ASN Executive Vice President's Update: Assessing KidneyX After Five Years

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To “boost innovation in the fight against cancer” as part of the reignited Cancer Moonshot, the Biden administration, on Thursday, February 2, 2023, announced CancerX (1). According to the administration, this public-private partnership “will build on previous models deployed by successful HHS [US Department of Health and Human Services] InnovationX program accelerators such as KidneyX [The Kidney Innovation Accelerator]” (2).

In the 5 years between Thursday, April 26, 2018—when ASN and the Trump administration established KidneyX (3)—and the announcement about CancerX earlier this year, HHS has launched several similar initiatives, including InnovationX, LymeX, PandemicX, and PreventionX. These public-private partnerships are housed in the Office of the Assistant Secretary for Health at HHS and structured similarly to highlight unmet needs, support innovation, advance solutions, and build community to overcome challenges that private markets and government agencies cannot solve alone.

Representatives Larry Bucshon, MD (R-IN), and Suzan DelBene (D-WA)—who co-chair the Congressional Kidney Caucus—and Senators Ben Cardin (D-MD) and Todd Young (R-IN) have been tireless advocates for the more than 37 million Americans with kidney diseases. Having successfully secured $20 million in funding to support KidneyX since fiscal year (FY) 2020, they are currently seeking an additional $25 million in funding for FY 2024. Their leadership has also resulted in congressional acclaim for KidneyX, making it synonymous with bold innovation. Due to this bipartisan, bicameral support, it is not surprising that the Biden administration used KidneyX as a model to spur innovation in other diseases, especially as part of reigniting the Cancer Moonshot.

To accomplish its mission of accelerating “innovation in the prevention, diagnosis, and treatment of kidney diseases,” KidneyX is built on four pillars (3):

1. Offering funding opportunities through prize competitions for unmet needs in kidney diseases

2. Coordinating regulatory and payment policies across HHS—including the National Institutes of Health, Food and Drug Administration (FDA), and Centers for Medicare & Medicaid Services (CMS)—to clarify pathways to commercializing innovations

3. De-risking commercialization to attract outside investment capital and partnerships

4. Creating a sense of urgency on behalf of people with kidney diseases

By evaluating each of these pillars, ASN can help HHS, the rest of the kidney community, the Congressional Kidney Caucus, and the Senate KidneyX champions assess the first 5 years of KidneyX and plan for its future.

Pillar 1: Offering Funding Opportunities. In its first 5 years, KidneyX designed, supported, and completed six separate prize competitions:

• COVID-19 Kidney Care Challenge

• Patient Innovator Challenge, which was funded by the National Kidney Foundation (NKF)

• Redesign Dialysis Phase One

• Redesign Dialysis Phase Two

• Artificial Kidney Prize Phase One

• Artificial Kidney Prize Phase Two

Through these six competitions, KidneyX has awarded approximately $17 million to 75 winners in 26 different US states (as well as one recipient in the United Kingdom, supported directly by ASN). Besides starting to bring new innovators into the kidney community, KidneyX's winners have included university-based start-ups, such as Relavo; researchers from other fields, such as those at VasoBio, who can apply their technologies toward unmet needs in treating kidney diseases; people ineligible for traditional government-funding mechanisms, such as the winners of the Patient Innovator Challenge; and innovators who explicitly sought advances in other artificial organs, such as the liver and heart, toward the development of an artificial kidney.

As you can imagine, ASN and HHS have learned a lot about administering prize competitions during the past 5 years. These insights will help inform future competitions, outreach efforts to attract non-traditional kidney innovators, and potential partnerships with other non-government organizations, as well as allow the KidneyX Steering Committee (Table 1) to consider other mechanisms for offering funding opportunities.

Table 1.

KidneyX Steering Committee

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Pillar 2, A: Coordinating Regulatory Policies across HHS. In 2012, ASN partnered with the FDA and more than 75 member organizations to launch the Kidney Health Initiative (KHI). With a mission “to catalyze innovation and the development of safe and effective patient-centered therapies for people living with kidney diseases,” KHI identifies surrogate endpoints for the eventual approval of new drugs, strengthens the pipeline of potential therapeutics, creates roadmaps to support product development and early-stage investment, and defines best practices to partner with people living with kidney diseases (4).

To coordinate regulatory policies—particularly for KidneyX's Redesign Dialysis and Artificial Kidney Prize—KHI helps the FDA, ASN, and the rest of the kidney community to frame how innovators improve care and quality of life for people with kidney failure through innovative kidney replacement technologies. Examples of this alignment include: A Technology Roadmap for Innovative Approaches to Kidney Replacement Therapies: A Catalyst for Change (5), Building Capacity to Incorporate Patient Preferences into the Development of Innovative Alternatives to Renal Replacement Therapy (a project that resulted in multiple publications and toolkits) (6), Human Centered Design Toolkit for Kidney Failure (7), and Xenotransplantation: Knowledge and Perception Assessment (which is expected to be finished later this year) (8).

