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PCORI funded the development of PCORnet^®, the National Patient-Centered Clinical Research Network, to make it easier and more efficient to conduct research. PCORnet is made up of Partner Networks that harness the power of large amounts of health data and patient partnerships.

Patient-Powered Research Networks (PPRNs) are networks operated and governed by patients, families, caregivers, and community members in close collaboration with clinical researchers, with the goal of shaping and conducting research to better serve their communities and improve their health outcomes. Several PPRNs, including the NephCure Kidney Network (NKN), participated as Partner Networks in PCORnet.

PCORI funded NKN’s participation in PCORnet from 2015 to 2019. This report outlines the NKN’s achievements in building its research infrastructure capacity to

1. Create ways to involve patients, families, and caregivers in decision making about the network
2. Create a way to collect and share data reported by patients for research
3. Involve patients, caregivers, families, and communities in the research process, including recruiting people to participate in research studies
4. Lead or join in research studies that focus on the community’s priorities

Network at a glance

Primary nephrotic syndrome (NS) is a group of rare but serious kidney diseases that often lead to kidney failure or other major medical problems. The NKN PPRN aimed to make it easier to conduct research about NS. The NKN made research findings about NS available to patients to help patients make more informed treatment and health decisions. The network included a registry of more than 1,300 patients with NS. Arbor Research Collaborative for Health led the NKN along with partners at NephCure Kidney International, Children’s Hospital of Philadelphia, and the University of Michigan.

How does the network operate?

The NKN created a steering committee to develop and approve all policies related to NKN activity, including guidelines for how the network would operate and how it would oversee and evaluate research studies. The steering committee also developed NKN’s policies on how to involve patients, share network activities with its members, conduct research, and share data. In addition, the steering committee

• Managed the development of the NKN mobile app
• Managed updates to the NKN patient and researcher databases
• Reviewed proposed research studies, conference presentations, and publication ideas

The NKN also created subgroups and task forces to work with the steering committee on specific projects, including

• Involving patients, community members, and clinicians in the network
• Updating the NKN mobile app, patient surveys, and the NKN website
• Recruiting patients for the database and keeping them involved
• Creating and sharing research reports with the NKN community

How did the network involve patients and other partners?

Patients could participate in the NKN in three ways: by becoming part of the steering committee, registering in the database, or being part of the broader NS community. Patient and caregiver members made up most of the steering committee. These members also worked in advisory roles in research projects and on work groups and task forces. Patients on the steering committee held network leadership positions and helped decide which studies the network should get involved with. The committee developed a training program to help train new committee members and to help experienced members get more involved in the network. Patients could access information about what was going on in the network through a biweekly newsletter containing project updates. The NKN also had meetings with its patient committee members via conference calls and during a large in-person kickoff meeting.

The NKN invited patients from the broader NS community to be part of the subgroups and task forces. Patients who had shared their information in the database could take part in monthly surveys that included questions about the research topics most important to them. Patient advocates, physicians, and researchers were also part of the steering committee.

Who is in the network?

The NKN had 1,310 enrolled patients as of July 3, 2019. Patients shared their information to the database through online surveys.

While a Partner Network in PCORnet, the NKN participated in 10 studies. The NKN also formed close collaborations with other PPRNs.

How does the network support future research?

PPRNs follow PCORI standards to enhance the sustainability of the network, including fostering patient and clinician engagement at all stages of the research process and building external collaborations.

Glossary

Institutional Review Board (IRB): A group that follows federal regulations, state laws, and institutional policy to review, monitor, and approve research in order to protect the ethical rights and privacy of the subjects involved.

Patient-Powered Research Networks (PPRN): PPRNs are operated and governed by patient groups and their partners and are focused on particular conditions or populations.

PCORnet: PCORnet is a network of networks that brings together patients, clinicians, researchers, and healthcare systems to share information and participate in research.

Overview

PCORI funded the development of PCORnet^®, the National Patient-Centered Clinical Research Network, to make it easier and more efficient to conduct research. PCORnet is made up of Partner Networks that harness the power of large amounts of health data and patient partnerships.

Patient-Powered Research Networks (PPRNs) are networks operated and governed by patients, families, caregivers, and community members in close collaboration with clinical researchers, with the goal of shaping and conducting research to better serve their communities and improve their health outcomes. Several PPRNs, including NephCure Kidney Network (NKN), participated as Partner Networks in PCORnet.

PCORI funded NKN’s participation in PCORnet from 2015 to 2019. This report outlines the NKN’s achievements toward the PPRN goals of developing and optimizing research and engagement infrastructure capacity to

• Establish a governance structure that fully engages patients and other stakeholders for the purposes of contributing to research
• Create a data infrastructure conducive to the ongoing collection of patient-reported data
• Meaningfully engage patients, caregivers, families, and community members in the research process, including identifying and recruiting participants for clinical trials and other research studies
• Lead or partner in the conduct of comparative effectiveness studies responsive to the research priorities of their communities

Network Infrastructure

Primary nephrotic syndrome (NS) is a collection of rare but serious kidney diseases that often lead to kidney failure or other major medical complications. The NKN PPRN aimed to facilitate timely, accurate, and efficient clinical research related to NS that provides evidence to help patients make more informed decisions. As of July 3, 2019, the network included a registry of more than 1,300 patients with NS. Arbor Research Collaborative for Health led the NKN along with partners NephCure Kidney International, Children’s Hospital of Philadelphia, and the University of Michigan.

Network Governance and Operations

A steering committee was responsible for the development and approval of all policies related to NKN activity, including establishing requirements for network operations, ongoing monitoring, and evaluation. The steering committee developed the NKN’s engagement, transparency, research, and data sharing policies. Likewise, the steering committee guided development of the NKN mobile application and updates to the NKN patient and researcher registry portals. The committee also reviewed proposed research studies, posters, and manuscript ideas, and had members who served on study committees.

The NKN created work groups dedicated to patient and community engagement and task forces focused on the NKN mobile app, survey toolkit, website enhancement, clinician engagement, recruitment and retention, and research deliverables. Both the work groups and task forces worked in partnership with the steering committee. In addition, the NKN developed various products for dissemination within the larger NS community, including an annual data report.

Stakeholder Engagement

Patients and caregivers made up a majority of the steering committee. These members also served in advisory roles in research projects, as well as on work groups and task forces. Patients on the steering committee held network leadership positions, took part in decision making about network participation in proposed studies, and partnered in the research process, including defining study questions, implementing study outcomes, and disseminating findings. The steering committee also developed a training module for new and current members to onboard new committee members and deepen all members’ engagement with the governance of the NKN. The network published a biweekly newsletter to share operational and project updates with patients. The NKN met with its patient committee members via teleconferences and a large in-person kickoff meeting. The steering committee also included patient advocates, physicians, pharmaceutical industry members, and researchers.

The NKN invited patients from the broader NS community to serve on the work groups and task forces. The NKN engaged with patient registrants by conducting monthly surveys within the network to learn more about the community’s research priorities. The NKN also developed a medical diary mobile app designed specifically for patients with NS to track appointments, medications, and laboratory data. The app is available for Apple and android devices.

Demographics and Research Capacity

The NKN had 1,310 enrolled patients as of July 3, 2019. The registry included predominantly patient-reported data from web-based patient surveys.

While a Partner Network in PCORnet, the NKN participated in 10 studies, including one engagement project, seven infrastructure awards, one randomized controlled trial, and one measure validation.

Future Opportunities

PPRNs follow PCORI standards to enhance the sustainability of the network, including fostering patient and clinician engagement at all stages of the research process and building external collaborations.