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Workman TA. Engaging Patients in Information Sharing and Data Collection: The Role of Patient-Powered Registries and Research Networks [Internet]. Rockville (MD): Agency for Healthcare Research and Quality (US); 2013 Sep.

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Engaging Patients in Information Sharing and Data Collection: The Role of Patient-Powered Registries and Research Networks [Internet].

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Defining Patient Registries and Research Networks

Patient Registries

Patient registries have been defined as “an organized system that uses observational study methods to collect uniform data (clinical and other) to evaluate specified outcomes for a population defined by a particular disease, condition, or exposure, and that serves a predetermined scientific, clinical, or policy purpose(s).”6 In brief, a patient registry is a collection—for one or more purposes—of standardized information about a group of patients who share a condition or experience. The use of “patient” in patient registries is often used to distinguish the focus of the data set on health information. Currently, there is no consistent definition of the term “patient registry” used in the health research field. Terms such as clinical registries, clinical data registries, disease registries, and outcomes registries are also used to describe the same data collection method.7 Examples of individual researcher-generated registries can be found at the AHRQ Registry of Patient Registries, available at https://patientregistry.ahrq.gov.

Traditional Patient Registries

Patient registries have traditionally been researcher-generated. Research institutions, academic clinical institutions, or individual research teams establish a registry, using private or Federal funds, for the purpose of observational data collection that can be used for a specific research agenda. These registries may be organized and operated in a variety of forms and formats. They may be operated by a single institution or by a collaborative of multiple institutions or clinics. Researcher-generated patient registries currently exist for a wide range of chronic or rare conditions, including many forms of cancer, diabetes, cystic fibrosis, acute coronary syndrome, and arthritis.

The purposes for patient registries can range widely. According to the National Institutes of Health, “Registries can be used to recruit patients for clinical trials to learn about a particular disease or condition; to develop therapeutics or to learn about population behavior patterns and their association with disease development; developing research hypotheses; or for improving and monitoring the quality of health care.”8 Patient registries can also be used to monitor outcomes and study best practices in care or treatment.9 They may pursue a specific, focused research agenda, collecting data for a limited time to answer a specific research question (or questions), or may collect data on an indefinite basis to answer a variety of existing and emerging research questions. Patient registries may also include the collection of tissue or blood samples collected in a variety of ways.

The creation and use of researcher-generated patient registries has grown steadily for several decades, although the actual number of existing registries in the United States is unknown. In 2012, the Agency for Healthcare Research and Quality launched an online registry of patient registries to provide a searchable database of patient registries in the United States.10

Patient-Powered Patient Registries (PPRs)

PPRs are similar in many ways to researcher-generated patient registries in definition, purpose, and features. At times, these registries are somewhat indistinguishable from traditional registries, with one exception: In patient-powered patient registries, patients and family members “power” the registry by managing or controlling the collection of the data, the research agenda for the data, and/or the translation and dissemination of the research from the data.

Experts in the field, however, differ in their individual conceptualizations of what constitutes a valid PPR. In the view of some, only registries that are created, maintained, and controlled by patients or patient advocacy organizations can be considered “patient-powered,” while others focus on the specific contributions of patients—that is, their involvement and contribution to all aspects of the registry—as the critical factor, regardless of the registry’s ownership or involvement by commercial or professional interests. These differences in conceptualization have added to the challenges of classifying PPRs for the broader research field. Terms such as “patient-generated,” “patient-run,” “patient-powered,” and “participant-controlled” can be found among various users.

Patient-powered patient registries are also organized and operated in a variety of forms and formats. They may be operated by a single organization or by a collaborative of multiple organizations. Like researcher-generated registries, patient-powered patient registries exist for a wide range of conditions.

PPRs may also pursue a specific research question or conduct ongoing data collection to answer a variety of existing and emerging research questions. Several PPRs have biobanks, or repositories, where patients can provide samples of blood or tissue to be used in research. Other patient advocacy organizations, such as the TMJ Association, use their registry as a recruitment vehicle for existing clinical trials, inviting members of the TMJ community whose profile matches a trial protocol.