Recognizing the challenges of coordinating regulatory policies, ASN and NKF on Tuesday, November 8, 2022, sent a joint letter to FDA Commissioner Robert M. Califf, MD—who served on the KidneyX Steering Committee before returning to the FDA last year—offering to help address public “concerns about FDA's recent decisions of new drug applications for therapies targeting kidney diseases” (9). ASN and NKF offered to help harmonize “endpoints among kidney patients, researchers, sponsor(s), and FDA”; sponsor “a Patient Focused Drug Development Meeting highlighting the preferences of people living with kidney failure on dialysis”; establish “processes for providing constructive feedback earlier in the regulatory review process to allow sponsors to make necessary adjustments to drug development studies and assure they demonstrate safety and efficacy”; utilize “FDA's labeling authority to convey risks and benefits of a therapeutic to kidney patients and health care professionals”; and ensure “participants in clinical trials designed for people living with kidney diseases accurately reflect the patient population.”

Dr. Califf responded to ASN and NKF on Tuesday, February 14, 2023, emphasizing: “FDA recognizes the morbidity and mortality associated with kidney disease, the unmet needs of patients living with kidney disease, and the urgent need to make additional treatment options available, particularly for underserved minorities.” He added, “FDA looks forward to continued productive and valued interactions with ASN and NKF to help facilitate the development and availability of effective and safe therapies for people living with kidney disease.”

Pillar 2, B: Coordinating Payment Policies across HHS. Based on its ongoing focus on the payment landscape, ASN included recommendations concerning this issue in a response to a CMS Request for Information (CMS-3409-NC) on Tuesday, February 1, 2022 (10). In its response, ASN called on CMS to “elevate the development of artificial kidneys as alternatives to dialysis to a national priority” and to “recognize that innovation is imminent for people with kidney failure but still in an early enough stage to be shaped by public policy.” ASN continues to collaborate with experts to identify potential payment pathways for artificial kidneys that can incentivize innovations for this much-needed kidney replacement option.

The broader problem, however, relates to the Medicare program's challenges in paying for innovative medical devices. In 2021, CMS withdrew—and has yet to replace—a federal regulation, “The Medicare Coverage of Innovative Technology (MCIT) Definition of ‘Reasonable and Necessary,'” due to concerns raised about insufficient patient protections (11). MCIT would have provided 4 years of Medicare coverage for medical devices approved through the FDA Breakthrough Device Designation (BDD).

According to the FDA, BDD is intended to provide patients and health professionals “with timely access to these medical devices by speeding up their development, assessment, and review, while preserving the statutory standards for premarket approval, 510(k) clearance, and De Novo marketing authorization, consistent with the Agency's mission to protect and promote public health” (12). The FDA uses a 510(k) clearance to demonstrate that a new medical device is similarly safe and effective in comparison with another cleared device with the same intended use.

For years, ASN and other members of the kidney community have also raised concerns about two new payment designations within the Medicare End Stage Renal Disease Prospective Payment System, which is also known as “the bundle.” Through the Transitional Drug Add-on Payment Adjustment (TDAPA), eligible new drugs can receive a temporary pass-through payment outside of the bundle, and the Transitional Add-on Payment Adjustment for New and Innovative Equipment and Supplies (TPNIES) is intended to incentivize early adoption of eligible new and innovative equipment, such as home dialysis machines.

For TPNIES, CMS requires evidence of improved care, specifically for people eligible for Medicare coverage. Additionally, by requiring evidence of substantial clinical improvement that is not clearly defined, TPNIES requires an even higher, and less clear, threshold than TDAPA. In fact, the Tablo Hemodialysis System from KidneyX winner Outset Medical is the only medical device to receive TPNIES approval from CMS.

On Thursday, May 11, 2023, the House Committee on Ways and Means' Subcommittee on Health (which has jurisdiction over Medicare) held a hearing on medical innovation and access to care. During the hearing, several members of Congress expressed their concerns about barriers faced by device companies in obtaining insurance coverage after the FDA BDD, thereby stifling innovation and limiting patient options. Among those demanding clear metrics for insurance coverage was Representative DelBene, who has previously sponsored legislation that would provide traditional Medicare coverage for breakthrough devices.

Pillar 3: De-Risking Commercialization. To encourage investment in the kidney arena, KidneyX has held annual in-person and virtual summits (including one on Monday, June 12, 2023, in Washington, DC), pitch sessions at ASN Kidney Week in 2019 and 2022, and Capital Market Days (in London, England, and virtually) and continues to connect winners and other entrepreneurs with experts in the kidney community. Last month, KidneyX initiated a webinar series on kidney entrepreneurship that focuses on common pitfalls preventing the advancement of kidney technologies with strategies to overcome them, trends shaping the xenotransplantation and artificial kidney markets, current patient flows for dialysis to understand where artificial kidneys can provide the most benefit, and the existing landscape of emerging technologies in home dialysis and kidney transplant (13).

In 2022, the FDA cleared devices developed by two KidneyX prize winners. VenoStent received BDD from the FDA on its innovative vascular access technologies, while Alio received FDA 510(k) clearance “for its remote monitoring system that collects data on skin temperature, auscultation, or internal body sounds, and heart rate” (14). Alio's current focus is remote monitoring for patients on dialysis, but “it is working on clinical studies to validate the system's use for other indications.”

