Pragmatic clinical trial

August 27, 2021: Milrinone as Compared with Dobutamine in the Treatment of Cardiogenic Shock: the DOREMI trial (Benjamin Hibbert, MD, PhD)

Posted on by terren.green@duke.edu

Speaker

Benjamin Hibbert, MD, PhD
Clinician Scientist and Interventional Cardiologist
University of Ottawa Heart Institute

Topic

Milrinone as Compared with Dobutamine in the Treatment of Cardiogenic Shock: the DOREMI trial

Keywords

Randomized pragmatic clinical trial; Milrinone; Dobutamine; CAPITAL DOREMI; Cardiogenic Shock; Evidenced based research

Key Points

  • Many therapies in cardiology and critical care are based on lower quality data, creating a large knowledge gap between the therapies used and the evidence to support their use.
  • Powerful randomized clinical trials are needed to evaluate and support the therapies used in clinical care.
  • Cardiogenic Shock, a common presentation with a mortality rate of 40% to 50%, has limited effective therapies and very limited data to support the therapies that are used.
  • The CAPITAL DOREMI trial was a double-blind randomized pragmatic clinical trial focused on the hypotheses that Milrinone would reduce the composite primary outcome compared to Dobutamine.
  • No significant differences were found between Milrinone and Dobutamine, and selection of one therapy over the other could be based on physician comfort and preference.
  • A future trial, CAPITAL DOREMI-2, will investigate the use of an inotrope vs placebo for Cardiogenic Shock.

Discussion Themes

Are medical physicians so anxious to help their desperately ill patients that they gravitate toward doing something, even when there isn’t high quality evidence that it helps?

The therapies used by critical care physicians do not always have a great foundation of evidence. As a field, critical care physicians should be developing this evidence base with pragmatic clinical trials.

Read more about Dr. Hibbert’s work with the CAPITAL DOREMI study.

Tags

#pctGR, @Collaboratory1

Grand Rounds August 27: Milrinone as Compared with Dobutamine in the Treatment of Cardiogenic Shock: the DOREMI trial

Speaker:
Benjamin Hibbert, MD, PhD
Clinician Scientist and Interventional Cardiologist
University of Ottawa Heart Institute

Topic: Milrinone as Compared with Dobutamine in the Treatment of Cardiogenic Shock: the DOREMI trial
Date: Friday, August 27, 2021, 1:00-2:00 p.m. ET

Meeting Info: To check whether you have the appropriate players installed for UCF (Universal Communications Format) rich media files, go to https://dukemed.webex.com/dukemed/systemdiagnosis.php.

To join the online meeting:
Go to https://dukemed.webex.com/dukemed/j.php?MTID=m228b52a4666c1e24046e11c01ae7bd0d

You must log in to the URL first.
Click ‘Audio Conference’
Choose ‘I will call in’, select the Toll number.
Dial in using the information from the dialog box that appears.
Be certain to use the Access Code AND the Attendee ID.

Troubleshooting:
If the URL above does not work, go to dukemed.webex.com and enter:
Meeting Number: 120 909 9614
Meeting Password: 1234

For Audio ONLY:
Call-in toll number (US/Canada): 1-650-479-3207
Access code: 120 909 9614

NOTE: For Toll-free users, the call-back (call me) services are also available.

August 2, 2021: Grand Rounds Podcast Expert Panel Discussion Summarizing the Diversity Workshop Series is Available

Posted on by terren.green@duke.edu

Robin Elizabeth Boineau, M.D., M.A.

In the latest episode of the NIH Collaboratory Grand Rounds podcast, Dr. Robin Boineau concludes the diversity workshop series, ‘Inclusion of Diverse Participants in Pragmatic Clinical Trials’ in a discussion with Dr. David Chambers, Dr. Lesley Curtis, Dr. Emily O’Brien, Dr. Wendy Weber, and Dr. Kanecia Zimmerman.

Listen to all 5 episodes in the Grand Rounds Diversity Workshop Series here:

 

June 11, 2021: The TSOS Effectiveness-Implementation Hybrid Study: Health Care System Level Theoretical Considerations & Pragmatic Trial Results (Douglas Zatzick, MD)

Posted on by terren.green@duke.edu

Speaker

Douglas Zatzick, MD
Professor of Psychiatry & Behavioral Sciences
Harborview Level I Trauma Center
University of Washington School of Medicine, Seattle

Topic

The TSOS Effectiveness-Implementation Hybrid Study: Health Care System Level Theoretical Considerations & Pragmatic Trial Results

Keywords

Pragmatic clinical trials; Implementation Science; Healthcare policy; TSOS; Effectiveness research; Longitudinal health care

Key Points

  • Combining Implementation Science theories with results from pragmatic clinical trials may be useful to target changes in healthcare policy and reduce the 17 year lag on translation from research to practice.
  • TSOS is a hybrid pragmatic clinical trial studying the effect of PTSD screening and implementation of screening procedures in healthcare systems.
  • TSOS study sites that implemented the study interventions better showed significant positive results in the experimental group as compared to the control group.
  • The results of the TSOS study have prompted the American College of Surgeons to require psychological sequelae screening in trauma centers nationally.

