IMPACCT Kids' Care: a real-world example of stakeholder involvement in comparative effectiveness research.
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SUBMITTER: Likumahuwa-Ackman S
PROVIDER: S-EPMC4538706 | biostudies-other | 2015 Aug
REPOSITORIES: biostudies-other
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Project description:Patients, providers and policy-makers rely heavily on comparative effectiveness research (CER) when making complex, real-world medical decisions. In particular, Medicaid providers and policy-makers face unique challenges in decision-making because their program cares for traditionally underserved populations, especially children, pregnant women and people with mental illness. Because these patient populations have generally been underrepresented in research discussions, CER questions for these groups may be understudied. To address this problem, the Agency for Healthcare Research and Quality commissioned our team to work with Medicaid Medical Directors and other stakeholders to identify relevant CER questions. Through an iterative process of topic identification and refinement, we developed relevant, feasible and actionable questions based on issues affecting Medicaid programs nationwide. We describe challenges and limitations and provide recommendations for future stakeholder engagement.
| S-EPMC5549719 | biostudies-literature
Project description: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.
| S-EPMC4242886 | biostudies-other
Project description:BackgroundThe Institute of Medicine recently recommended that comparative effectiveness research (CER) should involve input from consumers. While systematic reviews are a major component of CER, little is known about consumer involvement.ObjectiveTo explore current approaches to involving consumers in US-based and key international organizations and groups conducting or commissioning systematic reviews ('organizations').DesignIn-depth, semi-structured interviews with key informants and review of organizations' websites.Setting and participantsSeventeen highly regarded US-based and international (Cochrane Collaboration, Campbell Collaboration) organizations.ResultsOrganizations that usually involve consumers (seven of 17 in our sample) involve them at a programmatic level in the organization or in individual reviews through one-time consultation or on-going collaboration. For example, consumers may suggest topics, provide input on the key questions of the review, provide comments on draft protocols and reports, serve as co-authors or on an advisory group. Organizations involve different types of consumers (individual patients, consumer advocates, families and caregivers), recruiting them mainly through patient organizations and consumer networks. Some offer training in research methods, and one developed training for researchers on how to involve consumers. Little formal evaluation of the effects of consumer involvement is being carried out.ConclusionsConsumers are currently involved in systematic reviews in a variety of ways and for various reasons. Assessing which approaches are most effective in achieving different aims of consumer involvement is now required to inform future recommendations on consumer involvement in CER.
| S-EPMC5060681 | biostudies-literature
Project description:Comparative effectiveness research (CER) is intended to address the expressed needs of patients, clinicians, and other stakeholders. Representatives of 54 stakeholder groups with an interest in chronic obstructive pulmonary disease (COPD) participated in workshops convened by the COPD Outcomes-based Network for Clinical Effectiveness and Research Translation (CONCERT) over a 2-year period. Year 1 focused on chronic care and care coordination. Year 2 focused on acute care and transitions in care between healthcare settings. Discussions and provisional voting were conducted via teleconferences and e-mail exchanges before the workshop. Final prioritization votes occurred after in-person discussions at the workshop. We used a modified Delphi approach to facilitate discussions and consensus building. To more easily quantify preferences and to evaluate the internal consistency of rankings, the Analytic Hierarchy Process was incorporated in Year 2. Results of preworkshop and final workshop voting often differed, suggesting that prioritization efforts relying solely on requests for topics from stakeholder groups without in-person discussion may provide different research priorities. Research priorities varied across stakeholder groups, but generally focused on studies to evaluate different approaches to healthcare delivery (e.g., spirometry for diagnosis and treatment, integrated healthcare strategies during transitions in care) rather than head-to-head comparisons of medications. This research agenda may help to inform groups intending to respond to CER funding opportunities in COPD. The methodologies used, detailed in the online supplement, may also help to inform prioritization efforts for CER in other health conditions.
