A guide to research partnerships for pragmatic clinical trials.
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ABSTRACT:
SUBMITTER: Johnson KE
PROVIDER: S-EPMC4707716 | biostudies-literature |
REPOSITORIES: biostudies-literature
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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.
| S-EPMC9528570 | biostudies-literature
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.
| S-EPMC8543383 | biostudies-literature
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.
| S-EPMC4975022 | biostudies-literature
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.
| S-EPMC10363182 | biostudies-literature
Project description:We conducted formative research using in-depth interviews to identify preferences for and anticipated responses to receiving thank you notes and lay summaries of aggregate results among caregivers and adolescent participants of pragmatic pediatric studies conducted by the National Institute of Health-sponsored Pediatric Trials Network. We analyzed the data using qualitative thematic analysis. Nearly all participants said receiving a thank you note would make them feel valued, appreciated, and proud because they contributed to science. Similarly, nearly all participants said that receiving a lay summary of research results would make them aware of their role in improving the lives of children, feel like they are an active partner in research, and believe that researchers want to keep them informed. Participants also said that receiving a thank you note or lay summary may motivate them to participate in future research. Providing thank you notes as part of study participation should become a standard clinical trial practice, similar to the practice of providing lay summaries.
| S-EPMC8181207 | biostudies-literature
Project description:Rapidly accruing knowledge of the mutational landscape of malignant neoplasms, the increasing facility of massively parallel genomic sequencing, and the availability of drugs targeting many "driver" molecular abnormalities have spurred the oncologic community to consider how to use these new tools to improve cancer treatment. In order to assure that assignment of patients to a particular targeted treatment is likely to be beneficial to the patient, it will be necessary to conduct appropriate clinical research. It is clear that clinical (histology and stage) eligibility criteria are not sufficient for most clinical trials using agents that target mutations that are present in only a minority of patients. Recently, several clinical trial designs have been suggested to test the benefit of targeted treatment in molecular and/or clinical subgroups of patients. However, challenges remain in the implementation of such trials, including choice of assay, levels of evidence regarding gene variants, tumor heterogeneity, identifying resistance mechanisms, the necessity of screening large numbers of patients, infrastructure needs, and collaboration of investigators and industry. This article reviews current trial designs and discusses some of the considerations, advantages, and drawbacks of designing clinical trials that depend on particular molecular variants as eligibility criteria.
| S-EPMC4610034 | biostudies-literature
Project description:PurposeIn the wake of the coronavirus disease 2019 (COVID-19) pandemic, support in clinical trials by students of human medicine and related disciplines has become of even greater importance than in pre-pandemic times. Documentation in clinical trials adheres to the principles of Good Clinical Practice (GCP), and healthcare professionals involved in the conduct of clinical trials-including students-are obliged to perform documentation in accordance with GCP principles. Unprecedented challenges have arisen with regard to the appropriate training of students as training courses in presence had largely to be suspended due to social-distancing regulations during the heyday of the COVID-19 pandemic. Therefore, novel training formats and self-study training materials for students working in clinical trials are urgently warranted.MethodsTo overcome this shortcoming and to define a common quality standard, an interdisciplinary, multiprofessional (physicians, study nurses, medical students), and binational (Germany, The Netherlands) expert panel convened and devised the Students' guide to documentation in clinical trials.ResultsFollowing a brief description of the different roles in clinical trials (e.g., sponsor, (principal) investigator, monitor) and an introduction into the principles of GCP, the documentation of adverse events, concomitant medication, medical history, and quality control are comprehensively discussed. The Guide concludes with a trilingual medical dictionary (English, German, Dutch) and with recommendations of pertinent literature for further reading.ConclusionServing both as textbook for self-training and as (quick-) reference work for the daily routine, the Guide has specifically been designed to complement, but not to replace practical training courses for students. While primarily addressed at students of human medicine and related disciplines, the Guide can also be of high relevance and utility to other healthcare professionals involved in the conduct of clinical trials.
| S-EPMC9524727 | biostudies-literature
Project description:Randomized controlled trials in CKD lag in number behind those of other chronic diseases, despite the high morbidity and mortality experienced by patients with kidney disease and the exorbitant costs of their health care. Observational studies of CKD frequently yield seemingly paradoxic associations of traditional risk factors with outcomes, making it difficult to extrapolate the results of trials conducted in people with normal kidney function to patients with CKD. However, many completed trials in CKD have been limited by intermediate outcomes of unclear clinical significance or narrow eligibility criteria that limit external validity, and implementation of proven therapies remains a challenge. It is therefore imperative that the nephrology community capitalize on recent interest in novel approaches to trial design, such as pragmatic clinical trials. These trials are meant to promote research within real world settings to yield clinically relevant results with greater applicability than those of traditional trials, while maintaining many advantages, such as controlling for potential sources of bias. We provide a description of pragmatic clinical trials and a discussion of advantages, disadvantages, and practical challenges inherent to this study design, in the context of specific scientific questions relevant to patients with CKD.
| S-EPMC5042675 | biostudies-literature
Project description:ObjectivesAmong the challenges in health research is translating interventions from controlled experimental settings to clinical and community settings where chronic disease is managed daily. Pragmatic trials offer a method for testing interventions in real-world settings but are seldom used in OA research. The aim of this study was to evaluate the literature on pragmatic trials in OA research up to August 2016 in order to identify strengths and weaknesses in the design and reporting of these trials.MethodsWe used established guidelines to assess the degree to which 61 OA studies complied with pragmatic trial design and reporting. We assessed design according to the pragmatic-explanatory continuum indicator summary and reporting according to the pragmatic trials extension of the CONsolidated Standards of Reporting Trials guidelines.ResultsNone of the pragmatic trials met all 11 criteria evaluated and most of the trials met between 5 and 8 of the criteria. Criteria most often unmet pertained to practitioner expertise (by requiring specialists) and criteria most often met pertained to primary outcome analysis (by using intention-to-treat analysis).ConclusionOur results suggest a lack of highly pragmatic trials in OA research. We identify this as a point of opportunity to improve research translation, since optimizing the design and reporting of pragmatic trials can facilitate implementation of evidence-based interventions for OA care.
| S-EPMC5850497 | biostudies-literature
Project description:A randomized clinical trial is widely regarded as the most rigorous study design to determine the efficacy of intervention because spurious causality and bias associated with other experimental designs can be avoided. The purpose of this article is to provide clinicians and clinical researchers the types of randomized clinical trials used in stroke studies and to discuss the advantages and the limitations for each type of randomized stroke clinical trials.
| S-EPMC2837939 | biostudies-other