pragmatic clinical trials

Grand Rounds July 11, 2025: Novel Approaches to Recruiting Clinical Sites for Embedded Pragmatic Clinical Trials: Insights from the AIM-Back Trial (Trevor Lentz, PT, PhD and Tyler Cope, PT, DPT, ACT)

Posted on by terren.green@duke.edu

Speakers

Trevor Lentz, PT, PhD
Tyler Cope, PT, DPT, ACT
Duke Clinical Research Institute
Duke Department of Population Health Sciences
Durham Veterans Administration

Keywords

AIM-Back; Clinical site recruitment; Cluster randomized trial; Low back pain; Recruitment funnel

Key Points

  • Low back pain is an impactful condition that is more common in the veteran population. Typical low back pain care involves imaging and pharmacologic treatments that don’t always resolve pain issues and may lead to more invasive injection-based or surgical measures that often don’t result in better outcomes.
  • Research has shown that non-drug treatments (eg, cognitive behavioral therapy [CBT], yoga, physical therapy [PT]) are effective but not often used.
  • The AIM-Back trial (Improving Veteran Access to Integrated Management of Back Pain), an embedded pragmatic cluster randomized trial, sought to restructure care practices in Veteran’s Administration (VA) healthcare systems to promote and facilitate 2 clinical non-drug pathways that are supported by established guidelines as first-line treatment for low back pain.
  • Two care pathways were developed in coordination with VA clinicians, veterans, and care givers: (1) Sequenced Care Pathway – This pathway provided an initial onsite physical therapy evaluation and treatment session followed by weekly telehealth physical activity training for 6 weeks. The patient then saw the physical therapist again and was either discharged or provided with 6 weeks of training in psychologically-informed practices to help patients manage pain. (2) Pain Navigator Pathway – In this pathway, a local site clinician who was trained by the study team as a pain navigator discussed and facilitated alternative treatments for low back pain (eg, PT, yoga, CBT, massage). Patient follow up at both 6 and 12 weeks assessed progress and outcomes.
  • AIM-Back used a novel and intentional recruitment method, borrowing the concept of the business sales funnel, to generate as many site leads as possible. The recruitment process was systematic involving a 3 step framework: (1) Identify leads, (2) Approach leads, (3) Engage and select sites.
  • In step 1, leads were identified through Warm Market methods (sites known to the researchers), by Leveraging Data (evaluating lists of providers for potential fit), and through traditional Promotional Outreach efforts (advertising through networks and listservs). AIM-Back identified 184 leads from 53 VA healthcare systems.
  • Step 2 involved approaching leads through email messages. AIM-Back learned that promoting the trial in a way that helps clinicians solve their problems instead of asking clinicians to help with the research was more likely to yield the site. AIM-Back received responses from 23 VA healthcare systems.
  • In step 3, AIM-Back engaged personnel at all levels, from leadership to clinicians, to assess feasibility and buy-in at the site. AIM-Back selected 19 participant sites within 10 VA healthcare systems.
  • The Promotional Outreach strategy proved most effective with 9 (47.4%) of sites resulting from this strategy. The Leveraging Data strategy netted 6 (31.6%) sites, and 4 (21.1%) sites came from the Warm Market strategy. Site recruitment took approximately 3.6-3.8 months on average.
  • 17 sites enrolled 1817 Veterans with most sites (n=16) meeting or exceeding the minimum enrollment goal. When sites chose not to participate, they cited a reluctance to change their existing programs, a lack of clinicians or resources, or they were already participating in similar trials.

Discussion Themes

AIM-Back messaging evolved over the course of recruitment from a more traditional trial marketing email to an email that was more personal, short, and leveraged the standing of Duke University. This more personal approach to recruitment led to better relationships with sites during the trial.

Project management software can be helpful for tracking follow up with site leads and communication during the recruitment process.

One overall goal of AIM-Back was to set up a new clinical program that could continue after the end of the trial. Sites were given training materials for the centralized study components and support from AIM-Back was stepped down slowly. Sites that chose to continue the intervention trained a physical activity/whole health coach and a PT for the psychologically informed PT portion of the intervention.

Indicators of a potentially successful site included qualitative components that reflect a high level of engagement such as high interest and excitement in the study along with a sufficient patient population.

Read more about the AIM-Back trial design.

July 3, 2025: OPTIMUM Team Identifies Facilitators of Participant Retention and Engagement

Posted on by Damon Seils
Headshot of Dr. Natalia Morone, principal investigator for OPTIMUM
Dr. Natalia Morone, principal investigator for OPTIMUM

Trust in their clinicians, positive interactions with study staff, and opportunities for social connection can improve recruitment, retention, and engagement with research participants, according to focus groups of participants in the OPTIMUM trial. These factors can be especially important for first-time research participants and for groups that are historically underrepresented in clinical trials.

The article was published online in Global Advances in Integrative Medicine and Health.

