amazingly rapid application of the emerging science of patient insights, and the substantive effect it has already had as a legitimate body of evidence for policy and regulatory decision-making within the realm of kidney and KRT. Each article provides information on the ascendancy of kidney patients within the scientific and policy processes.
We believe, as kidney patients and advocacy leaders for the American Association of Kidney Patients within the national policy arena, that our fellow authors have accurately captured one of the most significant transformations to the underlying basis of kidney medicine and innovation policy and regulatory decision-making in the past 50 years. History demonstrates that the achievement of seemingly distant national policy visions often depends upon the evolution, legitimization, and application of new sciences, disciplines, and technologies years in advance. Public attention and imagination are easily captured by a compelling vision and a well-planned communications strategy. We know this from our careers in national issue campaigns, service in four presidential administrations, and multiple staff roles in the United States Congress. But it is the hard work of scientists, researchers, innovation leaders, and strategic thinkers outside the limelight thinking beyond process, whose persistence and courage as change agents produce the results necessary to maintain public and stakeholder interest and achieve a vision.
2020 marked the 1-year anniversary of the unipolar genetically optimistic vision for American kidney care articulated through the bipartisan July 10, 2019, Executive Order on Advancing American Kidney Care(https://www.whitehouse.gov/presidential-actions/executive-order-advancing-american-kidney-health/), signed by President Trump. The Executive Order served as a framework for significant improvements in kidney health outcomes and a new era in kidney treatment innovations, including disease prevention, precision medicine, artificial wearable and implantable kidneys, and new devices, diagnostics, and biologics. Most significantly, the Executive Order armed kidney patients with greater care choices and reasserted patients into their rightful place as decision-makers for their own care. We are certain that this bipartisan vision will continue under president Biden because the tools and processes within the kidney ecosystem necessary to realize the vision were in place before 2019.
Pic: Cistanche Herb
Over the past decade, the Food and Drug Administration (FDA), led by the FDA Center for Devices and Radiologic Health (CDRH), has pursued a purposeful strategy to capture, quantify, and value patient preference insights across multiple disease populations, and incorporate them into regulatory deliberations and decisions. Substantial efforts have been made to engage kidney patients so as to better understand patient burdens of disease and actual or perceived barriers to better health outcomes. Particular attention has been paid to issues of risk tolerance and acceptance levels among patients for new therapeutics and devices. The search for a “P” value for patient preferences, and insights on the trade-offs patients may be willing to make between the benefits and risks of new therapeutics, is ongoing. How these data will be modeled to accelerate innovations in new KRTs is outlined in “Incorporating Patient Preferences via Bayesian Decision Analysis”.
Pic:Cistanche Extract
The FDA knew that the government alone is not, nor can it be, the primary driver for changing the status of kidney care or improving outcomes through innovation. In 2012, the FDA utilized one of the government's most powerful tools, the power to convene, and entered into a collaborative agreement with the American Society of Nephrology to launch the Kidney Health Initiative (KHI). In 7 years, KHI became the largest, fully representative collaborative in the kidney ecosystem. KHI formally included kidney patient advocacy organizations from the start, and its diverse membership includes multiple federal government agencies, established care providers, start-up enterprises, and nearly every pharmaceutical, device, and diagnostic company involved in kidney disease. KHI and FDAhave leaned forward to provide those interested in developing the next generation of commercially viableKRTs with a detailed study on the path forward, or the“how,” for innovation leaders via the KHI “TechnologyRoadmap for Innovative Approaches to RRT” (4). The roadmap, coupled with a highly inclusive, forthcoming major patient community preference survey, will expand opportunities to embed insights from patient life experiences into the next generation of therapies envisioned by the 2019 Executive Order. The CJASN paper “UsingPatient Preference Information to Inform Regulatory Decision Making: An Opportunity to Spur Patient-CenteredInnovation in Kidney Replacement Therapy Devices” provides details on these strategic developments to benefit kidney patients and support the broader vision of disrupting the status.
In conjunction with KHI efforts, the FDA and CDRH have pursued an aggressive internal and external strategy to incorporate patient insights in their studies and formal decision-making, as appropriate. The CDRH encourages innovation leaders to seek out and directly incorporate patient insights at the start of any new endeavor, far in advance of new submissions to the FDA. This is part of the larger CDRH plan to involve patients across the full product development life cycle, including clinical trial design, so that they are more representative of populations affected by the disease. The CJASN paper “Integrating Patient Perspective into Medical DeviceRegulatory Decision Making to Advance Innovation in Kidney Disease” discusses some of the specific steps the FDA has taken to engage and sustain kidney patient community and advocacy organization involvement.
