A guide to research partnerships for pragmatic clinical trials.
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ABSTRACT:
SUBMITTER: Johnson KE
PROVIDER: S-EPMC4707716 | biostudies-literature | 2014 Dec
REPOSITORIES: biostudies-literature
Publications
A guide to research partnerships for pragmatic clinical trials.
Johnson Karin E KE Tachibana Chris C Coronado Gloria D GD Dember Laura M LM Glasgow Russell E RE Huang Susan S SS Martin Paul J PJ Richards Julie J Rosenthal Gary G Septimus Ed E Simon Gregory E GE Solberg Leif L Suls Jerry J Thompson Ella E Larson Eric B EB
BMJ (Clinical research ed.) 20141201
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Project description:The use of real-world evidence (RWE) studies, including pragmatic randomised controlled trials (RCTs; randomised RWE studies), to aid the development of treatment guidelines, is gradually becoming a mainstay within clinical practice. RWE is an integral part of patient-driven decision-making and offers important value to add complimentary evidence to traditional RCTs; these provide a more well-rounded view of the benefits to patient-reported outcomes and improve the external validity of a given treatment versus findings from traditional RCTs alone. Discussions in recent scientific workshops explored the importance of pragmatic RCTs in optimising guideline development and patient care in chronic obstructive pulmonary disease (COPD) and asthma. The Salford Lung Study in patients with COPD (NCT01551758) and asthma (NCT01706198) were the world's first prelicence pragmatic RCTs that compared novel investigational treatments with existing COPD and asthma treatments and, more recently (2021), RWE studies have been used by the American Thoracic Society and the US Food and Drug Administration to support the approval of an immunosuppressant drug in patients receiving lung transplants. This highlights the importance of RWE data in supporting clinical guideline development and emphasises the advantages for the use of pragmatic RCTs in guiding clinical practice.
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Project description:PurposeToday's clinical trial partnerships frequently join multi-disciplinary investigators and stakeholders, from different countries and cultures, to conduct research with a broad array of goals. This diversity, while a strength, can also foster divergent views about priorities and what constitutes success, thereby posing challenges for management, operations, and evaluation. As a sponsor and partner in such collaborations, we seek to assist and support their development and implementation of sound research strategies, to optimize their efficiency, sustainability, and public health impact. This report describes our efforts using an adaptation of the well-established Kaplan-Norton strategy management paradigm, in our clinical trials setting. We share findings from our first test of the utility and acceptance of this approach for evaluating and managing research strategies in a collaborative clinical research partnership.ResultsFindings from pilot studies and our first implementation in an ongoing clinical research partnership in Liberia, provide initial support for our hypothesis that an adapted version of the Kaplan-Norton strategy management model can have use in this setting. With leadership from within the partnership, analysis artifacts were gathered, and assessments made using standardized tools. Practical feasibility, resonance of the findings with partners, and convergence with other empirical assessments lend initial support for the view that this approach holds promise for obtaining meaningful, useable results for assessing and improving clinical research management.Conclusions and implicationsEngaged leadership, thoughtful timing to align with partnership planning cycles, support for the process, and an eye towards the collaboration's long-term goals appear important for developing model understanding and practice. Skepticism about evaluations, and unease at exposing weaknesses, may hinder the effort. Acceptance of findings and associated opportunities for improvement by group leadership, support a growing sense of validity. Next steps aim to test the approach in other partnerships, streamline the methodology for greater ease of use, and seek possible correlations of strategy management assessments with performance evaluation. There is hardly a better example than the COVID-19 pandemic, to spotlight the need for efficient and effective clinical research partnerships to address global health challenges. While heartened by the collaborative spirit driving the effort so far, we cannot let our enthusiasm lull us into thinking that nobility of purpose or an abundance of good will is sufficient. Careful monitoring and adjustment of clinical research strategy in response to changes (e.g., demographics, pathogen evolution, research acceptance, political and cultural environments) are vital to making the needed adjustments that can guide these programs toward successful outcomes. We hope that our work can raise awareness about the importance, relevance, and feasibility of sound strategy management in clinical research partnerships, especially during this time when there is so much at stake.
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Project description:AimPracticing physicians inevitably become involved in pragmatic clinical trials (PCTs), including comparative effectiveness research. We sought to identify physicians' perspectives related to PCTs.MethodsIn-depth semistructured interviews with 20 physicians in the USA.ResultsAlthough physicians are generally willing to participate in PCTs, their support is predicated on several factors including expected benefits, minimization of time and workflow burdens and physician engagement. Physicians communicated a desire to respect patients' rights and interests while maintaining a high level of care.ConclusionFuture work is needed to systematically assess the impact of PCTs on clinicians in meeting their ethical obligations to patients and the burdens clinicians are willing to accept in exchange for potential benefits.
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Project description:BackgroundPragmatic trials that combine electronic health record data and patient-reported data may be subject to selection bias due to the differential post-randomization exclusion of participants who are randomized in error. Such situations are often caused by inevitable reasons, such as incomplete patient medical records at the pre-randomization stage. This can lead to participants in the intervention arm being identified as ineligible after randomization, while randomized-in-error participants in the usual care are often not discernable. The differential exclusion can present analytic challenges and threaten result validity.MethodsUnder the potential outcomes framework, we developed a Bayesian model that jointly identifies the randomized-in-error status and estimates the average treatment effect among participants not randomized in error. We designed simulation studies with hypothesized proportions of 5 %-15 % randomization in error to evaluate the performance of our model across scenarios where the outcomes of participants randomized in error were either measured or unmeasured. Comparisons were made to intention-to-treat and covariate-adjusted estimators.ResultsSimulation results show satisfactory performance of our proposed models, where the estimated average treatment effects among participants not randomized in error have low bias (<1 %) and close to 95 % coverage. Estimates from the alternative approaches can exhibit notable biases and low coverage.ConclusionsDifferential exclusion in pragmatic clinical trials after randomization can lead to selection bias. Under certain assumptions, Bayesian methods provide a feasible solution to jointly identify randomized-in-error status and estimate the average treatment effect among participants not randomized in error, ensuring more reliable and valid inferences about intervention effects.
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Project description:BackgroundThere are unique opportunities related to the design and conduct of pragmatic trials embedded in health insurance plans, which have longitudinal data on member/patient demographics, dates of coverage, and reimbursed medical care, including prescription drug dispensings, vaccine administrations, behavioral healthcare encounters, and some laboratory results. Such trials can be large and efficient, using these data to identify trial-eligible patients and to ascertain outcomes.MethodsWe use our experience primarily with the National Institutes of Health Pragmatic Trials Collaboratory Distributed Research Network, which comprises health plans that participate in the US Food & Drug Administration's Sentinel System, to describe lessons learned from the conduct and planning of embedded pragmatic trials.ResultsInformation is available for research on more than 75 million people with commercial or Medicare Advantage health plans. We describe three studies that have used or plan to use the Network, as well as a single health plan study, from which we glean our lessons learned.ConclusionsStudies that are conducted in health plans provide much-needed evidence to drive clinically meaningful changes in care. However, there are many unique aspects of these trials that must be considered in the planning, implementation, and analytic phases. The type of trial best suited for studies embedded in health plans will be those that require large sample sizes, simple interventions that could be disseminated through health plans, and where data available to the health plan can be leveraged. These trials hold potential for substantial long-term impact on our ability to generate evidence to improve care and population health.
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