Smartphones, iPads, insulin pumps, and pharmacogenomics: these are all technology developments made in the past 50 years.
While the world around us has exploded with technological advancements, the way we provide dialysis has changed very little in 50 years. Most people with kidney failure still endure treatment sessions of 4 to 6 hours three times a week in a dialysis center. The process is intrusive and affects their quality of life as they are tethered to a schedule and a machine. Not only can travel be restrictive, but maintaining a job and family life can be a challenge as well. Additionally, patients undergoing dialysis often undergo numerous surgeries and experience many adverse effects from cramping to clotting and infections.
Beyond the personal and physical toll, there is also the societal and medical burden. In 2016, Medicare spent $34 billion, or 7%, of all expenditures on treatment of end stage renal disease. Despite this large investment in treatment, the US government spends less than 1% of Medicare kidney care costs in kidney research. According to a US Government Accountability Office study, more is spent on treating kidney failure than the entire National Institutes of Health (NIH) budget.
Despite these enormous costs, patient outcomes are poor. The 5-year survival rate for a hemodialysis patient is worse than that of most cancers. There is no cure for kidney failure, and while transplantation is the optimal form of renal replacement therapy (RRT) available, patients face long wait lists and organ shortages.
Can we break this cycle of a lack of innovation, high costs, poor quality, and poor outcomes? The Kidney Health Initiative (KHI) believes we can. Established in 2012 as a public-private partnership between the ASN and the US Food and Drug Administration (FDA), KHI aims to foster innovation and enhance patient safety for kidney diseases. KHI is a collaborative partnership that aims to bring together all the major stakeholders in kidney disease (patients and patient organizations, big pharma and small biotech, dialysis providers, health professional organizations, and federal agencies [FDA, NIH, the Centers for Disease Control, and the Centers for Medicare & Medicaid Services] as well as international partners).
In 2016, KHI submitted a commitment statement to the White House Summit on Organ Donation to “identify the scientific, technical, and regulatory milestones needed to achieve the goal of creating a bioengineered alternative to dialysis as renal replacement therapy”—in other words, to create a roadmap that would serve as a catalyst to develop patient-centered, patient-driven alternatives to RRT (Figure 1). According to Prabir Roy-Chaudhury MD, PhD, FRCP, Professor of Medicine and the Division Director for Nephrology at the University of Arizona and Co-Chair on the KHI Board of Directors of the American Society of Nephrology, “The only way to change the current construct of dialysis is to focus on patient-centered innovation. We need to identify the issues that are important to patients with kidney failure and then harness the multi-disciplinary strengths of all the stakeholders to develop technologies and pathways that allow patients to live; not just keep them alive.”
KHI’s goals are to:
■ Convene a diverse group of stakeholders, patients and care partners, academics, industry, and regulators.
■ Describe scientific, technical, reimbursement, and regulatory challenges for mechanical, cellular, and hybrid technologies.
■ Create a set of design criteria for future alternatives to RRT.
■ Identify ways to incorporate patient preferences and feedback on design features.
■ Create a roadmap with milestones and opportunities for creating a bioengineered alternative to dialysis.
In March 2017, KHI conducted a workshop of over 100 stakeholders to review the state of the science in RRT and gain input on challenges and potential design criteria for alternatives to RRT. There are numerous initiatives underway to bring advances in technology to a solution that will benefit our patients. These include academic consortia such as NIH’s (Re)Building a Kidney Consortium and translational approaches for cellular therapy or wearable/portable devices by start-up companies.
Despite this progress, many challenges remain. There are knowledge gaps in determining what is “necessary” vs. optimal RRT to minimize uremic symptoms and consequences, how cells can repair or replace a damaged kidney’s functions, and how to incorporate diverse patient needs. Financial coverage for innovative technologies by the single-payer system for ESRD and parallel review with the FDA are also areas that must be explored. The KHI collaboration started with the ASN and FDA and continues to draw a great deal of strength from the ongoing integral interaction.
KHI has established working groups to focus on mechanical, cellular, and vascular access patient-centered design criteria for components of RRT, and a 12-member Patient Advisory Committee is providing the very important patient perspective to these working groups. The necessary innovations are being positioned along a continuum that ranges from technical innovations needed for portable/wearable devices to implantable devices, including bio-hybrid approaches, to alternative directions including chimeric kidneys, xenotransplantation, and innovative biological repair/rebuilding.
“We are taking a systematic approach and being careful not to predetermine the form of the ultimate products that will accomplish our goal,” said Joseph V. Bonventre, MD, PhD, Chief of the Renal Unit and Director of the Bioengineering Division at Brigham and Women’s Hospital and Chair of the KHI RRT Roadmap Steering Committee. “Our patients have waited long enough, and we owe it to them to do all we can to bring new innovative solutions to alleviate the burden of loss of kidney function.”
The first draft of the RRT Roadmap will be available in late summer 2018. But the process is ongoing and will not only guide product development for industry but also be an important tool for early-stage companies with access to investors, business and manufacturing experts, scientists, engineers, patients, payers, and consumers.
In summary, the KHI RRT roadmap project is complex, but the objective is simple. We aim to serve as a catalyst for industry, academia, and other organizations to invest in RRT alternatives, and to help direct those investments to optimize solutions that will successfully address patient needs.