scholarly journals Pragmatic Clinical Trials—Ready for Prime Time?

2021 ◽  
Vol 4 (12) ◽  
pp. e2140212
Author(s):  
Mahmoud Al Rifai ◽  
Dipti Itchhaporia ◽  
Salim S. Virani
Keyword(s):  
Clinical Trials ◽  
Prime Time ◽  
Pragmatic Clinical Trials

scholarly journals Improving pragmatic clinical trial design using real-world data

2019 ◽  
Vol 16 (3) ◽  
pp. 273-282 ◽  
Author(s):  
Susan M Shortreed ◽  
Carolyn M Rutter ◽  
Andrea J Cook ◽  
Gregory E Simon
Keyword(s):  
Clinical Trials ◽  
Trial Design ◽  
Suicide Attempts ◽  
Real World Data ◽  
Electronic Health Record Data ◽  
Pragmatic Clinical Trial ◽  
Pragmatic Clinical Trials ◽  
Sample Size Calculations ◽  
Record Data ◽  
Electronic Health

Background Pragmatic clinical trials often use automated data sources such as electronic health records, claims, or registries to identify eligible individuals and collect outcome information. A specific advantage that this automated data collection often yields is having data on potential participants when design decisions are being made. We outline how this data can be used to inform trial design. Methods Our work is motivated by a pragmatic clinical trial evaluating the impact of suicide-prevention outreach interventions on fatal and non-fatal suicide attempts in the 18 months after randomization. We illustrate our recommended approaches for designing pragmatic clinical trials using historical data from the health systems participating in this study. Specifically, we illustrate how electronic health record data can be used to inform the selection of trial eligibility requirements, to estimate the distribution of participant characteristics over the course of the trial, and to conduct power and sample size calculations. Results Data from 122,873 people with patient health questionnaire (PHQ) responses, recorded in their electronic health records between 1 July 2010 and 31 March 2012, were used to show that the suicide attempt rate in the 18 months following completion of the questionnaire varies by response to item nine of the PHQ. We estimated that the proportion of individuals with a prior recorded elevated PHQ (i.e. history of suicidal ideation) would decrease from approximately 50% at the beginning of a trial to about 5%, 50 weeks later. Using electronic health record data, we conducted simulations to estimate the power to detect a 25% reduction in suicide attempts. Simulation-based power calculations estimated that randomizing 8000 participants per randomization arm would allow 90% power to detect a 25% reduction in the suicide attempt rate in the intervention arm compared to usual care at an alpha rate of 0.05. Conclusions Historical data can be used to inform the design of pragmatic clinical trials, a strength of trials that use automated data collection for randomizing participants and assessing outcomes. In particular, realistic sample size calculations can be conducted using real-world data from the health systems in which the trial will be conducted. Data-informed trial design should yield more realistic estimates of statistical power and maximize efficiency of trial recruitment.

scholarly journals Accounting for quality improvement during the conduct of embedded pragmatic clinical trials within healthcare systems: NIH Collaboratory case studies

Healthcare ◽  
2021 ◽  
Vol 8 ◽  
pp. 100432
Author(s):  
Leah Tuzzio ◽  
Catherine M. Meyers ◽  
Laura M. Dember ◽  
Corita R. Grudzen ◽  
Edward R. Melnick ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Quality Improvement ◽  
Case Studies ◽  
Healthcare Systems ◽  
Pragmatic Clinical Trials

Optimizing the Impact of Pragmatic Clinical Trials for Veteran and Military Populations: Lessons From the Pain Management Collaboratory

2021 ◽  
Author(s):  
Joseph Ali ◽  
Margaret Antonelli ◽  
Lori Bastian ◽  
William Becker ◽  
Cynthia A Brandt ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Pain Management ◽  
Pragmatic Trial ◽  
Dissemination And Implementation ◽  
Large Network ◽  
Military Health ◽  
Healthcare Needs ◽  
Unmet Healthcare Needs ◽  
Pragmatic Clinical Trials ◽  
The Impact

