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Digital Health Technologies and Innovations
Published in Kelly H. Zou, Lobna A. Salem, Amrit Ray, Real-World Evidence in a Patient-Centric Digital Era, 2023
Kelly H. Zou, Mina B. Riad, Shaantanu Donde, Joan van der Horn, Tarek A. Hassan
Third, Pragmatic Clinical Trial (PCT) is those clinical trials for which the hypothesis and study design are developed specifically to answer the questions faced by decision makers, and they are either pragmatic or practical. The characteristic features of PCTs are that they (1) select clinically relevant alternative interventions to compare, (2) include a diverse population of study participants, (3) recruit participants from heterogeneous practice settings, and (4) collect data on a broad range of health outcomes. See: Tunis et al. (2003).
Real-World Data and Real-World Evidence
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Pragmatic clinical trial (PCT) aims to estimate the effectiveness of an intervention in a setting close to routine clinical practice, which is a study design between RCT and observational study. Common design types for PCTs include17: (i) individual PCTs, which use individuals as observation units of trials; (ii) cluster PCTs, which use clusters as grouped units. Clusters in PCTs can be families, clinics, hospitals, or schools; (iii) stepped wedge PCTs is a special type of cluster PCTs, in which groups were randomly assigned to receive the intervention with different starting times. In stepped wedge PCTs, each group started the experimental intervention at different stage, and eventually all groups received the intervention. The type of design should be chosen on the basis of research purpose, research questions and research conditions. From the perspective of simpleness and efficiency, we give priority to individual PCTs.
Harnessing Real-World Data to Inform Platform Trial Design
Published in Zoran Antonijevic, Robert A. Beckman, Platform Trial Designs in Drug Development, 2018
Daphne Guinn, Subha Madhavan, Robert A. Beckman
Currently, many groups are interested in creating new sources of RWD to inform clinical practice and drug development. This may be through creation of registries, such as the RISE (Rheumatology Informatics System for Effectiveness) registry created by the American College of Rheumatology and Amgen, to facilitate research through the collection of health information captured from the EHR to support clinical care and payment.17,18 The American Society of Clinical Oncology also has developed an innovative approach to promote the use of big data to improve health care through the creation of ASCO CancerLinQ19. CancerLinQ collects information from participating physicians’ EHR systems to support clinical decision-making and answer research questions.19,20 Another method for RWD collection is to perform a prospective trial using existing infrastructure associated with routine clinical care or a registry system. These pragmatic trials can be an administrative and cost efficient way to perform clinical research and produce RWD.21 The Thrombus Aspiration in ST-Elevation Myocardial Infarction in Scandinavia (TASTE) trial is an example of a trial that used a registry platform to efficiently randomize patients and collect data for a large multicenter, prospective, open-label trial.22 The trial was able to collect information about thrombus aspiration, where previous underpowered traditional trials had failed.23 Another example is the PCORNET ADAPTABLE (Aspirin Dosing: A Patient-centric Trial Assessing Benefits and Long-term Effectiveness) trial, which is assessing two different daily doses of aspirin for prevention of heart attack or stroke24. This trial is patient-focused and has used patient feedback to develop all aspects of the design and implementation. Pragmatic clinical trial designs could be useful in generating data for label expansion or modification, when traditional trials cannot be efficiently executed.5
Progressing from “Whether to” to “How to” Conduct Pragmatic Trials
Published in The American Journal of Bioethics, 2023
Jonathan D. Casey, Todd W. Rice, Matthew W. Semler
In their manuscript, Morain and Largent address the ways in which the traditional model of a researcher-participant dyad is inapplicable to pragmatic clinical trials (Morain and Largent 2023). Through specific case studies, they highlight six key differences in the relationship between the researcher and the patient in traditional, explanatory trials vs pragmatic trials: (1) pragmatic clinical trials may be conducted with a waiver or alteration of consent, (2) pragmatic researchers may not have direct interaction with study participants, (3) addressing individual-level findings in a pragmatic clinical trial may be prohibitively costly to institutions and research enterprises, (4) pragmatic clinical trials are embedded within real-world settings where gaps in care are common, (5) individual-level findings in pragmatic clinical trials may have implications for others receiving care in that setting, and (6) the tools used to address individual-level findings in explanatory trials (e.g., prescribing a new medication) may not be available in pragmatic clinical trials. The authors conclude that the traditional paradigm used for researcher-patient dyads cannot be applied to pragmatic trials. We agree with the authors’ arguments and their conclusion that we must look beyond the researcher-participant dyad and explore the extent to which the institutions in which pragmatic clinical trials occur should bear additional responsibilities. As institutional leaders, we would welcome further scholarship and guidance in these areas.
Personalizing therapy selection in inflammatory bowel disease
Published in Expert Review of Clinical Immunology, 2023
For effective IBD care, we need to treat the patient as dynamic entity constantly interacting their internal and external environment. In order to accomplish this goal of personalizing IBD management, several steps would be important: Expand study populations beyond the current narrow pools (white, US and European ancestry).A holistic approach by funders to all aspects of IBD pathogenesis, including the exposome, diet, viruses and epithelial cell dysfunction.Pragmatic clinical trial design that allows subgroups to be selected a priori for one therapy over another, to generate subgroup-specific efficacy and safety data.Global collaboration on outcomes research that encompasses diverse patient populations.Equitable access to new technologies such as machine learning models that may be able to predict disease expression in at risk patients.
Patient Ineligibility as a Barrier to Participation in Clinical Trials
Published in The American Journal of Bioethics, 2023
Misalignment between clinical and research populations carries a deeper risk than slowing recruitment efforts: Clinicians and researchers might feel like they are pursuing different goals. Garland and colleagues dismiss as a “bad reason” for nonparticipation a concern that “Clinicians might … believe they or their current patients will not benefit from the research themselves.” However, this concern has legitimacy to the extent that clinicians and researchers see different patient populations and focus on different questions. It will be easier to convince clinicians to participate in a pragmatic clinical trial if the project has clear relevance to the clinician’s caseload.