NIH Collaboratory One-day Workshop

Changing Trials for Changing Times:
Essentials of Embedded Pragmatic Clinical Trials Workshop
February 20, 2023

Program Description

Recent rapid changes in the challenges facing healthcare have made it even more critical to have a highly efficient mechanism for clinical research that can deliver much-needed evidence faster and with minimal additional resources. This workshop introduces concepts in the design, conduct, and implementation of embedded pragmatic clinical trials (ePCTs), with a particular focus on methods relevant to health services researchers. ePCTs are randomized trials conducted within health care systems and use streamlined procedures and existing infrastructure to answer important medical questions for patients, providers, and health system leaders. Such trials have the potential to inform policy and practice with broadly generalizable, high-quality evidence at lower cost and greater efficiency compared with traditional explanatory clinical trials. The workshop will provide an introduction to the investigative opportunities for embedded health systems research, along with strategies for conducting clinical trials that provide real-world evidence necessary to inform both practice and policy. Firsthand ePCT experiences and case studies from the NIH Pragmatic Trials Collaboratory will support and illustrate the topics presented and demonstrate the importance of leveraging networks throughout the course of ePCTs.

Learning Objectives

  1. To clarify the definition of ePCTs and explain their utility.
  2. To introduce attendees to the unique characteristics and challenges of designing, conducting, and implementing ePCTs within diverse health care systems.
  3. To increase the capacity of health services researchers to address important clinical questions with ePCTs and to leverage networks and their resources to support trial success.

Agenda

DURATION

AGENDA TOPIC

SPEAKERS

GOALS

8:00-8:45 am
(45 min)

What are Embedded Pragmatic Clinical Trials (ePCTs)?

Wendy Weber

  • Welcome and introduction of agenda and objectives
  • Identify key considerations in the design and conduct of ePCTs and how they differ from explanatory trials
  • Learn about the advantages and disadvantages of ePCTs, when a pragmatic approach can be used to answer the research question

8:45-9:05 am
(20 min)

Engaging Stakeholders & Aligning with Health System Partners Emily O’Brien
  • Describe the breadth of stakeholders to engage as partners and approaches for engaging them through all phases of the study
  • Understand the real-world priorities and perspectives of healthcare system leaders and how to obtain their support
  • Identify engagement practices to obtain patient and community perspectives
  • Highlight challenges of partnering with diverse healthcare systems

9:05-9:30 am
(25 mins)

Objectives and Trial Design: An Overview of Hybrid Designs

Patrick Heagerty

  • Overview of the 3 types of effectiveness-implementation hybrid trial designs and when they may be appropriate for ePCTs

9:30-10:00 am
(30 min)

Measuring Outcomes Emily O’Brien
  • Describe methods for measuring outcomes using data sources such as electronic health records (EHRs) and patient-reported outcomes (PROs)
  • Discuss the integration of a health equity lens in evaluating outcomes

10:00-10:30 am
(30 min)

ePCT Design

Patrick Heagerty

  • Learn about cluster randomized and stepped-wedge study designs 

10:30-10:45 am
(15 min)

Break  
  • Networking among attendees and presenters

10:45-11:15 am
(30 min)

ePCT Analysis

Patrick Heagerty

 

  • Recognize the analytical challenges and trade-offs of pragmatic study designs, focusing on what principal investigators (PIs) need to know

11:15 am- 12:15 pm
(60 min)

ePCTs in Context: Panel Discussion with Collaboratory Demonstration Project PIs

Moderator:
Kevin Weinfurt

Panel:
Arne Beck (GGC4H)
Michael Ho (Nudge)Robert Toto (ICD-Pieces)Angelo Volandes (ACP PEACE)
  • Introduce PIs of 4 ongoing ePCTs to reflect on the morning topics and discuss challenges, solutions, lessons learned, and how they leveraged networks and resources to support trial success
  • Q & A with attendees

12:15-1:15 pm
(60 min)

Lunch  
  • Networking among attendees and presenters

1:15-1:45 pm
(30 min)

Pilot & Feasibility Testing  Wendy Weber
  • Identify approaches to evaluating the capabilities of the partner healthcare system and testing key elements of various types of interventions 

1:45-2:15 pm
(30 min)

Ethical & Regulatory Oversight Considerations

Stephanie Morain

  • Learn about the regulatory and ethical challenges of conducting ePCTs
  • Discuss unique needs of historically underrepresented and mistreated groups

2:15-3:15 pm
(60 min)

ePCTs in Context: Panel Discussion with Collaboratory Demonstration Project PIs

Moderator:
Kevin Weinfurt

Panel:
Arne Beck (GGC4H)
Michael Ho (Nudge)Robert Toto (ICD-Pieces)Angelo Volandes (ACP PEACE)
  • Introduce PIs of 4 ongoing ePCTs to reflect on the afternoon topics and discuss challenges, solutions, lessons learned, and how they leveraged networks and resources to support trial success
  • Q & A with attendees

3:15-3:30 pm
(15 min)

Break  
  • Networking among attendees and presenters

CLICK HERE TO REGISTER

Please note this is a separate event from the 2023 Conference.