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01-08-2014 | Review Article

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

Authors: Laura P. Forsythe, PhD, MPH, Victoria Szydlowski, BS, Mohammad Hassan Murad, MD, MPH, Stanley Ip, MD, Zhen Wang, PhD, Tarig A. Elraiyah, MBBS, Rachael Fleurence, PhD, David H. Hickam, MD, MPH

Published in: Journal of General Internal Medicine | Special Issue 3/2014

ABSTRACT

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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Title: A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases
Authors: Laura P. Forsythe, PhD, MPH
Victoria Szydlowski, BS
Mohammad Hassan Murad, MD, MPH
Stanley Ip, MD
Zhen Wang, PhD
Tarig A. Elraiyah, MBBS
Rachael Fleurence, PhD
David H. Hickam, MD, MPH
Publication date: 01-08-2014
Publisher: Springer US
Published in: Journal of General Internal Medicine / Issue Special Issue 3/2014
Print ISSN: 0884-8734
Electronic ISSN: 1525-1497
DOI: https://doi.org/10.1007/s11606-014-2895-9

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ABSTRACT

BACKGROUND

OBJECTIVES

METHODS/RESEARCH DESIGN

RESULTS

CONCLUSIONS

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