Although the genesis of PPRs has not been studied or documented, most seem to originate from a patient support or advocacy organization, either as the direct intention of the organization or as an added component. The goal of these registries is to enhance translational research by providing data that could better characterize the disease, discover biomarkers, or provide information to assist patient and family decisionmaking.11 In several cases, the creation of a PPR followed the formation of a support and information-sharing network of patients or families who shared a set of experiences with a disease or condition. The earliest documented PPR is the Hereditary Disease Foundation, created in 1983. While the support and advocacy network met a variety of patient and family needs, the foundation’s co-founder, Nancy Wexler, also collected samples from people who were affected by Huntington Disease for the purpose of advancing research.4 An example of the typical genesis of a PPR can be found in the story of Pat Furlong, a registered nurse and the mother of two sons, Patrick and Christopher, who were both diagnosed with Duchenne muscular dystrophy when little was known about the condition. Refusing to accept that answers could not be found, she reached out to other families of the rare disease, founded the Parent Project Muscular Dystrophy, and eventually created a patient and family registry, DuchenneConnect (www.duchenneconnect.org), which is both an information sharing network and registry.12 An additional examples of patient-powered individual condition registries is the Life Raft Group Patient Registry and Tissue Bank (http://liferaftgroup.org/patient-registry), which collects data from patients affected by Gastrointestinal Stromal Tumor (GIST), a rare family of cancers.

Like researcher-generated patient registries, there is no single complete listing or documented number of PPRs in the United States. An effort to document patient-powered patient registries is being undertaken by the American Association for the Advancement of Science through funding by the Agency for Healthcare Research and Quality.13 One study of 201 disease advocacy organizations found that forty-five percent had supported a research registry or biobank.11

Concerns About Patient Registries

Patient registries have been promoted and praised in both chronic14,15 and rare16,17 disease practice communities, and debated among comparative effectiveness researchers as to whether they provide valid data to compare treatments.2,1820 Patient-generated patient registries in particular have been criticized on several levels, including the concern that only a small minority of patients with sufficient education and ability are able to participate, and that data may be biased for a variety of reasons.21 Experts have noted the lack of standardization in data collection and potential competition for registered patients across registries, which could create a fractured set of patient data. For example, if multiple patient registries exist for a single condition, there is a greater likelihood that competition for patients may limit any given data set. This is a particular concern for rare diseases, where an affected population may be very small, resulting in small data sets for each registry that are less able to draw valid conclusions for the population. Meta-analysis across registries may be challenging or impossible should each competing registry collect different data from the same patients, or collect the data under different timeframes.16 More specific concerns have been expressed regarding issues of patient consent and rights violations relative to tissue samples submitted to biobanks, in particular the concern about whether patients are fully aware of the possible subsequent uses of their specimens.22 Despite these concerns, patient registries have yielded a significant amount of research that meets the needs of patients and families. PPRs have also added to disease and treatment knowledge. In addition to self-published monographs, several PPRs have used their data or had their data used in published research, although no formal list of publications across PPRs exists.4,11,23

Research Networks

Some patient registries are part of broader research collaboratives that connect individual registries into a larger network of registries that collect data on one or more conditions. The network provides a shared infrastructure and standardized data collection across registries. Collected data from each registry may be combined for analysis, although participation in a research network does not eliminate the ability of any individual patient registry from analyzing only the data from the registry alone. Research networks may also have existing and emerging research agendas that are realized through an established collection of researchers who study the collective or registry-specific data based on the wishes of the patient network or each registry. In some cases, the network may make data available to researchers who request use for purposes that are unrelated to the agenda of those operating the registries.

Both researcher-generated and patient-powered research networks exist. An example of a researcher-generated research network is the SEER registries (seer.cancer.gov/registries), a collaboration of 19 registries created and managed by the National Cancer Institute. An example of a patient-generated research network is the Genetic Alliance Registry and BioBank (www.biobank.org), a collaboration of more than 1,200 individual disease advocacy organizations.

There is little published literature to date on PPRNs, although several large networks have emerged in the past five years. A report created for the Patient-Centered Outcomes Research Institute (PCORI), developed a taxonomy of research networks in an effort to create an inventory of existing networks. The taxonomy classified three distinct categories: Clinical Data Research Networks (CDRN), Patient-Powered Research Networks (PPRN), and Patient Registries based on a set of distinguishing characteristics.24 Although the taxonomy and characteristics vary somewhat from other descriptions and opinions, the taxonomy serves as a critical starting point for future research on the use of patient-powered networks and patient registries as data collection tools.

The Genetic Alliance Registry and BioBank (GARB) serves as an example of the evolution that can occur from an individual patient-powered disease registry to a PPRN. In 1995, PXE International, identifying itself as a “research advocacy organization,” created a patient-powered registry and biobank to accelerate translational research in pseudoxanthoma elasticum, a rare genetic metabolic disorder. The organization, led by founder Sharon Terry, became a mentor to a number of other organizations that also wished to create PPRs. This soon led to the formation of GARB in 2003 with eight disease advocacy organizations, using their infrastructure, model, and methods to create the broader network.4 In a recent expansion of GARB, the registry platform has become disease-agnostic and now includes more than 2,000 diseases.

