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A systematic review of stakeholder engagement in comparative effectiveness and patient-centered outcomes research.

ABSTRACT: We conducted a review of the peer-reviewed literature since 2003 to catalogue reported methods of stakeholder engagement in comparative effectiveness research and patient-centered outcomes research.We worked with stakeholders before, during and after the review was conducted to: define the primary and key research questions; conduct the literature search; screen titles, abstracts and articles; abstract data from the articles; and analyze the data. The literature search yielded 2,062 abstracts. The review was conducted on 70 articles that reported on stakeholder engagement in individual research projects or programs.Reports of stakeholder engagement are highly variable in content and quality. We found frequent engagement with patients, modestly frequent engagement with clinicians, and infrequent engagement with stakeholders in other key decision-making groups across the healthcare system. Stakeholder engagement was more common in earlier (prioritization) than in later (implementation and dissemination) stages of research. The roles and activities of stakeholders were highly variable across research and program reports.To improve on the quality and content of reporting, we developed a 7-Item Stakeholder Engagement Reporting Questionnaire. We recommend three directions for future research: 1) descriptive research on stakeholder-engagement in research; 2) evaluative research on the impact of stakeholder engagement on the relevance, transparency and adoption of research; and 3) development and validation of tools that can be used to support stakeholder engagement in future work.

SUBMITTER: Concannon TW

PROVIDER: S-EPMC4242886 | biostudies-other | 2014 Dec

REPOSITORIES: biostudies-other

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A systematic review of stakeholder engagement in comparative effectiveness and patient-centered outcomes research.

Concannon Thomas W TW Fuster Melissa M Saunders Tully T Patel Kamal K Wong John B JB Leslie Laurel K LK Lau Joseph J

Journal of general internal medicine 20140604 12

<h4>Objectives</h4>We conducted a review of the peer-reviewed literature since 2003 to catalogue reported methods of stakeholder engagement in comparative effectiveness research and patient-centered outcomes research.<h4>Methods and results</h4>We worked with stakeholders before, during and after the review was conducted to: define the primary and key research questions; conduct the literature search; screen titles, abstracts and articles; abstract data from the articles; and analyze the data. T ...[more]

PMID: 24893581

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Project description:Introduction/objectivesThe engagement of patients and other stakeholders is a critical element in the design of patient-centered outcomes research studies. However, methodology for scalable engagement in research management particularly activities such as operationalization of principles and setting of priorities is not well-developed. The objective of this study is to describe a novel approach for scalable stakeholder engagement in research aligned with the Patient-Centered Outcomes Research Institute (PCORI) engagement principles, which was evaluated in a national clinical data research network.Materials and methodsPatient, patient advocate, clinician, and researcher stakeholders were recruited from clinical sites, as well as social media sites related to the 3 conditions of focus, heart failure, obesity, and Kawasaki disease. The engagement strategy was designed, implemented, and mapped to the PCORI engagement principles. Evaluation included internal assessment and quantitative measures of online engagement.ResultsWe operationalized the PCORI principles with 12 stakeholder engagement strategies and convened stakeholder advisory boards and online research prioritization panels to determine research priorities in a rigorous, deliberative process. A total of 46 advisors (20 patients) and 339 panelists (159 patients) actively participated. There were not significant differences between patients and clinicians in level of online engagement. Nonetheless, while patients reported a slightly greater challenge with following online discussion, they overall had a more favorable opinion about use of the online format.Discussion/conclusionAn efficient way to engage large numbers of representative stakeholders in research is a necessary first step to assure the public of trustworthy use of data networks for health research. This paper describes a comprehensive approach to engagement in patient-centered outcomes research management that informs ongoing development of rigorous methodologies in this area.

Project description:IntroductionThe SCL16A2 gene encodes the thyroid hormone (TH) transporter MCT8. Pathogenic variants result in a reduced TH uptake into the CNS despite high serum T3 concentrations. Patients suffer from severe neurodevelopmental delay and require multidisciplinary care. Since a first compassionate use study in 2008, the development of therapies has recently gained momentum. Treatment strategies range from symptom-based approaches, supplementation with TH or TH-analogs, to gene therapy. All these studies have mainly used surrogate endpoints and clinical outcomes. However, the EMA and FDA strongly encourage researchers to involve patients and their advocacy groups in the design of clinical trials. This should strengthen the patients' perspective and identify clinical endpoints that are clinically relevant to their daily life.MethodsWe involved patient families to define patient-relevant outcomes for MCT8 deficiency. In close collaboration with patient families, we designed a questionnaire asking for their five most preferred therapeutic goals, which, if achieved at least, make a difference in their lives. In addition, we performed a systematic review according to Cochrane recommendations of the published treatment trials.ResultsWe obtained results from 15 families with completed questionnaires from 14 mothers and 8 fathers. Improvement in development, especially in gross motor skills, was most important to the parents. 59% wished for head control and 50% for sitting ability. Another 36% wished for weight gain, 32% for improvement of expressive language skills, and 18% for a reduction of dystonia/spasticity, less dysphagia, and reflux. Paraclinical aspects were least important (5-9%). In a treatment trial (n=46) and compassionate use cases (n=83), the results were mainly inconclusive, partly due to a lack of predefined patient-centered clinical endpoints.DiscussionWe recommend that future trials should define a relevant improvement in "development" and/or other patient-relevant outcomes compared to natural history as treatment goals.

