Overcome Barriers to Innovation

Since 2012, projects have contributed recommendations and resources that are addressing the barriers to innovation for people living with kidney diseases. For innovators, projects provide subject matter expertise on the best methods of designing and conducting clinical trials or advice on how to include the patient perspective in the development of new products. KHI intends to outline a pathway to develop new therapies for kidney diseases by increasing the number of clinical trials and products in development.

Download and share these recommendations and resources with colleagues and innovators to transform the available therapies and help KHI meet its vision to prevent, treat, and cure kidney diseases.

Clinical Trial Endpoints for Dialysis Vascular Access

This project convened diverse stakeholders (health professionals, industry, dialysis organizations, and federal agencies) to clarify appropriate trial endpoints for vascular access trials best suited to inform clinical, regulatory, and coverage decisions, in those circumstances where clinical data is required to support the decisions. The workgroup developed parallel white papers on catheters, fistulas, and grafts endpoints. Learn more about this project here.

Clinical Trial End Points for Hemodialysis Vascular Access FDA Regulatory Perspectives for Studies on Hemodialysis Vascular Access Recommended Clinical Trial End Points for Dialysis Catheters Definitions and End Points for Interventional Studies for Arteriovenous Dialysis Access

Outcome Measures in Lupus Nephritis Trials

In partnership with the Lupus Nephritis Trial Network, a workgroup analyzed existing data to test for clear, valid endpoints for lupus nephritis trials. The workgroup contacted pharmaceutical companies that have completed trials in lupus nephritis to discuss the inclusion of their data in this project. Recognizing the value of including physicians treating these patients, the workgroup recruited global cohort sites to participate in the project.

Upon completion of the data analysis, the workgroup recommended a core set of outcome measures, biomarkers, surrogate markers, and clearly defined terms that should be incorporated into all future lupus nephritis trials. Developing these measures will make it much more feasible for companies to invest in the research necessary to improve treatments for patients with this serious disease.

Establishing Surrogate Kidney Endpoints for Lupus Nephritis Clinical Trials: Development and Validation of a Novel Approach to Predict Future Kidney Outcomes

Data Harmonization in Kidney Transplantation

In coordination with the Clinical Data Interchange Standards Consortium (CDISC), Coalition for Accelerating Standards and Therapies (CFAST), FDA, and TransCelerate BioPharma, Inc., KHI assembled a workgroup to partner with these groups and helped define appropriate standards in the therapeutic area of kidney transplant. Learn more about this project here.

CDISC Therapeutic Area User Guide for Kidney Transplantation

Data Standards in Diabetic Kidney Disease

In coordination with the Clinical Data Interchange Standards Consortium (CDISC), Coalition for Accelerating Standards and Therapies (CFAST), FDA, and TransCelerate BioPharma, Inc., KHI assembled a workgroup that helped define appropriate standards in the therapeutic area of diabetic kidney disease. KHI's specific role was providing clinical and medical expertise. Learn more about this project here.

CDISC Therapeutic Area User Guide for Diabetic Kidney Disease

Pragmatic Trials in Dialysis: Challenges and Opportunities

Few randomized clinical trials have been conducted in this setting due to various perceived barriers – including FDA regulations related to data collection, lack of clarity around best practices for ensuring human subject protections, and cumbersome CMS requirements related to billing for patients involved in research. Learn more about this project here.

Pragmatic Trials in Maintenance Dialysis: Perspectives from the Kidney Health Initiative

Pharmacokinetics in Patients Receiving Continuous Renal Replacement Therapy

Patients in the intensive care unit often develop acute kidney injury (AKI) and require continuous renal replacement therapy (CRRT), a procedure that differs significantly from outpatient dialysis. While the rate of acute kidney injury in the United States has doubled during the last 10 years, less than 20% of the drugs used have been studied with regard to appropriate dosing for patients in intensive care units (ICUs) receiving CRRT. As a result, clinicians and pharmacists often end up making best guesses in terms of dosages of life saving drugs in critically ill patients.

The growing number of cases of AKI, increasing use of CRRT, and reliance on a large number of potentially life-saving medications in these critically ill patients necessitate a better understanding of basic pharmacokinetics in this setting. Standardized pharmacokinetics guided drug dosing studies during the pre-approval development process are urgently needed for drugs likely to be used in critically ill patients, and similar studies should be conducted on currently approved drugs commonly used in these patients.

A workgroup that included FDA staff, evaluated medication dosing during CRRT and prioritized drugs or classes of drugs to help generate key data to improve treatment for these critically ill patients.

Pharmacokinetic Assessment in Patients Receiving Continuous RRT: Perspectives from the Kidney Health Initiative

Regulatory Policies and Positions Affecting Device Approval in the US

For medical device development, it is essential that device manufacturers and other stakeholders have optimal information regarding the standards and requirements for approval of clinical trial protocols and market approval for devices at the U.S. Food and Drug Administration. A workgroup was assembled that defined and studied a few broad categories of devices that have been marketed outside of the United States. The workgroup is determining the value of these devices in patient care, ascertaining pathways to expedite approvals in the United States (if appropriate), and then summarizing their findings in a white paper. Learn more about this project here.

Hemodiafiltration to Address Unmet Medical Needs ESKD Patients Regulatory Considerations for Hemodiafiltration in the United States

Prioritizing Symptoms of ESRD Patients for Developing Therapeutic Interventions

While dialysis is a life-saving therapy for patients with end stage kidney disease (ESKD), a variety of symptoms occur following treatment that significantly diminishes the quality of life of people living with kidney disease. Patients have identified the need to alleviate these symptoms as a key research area, prioritizing it above other health outcomes. Alleviation of these symptoms could result in increased quality of life and better patient-reported outcome measures. Despite the patient demand for new therapies to address these symptoms, no drug or device has been approved by the US Food and Drug Administration (FDA) to date. KHI assembled a workgroup to confirm and prioritize symptoms experienced by ESKD patients. After identifying three prioritized symptoms, the group then identified near-term, actionable research goals for these prioritized symptoms. Learn more about this project here.

Symptom Prioritization among Adults Receiving In-Center Hemodialysis: A Mixed Methods Study Fostering Innovation in Symptom Management among Hemodialysis Patients Paths Forward for Insomnia, Muscle Cramps, and Fatigue

Advancing Technologies to Facilitate Remote Management of Patient Self-Care in Renal Replacement Therapy (RRT)

There is uncertainty regarding the approval of advanced medical devices, accessories, software, and mobile medical applications for consumer use. Reimbursement policies vary significantly across Medicaid, Medicare, and commercial insurance. This regulatory and payer misalignment and the knowledge gap is hindering patient access to technologies and services that have potential to improve outcomes for patients on home based therapies. KHI assembled a workgroup tasked with addressing the state of remote monitoring wireless health/telemedicine technology that can be utilized to treat patients with kidney disease and assessing potential clinical and economic value. This information will be used to address key regulatory, legal, legislative, and reimbursement barriers affecting the adoption and usage of such technologies. The workgroup developed a white paper and presented their findings at a workshop. Learn more about this project here.

Perspectives from the Kidney Health Initiative on Advancing Technologies to Facilitate Remote Monitoring of Patient Self-Care in RRT