Discussion Themes

Healthcare systems want to see beneficial changes from pragmatic clinical trials implemented more quickly. 

To quickly implement procedures from a pragmatic clinical trial, we need a trial design that is feasible and easy to implement into clinical care and easy to incorporate into health care systems.

The focus should be on implementing the possible changes that you know you can get done in order to start a cascade of changes going forward.

Read more about TSOS.

Tags

#pctGR, @Collaboratory1

May 18, 2020: Two New Sections in the Living Textbook Describe Incentives for Sharing Data Sets and Preparing for Data Sharing

Posted on by Karen Staman

Drs. Adrian Hernandez, Greg Simon, and Rich Platt of the NIH Collaboratory have authored two new sections of the Living Textbook as part of an ongoing commitment to sharing resources and data.

  • Incentive Structure and Citations for Data Sets
    • This section calls for a revision of the appointment, promotion, and tenure (APT) process to incorporate effective data sharing into decision-making and to recognize and credit creators of data sets that gain meaningful use by others.
  • Preparing for Data Sharing
    • This section describes how to prepare for data and resource sharing throughout the embedded PCT (ePCT) lifecycle, including during grant submission, trial registration, conduct, and dissemination.

All NIH Collaboratory Trials are expected to share data and resources, such as protocols, consent documents, public use datasets, computable phenotypes, and analytic code. During the onboarding process, NIH Collaboratory Trials are given a Data and Resource Sharing Informational Document and an Onboarding Data and Resource Sharing Questionnaire to assist clinical investigators in developing data sharing plans. At closeout, NIH Collaboratory Trials are provided a Closeout Data and Resource Sharing Checklist and are expected to utilize this checklist to provide a final data share package.

For more on data sharing, see the Living Textbook chapter, Data Sharing and Embedded Research.

April 23, 2020: New Workshop Summary on the Design and Analysis of Pragmatic Clinical Trials

Posted on by Karen Staman

In 2019, NIH Health Care Systems Research Collaboratory held a comprehensive workshop to explore and discuss statistical issues encountered with embedded pragmatic clinical trials (ePCTs). The new Workshop Summary describes panel discussions with the principal investigators and statisticians of NIH Collaboratory Trials and the challenges and solutions encountered during the design and analysis of their trials.

The 4 panel discussions covered the following topics:

  • Measurement and Data: Outcomes, Exposures, and Subgroups Based on EHR Data
  • To Cluster or Not to Cluster?
  • Choosing a Parallel Group or Stepped-Wedge Design
  • Unique Complications

This Workshop Summary also provides lessons learned and recommends tools to help others design and analyze future ePCTs. For more on the design and analysis of pragmatic clinical trials, see the tools provided by the Biostatistics and Study Design Core and Living Textbook chapters on Experimental Designs and Randomization Schemes and Analysis Plans.

January 9, 2020: NIH Collaboratory Investigators Respond to Draft NIH Policy on Data Sharing

Posted on by Karen Staman

NIH Collaboratory leadership and NIH Collaboratory Trial principal investigators, along with their colleagues, responded this week to the recently released Draft NIH Policy for Data Management and Sharing and supplemental draft guidance. The draft policy proposes that applicants for research funding submit a plan describing how scientific data will be managed and shared.

“We applaud the NIH’s policy and commitment to making the results and outputs of the research it funds and conducts available to the public. We enthusiastically support data sharing and agree with the principles of this policy. However, we believe more detail is warranted about the different types of research (ie, embedded pragmatic research), the associated protections, and acceptable mechanisms for sharing data, such as public and private archives and enclaves.” —Response to Draft NIH Policy for Data Management and Sharing

The main topics covered in the response are:

  • Support for the goals of the draft data sharing policy
  • Assessing and mitigating re-identification risk
  • Protecting secondary subjects
  • Use of data enclaves
  • Crediting those who share data

Other signatories include participants in the National Academy of Medicine’s Clinical Effectiveness Research Innovation Collaborative of the Leadership Consortium for Value and Science-Driven Health Care, and leaders of the Health Care Systems Research Network.

The full letter is available for download and includes the list of signatories.

Comments are due no later than January 10, 2020, and may be submitted online.

For more on data sharing, see the Living Textbook chapter, Data Sharing and Embedded Research.

