In total, 50 individuals participated, representing patient organizations (n = 21), industry (n = 12), research entities (n = 3), regulatory agencies (n = 1), and other healthcare organizations (n = 3). The resulting draft themes were circulated to 38 organizations, with six providing comment (two patient organizations, three biopharmaceutical organizations, one US government agency). Note that, in this context, patient refers to patient participants in our roundtable rather than patients at large, though typically patients and family caregivers are common among patient-group staff representatives.
Patient Perspectives on Real-World Evidence (RWE)
Our roundtable discussion identified ten themes with implications for policy makers, researchers, clinicians, and patient groups.
Raising Patient-Community Awareness
Most Patients are Unaware of RWE and its Actual or Potential Uses
Most patients have little understanding of RWE or that controversies exist regarding selection of types of evidence used in decision making about availability of treatment, payments based on value, or choices among various treatment options. Participants noted that, while educating individual patients (i.e., the public) may not be feasible, patient organizations can play an important role in disseminating important insights from RWE.
Enhancing Patient-Community Capacity
Common Definitions for RWD and RWE are Vital
Efforts to clearly define RWD, its sources, and how such data can be interpreted to yield useful RWE are needed. Patient advocates believe that better defining RWE is important to increasing their confidence in the data collection methods and applications.
Skills and Tools are Needed by Patient Organizations to Facilitate Uptake of RWE
Table 1 lists the skill sets and tools participants suggested for patient groups to enhance communication, understanding, and use of insights from RWE among constituents. This list can be viewed as a broad first step to public understanding of RWE. Similar to how patient groups translate findings from clinical trials to their patient members, patient groups can serve as arbiters of findings from RWE. To make the best use of and communicate about RWE to their constituents, patient organizations require education and tools related to (1) introduction to RWE and RWD sources; (2) evaluating and disseminating RWE to patients; (3) RWE for shared decision making; and (4) co-developing RWE. Suggested formats should be easy to understand, brief, and created with patient input to ensure accessibility and usability. The goal is not to make patients or patient advocacy organizations RWE “method experts”, but rather to support patient advocacy organizations in discerning between good-quality RWE and “junk science”.
Partnerships with the Scientific Community to Support High-Quality RWE
Participants noted the importance of partnerships between patient groups and the scientific community being developed to guide interpretation and understanding of RWE. For example, patient groups may need scientific advisory boards or to rely on author-provided summaries of RWE to help them interpret study findings.
Patient-Specific Uses of RWE
RWE Should Support Informed Decision Making
RWE is intended to discern how things work in real-world settings and in diverse patient populations to help guide decision making. Patient groups saw the possibility of using RWD to understand how a treatment works in diverse patient populations—to find someone that “looks like me”—as an assurance of how a treatment might benefit them personally. Studies should be designed in partnership with patients and with the intent to communicate results to patients in a balanced manner to inform decision making. Table 3 lists guiding questions that can improve trustworthiness and uptake of research findings.
Clinicians Must be Champions for Dissemination and Use
Education efforts on RWE must target the clinician community, as clinicians are a primary conduit of information to patients. For RWE to be impactful, participants emphasized the need for clinicians to understand the value of RWE to identify potential treatment options and support patient decision making.
Communications to Patients Should be Balanced and Empowering
Roundtable participants identified important principles to guide communication of RWE to patients. These recommendations are summarized in Table 2.
Acceptable Uses of RWE Must be Linked to the Context of its Use
RWE may serve different purposes for different diseases (e.g., inform surrogate outcomes for future trials for rare conditions). Patient representatives generally agreed that RWE should not be used for clinical evaluation of new, unapproved therapies. However, patients saw opportunity for RWE to inform new uses of approved therapies in, for example, new patient subgroups (e.g., children), for treatment of comorbid conditions, and to achieve patient-defined endpoints not part of completed clinical studies (Table 3).
Privacy Must be Protected and Data Ownership Clear to Promote Trust
Patients often lack clarity about who owns data about their health and often struggle to access their own medical records. Patient groups were concerned about efforts that seek to commoditize data about them, without a clear focus on improving their health outcomes and care. While the Health Insurance Portability and Accountability Act (HIPAA) may allow for data sharing, privacy issues remain a concern for patients, and standards of protection to ensure anonymity must be clarified.
RWE Should Include Authentic Sources of Patient Data
Electronic health records and claims data, two primary sources of RWD, are only “real” to an extent and may not reflect the diversity of disease experiences, preferences, or outcomes. Participants recommended RWE should aggregate these data with additional real-world sources, including patient-generated data (e.g., patient-organization registries).