Developing Patient-Centered Real-World Evidence: Emerging Methods Recommendations From a Consensus Process

Oehrlein, Elisabeth M.; Schoch, Silke; Burcu, Mehmet; McBeth, J; Bright, Jennifer; Pashos, Chris L.; Willke, Richard J.; Love, T.R.; Mattingly, T. Joseph; Perfetto, Elèanor M.

doi:10.1016/j.jval.2022.04.1738

Cited by 13 publications

(7 citation statements)

References 37 publications

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“…Most important among this list is the second recommendation to: “Prioritize patient‐identified questions aligned with study objectives/audience” (tab. 3, p. 7) 12 . For experienced community‐engaged researchers, active involvement of stakeholders in the formation of study questions, the nexus of all discovery, is a clear litmus of meaningful participation 12 .…”

Section: Discussionmentioning

confidence: 99%

“…In the area of patient‐centered real‐world evidence, Oehrlein, et al 12 propose 13 recommendations to guide future research. The recommendations are organized into four categories (refinement of the research question, development of the research protocol, translation of research findings, and general recommendations).…”

Section: Discussionmentioning

confidence: 99%

“…12 For experienced communityengaged researchers, active involvement of stakeholders in the formation of study questions, the nexus of all discovery, is a clear litmus of meaningful participation. 12 It is also very much in line with current developments in academia on diversity, equity, and inclusion which name community engagement as critical to antiracist science. In the next phase of highly structured community engagement approaches, such as CBPR, expansion lies in work to "Engage for Equity," also known as E2.…”

Section: Rise Websitementioning

confidence: 99%

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A community‐engaged approach to the design of a population‐based prospective cohort study to promote bladder health

Klusaritz

Maki

Levin³

et al. 2022

Neurourology and Urodynamics

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Introduction: Community engagement is increasingly recognized as a critical component of research, but few studies provide details on how to successfully incorporate community perspectives in urological research. This manuscript describes the community engagement strategy used by the Prevention of Lower Urinary Tract Symptoms Research Consortium (PLUS) to design RISE FOR HEALTH (RISE), a multicenter, population-based, prospective cohort study to promote bladder health. Methods and Results:The PLUS Community Engagement Subcommittee, guided by a set of antiracist community engagement principles and practices, organized, implemented, and communicated findings for all RISE community engagement activities. Community engagement was conducted through a diverse network of community partners at PLUS clinical research centers called Rapid Assessment Partners (RAPs). Via online surveys (4), virtual discussion groups ( 14), and one-on-one interviews (12), RAPs provided input on RISE processes and materials, including in-person visit procedures,

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Rise Websitementioning

confidence: 99%

See 1 more Smart Citation

A community‐engaged approach to the design of a population‐based prospective cohort study to promote bladder health

Klusaritz

Maki

Levin³

et al. 2022

Neurourology and Urodynamics

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“…A broader RDR equity lens could be achieved by using equity tools such as the PROGRESS (Place of resident, Race, Occupation, Gender, Religion, Education, Socioeconomic status, Social capital) framework [ 95 ], which would facilitate a greater understanding of how equity-deserving populations are affected by RDs or represented in RDR registries. Finally, the integration of patients’ experiences and insights when designing and interpreting results is an important avenue of research to enhance the quality and acceptance of RDR studies by generating patient-centered RWE [ 96 ].…”

Section: Limitationsmentioning

confidence: 99%

Scoping review of the recommendations and guidance for improving the quality of rare disease registries

Tarride,

Okoh,

Aryal

et al. 2024

Orphanet J Rare Dis

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Background Rare disease registries (RDRs) are valuable tools for improving clinical care and advancing research. However, they often vary qualitatively, structurally, and operationally in ways that can determine their potential utility as a source of evidence to support decision-making regarding the approval and funding of new treatments for rare diseases. Objectives The goal of this research project was to review the literature on rare disease registries and identify best practices to improve the quality of RDRs. Methods In this scoping review, we searched MEDLINE and EMBASE as well as the websites of regulatory bodies and health technology assessment agencies from 2010 to April 2023 for literature offering guidance or recommendations to ensure, improve, or maintain quality RDRs. Results The search yielded 1,175 unique references, of which 64 met the inclusion criteria. The characteristics of RDRs deemed to be relevant to their quality align with three main domains and several sub-domains considered to be best practices for quality RDRs: (1) governance (registry purpose and description; governance structure; stakeholder engagement; sustainability; ethics/legal/privacy; data governance; documentation; and training and support); (2) data (standardized disease classification; common data elements; data dictionary; data collection; data quality and assurance; and data analysis and reporting); and (3) information technology (IT) infrastructure (physical and virtual infrastructure; and software infrastructure guided by FAIR principles (Findability; Accessibility; Interoperability; and Reusability). Conclusions Although RDRs face numerous challenges due to their small and dispersed populations, RDRs can generate quality data to support healthcare decision-making through the use of standards and principles on strong governance, quality data practices, and IT infrastructure.

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“…Not doing so is a missed opportunity for learning about the therapeutic use of interventions. Patient experience data reflect patients' lived experience, their symptoms, interactions with the healthcare system, information-seeking behaviours, any misdiagnoses, lifestyle changes, treatments, side effects, and comorbidities adding to the completeness of the data to enhance person-centred care [99]. Patient groups themselves can collect data, as with Duchenne disease [100].…”

Section: Access To Innovative Medicines: What Makes One Need This?mentioning

confidence: 99%

Perspective Chapter: Patient Advocacy - From a Seat at the Table to Equal Stakeholders

L. Wale,

Hamerlijnck

2024

Economics of Healthcare, Studies and Cases [Working Title]

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Much has changed in individual and policy level patient advocacy in the years since the late 1980s when HIV/AIDS activists challenged the United States medical product regulator and aspects of how randomised controlled trials are conducted. Delays in access to innovative, life-saving medical interventions continue to be a key topic. We provide storylines to explore three interrelated pathways. The first follows the involvement of patients in the medical product lifecycle and clinical trial regulation, where the biopharmaceutical industry is a major stakeholder. The second follows the course of evidence-based practice and patients, the need for outcome measures of patient experience data and patient-relevant outcome measures that incorporate the patient voice into person-centred models of healthcare; and how regulators and HTA bodies are accelerating access to innovative medical products. The third storyline uses the European Union with its public-private funding of medical and healthcare research as a case study to highlight how patient advocacy is changing. Following the maturation of patient advocacy from patients as research subjects to patient involvement and centredness throughout the medical product lifecycle, we suggest possible next steps to continue to evolve patient advocacy into equal stakeholders within healthcare and innovative medical product development.

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Developing Patient-Centered Real-World Evidence: Emerging Methods Recommendations From a Consensus Process

Cited by 13 publications

References 37 publications

A community‐engaged approach to the design of a population‐based prospective cohort study to promote bladder health

A community‐engaged approach to the design of a population‐based prospective cohort study to promote bladder health

Scoping review of the recommendations and guidance for improving the quality of rare disease registries

Perspective Chapter: Patient Advocacy - From a Seat at the Table to Equal Stakeholders

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