December 18, 2019: National Institute on Aging IMPACT Collaboratory Launches Grand Rounds Series and New Website

Posted on by Gina Uhlenbrauck

The National Institute on Aging (NIA) IMbedded Pragmatic Alzheimer’s disease (AD) and AD Related Dementias (AD/ADRD) Clinical Trials (IMPACT) Collaboratory has launched a new website highlighting the project.

The IMPACT Collaboratory recently invited applications for pilot grant funding for projects that aim to generate preliminary data necessary to design and conduct full-scale embedded pragmatic clinical trials of nonpharmacologic interventions for persons living with Alzheimer’s disease and related dementias and/or their caregivers. Pilot grants will help IMPACT Collaboratory’s mission to build the nation’s capacity to conduct pragmatic clinical trials of interventions embedded within health care systems for people living with dementia and their caregivers.

Since the IMPACT Collaboratory started this September, the program has been hard at work launching its new website, as well as a series of specialized Grand Rounds focused on the topic of pragmatic research in the area of Alzheimer’s disease and related dementias.

IMPACT Collaboratory Grand Rounds occur the 3rd Thursday of each month at 12 noon ET. Join the next webinar on Thursday, December 19, “Stepped Wedge Cluster Trials: What, How, And When?,” by Monica Taljaard, PhD, Senior Scientist, Ottawa Hospital Research Institute and Associate Professor, University of Ottawa from the IMPACT Collaboratory Design and Statistics Core.

Following in the footsteps of the NIH Collaboratory, the IMPACT Collaboratory has also launched a podcast series to correspond with its Grand Rounds Series. Both the Grand Rounds webinar recordings and accompanying podcasts for all the sessions are available on the website.

Those interested in getting updates about the IMPACT Collaboratory can also join the program’s mailing list .

October 15, 2019: Postdoctoral Fellowship in the Ethics and Regulatory Aspects of Pragmatic Clinical Trials at Johns Hopkins

Posted on by Gina Uhlenbrauck

The Johns Hopkins Berman Institute of Bioethics invites applications for a Postdoctoral Fellowship in the Ethics and Regulatory Aspects of Pragmatic Clinical Trials. This position includes pursuing independent research, working alongside faculty members involved with the ethics and regulatory aspects of large-scale pragmatic clinical trials (PCTs), and participating in the Hecht-Levi Postdoctoral Fellowship in Bioethics. The postdoctoral fellow is expected to pursue one or more projects addressing the ethics and regulatory aspects of PCTs in collaboration with Berman Institute faculty members. The Fellow will actively engage with the Ethics and Regulatory Core of the NIH Health Care Systems Research Collaboratory and the Pragmatic and Implementation Studies for the Management of Pain to Reduce Opioid Prescribing (PRISM) Resource Coordinating Center.

Qualifications: Applications are welcome from candidates who will have an MD, PhD, or JD or their equivalent by the start date of the fellowship. Applicants should not have completed their terminal degree more than 3 years before the start date of the appointment. Physicians should not have completed a formal residency training program more than 3 years by the start date of the appointment.

Start date: September 1, 2020.

Terms of Appointment: The fellowship is guaranteed for 1 year with the expectation of a second year of funding, contingent on review. Applicants may not be employed by another institution and are expected to be in residence for the duration of the appointment.

How to apply: For details on how to apply see: https://bioethics.jhu.edu/education-training/fellowships/#fellowship-pragmatic. Applications must be submitted by December 16, 2019.

August 2, 2019: AI and the Future of Psychiatry (Murali Doraiswamy, MBBS)

Posted on by Gina Uhlenbrauck

Speaker

Murali Doraiswamy, MBBS
Professor of Psychiatry and Behavioral Sciences
Duke School of Medicine

Topic

AI and the Future of Psychiatry

Keywords

Artificial intelligence; Machine learning; Psychiatry; Ethical adoption of technologies; Mental health; Wearables; Mobile health

Key Points

  • There is growing evidence from randomized controlled trials of the efficacy of using digital tools in mental health diagnosis and treatment.
  • Could artificial intelligence (AI) and machine learning technologies be used to:
    • Reduce the stigma associated with mental health treatment?
    • Predict the risk for future suicide?
    • Detect Alzheimer’s years before diagnosis?
  • Categories of AI applications include low-risk apps that measure but do not diagnose, and apps used in diagnosis or treatment that must meet the same high standards of evidence as medications.
  • Clinicians still struggle with how to integrate patient data from wearable devices. AI technology might help if it could be used to synthesize the data into a risk profile for an individual.

Discussion Themes

What are the roles of stress, exercise, and sleep in mental health, and can autonomic data from wearables help explain the variance in mental health symptoms?

To develop evidence thresholds for AI, we need larger scale public-private partnerships as well as pragmatic trials addressing key clinical questions.

Read more from Dr. Doraiswamy in How to Use Technology Ethically to Increase Access to Mental Healthcare.
Tags

#AI, #pctGR, @Collaboratory1