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

Forsythe, Laura P.; Szydlowski, Victoria; Murad, M. Hassan; Ip, Stanley; Wang, Zhen; Elraiyah, Tarig; Fleurence, Rachael; Hickam, David H.

doi:10.1007/s11606-014-2895-9

Cited by 113 publications

(150 citation statements)

References 33 publications

(116 reference statements)

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“…However, it is arguably more critical within a team in which people may not have worked together before and may be bringing different expectations about how work ought to proceed. 9. Think carefully about labels, as they convey implicit values.…”

Section: Theme 3: Facilitating Good Communicationmentioning

confidence: 99%

“…8 Third, benefits of partnership may be difficult to formally assess. 9 Fourth, time requirements are a frequent concern for everyone. 7,10 These reviews offer valuable evidence syntheses relevant to partnership, as do recommendations from long-standing traditions of methods such as community-based participatory research 11 and participatory action research.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Twelve Lessons Learned for Effective Research Partnerships Between Patients, Caregivers, Clinicians, Academic Researchers, and Other Stakeholders

Witteman

Dansokho

Colquhoun³

et al. 2018

J GEN INTERN MED

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Research increasingly means that patients, caregivers, health professionals, other stakeholders, and academic investigators work in partnership. This requires effective collaboration rooted in mutual respect, involvement of all participants, and good communication.Having conducted such partnered research over multiple projects, and having recently completed a project together funded by the Patient-Centered Outcomes Research Institute, we collaboratively developed a list of 12 lessons we have learned about how to ensure effective research partnerships. To foster a culture of mutual respect, hold early in-person meetings, with introductions focused on motivation, offer appropriate orientation for everyone, and maintain awareness of individual and project goals. To actively involve all team members, it is important to ensure sufficient funding for everyone's participation, to ask for and recognize diverse contributions, and to seek the input of quiet members. To facilitate good communication, teams should carefully consider labels, avoid jargon and acronyms, judiciously use homogeneous and heterogeneous subgroups, and keep progress visible. In offering pragmatic, actionable lessons we have learned through our separate and shared experiences, we hope to help foster more patient-centered research via productive and enjoyable research collaborations. INTRODUCTIONHealth research teams increasingly include patients, caregivers, clinicians, and other stakeholders whose primary careers are not health research. This may occur because team leaders are convinced of the merits of such an approach, because funders or publishers require it, or both. 1-4 Such partnerships are intended to increase the relevance of research to those who might benefit from it and thus to reduce research waste. 5 This is an excellent and laudable aim; however, there is relatively little practical guidance available about how to effectively conduct such partnered research.Previous reviews and evaluations demonstrate four points about conducting partnered research. First, research teams with patients, caregivers, and other stakeholder team members tend to involve these stakeholders more at earlier stages in the project than at later stages. 6,7 Second, people coming into projects without a research background may require orientation in order to participate fully. 8 Third, benefits of partnership may be difficult to formally assess. 9 Fourth, time requirements are a frequent concern for everyone. 7,10 These reviews offer valuable evidence syntheses relevant to partnership, as do recommendations from long-standing traditions of methods 558JGIM such as community-based participatory research 11 and participatory action research. 12 However, as partnered research expands across research types and funding opportunities, more people are engaging in team structures that are new to them. Available frameworks suggest structures for partnerships, [13][14][15][16] and literature promotes broad principles such as addressing issues of power and equity [17][18][19...

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Section: Theme 3: Facilitating Good Communicationmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Twelve Lessons Learned for Effective Research Partnerships Between Patients, Caregivers, Clinicians, Academic Researchers, and Other Stakeholders

Witteman

Dansokho

Colquhoun³

et al. 2018

J GEN INTERN MED

View full text Add to dashboard Cite

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“…The effective engagement of patients in research on rare diseases is currently an area of increasing interest [8, 20]. This has come about as a result of governments giving greater priority and incentives for rare disease research, including through orphan drug legislation, [21, 22] and promotion by policy makers [20, 23].…”

Section: Discussionmentioning

confidence: 99%

“…This has come about as a result of governments giving greater priority and incentives for rare disease research, including through orphan drug legislation, [21, 22] and promotion by policy makers [20, 23]. This has occurred in parallel with a recent shift from a passive role for patients in research towards a desire to play a more involved role in the research process, as demonstrated by the survey outcomes.…”

