Results Summary
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 CENA, participated as Partner Networks in PCORnet.
PCORI funded CENA participation in PCORnet from 2015 to 2019. This report outlines CENA achievements in building its research infrastructure capacity to
- Create ways to involve patients, families, and caregivers in decision making about the network
- Create a way to collect and share data reported by patients for research
- Involve patients, caregivers, families, and communities in the research process, including recruiting people to participate in research studies
- Lead or join in research studies that focus on the community’s priorities
Network at a glance
CENA is a PPRN made up of patients from disease-specific advocacy groups. Each group represents patients with a specific health condition. CENA supported the groups by working with them in a network. More than 10,000 patients and caregivers from 16 groups were involved in CENA. The goal of CENA was to help the groups conduct studies that answer research questions of interest to people with those health conditions.
| Title | Community Engaged Network for All (CENA) |
| Network design | Patient-Powered Research Network (PPRN) |
| Lead partner | Genetic Alliance, Inc. |
| Other partners | 16 disease advocacy organizations Private Access University of California San Francisco (UCSF) University of California Davis (UCD) |
| Participants | 10,153 |
| Area(s) of focus | Patients with Alström syndrome Asthma and allergies Celiac disease Dyskeratosis congenita Fibrolamellar hepatocellular carcinoma |
How does the network operate?
People with lived experience of disease lead CENA. CENA created a board to govern the network. Each advocacy group named one patient to this board. These patients met throughout the project and discussed what information was important for the network to collect. The patients also gave input on topics like data collection and who can access data, as well as how CENA operates.
All of the advocacy groups used an online system, called Platform for Engaging Everyone Responsibly, or PEER, to build registries. These registries stored information patients chose to share about their health and the care they received. The PEER database collected data in PCORI’s standard format. It also allowed patients to control who could see and use their health data.
About half of the advocacy groups also collected and stored biological samples like blood or saliva from patients who agreed to share them. The groups collected these samples at their conferences or social events.
CENA used a web-based platform to communicate with patients, caregivers, clinicians, and researchers participating within the advocacy groups. Using the platform, the participants and researchers designed and conducted research studies and shared the results.
Researchers who wanted to use CENA data for studies requested permission through a standard process. CENA worked with Genetic Alliance’s Institutional Review Board, or IRB, to review all studies and ensure that they protected the rights and privacy of people who participated. Using one IRB to do all reviews helped make sure they were quick and low cost.
How did the network involve patients and other partners?
Through CENA, the advocacy groups connected patients and community members to research teams to serve as advisors on projects. Patients also gave input to guide research priorities. This input included talking about how important potential research topics are, how practical it is to do some studies, and how studies might affect a community. CENA helped guide researchers in how to pay patients who advise study teams, clearly define what patients will do on the teams, and make sure patients stay informed about study progress.
More than half of the advocacy groups involved patients in designing research projects. The groups connected their patients to research teams to design study methods, write information about studies, and create surveys and other ways of collecting information from patients.
Who is in the network?
As of March 31, 2019, CENA had 10,153 patients from the advocacy groups who gave consent for CENA to use their data.
| Population (as of 03/31/19) |
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Race/ethnicity
Age
Sex assignment at birth
Gender identity
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While a Partner Network in PCORnet, CENA participated in 22 studies.
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, participating in multinetwork PCORnet research, and building external collaborations.
Glossary
Patient Registry: Patient registries store data about people with a specific health problem.
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.
Professional Abstract
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 Community Engaged Network for All (CENA), participated as Partner Networks in PCORnet.
PCORI funded CENA’s participation in PCORnet from 2015 to 2019. This report outlines CENA’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
CENA is a PPRN made up of patients from disease-specific advocacy organizations (DAOs), such as patients with Lynch syndrome, celiac disease, and other health conditions. CENA was designed as a network of networks. Each DAO built its own network while supporting others in CENA’s collaborative community. More than 10,000 participants, including patients and caregivers, represented 16 different DAOs in CENA. The goal of the network was to increase the capacity of disease-specific communities to advance people-driven research on the conditions of interest.
| Title | Community Engaged Network for All (CENA) |
| Network design | Patient-Powered Research Network (PPRN) |
| Lead partner | Genetic Alliance, Inc. |
| Other partners | 16 disease advocacy organizations (DAOs) Private Access University of California San Francisco (UCSF) University of California Davis (UCD) |
| Participants | 10,153 |
| Area(s) of focus | Patients with Alström syndrome Asthma and allergies Celiac disease Dyskeratosis congenita Fibrolamellar hepatocellular carcinoma Gaucher disease Hepatitis Inflammatory breast cancer Joubert syndrome Lyme disease Lynch syndrome Metachromatic leukodystrophy Pancreatic cancer Pseudoxanthoma elasticum Psoriasis X and Y chromosome variations |
Network Governance and Operations
Individuals with lived experience of disease founded and lead CENA. CENA established a governance board to provide operational, privacy, data protection, outreach, and strategic guidance. Each of the DAOs named one patient representative to this board. These patients met throughout the project and discussed what patient-reported outcomes (PROs) were important across the network. They also identified data, access, and operational questions.
The DAOs used an online database called the Platform for Engaging Everyone Responsibly (PEER) to build patient registries. DAOs used this system to store PROs, phenotypic and longitudinal data, and clinical data. PEER enabled patients to control who can see their health information, when, and for what purpose. CENA mapped the PEER database, where applicable, to PCORI’s Common Data Model (CDM). About half of the DAOs also collected specimens from patients to store in a partner biobank or their own biorepository. The DAOs collected specimens at their organizational conferences and social events.
CENA used a web-based platform called MOSAIC as a centralized communication tool. DAOs used MOSAIC to help patients, caregivers, clinicians, and researchers work together in developing, designing, implementing, and disseminating research studies. With support from CENA, each of the organizations addressed research questions that emerged from their communities.
To streamline the process for approving research and use of registries, CENA used Genetic Alliance’s Institutional Review Board (IRB). Centralizing IRB reviews ensured that reviews occurred in a timely manner, with low costs and minimal restrictions. When requesting use of registry data for studies, organizations completed a template-based application for expedited IRB review. The IRB completed most approvals within two working days.
Stakeholder Engagement
Through CENA, DAOs connected participants to research teams to serve as advisors on projects and recruited and convened participant advisory boards. DAOs provided training, support, and mentoring to help members of communities affected by their disease of focus to participate in research as full partners. To support successful partnerships among patients and researchers, CENA provided guidance on adequate compensation for participant involvement, a template agreement outlining roles and responsibilities for participants partnering on research studies, and ensured participants received an orientation to each project and were regularly included in team meetings.
All DAOs worked with both patients and the clinicians who treat them to establish formal research priorities. They also facilitated discussions among participants about the importance of identified topics, the feasibility of projects, and their potential effect on, or uptake by, communities.
More than half of the DAOs reported having patients involved in the design phase of research projects. The DAOs connected their participant community to research teams to develop methods, design messaging that was participant-centered and community appropriate, and design surveys and other data collection instruments.
Demographics and Research Capacity
As of March 31, 2019, CENA had 10,153 participants from the DAOs who gave consent for their data to be used in CENA activities.