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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 DuchenneConnect Network (now known as the Duchenne Registry), participated as Partner Networks in PCORnet.

PCORI funded the DuchenneConnect Network’s participation in PCORnet from 2014 to 2018. This report outlines the DuchenneConnect Network’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

The DuchenneConnect Network is a PPRN made up of patients with Duchenne and Becker muscular dystrophy, or DBMD. DBMD causes skeletal and heart muscles to weaken over time. The DuchenneConnect Network collects data through an online patient registry. Registries store data about people with a specific health problem. The DuchenneConnect Network began collecting data in 2007. Patients submit data through online surveys. The Network has more than 5,000 members and adds about 50 new members each month.

How does the network operate?

The DuchenneConnect Network is a program of Parent Project Muscular Dystrophy, or PPMD, a nonprofit focused on DBMD. PPMD is governed by a 20-member board of directors who oversee all PPMD programs. Most board members are parents of a child with DBMD.

The DuchenneConnect Network created a standard application for researchers who want to use registry data for studies. The PPRN also created standard methods for data management, which included standard ways to train team members, review registry data, and recruit patients for studies.

Geisinger Health System’s Institutional Review Board, or IRB, oversees registry data to ensure that patients’ rights and privacy are protected. Researchers may use their own institution’s IRB if needed.

How did the network involve patients and other partners?

The DuchenneConnect Network has a steering committee to direct all registry functions. This committee includes 12 clinicians, researchers, patients with DBMD, and parents of patients with DBMD.

The Network also created a family advisory group for the PCORnet project. This group includes four patients with DBMD and five parents of patients with DBMD. This group met monthly by phone and once a year in person. The group helped guide what research the Network should support, and how PCORnet could help researchers. The group also worked on outreach to underserved communities, ways to encourage more people to participate in research, improvements to research consent processes and surveys, and future directions of the program.

The DuchenneConnect Network also created a process for patients and families to help decide what research is most important, to create research protocols, and to plan requests for research funding. The Network also provided patients and family members with conferences, support groups, and webinars. Through these events, patients and families discussed and guided registry activities.

Who is in the network?

The DuchenneConnect Network had 5,123 patients in its registry as of March 29, 2019. Patients completed online surveys that collected data about their medical history; symptoms; physical function; and traits like age, sex, race, and ethnicity. The Network also collected results from genetic tests and offered free genetic testing to those in need through the Decode Duchenne genetic testing program.

While a Partner Network in PCORnet, DuchenneConnect participated in 39 studies.

How does the network support future research?

The DuchenneConnect Network is now the Duchenne Registry. The Duchenne Registry is able to sustain itself with financial support from PPMD and private companies and with fees from researchers who pay to use the registry for data access and recruitment.

The registry works with other networks that are part of PCORnet.

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 the DuchenneConnect Network (now known as the Duchenne Registry), participated as Partner Networks in PCORnet.

PCORI funded the DuchenneConnect Network’s participation in PCORnet from 2014 to 2018. This report outlines the DuchenneConnect Network’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

The DuchenneConnect Network is a PPRN made up of patients with Duchenne and Becker muscular dystrophy (DBMD). The DuchenneConnect Network is a self-report registry that began collecting data in 2007 as a community resource to facilitate and expedite research in DBMD. The Network has more than 5,000 members and adds approximately 50 new registrants per month.

Network Governance and Operations

The DuchenneConnect Network is a program of Parent Project Muscular Dystrophy (PPMD), the leading nonprofit organization focused solely on DBMD. PPMD is governed by a 20-member board of directors who oversee all PPMD programs. A majority of the board members are parents of a child with Duchenne.

In addition, DuchenneConnect Network established a steering committee to oversee all registry functions. This 12-member committee is composed of clinicians, researchers, patients with DBMD, and parents of patients with DBMD.

To streamline requests for data, the DuchenneConnect Network created a standardized application for requesting access to registry data and a data access agreement. It also established standard operating procedures to streamline data management activities, including training team members, curating registry data, and recruiting PPRN members.

All research requests involving registry recruitment of participants must be approved by an Institutional Review Board (IRB). The DuchenneConnect Network has a long-standing connection with Geisinger Health System’s IRB, but researchers may use their own institution’s IRB if needed.

Stakeholder Engagement

In addition to PPMD’s board of directors and the DuchenneConnect Network’s steering committee, DuchenneConnect also created a family advisory group specific to the PCORnet project. This family advisory group consisted of four patients with DBMD and five parents of patients with DBMD. This group met monthly by telephone and once a year in person to guide research prioritization and potential uses of the PCORnet infrastructure to benefit stakeholders. The group also guided engagement and outreach to underserved communities and participated in other aspects of the network, including retention improvement, consent and survey revision, and programmatic direction.

The DuchenneConnect Network created a process that incorporates patients and caregiver teams in identifying, developing, and supporting research priorities, protocols, and funding requests. The PPRN also hosted events, like conferences and events with local grassroots groups, and hosted educational webinars to create a two-way dialogue that enables patients and families to guide registry activities.

Demographics and Research Capacity

The DuchenneConnect Network had 5,123 registrants as of March 29, 2019. To collect information from patients about their medical history, symptoms, function, and demographics, the PPRN used a web-based research portal to administer surveys to Network participants. The DuchenneConnect Network also collected results from genetic tests and offered free genetic testing to those in need through the Decode Duchenne genetic testing program.

While a Partner Network in PCORnet, the DuchenneConnect Network participated in 11 randomized controlled trials, 11 descriptive studies, 8 observational prospective studies, 5 observational retrospective studies, and 4 qualitative studies.

Future Opportunities

The DuchenneConnect Network is now the Duchenne Registry. The registry is able to sustain itself financially with support from PPMD and from industry and with fees from researchers for data access and study recruitment.

The registry works with other networks that are part of PCORnet.