FDA and KHI efforts have had significant spillover effects on efforts other federal agencies have launched to sub-substantively incorporate kidney patient insights. The NationalInstitutes of Health, National Institute on Diabetes andDigestive and Kidney Diseases have anchored much of theKidney Precision Medicine Project (KPMP) on patient insights as experts in kidney disease and risk acceptance for clinical research, including elective biopsies. This includes patient involvement in formal decision mechanisms, including the KPMP Community Engagement Committee and the KPMP External Expert Panel that guides the project. At the Centers for Medicare and Medicaid Services and the Center for Medicaid and Medicaid Innovation, kidney patients are regularly enlisted to chair or serve on technical evaluation panels aimed at making dialysis quality measurements more relevant to patient decision-making and consumer preferences. The Veterans Administration, a long-time leader in telemedicine, has demonstrated renewed efforts to engage kidney patients and share their insights with other federal agencies, as appropriate.
In practical terms, what does this research and scientific activity around patient preference, as chronicled across this group of CJASN papers, really mean for kidney patients, their caregivers, and the future of status-quo kidney care? The power of the answer is clear in its simplicity. The lives of kidney patients, disproportionately from minority communities, will improve as a new generation of therapeutics and care options, shaped by the insights of patients like themselves, are made available. As authors, we believe a new generation of care solutions, developed with the science of patient insights, will drive diversification in care choices and concurrent demand for coverage by payors, both federal and private, upon their approval by the FDA.
Unfortunately, for kidney patients caught in status kidney care, the pursuit of aspiration is severely limited by the human body and spirit, especially when kidneys fail, as CJASN readers who care for patients know firsthand. But to us, as the authors and national American Association of Kidney Patients advocacy leaders with decades of both dialysis and transplant experience, there are two factors that exacerbate the negative physiologic and potential psycho-logic effects that accompany kidney disease. First, many patients have put on care regimens, like in-center dialysis, without being asked what their aspirations are, or without having a complete discussion about which type of treatment will best positions them to achieve their larger plans, like obtaining a kidney transplant or continuing their work. Second, status quo technologies severely limit the types of care presently available to home and in-center therapies, such as peritoneal dialysis and hemodialysis, and kidney transplant (pre-emptive for those fortunate enough to find a donor ahead of dialysis). These technologies are over 50 years old and, apart from organ transplantation, have serious drawbacks to the freedoms necessary to live a truly active and full life.
Pic: Cistanche can relieve chronic kidney disease
At the American Association of Kidney Patients, were defined ideal and quality kidney disease therapy solutions that best enable patients to fully pursue their aspirations. Aspirations are not merely short-term goals: we mean the ability to pursue a career, travel, start a family, have children, own a home, retire securely, and live an active life despite having to manage a life-threatening disease. We take pride in the fact that our coauthors in thisCJASNseriesare change agents working together to transform the kidney care status quo. Each has consistently welcomed Sidney patients, including us, into their professional endeavors in highly substantive roles: not as spectators, but as expert collaborators whose insights are highly valued and respected. Through their efforts, and their allied networks, we are highly optimistic about the future of kidney care for all patients, especially those yet to be diagnosed.
Disclosures:
P.T. Conway reports roles with the following entities: Chair, Patient Engagement Advisory Committee, US Food, and DrugAdministration; Co-Chair, Global Innovations in Patient-CenteredKidney Care Summit, American Association of Kidney Patients/George Washington University School of Medicine and HealthSciences; Board Member, Kidney Health Initiative; External ExpertPanel, Kidney Precision Medicine Project (KPMP), National Institutes of Health/National Institute of Diabetes and Digestive and Kidney Diseases (NIH/NIDDK); Contract Management Board, NIH/NIDDK, United States Renal Data System (USRDS); Member, Nephrology Specialty Board, American Board of Internal Medicine; Member, Patient Advisory Board, Center for Dialysis Innovation, University of Washington; Moderator, KidneyX 2019Summit; and Policy Stakeholder, Executive Order on AdvancingAmerican Kidney Health. R. Knight reports roles with the following entities: Member, Advisory Council, NIH/NIDDK; Co-Chair, Strategic Plan Stakeholder Engagement Subgroup, NIH/NIDDK; Co-Chair, Community Engagement Committee, KPMP, NIH/NIDDK; Member, Visiting Committee, ScientifificRegistry for transplant Recipients; Member, Patient Advisory Committee, Network 5, Centers for Medicare and Medicaid Services; and policy Stakeholder, Executive Order on Advancing AmericanKidney Health.
References:
1. Tarver ME, Neuland C: Integrating patient perspectives into medical device regulatory decision making to advance innovation in kidney disease. Clin J Am Soc Nephrol 16: 636–638, 202110.2215/CJN.11510720
2. Chaudhuri SE, Lo AW: Incorporating patient preferences via Bayesian decision analysis. Clin J Am Soc Nephrol 16: 639–641,2021 10.2215/CJN.12110720
3. Flythe JE, West M: Using Patient preference information to inform regulatory decision making: An opportunity to spur patient-centered innovation in kidney replacement therapy devices. Clin J Am Soc Nephrol 16: 642–644, 2021 10.2215/CJN.11930720
4. Kidney Health Initiative: Technology Roadmap for Innovative
Approaches to Renal Replacement Therapy, 2018. Available at:
https://www.asn-online.org/g/blast/fifiles/KHI_RRT_Roadmap1.
0_FINAL_102318_web.pdf. Accessed September 11, 2020
