Continuous Patient Engagement in Comparative Effectiveness Research

Mullins, C. Daniel; Abdulhalim, Abdulla M.; Lavallee, Danielle C.

doi:10.1001/jama.2012.442

Cited by 168 publications

(86 citation statements)

References 55 publications

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“…Although other researchers have reported similar expectations based on their experiences with research engagement in other settings (not specific to rare diseases), definitive evidence to support this is not yet available. 9,53 Very few studies reported training researchers, patients, or other stakeholders for engagement. Other literature supports training as increasing the impact of engagement; while some training opportunities are available for patients 43 and researchers, 54 these are not specific to research on rare diseases.…”

Section: Discussionmentioning

confidence: 99%

A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

et al. 2014

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BACKGROUND: Patients with rare diseases have limited access to useful information to guide treatment decisions. Engagement of patients and other stakeholders in clinical research may help to ensure that research efforts in rare diseases address relevant clinical questions and patient-centered health outcomes. Rare disease organizations may provide an effective means to facilitate patient engagement in research. However, the effectiveness of patient-engagement approaches, particularly for the study of rare diseases, has not been well studied. OBJECTIVES: To synthesize evidence about engagement of patients and other stakeholders in research on rare diseases, including the role of rare disease organizations in facilitating patient-centered research. METHODS/RESEARCH DESIGN: A systematic review and gray literature search were guided by a technical expert panel composed of patient representatives, clinicians, and researchers. English-language studies that engaged patients or other stakeholders in research on rare diseases or evaluated engagement were included. Studies were assessed on how well key research questions were answered, based on the level of detail describing engagement activities and whether outcomes from engagement were assessed. RESULTS: Thirty-five studies were included, although many reported minimal information on engagement. Patients and other stakeholders were most commonly engaged to identify patient-centered research agendas, to select which study outcomes were important to patients, to provide input on study design, and to identify strategies for increasing enrollment in trials. Rare disease organizations mainly helped provide access to patients and communicated research opportunities and findings. They also helped promote collaborative networks and provided financial support for research infrastructures. Although authors reported benefits of engagement and identified changes to their research processes, no empirical assessments of engagement practices and their effectiveness were found. CONCLUSIONS: Researchers studying rare diseases can obtain patient input regarding which research questions and health outcomes to study; however, the most effective approaches to engagement have not been well defined.

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Section: Discussionmentioning

confidence: 99%

A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

et al. 2014

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“…A more patient-centered approach to participant involvement in outcomes research has been proposed, which would lead to the empowerment of participants throughout the research process. ii Study design elements of clinical trials intended for regulatory approval of drugs and health care technologies, however, traditionally do not reflect a patient-centered approach.…”

Section: Introductionmentioning

confidence: 99%

Patient-Centeredness in the Design of Clinical Trials

et al. 2014

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Evidence from clinical trials should contribute to informed decision making and a learning health care system. People frequently, however, find participating in clinical trials meaningless or disempowering. Moreover, people often do not incorporate trial results directly into their decision making. The lack of patient centeredness in clinical trials may be partially addressed through trial design. For example, Bayesian adaptive trials designed to adjust in a pre-specified manner to changes in clinical practice could motivate people and their health care providers to view clinical trials as more applicable to real-world clinical decisions. The way in which clinical trials are designed can transform the evidence generation process to be more patient centered, providing people with an incentive to participate or continue participating in clinical trials. In order to achieve the transformation to patient-centeredness in clinical trial decisions, however, there is a need for transparent and reliable methods and education of trial investigators and site personnel.

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“…Others have suggested it is neither necessary nor advisable for clinicians to have this role, and patients can and should decide for themselves. 27 Interestingly, patients do not seem to distinguish between research and clinical care from the same site, such that positive clinical experiences may facilitate participation, but negative clinical experiences may thwart participation. To overcome research inertia- not having time or not seeing sufficient benefit to taking part in research, particularly in the new and harder to understand world of Big Data, stakeholders believe clinicians hold a very important role.…”

Section: Discussionmentioning

confidence: 99%

A Tale of 2 Constituencies

et al. 2018

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Background:Patient and clinician stakeholders are inadequately engaged in key aspects of research, particularly regarding use of Big Data to study and improve patient-centered outcomes. Little is known about the attitudes, interests, and concerns of stakeholders regarding such data.Research Design:The New York City Clinical Data Research Network (NYC-CDRN), a collaboration of research, clinical, and community leaders built a deidentified dataset containing electronic health records from millions of New Yorkers. Guided by a patient-clinician advisory board, we developed a question guide to explore patient and clinician experiences and ideas about research using large datasets. Trained facilitators led discussions during preexisting patient, community, and clinician group meetings. The research team coded meeting notes and identified themes.Results:Fully 272 individuals participated in 19 listening sessions (139 patients/advocates, 133 clinicians) at 6 medical centers with diverse NYC communities: 76% were female and 63% were nonwhite. Clinicians and patients agreed on all major themes including the central role of clinicians in introducing patients to research and the need for public campaigns to inform stakeholders about Big Data. Stakeholders were interested in using granular data to compare the care and clinical outcomes of their neighborhoods with others across NYC, but were also concerned that data could not truly be deidentified.Conclusions:Clinicians and patients agree on potential benefits of stakeholder-engaged Big Data research and provided suggestions for further research and building stakeholder research capacity. This evaluation demonstrated the potential of brief meetings with existing patient and clinical groups to explore barriers and facilitators to patient and clinician engagement.

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Continuous Patient Engagement in Comparative Effectiveness Research

Cited by 168 publications

References 55 publications

A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

Patient-Centeredness in the Design of Clinical Trials

A Tale of 2 Constituencies

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