| S-EPMC3603554 | biostudies-literature
Project description:Aim: Stakeholder engagement is central to comparative effectiveness research yet there are gaps in definitions of success. We used a framework developed by Lavallee et al. defining effective engagement criteria to evaluate stakeholder engagement during a pragmatic cluster-randomized trial. Methods: Semi-structured interviews were developed from the framework and completed to learn about members' experiences. Interviews were analyzed in a deductive approach for themes related to the effective engagement criteria. Results: Thirteen members participated and described: respect for ideas, time to achieve consensus, access to information and continuous feedback as areas of effective engagement. The primary criticism was lack of diversity. Discussion: Feedback was positive, particularly among themes of respect, trust and competence, and led to development of a list of best practices for engagement. The framework was successful for evaluating engagement. Conclusion: Standardized frameworks allow studies to formally evaluate their stakeholder engagement approach and develop best practices for future research.
| S-EPMC9832319 | biostudies-literature
Project description:BackgroundThere is an urgent need to understand the real-world effectiveness of remdesivir in the treatment of SARS-CoV-2.MethodsThis was a retrospective comparative effectiveness study. Individuals hospitalized in a large private healthcare network in the US from February 23, 2020 through February 11, 2021 with a positive test for SARS-CoV-2 and ICD-10 diagnosis codes consistent with symptomatic COVID-19 were included. Remdesivir recipients were matched to controls using time-dependent propensity scores. The primary outcome was time to improvement with a secondary outcome of time to death.ResultsOf 96,859 COVID-19 patients, 42,473 (43.9%) received at least one remdesivir dose. The median age of remdesivir recipients was 65 years, 23,701 (55.8%) were male and 22,819 (53.7%) were non-white. Matches were found for 18,328 patients (43.2%). Remdesivir recipients were significantly more likely to achieve clinical improvement by 28 days (adjusted hazard ratio [1.19, 95% confidence interval (CI), 1.16-1.22]). Remdesivir patients on no oxygen (aHR 1.30, 95% CI 1.22-1.38) or low-flow oxygen (aHR 1.23, 95% CI 1.19-1.27) were significantly more likely to achieve clinical improvement by 28 days. There was no significant impact on the likelihood of mortality overall (aHR 1.02, 95% CI 0.97-1.08). Remdesivir recipients on low-flow oxygen were significantly less likely to die than controls (aHR 0.85, 95% CI 0.77-0.92; 28-day mortality 8.4% [865 deaths] for remdesivir patients, 12.5% [1,334 deaths] for controls).ConclusionsThese results support the use of remdesivir for hospitalized COVID-19 patients on no or low-flow oxygen. Routine initiation of remdesivir in more severely ill patients is unlikely to be beneficial.
| S-EPMC8754724 | biostudies-literature
Project description:Novel agents, including Bruton tyrosine kinase inhibitors (BTKis), have become the standard of care for patients with chronic lymphocytic leukemia (CLL). We conducted a real-world retrospective analysis of patients with CLL treated with acalabrutinib vs ibrutinib using the Flatiron Health database. Patients with CLL were included if they initiated acalabrutinib or ibrutinib between 1 January 2018 and 28 February 2021. The primary outcome of interest was time to treatment discontinuation (TTD). Kaplan-Meier analysis was used to estimate unweighted and weighted median TTD. A weighted Cox proportional hazards model was used to compare the TTD between cohorts. Of the 2509 patients included in the analysis, 89.6% received ibrutinib, and 14.1% received acalabrutinib. TTD was not significantly different between cohorts in the unweighted analysis. After weighting, the cohorts were balanced on all baseline characteristics except cardiovascular risk factors and baseline medications use. The median (95% confidence interval [CI]) TTD was not reached (NR; 95% CI, 25.1 to NR) for the acalabrutinib cohort and was 23.4 months (95% CI, 18.1-28.7) for the ibrutinib cohort. The discontinuation rate at 12 months was 22% for the weighted acalabrutinib cohort vs 31% for the weighted ibrutinib cohort (P = .005). After additional adjustment for prior BTKi use, the acalabrutinib cohort had a 41% lower risk of discontinuation vs ibrutinib (hazard ratio, 0.59; 95% CI, 0.43-0.81; P = .001). In the largest available study comparing BTKis, patients with CLL receiving acalabrutinib demonstrated lower rates of discontinuation and a prolonged time to discontinuation vs those receiving ibrutinib.
