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Introduction

There is limited reliable information regarding causes, progression, or treatment options for patients and family members coping with a variety of conditions. In some cases, limited information is a function of the rare nature of the condition and the scarcity of trustworthy data. In other cases, research on the condition has not focused on finding answers to the questions that patients most value due to challenges in collecting or analyzing such data, methodological constraints, or to limited researcher interest in such questions. Funding for such research across conditions may also be limited or virtually non-existent, which leads researchers to focus on other priorities.

In response, many patients and families have sought to contribute to the research process, hoping either to advance current options or to contribute to the options available for future patients. Many patients and their support and advocacy organizations seek increased attention or funding for focused research on conditions or treatments. Patients, family members, and consumer advocates also serve as advisors, informants, and partners with researchers on studies such as those funded by the Effective Health Care Program at the Agency for Healthcare Research and Quality (AHRQ) and the Patient-Centered Outcomes Research Institute (PCORI).

Patients are often encouraged by support and advocacy organizations to participate in research, particularly in clinical trials. Clinical trials, however, although an important source of new knowledge, may have a limited reach in garnering participants to yield a robust data set. Only about 6 percent of patients with a severe disease participate in clinical trials. Patients may or may not fit the inclusion criteria for a clinical trial, and results from these studies can take years before being translated into useable information for patients and family members. More importantly, the nature of the clinical trial may or may not yield information that patients and family members find most relevant or useful in making treatment decisions.

Patient registries are additional or supplemental data sources. Registries use existing or contributed clinical data to provide information on “real world” practice and the effectiveness of treatments and procedures, and are to be distinguished from randomized controlled trials or scientific experiments that cannot measure real-life experience, in some cases, to the same degree. Data included in patient registries may come from a variety of sources, including electronic medical records, clinician or patient reported clinical outcomes, diagnostic reports or images, hospital records, collected/donated blood or tissue samples, or questionnaires/surveys completed by clinicians or patients (or both). Traditionally, many of these registries are generated by researchers, funders, or institutions for scientific purposes. Such registries resolve many of the limitations posed by clinical trials, and offer new ways to answer salient patient questions. However, when managed by researchers, the registry may provide little or no opportunity for involvement or control by patient or family members or patient support and advocacy organizations. As a result, the registries may not meet the needs of patients and their caregivers.

To focus research more directly on patient and family member needs, patient and family advocates and organizations have created and operated “patient-powered” patient registries and research networks since as early as 1995. These registries and networks are distinguished from researcher-generated registries in that the registry (or network) and the research it yields is managed by patients and family members themselves, often through a disease advocacy organization or a network of organizations that receives advice and input from a scientific board of advisors. Patient-powered registries (PPRs) and patient-powered research networks (PPRNs) offer new directions for patient-centered outcomes research, and contribute to translational science in important ways. Experts agree that these registries are transforming patient/caregiver support and advocacy groups into research organizations. They also provide patients and family members another way to become engaged in research beyond the role of advisor or informant to researcher-generated studies.

This paper describes PPRs and PPRNs and outlines the considerations for patient advocacy and support organizations wishing to create or participate in these entities. The first section of the paper offers a definition and shared characteristics of both researcher-generated and patient-generated patient registries and research networks. The second section outlines the current pathways that exist for the creation of or involvement with a PPR and/or PPRN, and the advantages and disadvantages of each path. The final section reviews emerging issues in the rapid evolution of patient-powered registries and research networks.