ABSTRACT Pragmatic clinical trials (PCTs) are well-suited to address unmet healthcare needs, such as those arising from the dual public health crises of chronic pain and opioid misuse, recently exacerbated by the COVID-19 pandemic. These overlapping epidemics have complex, multifactorial etiologies, and PCTs can be used to investigate the effectiveness of integrated therapies that are currently available but underused. Yet individual pragmatic studies can be limited in their reach because of existing structural and cultural barriers to dissemination and implementation. The National Institutes of Health, Department of Defense, and Department of Veterans Affairs formed an interagency research partnership, the Pain Management Collaboratory. The partnership combines pragmatic trial design with collaborative tools and relationship building within a large network to advance the science and impact of nonpharmacological approaches and integrated models of care for the management of pain and common co-occurring conditions. The Pain Management Collaboratory team supports 11 large-scale, multisite PCTs in veteran and military health systems with a focus on team science with the shared aim that the “whole is greater than the sum of the parts.” Herein, we describe this integrated approach and lessons learned, including incentivizing all parties; proactively offering frequent opportunities for problem-solving; engaging stakeholders during all stages of research; and navigating competing research priorities. We also articulate several specific strategies and their practical implications for advancing pain management in active clinical, “real-world,” settings.

scholarly journals Data monitoring committees for pragmatic clinical trials

2015 ◽  
Vol 12 (5) ◽  
pp. 530-536 ◽  
Author(s):  
Susan S Ellenberg ◽  
Richard Culbertson ◽  
Daniel L Gillen ◽  
Steven Goodman ◽  
Suzanne Schrandt ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Data Monitoring ◽  
Pragmatic Clinical Trials

Use of pragmatic clinical trials in reimbursement decisions

2021 ◽  
pp. 455-460
Author(s):  
Catherine Copley-Merriman ◽  
Margaret Mordin
Keyword(s):  
Clinical Trials ◽  
Pragmatic Clinical Trials

Pragmatic Clinical Trials in Osteoporosis

2019 ◽  
Vol 17 (6) ◽  
pp. 521-526 ◽  
Author(s):  
Giovanni Adami ◽  
Kenneth G. Saag ◽  
Maria I. Danila
Keyword(s):  
Clinical Trials ◽  
Pragmatic Clinical Trials

scholarly journals Statistical Considerations for Embedded Pragmatic Clinical Trials in People Living with Dementia

2020 ◽  
Vol 68 (S2) ◽  
Author(s):  
Heather G. Allore ◽  
Keith S. Goldfeld ◽  
Roee Gutman ◽  
Fan Li ◽  
Joan K. Monin ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Pragmatic Clinical Trials

scholarly journals Poor implementation of the EU clinical trial regulation is a major threat for pragmatic trials in European countries

2020 ◽  
Vol 29 ◽  
Author(s):  
Giovanni Ostuzzi ◽  
Chiara Gastaldon ◽  
Carlo Petrini ◽  
Brian Godman ◽  
Corrado Barbui
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
European Countries ◽  
Pragmatic Trials ◽  
Research And Practice ◽  
Major Threat ◽  
Pragmatic Clinical Trials ◽  
Clinical Trial Regulation ◽  
The Eu

Abstract The principle of pragmatism in clinical trials has been broadly recognised as a way to close the gap between research and practice. In this contribution, we argue that the conduct of pragmatic clinical trials in Europe may be hampered by poor implementation of current European Union's Clinical Trial Regulation No. 536/2014.

scholarly journals Addressing guideline and policy changes during pragmatic clinical trials

2019 ◽  
Vol 16 (4) ◽  
pp. 431-437 ◽  
Author(s):  
Lesley H Curtis ◽  
Laura M Dember ◽  
Miguel A Vazquez ◽  
David Murray ◽  
Lynn DeBar ◽  
...  
Keyword(s):  
Public Health ◽  
Clinical Trials ◽  
Large Scale ◽  
Well Being ◽  
Clinical Equipoise ◽  
Opioid Use ◽  
Pragmatic Clinical Trials ◽  
The Face ◽  
Stage Renal Disease ◽  
End Stage

While conducting a set of large-scale multi-site pragmatic clinical trials involving high-impact public health issues such as end-stage renal disease, opioid use, and colorectal cancer, there were substantial changes to both policies and guidelines relevant to the trials. These external changes gave rise to unexpected challenges for the trials, including decisions regarding how to respond to new clinical practice guidelines, increased difficulty in implementing trial interventions, achieving separation between treatment groups, and differential responses across sites. In this article, we describe these challenges and the approaches used to address them. When deliberating appropriate action in the face of external changes during a pragmatic clinical trial, we recommend considering the well-being of the participants, clinical equipoise, and the strength and quality of the evidence associated with the change; involving those charged with data and safety monitoring; and where possible, planning for potential external changes as the trial is being designed. Any solution must balance the primary obligation to protect the well-being of participants with the secondary obligation to protect the integrity of the trial in order to gain meaningful answers to important public health questions.

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