Patients at the Center: Kidney Health Initiative Holds Sixth Annual Stakeholders Meeting

Zach CahillZach Cahill is Marketing and Communications Specialist, Kidney Health Initiative.

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Urgency and innovation are frequent themes in the kidney community. The Kidney Health Initiative (KHI) holds an annual meeting to translate these themes into reality. A public-private partnership with the U.S. Food and Drug Administration (FDA) and over 90 companies and organizations from various stakeholder groups in the kidney community, KHI was established to spur innovation and enhance patient safety.

Patients took center stage at the 2018 KHI Stakeholders Meeting, held in Silver Spring, MD, May 14–15. Each session at the meeting included a patient or care partner who provided the patient perspective on the session topic. Their personal stories and experiences highlighted the collective sense of urgency and communicated the need for advances in therapies for people living with kidney diseases.

KHI values the input of patients by integrating them into every aspect of the initiative. They serve on the KHI Board of Directors and in project workgroups. In 2015, KHI formed the Patient and Family Partnership Council (KHI PFPC), which is made up of 10 patient and care partner advocates representing different therapies and kidney diseases.

“Has there been enough progress? No.”—Pamela Duquette, KHI PFPC member

Pamela Duquette, a member of the KHI PFPC, described her daughter’s 14-year journey with focal segmental glomerulosclerosis (FSGS) during the opening session. Duquette expressed the fear that comes from being a parent of a child who has a disease without a cure. She described the exhausting hospitalizations, debilitating side effects, and fear of infection that come with treating the disease. Her daughter received a living donor transplant that dramatically improved her life and opened the door to school and a social life. Duquette knows the transplant will not last forever and is hopeful for new innovations in the future.

“We are grateful for the progress that has been made, but [my daughter] asks for more, not just for her but for others,” Duquette said.

KidneyX, a new public-private partnership between the U.S. Department of Health and Human Services (HHS) and the American Society of Nephrology, could provide the progress Duquette and other patients and caregivers seek. KidneyX will launch a pilot prize competition around next generation dialysis to provide seed funding to companies that are creating innovations to improve patients’ lives and disrupt the market for potential new therapies for kidney diseases.

KidneyX seeks to accelerate innovation by providing non-dilutive funds to innovators, and by coordinating the various agencies within HHS to accelerate new products, said Bruce Greenstein, HHS Chief Technology Officer. The hope is that innovators and entrepreneurs will receive the funding and approval support from HHS to accelerate new therapies into the hands of clinicians and people living with kidney diseases.

It sometimes takes a long time for an idea to make it through the regulatory process and ultimately start benefiting patients. From the perspective of the FDA, it not only takes time to get new drugs, devices, and therapies approved, but many more years to develop best practices for a new treatment.

The wait time is too long for people living with any disease, according to Janet Woodcock, MD, Director of the FDA Center for Drug Evaluation and Research and keynote speaker at the KHI Stakeholders meeting. Woodcock said consortia like KHI can be effective in moving the needle on innovation and shortening the translation time for new discoveries. She noted that each stage in the approval process can be improved when consortia bridge the gaps and bring together all the important players in the field. KHI can help the translation process in many ways by developing frameworks to track the natural history of a disease, acceptable trial designs and endpoints, and patient-reported outcomes.

“When I first heard of the artificial kidney, my first thought was my wait for a kidney is over.” —Caleb Davy, 16-year-old kidney patient on dialysis for seven years

Caleb Davy has battled kidney diseases his entire life and has been waitlisted for a kidney for seven years. He spoke of the realities of life on dialysis: his four vascular access surgeries, the fluid limitations of only being able to drink a liter a day, and the emotional toll of waiting for a kidney for the past seven years.

Yet he also described the hope he felt when he first heard of the artificial kidney. He called on the researchers and innovators present at the meeting to develop and obtain approval for an artificial kidney. Patients understand that innovations take time, Davy noted, but “if patients must wait, researchers and innovators must persevere to overcome barriers and create results.”

Work is ongoing across fields and around the world to develop alternatives to renal replacement therapy. The KHI Renal Replacement Therapy (RRT) Roadmap project seeks to capture the scientific, technical, and regulatory milestones required to develop a viable replacement for dialysis. The RRT Roadmap will help to identify the challenges, design requirements, and scientific priorities to link with fundable projects through programs such as KidneyX.

“What we don’t take seriously enough is the timeframe for patients.” —Murray Sheldon, MD, Associate Director of Technology, FDA

Karin Hehenberger, MD, PhD, type 1 diabetic and pancreas and kidney transplant recipient, spoke to KHI members about the sense of isolation she has felt as a patient. After being diagnosed with type 1 diabetes at age 14, Hehenberger tried to live a normal life, but ultimately realized that she was hiding her diabetes because she didn’t think others would understand. After earning her medical degree and working in industry, Hehenberger realized there was another way to live with her disease and provide purpose to many others. She founded Lyfebulb to foster patient entrepreneurs—individuals living with a disease who influence product design or develop innovative products—by connecting them with industry or sources of capital. Hehenberger encouraged KHI members to engage with people living with kidney diseases so that products are truly patient-centered and patients can become empowered to live fully and avoid the isolation she experienced.

“I didn’t know my options for treatment and I have never been asked to participate in a research trial.”—Vanessa Evans, home hemodialysis patient and Patient Manager with NxStage

The KHI meeting concluded with a strong call to action from Vanessa Evans. As an individual who has worked in industry and lived with kidney failure, she called on KHI members to help stop the progression to kidney failure. She shared her personal story and interest in seeing more therapies developed to help patients earlier.

“By intervening earlier, patients can be more prepared for dialysis or request preemptive transplants,” Evans said. “Patients need to know the options for RRT, including peritoneal dialysis and home hemodialysis.” When asked if she had the opportunity to participate in a clinical trial, she stated, “I have never been asked, but would gladly enroll. Patients want to help. We just need to ask them to be involved and support their partnership.”

With more than 150 attendees, the Sixth Annual KHI Stakeholders Meeting was the largest in KHI history, providing an opportunity for representatives from industry, government agencies, researchers, patients and care partners, and clinicians to network and build relationships that will create collaboration and innovation. The meeting provided hope for new and innovative solutions to improve lives for people with kidney diseases.

Video and audio recordings of the KHI Sixth Annual Stakeholders Meeting are available on the KHI website at