In addition to funding the KidneyX Patient Innovator Challenge, NKF in 2022 launched the NKF Innovation Fund, “a new impact investment program aimed at fundamentally disrupting the fight against kidney disease” (15). During its first year, the NKF Innovation Fund supported three KidneyX winners: 34 Lives (formerly Renovera), Kuleana/University of Washington, and Relavo. Relavo has also received $1.25 million in Phase 1 and 2 Small Business Innovation Research funding from the National Science Foundation.

Demonstrating KidneyX's unique potential to excite, catalyze, and activate private markets to support innovation in kidney health, other winners to receive additional funding after their prize award include:

• VenoStent: $2.3 million in seed funding

• VasoBio: $3 million of follow-on grant funding from the California Institute for Regenerative Medicine

• NitriCap Medical: $3.2 million from the Michigan Biomedical Venture Fund

• The Kidney Project/University of California, San Francisco, School of Medicine: $6.7 million from Amgen Ventures, the John and Marcia Goldman Foundation, and other contributors

• Miromatrix Medical: $20 million in Series C financing, followed by $43 million initial public offering (IPO)

• Outset Medical: $277.9 million in its IPO

In evaluating KidneyX, it is important to question whether this level of private funding is enough. Have KidneyX's prize competitions (jointly administered by ASN and HHS) done enough to de-risk commercialization to attract outside investment capital and partnerships? The KidneyX Steering Committee is well positioned to consider this and related questions.

Pillar 4: Creating a Sense of Urgency. On Wednesday, July 10, 2019, the Executive Order on Advancing American Kidney Health (EO 13879) was signed, making it the nation's first presidential directive focused on overarching policy objectives for one disease. On that day, the success of KHI, HHS's commitment to KidneyX, and unified advocacy by ASN and the rest of the kidney community helped make improving kidney health federal policy in the United States.

To “encourage the development of an artificial kidney,” the executive order requested that HHS “produce a strategy for encouraging innovation in new therapies through the Kidney Innovation Accelerator (KidneyX), a public-private partnership between the Department and the American Society of Nephrology” (16). This request helped amplify the community's advocacy efforts, galvanize support in Congress, capture the attention of the media and investors, and focus the KidneyX Steering Committee on the Redesign Dialysis and Artificial Kidney Prize.

Beyond the executive order, KidneyX has involved people with kidney diseases in everything it does: serving as members of the KidneyX Steering Committee (NKF Chief Executive Officer Kevin Longino and musical artist David Rush), incorporating the patient perspective as scored criteria in all submissions, including patients as judges on review panels, and offering a Patient Innovator Prize. As KHI Strategy Committee Member Glenda V. Roberts said when she received the ASN President's Medal at Kidney Week 2022, “I think that the most exciting project that's going on is KidneyX, because KidneyX is facilitating innovation.”

After 5 years, KidneyX has made considerable progress despite the COVID-19 pandemic, a change in presidential administrations, and an expanded portfolio of innovator accelerators at HHS. During the Biden administration, the Office of the Assistant Secretary for Health at HHS has been responsible for KidneyX as a public-private partnership with ASN. HHS Assistant Secretary for Health Admiral Rachel L. Levine, MD, has been hugely supportive of KidneyX, and ASN members, leadership, staff, and I, as well as the rest of the kidney community, owe her—and Representatives Bucshon and DelBene and Senators Cardin and Young—our gratitude and appreciation.

Given Admiral Levine's support, two consecutive presidential administrations' interest in public-private innovation accelerators, like KidneyX; backing from both the Congressional Kidney Caucus and Senate champions; the Executive Order on Advancing American Kidney Health; and KidneyX's first 5 years of success, the time is right for ASN and the rest of the kidney community to advocate for the establishment of the HHS Office of Kidney Health and Transplantation. The announcement of the Organ Procurement and Transplantation Network Modernization Initiative by HHS's Health Resources and Services Administration on Wednesday, March 22, 2023, creates even more momentum, potential, and need for this approach (17).

As I noted in the April 2023 issue of ASN Kidney News, “the oversight, administration, and delivery of care for the more than 37 million Americans with kidney diseases, kidney failure, and kidney transplants are spread across the federal government” (18). Besides offering an ideal home for KidneyX, the HHS Office of Kidney Health and Transplantation would ensure that every HHS entity with a role to play in kidney and transplant health works synergistically. It would also amplify the patient voice and guarantee that ASN and the rest of the kidney community have a centralized way to share their experiences for driving access, accelerating innovation, and maximizing scientific advancements.

With KidneyX as a cornerstone and maximizing access to transplantation a key focus, the HHS Office of Kidney Health and Transplantation would also enhance efforts to offer funding opportunities through prize competitions and other mechanisms, coordinate regulatory and payment policies across HHS, de-risk commercialization to attract outside investment capital and partnerships, and create an even greater sense of urgency on behalf of people with kidney diseases. Such an approach would prove that “we're united 4 kidney health” and committed to intervening earlier, transforming transplant, accelerating innovation, and achieving equity (19).