Primary Immunodeficiency Connect (PI CONNECT)

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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 Primary Immunodeficiency Connect (PI CONNECT), participated as Partner Networks in PCORnet.

PCORI funded PI CONNECT’s participation in PCORnet from 2015 to 2019. This report outlines PI CONNECT’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 Immune Deficiency Foundation (IDF) led the network. The IDF gathered information from patients with primary immunodeficiency (PI) using an online portal and electronic health records. PI diseases are a group of more than 350 rare genetic disorders that affect the immune system. Patient data collected by the network became part of the United States Immunodeficiency Network (USIDNET) patient registry. As of March 31, 2019, the network included 2,447 people with a diagnosed PI disease who had contributed data.

How does the network operate?

A governance committee made up of patients, healthcare provides, and key PI CONNECT staff oversaw network activities. The governance committee elected two co-chairs, one of whom was a patient. The committee reviewed USIDNET data requests that involved PI CONNECT patient data. It also approved the network’s participation in other PCORnet projects. The governance committee had four subcommittees. They focused on engagement, research, communications, and protecting people and data during research. The two head researchers of PI CONNECT provided overall leadership to the governance committee.

To collect data from patients, the IDF used an electronic personal health record platform. This online portal gave patients greater flexibility and control of their online health records. The platform provided data to USIDNET that could not be traced to individual patients. The platform also made it easier for researchers to use the data.

How did the network involve patients and other partners?

Patients with PI or their caregivers made up at least half of the governance committee. All subcommittees had at least one patient member. PI CONNECT had two full-time positions dedicated to working with people with PI and their families. They helped patients and families participate in research, including using the online data platform.

The network also created the PI CONNECT Research Forum, a secure, online place where patients and clinicians like doctors and nurses could discuss research. The site also provided links to articles that used patient data and posted open clinical trials. PI CONNECT played an active role in the IDF’s annual patient meetings. At the meetings, PI CONNECT helped people with PI to better understand the research process.

The network translated all PI CONNECT materials including the online data platform into Spanish. The goal was to increase participation in research among Spanish-speaking patients and their families.

Who is in the network?

PI CONNECT collected data from 2,447 enrolled participants as of March 31, 2019. Data came from two sources: patient medical records and the online data platform. All PI CONNECT data aligned with the Common Data Model.

While a Partner Network in PCORnet, PI CONNECT participated in three studies.

How does the network support future research?

The IDF continues to work with patients, researchers, and other organizations to make patient needs and concerns a meaningful part of the research process.

Glossary

Common Data Model (CDM): A CDM establishes a standard way of defining and formatting data.

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 Primary Immunodeficiency Connect (PI CONNECT), participated as Partner Networks in PCORnet.

PCORI funded PI CONNECT’s participation in PCORnet from 2015 to 2019. This report outlines PI CONNECT’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

Led by the Immune Deficiency Foundation (IDF), the network collected and combined clinical and patient-reported outcome data from patients with primary immunodeficiency (PI) diseases. PI diseases are a group of more than 350 rare genetic disorders that affect the immune system. Patient data collected by the network became part of the United States Immunodeficiency Network (USIDNET) patient registry. As of March 31, 2019, the network included data from 2,447 participants with a diagnosed PI disease.

Network Governance and Operations

A governance committee composed of patients, clinicians, and key PI CONNECT staff oversaw network activities. The governance committee elected two co-chairs, one of whom was a patient. The committee reviewed USIDNET data requests that involved PI CONNECT patient data. It also approved the network’s participation in other PCORnet networks. The governance committee had four subcommittees. They focused on engagement, research, communications, and human subjects and data privacy. The PI CONNECT co-principal investigators provided overall leadership to the governance committee.

To collect data from patients, the IDF used an electronic personal health record (ePHR) platform, which gave patients greater flexibility and control of their online records. Using the ePHR platform to provide de-identified data led to improved ways to connect data points and retrieve data within the USIDNET patient registry.

Stakeholder Engagement

Patients with PI or their caregivers made up at least half of the governance committee. All subcommittees had at least one patient member. PI CONNECT had two full-time positions dedicated to connecting people with PI and their families to research processes, including the ePHR.

The network also established the PI CONNECT Research Forum, a secure online environment where patients and clinicians could discuss research. The site also provided links to articles that used patient data and posted information on open clinical trials. PI CONNECT played an active role in the IDF’s annual patient meetings during which they helped people with PI to better understand the research process.

The network translated PI CONNECT materials, consent forms, and the content of the ePHR into Spanish to increase participation among Spanish-speaking patients and their families.

Demographics and Research Capacity

PI CONNECT collected data from 2,447 enrolled participants as of March 31, 2019. Network staff abstracted clinical data from patient medical records to enter into USIDNET. The ePHR collected patient-reported outcomes such as physical function, fatigue, and mental health. All PI CONNECT data aligned with the Common Data Model.

While a Partner Network in PCORnet, PI CONNECT participated in one clinical trial; one observational study; and PCORnet’s Healthy Mind, Healthy You, a mindfulness study involving several PCORnet entities.

Future Opportunities

The IDF continues to work with patients, researchers, and other organizations to make patient needs and concerns a meaningful part of the research process.