pragmatic clinical trials

May 13, 2022: Ethics and Collateral Findings in Pragmatic Clinical Trials: Implications of a Multi-Method Exploration

Posted on by Elizabeth Mccamic

Speaker

Jeremy Sugarman, MD, MPH, MA
Deputy Director for Medicine
Harvey M. Meyerhoff Professor of Bioethics and Medicine
Berman Institute of Bioethics
Johns Hopkins University

Stephanie Morain, PhD, MPH
Assistant Professor
Berman Institute of Bioethics
Johns Hopkins University

Juli Bollinger, MS
Research Scientist
Berman Institute of Bioethics
Johns Hopkins University

Kevin P. Weinfurt, PhD
Professor and Vice Chair of Research
Department of Population Health Sciences
Duke University School of Medicine

 

 

Keywords

PragmaticClinicalTrials; Ethics

 

Key Points

  • There are many ethical complexities for pragmatic clinical trials, including consent, gatekeepers/relationship to patients, privacy, scale and incidental and secondary findings that may have implications for health, but were not generated to address the PCT’s primary research questions.
  • MOTIFS: Management of Trial Incidental Findings study aimed to asses gaps in current guidance for managing incidental findings that are relevant to PCTs by holding stakeholder interviews and focus groups.
  • In interviews with health care provider stakeholders, MOTIFS investigators learned that PCT-CFs are not on peoples’ radar, but there is a shared belief anticipation/planning is critical. A lack of shared language and not having clear “ownership” further complicates communicating about PCT-CFs.
  • Focus groups were held with patient participants from cities in three different geographic areas of the U.S. Participants had mixed reactions to learning about possible incidental findings, preferences for who and how the findings were communicated varied, but the timeline for delivery of the finding was important. Communication should include clear action steps and not have unneeded details, and a multi-modal approach of communicating was preferred.
  • Policy recommendations include PCT-CFs should anticipate and plan for incidental findings before study implementation; clincians should be prepared to discuss PCT-CFs with patients and for subsequent management; multiple modes of communication with patients is preferred.

Discussion Themes

– For at least some patients, the value of the information went beyond actionability–they wanted the information, even if not of immediate clinical relevance, as it could influence their future decisions about where/from whom to seek care.

– The study did not factor for race and ethnicity. Even with the large sample size, the experiment was a complex multifactorial design, and so adding new variables implies a multiplicative increase in study size.

– Incidental findings can be introduced as a possibility in the consent process of PCT-CFs when there is a full IC process.

Read more: 1) Ethics & Collateral Findings in Pragmatic Clinical Trials 2) Stakeholder perspectives regarding pragmatic clinical trial collateral findings 3) Patients’ Views About the Disclosure of Collateral Findings in Pragmatic Clinical Trials: a Focus Group Study 4) Patients’ Reactions to Letters Communicating Collateral Findings of Pragmatic Clinical Trials: a National Web-Based Survey.

Tags

#pctGR, @Collaboratory1

May 17, 2022: NIH and Program Leaders Discuss PRISM’s Role in NIH HEAL Initiative

Posted on by Elizabeth Mccamic

In an interview at the NIH Pragmatic Trials Collaboratory Steering Committee’s annual meeting in April, Dr. Rebecca Baker, Dr. Adrian Hernandez, and Dr. Wendy Weber discussed how the PRISM program and the NIH HEAL Initiative are working together to answer questions about pain management and the national opioid public health crisis.

Baker is the director of the NIH HEAL Initiative, and Hernandez is co-principal investigator of the NIH Pragmatic Trials Collaboratory Coordinating Center, for which Weber is the NIH Project Officer.

“I think the PRISM program is a great piece of the NIH HEAL initiative,” said Weber. “The HEAL initiative has everything from basic science all the way up to what PRISM is doing, which is taking these effective interventions and actually figuring out how do you put them into the healthcare system.”