Project description:BACKGROUND: A compelling ethical rationale supports patient engagement in healthcare research. It is also assumed that patient engagement will lead to research findings that are more pertinent to patients' concerns and dilemmas. However; it is unclear how to best conduct this process. In this systematic review we aimed to answer 4 key questions: what are the best ways to identify patient representatives? How to engage them in designing and conducting research? What are the observed benefits of patient engagement? What are the harms and barriers of patient engagement? METHODS: We searched MEDLINE, EMBASE, PsycInfo, Cochrane, EBSCO, CINAHL, SCOPUS, Web of Science, Business Search Premier, Academic Search Premier and Google Scholar. Included studies were published in English, of any size or design that described engaging patients or their surrogates in research design. We conducted an environmental scan of the grey literature and consulted with experts and patients. Data were analyzed using a non-quantitative, meta-narrative approach. RESULTS: We included 142 studies that described a spectrum of engagement. In general, engagement was feasible in most settings and most commonly done in the beginning of research (agenda setting and protocol development) and less commonly during the execution and translation of research. We found no comparative analytic studies to recommend a particular method. Patient engagement increased study enrollment rates and aided researchers in securing funding, designing study protocols and choosing relevant outcomes. The most commonly cited challenges were related to logistics (extra time and funding needed for engagement) and to an overarching worry of a tokenistic engagement. CONCLUSIONS: Patient engagement in healthcare research is likely feasible in many settings. However, this engagement comes at a cost and can become tokenistic. Research dedicated to identifying the best methods to achieve engagement is lacking and clearly needed.

Project description:BackgroundWe used various methods for identifying and prioritizing patient-centered outcomes (PCOs) for comparative effectiveness research (CER).MethodsWe considered potential PCOs ("benefits" and "harms") related to (1) gabapentin for neuropathic pain and (2) quetiapine for bipolar depression. Part 1 (April 2014 to March 2015): we searched for PCO research and core outcome sets (COSs). We conducted electronic searches of bibliographic databases and key websites and examined FDA prescribing information and reports of clinical trials and systematic reviews. We asked patient and clinician co-investigators to identify PCOs. Part 2 (not part of our original study protocol): in 2015, we surveyed members of The TMJ Association, Ltd., a patient group associated with temporomandibular disorders (4130 invitations sent). Participants prioritized (1) the importance of six potential benefits and (2) 21 potential harms selected by the investigators in part 1, using stated preference methods. We calculated descriptive statistics.ResultsIn part 1, we identified a COS for pain, the Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials (IMMPACT) recommendations. The COS identified several important benefits, but it lacked specific recommendations about which potential harms to include in CER. We did not identify a COS for bipolar depression. Research reports, prescribing information, and patient co-investigators helped identify but not prioritize outcomes. We abandoned our electronic search for PCO research because we found it would be resource-intensive and yield few relevant reports. In part 2, surveying patients was useful for prioritizing PCOs. Members of The TMJ Association, Ltd., completed the survey (N =?746) and successfully prioritized both benefits and harms. Participants did not identify many benefits other than those we identified in part 1; several participants identified additional harms.ConclusionsThese exploratory results could inform future research about identifying and prioritizing PCOs. We found that stakeholder co-investigators and research reports contributed to identifying PCOs; surveying a patient group contributed to prioritizing PCOs. Prioritizing potential harms was particularly challenging because there are many more potential harms than potential benefits. Methods for identifying and prioritizing potential benefits for CER might not be appropriate for harms. Further research is needed to determine the generalizability of these results.

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A systematic review of stakeholder engagement in comparative effectiveness and patient-centered outcomes research.

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A systematic review of stakeholder engagement in comparative effectiveness and patient-centered outcomes research.

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