The Vasculitis Patient-Powered Research Network

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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 Vasculitis Patient-Powered Research Network (VPPRN), participated as Partner Networks in PCORnet.

PCORI funded VPPRN’s participation in PCORnet from 2015 to 2019. This report outlines VPPRN’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 VPPRN is a PPRN made up of people affected by vasculitis, a family of almost 20 rare diseases. Vasculitis is inflammation of the blood vessels that occurs when the body’s immune system attacks blood vessels by mistake, damaging organs and tissues. Many types of vasculitis exist, and symptoms and severity vary.

Led by the University of Pennsylvania, the network is a partnership between the Vasculitis Clinical Research Consortium, which is a group of medical centers, patient organizations, and researchers, and the Vasculitis Foundation, a patient advocacy organization. As of March 2019, the network included 2,760 patient partners. These patient partners are people diagnosed with vasculitis, parents of children with vasculitis, and caregivers of adult patients. Recruiting patients with rare diseases for research is difficult, and the goal of the VPPRN was to collect patient-reported information for patient-centered research studies.

How does the network operate?

The VPPRN Steering Committee, made up of investigators, patients, and research staff, makes decisions about how the network operates. Three working groups also contribute. One working group plans research projects; another focuses on information supplied by patients for use in clinical studies; and a third encourages patient recruitment, involvement, and communications.

People with vasculitis provide information for use in research studies. When they join the network, they submit information about their health through a secure online portal. Patients gave permission to the network to share information. Information collected when patients join the network includes

• Basic information such as age, sex, job, and marital status
• Medical history
• Diagnosis
• Extent of disease
• Medications

The VPPRN also collects information from pregnant women with vasculitis. This information helps researchers understand how vasculitis affects the health of pregnant women and their babies.

Patients participating in the network also complete surveys that collect information for specific studies. For example, participants in a study on diagnosis completed surveys about how long it took them to be diagnosed with vasculitis and how the time from first symptoms to diagnosis affected them.

The VPPRN also collaborated with other PPRNs, research networks, universities, and the pharmaceutical industry on studies that used network data. The VPPRN scored research proposals based on the importance of the research to patients and its scientific value.

How did the network involve patients and other partners?

Patient partners participate in every level of VPPRN leadership. They work on strategic planning, and developed, reviewed, and approved research studies. The Steering Committee includes four patient partners, and one of the two main network researchers is a patient partner. The VPPRN also set up several working groups that include patient partners and representatives from advocacy organizations.

Patient partners received online orientation training about the research process and how the network operates.

While a Partner Network in PCORnet, VPPRN participated in 13 studies.

Who is in the network?

As of March 2019, the network includes 2,760 patient partners.

How does the network support future research?

The VPPRN has agreements with government health agencies and the pharmaceutical industry to help the network continue its work. It continues to support research by working with other PPRNs and other partners.

Glossary

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 Vasculitis Patient-Powered Research Network (VPPRN), participated as Partner Networks in PCORnet.

PCORI funded VPPRN’s participation in PCORnet from 2015 to 2019. This report outlines VPPRN’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 VPPRN is a PPRN made up of people affected by vasculitis, a family of almost 20 rare diseases that cause inflammation of blood vessels. These diseases can potentially affect any organ or system in the body. Led by the University of Pennsylvania, the network is a partnership between the Vasculitis Clinical Research Consortium, a group of academic medical centers, patient organizations, and clinical research resources, and the Vasculitis Foundation, a patient advocacy organization. As of March 2019, the network included 2,760 patient partners: people diagnosed with one of 12 types of vasculitis, parents of children with vasculitis, and caregivers of adult patients. Recruiting patients for research on rare diseases is challenging, and the goal of the VPPRN was to set up an infrastructure to collect patient-reported data to conduct patient-centered research.

Network Governance and Operations

VPPRN established a Steering Committee that consulted with the network’s two co-principal investigators to make decisions about network governance. The Steering Committee, made up of investigators, patients, and research staff, has responsibility for continuous oversight of working groups and the greater patient network and seeks to foster a culture that supports good faith dissent and empowers all perspectives to be openly shared. The network also established three working groups. One prioritizes and plans research projects; another focuses on patient-reported outcomes and data for use in clinical trials; and a third fosters patient recruitment, engagement, and communications.

The VPPRN’s primary data-gathering mechanism is the network’s online registry, which collects information from people with all forms of vasculitis through its HIPAA-compliant online portal. Baseline information collected when patient partners join the network includes

• Demographics
• Medical history
• Diagnosis
• Extent of disease
• Medications
• Patient global assessment

The VPPRN has also maintained a vasculitis pregnancy registry, which gathers information about how vasculitis affects reproductive health and pregnancy outcomes.

Patient partners registered with the network completed surveys that collected data for specific studies. For example, to conduct the Journey to Diagnosis study, the network surveyed patient partners about the time from first symptoms to diagnosis, the factors that affected how long it took to receive a diagnosis, and their perspectives on the consequences of diagnostic delay.

Researchers may submit proposals for studies to answer research questions with data collected from the VPPRN. The Steering Committee and the research working group evaluates these proposals based on the importance of the research to patients and its scientific merit, feasibility, and fundability. The network collaborates with other PPRNs, research networks, universities, and the pharmaceutical industry in such studies.

Stakeholder Engagement

Patient partners have participated at every level of VPPRN governance, including participating in strategic planning and developing, reviewing, and approving research studies. As of March 2019, the Steering Committee included four patient partners, and one of the two network co-principal investigators is a patient partner. Patient partners and representatives from advocacy organizations have served on working groups.

The VPPRN conducts online orientation training to help patient partners understand research and network operations. The network regularly surveys patient partners to gather feedback and assess the effectiveness of the network’s patient engagement efforts.

Demographics and Research Capacity

While a Partner Network in PCORnet, VPPRN participated in 13 studies, including 10 observational studies, one qualitative study, and two data analyses.

Future Opportunities

The VPPRN has agreements with federal agencies and industry to fund the network in the future. It continues to enhance the sustainability of the network by participating in multinetwork PCORnet research and building external collaborations.