The Helping to End Addiction Long-term Initiative (NIH HEAL Initiative) is an aggressive, trans-agency effort to speed scientific solutions to stem the national opioid public health crisis. It has launched more than 600 different research projects, totaling a more than $2 billion investment.

The PRISM program (Pragmatic and Implementation Studies for the Management of Pain to Reduce Opioid Prescribing) is a component of the NIH HEAL Initiative, and the NIH Pragmatic Trials Collaboratory serves as the PRISM Resource Coordinating Center, supporting large-scale embedded pragmatic clinical trials to determine the effectiveness of non-opioid interventions for pain.

“When we talk about learning in the healthcare system, this is an area that we see is going to have continuous learning, and so, as there are new options for pain management, how do you get them implemented into practice, what are the gaps, what else is needed for these areas,” said Hernandez. “This becomes a hallmark of what we are aiming to do for the NIH Pragmatic Trials Collaboratory.”

PRISM and the NIH HEAL Initiative have developed common measures across studies in order to create a HEAL data ecosystem that will allow investigators to do meta-analyses with shared outcome measures across studies.

“One thing that we’ve learned through HEAL, especially in our focus on pain, but also for addiction, is that these conditions don’t exist in isolation, so people rarely have just pain—they have pain plus other health conditions,” said Baker. “Being able to study that in a pragmatic way and iterate around different types of patients enriches the knowledge that comes out of the study and then enables clinicians to provide great care, too.”

Through key partnerships with the NIH Pragmatic Trials Collaboratory, PRISM, and other federal partners, the NIH HEAL Initiative is now integrating the experience of patients at the outset of studies and focusing on health disparities and health inequity issues.

“If we just conduct research, publish the papers, go to academic meetings and discuss them, we’re not going to have the impact we’re hoping to have through this initiative, and so the next phase is continuing to expand our research into these domains that we weren’t able to start with at the beginning but also continue to build those partnerships for dissemination and implementation,” said Baker.

View the full interview.

See the complete materials from the 2022 Steering Committee meeting.

December 3, 2021: Primary Care-Based Behavioral Treatment for Long Term Opioid Users with Chronic Pain: Primary Results and Lessons Learned from the PPACT Pragmatic Trial (Lynn DeBar, PhD MPH)

Posted on by terren.green@duke.edu

Speaker

Lynn DeBar, PhD MPH
Senior Scientist
Kaiser Permanente Washington Health Research Institute
Seattle, WA

Keywords

PPACT, Chronic pain, Opioid use, Patient reported outcome data, Patient-centered research, Cognitive behavioral therapy

Key Points

  • Chronic Pain is responsible for far more years lived with disability than a large number of other diseases and injuries, such as COPD, diabetes, dementia, stroke and others, combined.
  • PPACT was a cluster randomized study aimed at integrating interdisciplinary pain management methods into primary care to improve care for patients with chronic pain.
  • Participants had been prescribed long-term opioids for mixed chronic pain conditions.
  • Study intervention lasted 12 weeks and included cognitive behavioral therapy, yoga-based exercises, physical therapy, a medication review, and support from a primary care provider.
  • Participants in the treatment group of the PPACT study showed a modest reduction of pain that was sustained over a 12 month period.
  • The PPACT intervention was cost effective versus usual treatment methods.

Discussion Themes

An in-person enhanced enrollment session was conducted to give potential subjects a comprehensive overview of the study procedures. This enrollment session led to fewer people enrolling in the study, but of those that did, more completed the study.

The key to successful CBT is an interactive patient-centered approach.

 

Read more about the PPACT study.