| S-EPMC10424141 | biostudies-literature
Project description:OBJECTIVE:Head-to-head randomized controlled trials (RCTs) are the gold standard for assessing comparative treatment effects. In the absence of direct comparisons between all possible antiepileptic drugs (AEDs), however, clinical decision-making in focal (partial onset) epilepsy relies on alternative evidence borne from indirect comparisons including network meta-analyses (NMAs) and from real-world evidence (RWE) studies. We review NMAs and observational RWE studies comparing AEDs in the adjunctive setting to compare the robustness of these methods and to formulate recommendations for future evidence development. METHODS:A literature review identified NMAs and RWE studies comparing AEDs for the adjunctive treatment of focal seizures published between January 2008 and October 2018. NMAs were evaluated for robustness using a framework based on guidelines from the National Institute for Health and Care Excellence Decision Support Unit and the International Society for Pharmacoeconomics and Outcomes Research. RWE studies were evaluated using the GRACE checklist. RESULTS:From a total of 1993 records, 11 NMAs and six RWE studies were eligible. Key limitations identified in the NMAs include nonsystematic selection of RCTs, unexplored heterogeneity between included RCTs in terms of study and patient characteristics, and selection of AEDs and AED doses or dosing strategies that are not reflective of clinical practice. The main limitations of RWE studies concern sample size, design, and analysis methods. Approximately 90% of comparisons between individual AEDs were nonsignificant in the NMAs. None of the RWE studies adjusted for baseline differences between comparator groups; therefore, they lack the validity to make comparative conclusions. SIGNIFICANCE:Current NMAs and RWE studies provide only nominal comparative evidence for AED treatments in focal epilepsy, and should be used with caution for decision-making due to their methodological limitations. To overcome these hurdles, adherence to methodological guidelines and concerted efforts to collect relevant outcome data in the real world are needed.
| S-EPMC7216985 | biostudies-literature
Project description:Several of the most important conservation prioritization approaches select markedly different areas at global and regional scales. They are designed to maximize a certain biodiversity dimension such as coverage of species in the case of hotspots and complementarity, or composite properties of ecosystems in the case of wilderness. Most comparisons between approaches have ignored the multidimensionality of biodiversity. We analyze here the results of two species-based methodologies-hotspots and complementarity-and an ecosystem-based methodology-wilderness-at local scale. As zoning of protected areas can increase the effectiveness of conservation, we use the data employed for the management plan of the Peneda-Gerês National Park in Portugal. We compare the approaches against four criteria: species representativeness, wilderness coverage, coverage of important areas for megafauna, and for regulating ecosystem services. Our results suggest that species- and ecosystem-based approaches select significantly different areas at local scale. Our results also show that no approach covers well all biodiversity dimensions. Species-based approaches cover species distribution better, while the ecosystem-based approach favors wilderness, areas important for megafauna, and for ecosystem services. Management actions addressing different dimensions of biodiversity have a potential for contradictory effects, social conflict, and ecosystem services trade-offs, especially in the context of current European biodiversity policies. However, biodiversity is multidimensional, and management and zoning at local level should reflect this aspect. The consideration of both species- and ecosystem-based approaches at local scale is necessary to achieve a wider range of conservation goals.
| S-EPMC4392121 | biostudies-literature
Project description:IntroductionResilience in healthcare (RiH) is understood as the capacity of the healthcare system to adapt to challenges and changes at different system levels, to maintain high-quality care. Adaptive capacity is founded in the knowledge, skills and experiences of the people in the system, including patients, family or next of kin, healthcare providers, managers and regulators. In order to learn from and support useful adaptations, research is needed to better understand adaptive capacity and the nature and context of adaptations. This includes research on the actors involved in creating resilient healthcare, and how and in what circumstances different groups of patients and other key healthcare stakeholders enact adaptations that contribute to resilience across all levels of the healthcare system.Methods and analysisThis 5-year study applies an interactive design in a two-phased approach to explore and conceptualise patient and stakeholder involvement in resilient healthcare. Study phase 1 is exploratory and will use such data collection methods as literature review, document analysis, interviews and focus groups. Study phase 2 will use a participatory design approach to develop, test and evaluate a conceptual model for patient and stakeholder involvement in RiH. The study will involve patients and other key stakeholders as active participants throughout the research process.Ethics and disseminationThe RiH research programme of which this study is a part is approved by the Norwegian Centre for Research Data (No. 864334). Findings will be disseminated through scientific articles, presentations at national and international conferences, through social media and popular press, and by direct engagement with the public, including patient and stakeholder representatives.
| S-EPMC8183273 | biostudies-literature