Section: Discussionmentioning

confidence: 99%

An innovative and collaborative partnership between patients with rare disease and industry-supported registries: the Global aHUS Registry

Woodward¹,

Johnson

Walle

et al. 2016

Orphanet J Rare Dis

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Background: Patients are becoming increasingly involved in research which can promote innovation through novel ideas, support patient-centred actions, and facilitate drug development. For rare diseases, registries that collect data from patients can increase knowledge of the disease's natural history, evaluate clinical therapies, monitor drug safety, and measure quality of care. The active participation of patients is expected to optimise rare-disease management and improve patient outcomes. However, few reports address the type and frequency of interactions involving patients, and what research input patient groups have. Here, we describe a collaboration between an international group of patient organisations advocating for patients with atypical haemolytic uraemic syndrome (aHUS), the aHUS Alliance, and an international aHUS patient registry (ClinicalTrials.gov NCT01522183).

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“…In general, PE offers an opportunity to improve the science of PRO measure development, and to reinforce the patient centeredness of the process [44,45]. For example, PE may facilitate identification of topics/areas for future research, development of optimal study design, selection of appropriate outcomes/domains to include in a PRO instrument, and clarification of patients' description of symptoms.…”

Section: Patient Engagementmentioning

confidence: 99%

Streamlining the Validation of Patient Reported Outcome (PRO) Measures in Drug Regulatory Processes

Salek

Kamudoni²

2015

Pharm Med

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The context and setting in which patient-reported outcome (PRO) measures applied in drug development programmes are developed and applied are undergoing rapid changes. The emergence of social media, coupled with advancement of patient-centred methodologies, has led to more connected and empowered patients than before. Drug regulatory agencies are beginning to embrace a new role as collaborators in the drug development process, and new models for defining core outcomes in various disease areas are emerging. Technological advances and social changes have led to the wide use of personal gadgets with greater internet connectivity and computing power than most computers had a generation ago. To explore how progress in the measurement of PROs might be achieved amidst such changes, lessons were drawn from the PRO labelling claims reviewed by the US FDA following the publication of the PRO guidance in 2009. In addition, semi-structured interviews were carried out with seven experts working with drug regulatory agencies and in the pharmaceutical industry to identify major issues and possible solutions. The following recommendations are proposed: (1) that comprehensive hypotheses about the PRO concept be developed; (2) that PRO concepts/measures maintain substantive and clinical validity; (3) that the use of advanced psychometric methods be considered as the new gold standard in the development of PRO measures; (4) that psychometric properties of measures established through ''validation by application'' be enhanced; (5) that the generalizability of PRO measures be enhanced; (6) that the assessment of ability to detect change, and the interpretability of scores, be improved; and (7) that PRO guidelines by major regulatory authorities be aligned. These entail a greater transparency on the decisions taken during the PRO measure development process and greater understanding of the integration between qualitative aspects of the PRO measure and the score/measurement attributes. Key PointsMoving the science of patient-reported outcome (PRO) measurement within drug regulatory settings forward requires greater transparency on the decisions taken during the PRO measure development process and greater understanding of the integration between qualitative aspects of the PRO measure and the score/measurement attributes.Enhancing the validation of PROs will require ensuring that PRO measures have a strong conceptual foundation, that their clinical meaningfulness and relevance is adequately demonstrated, that a high degree of precision is established and that measurement equivalence across groups is determined. Furthermore, there is an urgent need for alignment in guidelines on PROs across medicines regulatory agencies supporting generalizability of measures.Electronic supplementary material The online version of this article (

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

Cited by 113 publications

References 33 publications

Twelve Lessons Learned for Effective Research Partnerships Between Patients, Caregivers, Clinicians, Academic Researchers, and Other Stakeholders

Twelve Lessons Learned for Effective Research Partnerships Between Patients, Caregivers, Clinicians, Academic Researchers, and Other Stakeholders

An innovative and collaborative partnership between patients with rare disease and industry-supported registries: the Global aHUS Registry

Streamlining the Validation of Patient Reported Outcome (PRO) Measures in Drug Regulatory Processes

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