 

Tags

#pctGR, @Collaboratory1

Podcast July 8, 2020: Reflection on Advances at the Intersection of Digital Health, Electronic Health Records, and Pragmatic Clinical Trials (Wendy Weber, ND, PhD, MPH; Lesley Curtis, PhD; Patrick Heagerty, PhD; Keith Marsolo, PhD)

Posted on by Gina Uhlenbrauck

This episode of the NIH Collaboratory Grand Rounds Podcast, led by Wendy Weber, ND, PhD, MPH, reflects on the Grand Rounds EHR workshop series, “Advances at the Intersection of Digital Health, Electronic Health Records, and Pragmatic Clinical Trials.” The podcast features panelists Lesley Curtis, PhD, Keith Marsolo, PhD and Patrick Heagerty, PhD, who all moderated webinars included in this Grand Rounds series.

Click on the recording below to listen to the podcast.

Want to hear more? Access the full Grand Rounds presentations for this series here.

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Read the transcript.

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January 24, 2020: Special Grand Rounds Series Begins January 31st With ‘Pragmatic Clinical Trials: How Do I Start?’

Posted on by Gina Uhlenbrauck

Join us Friday, January 31st, for “Pragmatic Clinical Trials: How Do I Start?”—the first in our special 5-part Grand Rounds series focused on the Living Textbook. NIH Collaboratory investigators Dr. Greg Simon and Dr. Lesley Curtis will offer guidance on what researchers need to begin an embedded pragmatic clinical trial, including:

  • Developing your research question
  • Establishing close partnerships with participating healthcare system leaders and staff
  • Putting together your trial team

Visit our events calendar here for details about how to join this webinar. See below for the full schedule of sessions and a special message from Dr. Kevin Weinfurt.

Full Schedule of sessions

Title Speakers Date
Pragmatic Clinical Trials:
How Do I Start?		 Greg Simon, MD, MPH, KPWHRI

Lesley Curtis, PhD, Duke University

 January 31, 2020
Preparing for the Unknown: Conducting Pragmatic Research in Real-World Contexts Jerry Jarvik, MD, MPH, University of Washington

Vince Mor, PhD, Brown University

Leah Tuzzio, MPH, KPWHRI

 February 28, 2020
Tips for Putting Together a Successful PCT Grant Application Wendy Weber, ND, PhD, MPH, NCCIH March 27, 2020
Choosing What to Measure
and Making It Happen: Your Keys to Pragmatic Trial Success		 Rachel Richesson, PhD, MPH, Duke University

Emily O’Brien, PhD, FAHA, Duke University

 May 1, 2020
Demystifying Biostatistical Concepts for Embedded Pragmatic Clinical Trials Liz Turner, PhD, Duke University

Patrick Heagerty, PhD, University of Washington

David Murray, PhD, NIH

 June 19, 2020

 

December 20, 2019: Embedded Pragmatic Clinical Trials: From Idea to Funding to Implementation: A Living Textbook Grand Rounds Series

Posted on by Gina Uhlenbrauck

Coming in 2020, the NIH Collaboratory will host a 5-part Grand Rounds Series focused on the Living Textbook. The series will feature Collaboratory expert speakers guiding the audience through the life cycle of an ePCT by way of the textbook—from idea to funding to implementation.

The series begins January 31 with “Pragmatic Clinical Trials: How Do I Start?” and will end June 19 with “Demystifying Biostatistical Concepts for Embedded Pragmatic Clinical Trials.”

See below for the full schedule of sessions and a special message from Dr. Kevin Weinfurt.

Full Schedule of sessions

Title Speakers Date
Pragmatic Clinical Trials:
How Do I Start?		 Greg Simon, MD, MPH, KPWHRI

Lesley Curtis, PhD, Duke University

 January 31, 2020
Preparing for the Unknown: Conducting Pragmatic Research in Real-World Contexts Jerry Jarvik, MD, MPH, University of Washington

Vince Mor, PhD, Brown University

Leah Tuzzio, MPH, KPWHRI

 February 28, 2020
Tips for Putting Together a Successful PCT Grant Application Wendy Weber, ND, PhD, MPH, NCCIH March 27, 2020
Choosing What to Measure
and Making It Happen: Your Keys to Pragmatic Trial Success		 Rachel Richesson, PhD, MPH, Duke University

Emily O’Brien, PhD, FAHA, Duke University

 May 1, 2020
Demystifying Biostatistical Concepts for Embedded Pragmatic Clinical Trials Liz Turner, PhD, Duke University

Patrick Heagerty, PhD, University of Washington

David Murray, PhD, NIH

 June 19, 2020

 

We hope you will join us to learn more about the fundamentals for designing and launching a successful ePCT. Please download the flyer for the Living Textbook Grand Rounds series and share with your colleagues and institution.