Other networks have had similar beginnings25. In 1998, Stephen Haywood was diagnosed with ALS (Lou Gehrig’s Disease). The Heywood family began searching across the globe for information that might extend or improve Stephen’s life. In 2004, frustrated by the lack of open and accessible information, Stephen’s two brothers, Benjamin and James Heywood, and a lifelong friend Jeff Cole, all MIT graduates, founded PatientsLikeMe with 12 disease communities.26 In 2011, PatientsLikeMe opened to all conditions and now includes approximately 1,200 individual disease registries. PatientsLikeMe is a for-profit corporation.25 A new not-for-profit network entitled Registries For All (www.reg4all.org) provides a single platform available to all patient-powered disease registries. The network was formed through a partnership of organizations including GARB, CFIDS Association of America, National Psoriasis Foundation, and the Inflammatory Breast Cancer Research Foundation. The team was awarded $300,000 in early 2013 from the Partners for Patient Health Innovation Challenge, funded by Sanofi US, an international pharmaceutical company.

It is important to note that PPRNs differ in their structure and operation. Some PPRNs such as GARB enable individual organizations and registries who are part of the network to maintain their autonomy and identity. Others, such as PatientsLikeMe, organize the network by disease communities that may be supported by multiple disease-advocacy organizations.

Elements of Successful Patient-Powered Registries and Research Networks

Experts in PPRs and research network development and management share the belief that four fundamental elements are common to the development and management of a successful registry or network.

  1. Well-designed technology. Critical to the success of a patient registry is the digital technology used to enable patients to join the network, report and store (and display) information, search for patients with similar experiences or conditions, and/or link to other resources. The design of a successful virtual platform requires technical expertise, patient-user involvement, and significant funding. Models of effective technical solutions and platforms for registries and networks vary widely, and have not yet been formally evaluated or compared. Patient/caregiver use of the platform, however, serves as a critical guidepost for the success or failure of the registry or network.
  2. Recruitment, encouragement, and gratitude for participation. Without exception, registry and network founders and managers point to the need for ongoing promotion of the registry to grow the membership and activate members to report data on a regular basis. Larger or more active patient support or advocacy organizations may be more likely to find success than organizations with small or limited constituencies, but all registries must regularly promote and encourage ongoing participation to maintain a robust data set. Most registries or networks send regular reminder emails encouraging members to report their latest symptoms, lab results, or changes in treatment, and/or to thank them for their participation. Others send specific requests for information relative to a study or a question raised either by the network or by a researcher who is using the network for data collection. Those managing the daily operations of registries and networks note that promotion takes a significant amount of their time and attention, as it is critical to the success or failure of each registry. They note that simply having a network is not sufficient; it must have a robust membership of active participants who are contributing to and using the information.
  3. Collaborative relationships with researchers. To be effective as data sources for researchers—and ultimately meet the information or knowledge needs of patients and caregivers—registries and networks must work diligently to ensure that the data collected can be used for research. Some registries use scientific advisors or advisory teams to oversee the standardization of data or tissue collection. An example is the International Pemphigus and Pemphigoid Foundation (www.pemphigus.org) which has a large Medical Advisory Board of academic dermatologists. A second purpose for these advisors is their connection to similar researchers with interests in the condition or treatment who may serve as investigators on studies or connect the data set to other research products such as clinical trials, reports, or peer-reviewed articles. The establishment and maintenance of relationships with the scientific community often leads to affiliations with professional or condition-specific organizations, which may serve to advance connections with key scientists, particularly those interested and willing to work within the goals and interests of the registry in designing and conducting research.
  4. Partnerships with a broad range of stakeholders. Many of the registries and networks make it their goal to establish collaborative relationships with similar organizations for the purpose of sharing resources, avoiding competition for members, and reducing the fracturing of efforts to collect data, raise funds, or advance knowledge. For example, the DuschenneConnect lists more than 50 domestic and international partner organizations that promote the registry and contribute to its content. The Genetic Alliance is a network of more than 10,000 health organizations, of which 200 are disease-specific advocacy organizations that utilize its materials and help connect patients to the registries. Several experts suggest that the “network” of a PPRN must be significantly more than its patient or caregiver members. Attention to maintaining ongoing partnerships with a broad range of stakeholders is needed to be successful at all aspects of information-sharing and research.


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