IMPACT Collaboratory: Are You Ready for a Pragmatic Trial? The RAPT Model and Implementation Considerations

Speakers:

Rosa Baier, MPH
Associate Director, Center for Long-Term Care Quality & Innovation
Associate Professor, Health Sciences, Policy & Practice
Brown University School of Medicine

Eric Jutkowitz, PhD
Assistant Professor
Brown University School of Public Health

Laura Gitlin, PhD, FGSA, FAAN
Dean, Distinguished Professor, Drexel University
Adjunt Professor, Johns Hopkins University

Read more about the RAPT Model.

Zoom Conferencing

Join from PC, Mac, iOS or Android: https://hebrewseniorlife.zoom.us/j/5479652617 

Dial-In: +1 646 876 9923 (US Toll) or +1 669 900 6833 (US Toll)

Meeting ID:  547 965 2617

January 11, 2019: FDA Releases Framework for Evaluating the Use of Real-World Evidence

Posted on by Karen Staman

To help fulfill the requirements of the 21st Century Cures Act by accelerating medical product development and fostering innovation and advances in medicine, FDA recently created a framework for evaluating the use of real-world evidence. The framework is intended to help evaluate trials that use real-world data for the creation of real-world evidence.

  • Real-world data: routinely collected information about a person’s health status in the electronic health record, claims, registries, and other sources, including patient-generated sources.
  • Real-world evidence: reliable, clinical information derived from real-world data about risks, benefits, and burdens of therapies.

This framework will apply to various pragmatic clinical trials embedded in health care systems and conducted as part of routine care (and will not apply to more traditional clinical trials conducted parallel to care).

Three main considerations are included in the framework:

  1. Will the real-world data be fit for use (do they reliably and adequately represent the concept)?
  2. Will the evidence generated by the trial provide adequate evidence to help answer regulatory questions?
  3. Will the conduct of the study meet FDA regulatory requirements?

December 11, 2018: Two New NIH Funding Opportunity Announcements for Pragmatic Trials Address the Opioid Crisis

Posted on by Karen Staman

The NIH has announced two new funding opportunity announcements (FOAs) for 7 or more embedded pragmatic clinical trials that address pain management and the opioid crisis. These projects will become part of the NIH Health Care Systems Research Collaboratory as phased UG3/UH3 cooperative research.

The two announcements are:

The announcements are part of the NIH Heal (Helping to End Addiction Long-term) Initiative, which was created in April 2018 in an effort to speed scientific solutions for addressing the national opioid public health crisis.

November 13, 2018: Summary of Workshop on Pragmatic Trials of Therapeutic A vs B Interventions Now Available

Posted on by Karen Staman

The NIH Collaboratory recently convened a workshop to explore embedded pragmatic clinical trials comparing two or more therapeutic medical interventions. These “A vs B” trials are meant to test existing, viable treatment alternatives where there is uncertainty about which treatment is best in which populations. There are unique barriers that make these types of pragmatic trials especially challenging to implement. For the workshop, a panel of experts gathered to discuss challenges and solutions regarding partnering with healthcare systems to conduct the trials, unique legal and ethical issues, and design and operational considerations. The summary of the workshop is now available: Workshop Summary: Embedded Pragmatic Clinical Trials of Therapeutic A vs. B Interventions

 

Additional Resources:

Embedded pragmatic clinical trials of therapeutic A vs. B interventions workshop videocast.