OPTIMUM, an NIH Collaboratory Trial, is studying the addition of mindfulness-based stress reduction to usual care for patients with chronic low back pain, compared with usual care alone, with the goal of improving pain outcomes and reducing opioid prescriptions. Patients in the control group were also offered free mindfulness sessions after the end of the 12-month follow-up period.

Understanding of randomized controlled trials and general research processes emerged as another important theme in the focus groups. In particular, appreciating the value of the control group in a randomized controlled trial can support participants’ ongoing engagement.

“Consistent, positive study staff interactions were particularly important for control group participants who were not immediately receiving the intervention that initially interested them in the trial,” the authors wrote.

The OPTIMUM trial, which is embedded in safety net hospitals and federally qualified health centers, includes a large number of first-time participants in research and patients from racial and ethnic groups that tend to be underrepresented in research. Understanding the needs and perspectives of first-time research participants and those from underrepresented groups can improve recruitment and retention strategies and help ensure the representativeness of study populations.

Read the full report.

OPTIMUM is supported within the NIH Pragmatic Trials Collaboratory through the NIH HEAL Initiative by a grant award administered by the National Center for Complementary and Integrative Health. Learn more about OPTIMUM.

July 2, 2025: Researchers Share Tips for Embedding Pragmatic Trials Into Primary Care Settings

Posted on by Karen Staman

Headshots of Drs. Wendy Weber, Julie Fritz, Natalia Morone, and Sebastian TongAlthough the need for pragmatic research in primary care settings is great, conduct of pragmatic clinical trials in these settings remains nascent. In a session at the NIH Pragmatic Trials Collaboratory’s 2025 Annual Steering Committee Meeting, Wendy Weber of the National Center for Complementary and Integrative Health (NCCIH) led a discussion about how to expand pragmatic research into primary care settings.

The panelists included Julie Fritz, principal investigator (PI) for BeatPain Utah; Natalia Morone, PI for OPTIMUM; and Sebastian Tong, co-PI for AIM-CP. They shared some of the challenges of conducting research in primary care.

  • Primary care physicians have multiple competing demands for attention and focus, and a range of topics to cover in a single visit.
  • The expectation of being offered participation in clinical trials is lower in primary care than in specialty visits.
  • There are fewer resources that can be diverted to research in primary care, especially time and personnel.

Key Takeaways

How do we adapt pragmatic clinical trials to primary care?

Identify relevant and meaningful research questions

The panelists suggested that, when planning a trial, investigators should think about the primary care setting and consider what problems keep clinicians up at night. Research will be easier to implement if it helps alleviate these problems.

“In primary care, [primary care physicians] are looking at the entire person,” Tong said. “There has to be value added in how we care for the patients with the condition under study, either where the [physician] sees a difference in their patients’ lives or their own lives with respect to reduced burden,” he said.

The panelists also suggested that, for community health centers, there are certain conditions that are linked to data reporting standards, and aligning research with these standards will make studies easier to champion.

Adapt to different kinds of primary care settings

The panelists stressed the need for adaptably and flexibility in intervention delivery because all primary care settings are different. If  possible, researchers should tailor the workflow at each site in a way that is familiar to providers. the panelists also suggested that initial engagement by external researchers is best made with someone who is trusted at the site.

Some clinics, including community health centers, may be new to research and may need additional support. Many do not have an institutional review board or a Federalwide Assurance number, and many do not have experience using the electronic health record for research and developing data use agreements.

About the Trials

  • BeatPain Utah, supported by the National Institute of Nursing Research (NINR), is comparing the effectiveness of nonpharmacologic interventions delivered via telehealth for patients with back pain seeking care in federally qualified health centers in Utah.
  • OPTIMUM, supported by NCCIH, is assessing the impact of a group-based mindfulness intervention for patients with chronic back pain under usual care circumstances in the primary care setting.
  • AIM-CP, supported by NINR, is testing the implementation of a care management program to address disparate access to nonpharmacological treatments for chronic pain in rural populations.

This summer, we are sharing highlights from the 2025 Annual Steering Committee Meeting.  Access the complete collection of meeting materials.

July 1, 2025: As Their Studies Wrap Up, Researchers Share Lessons for Pragmatic Trials

Posted on by Damon Seils

Headshots of Drs. Christine Goertz, Andrea Cook, Shruti Gohil, and Stacy SterlingA key feature of the NIH Pragmatic Trials Collaboratory is its culture of learning and knowledge sharing among investigators who are planning and conducting pragmatic clinical trials. In a session at the program’s 2025 Annual Steering Committee Meeting, a panel of investigators discussed their recently completed NIH Collaboratory Trials and their thoughts about future directions for pragmatic research.

The panelists included Andrea Cook, co–principal investigator (PI) for BackInAction; Shruti Gohil, lead investigator for INSPIRE; and Stacy Sterling, co-PI for GGC4H. Christine Goertz, a co-PI for IMPACt-LBP, moderated the discussion.

Key Takeaways

What do investigators wish they had known before starting a pragmatic trial? Sterling highlighted a need for ongoing engagement with partnering healthcare systems’ senior leaders to ensure they understand the challenges of adopting and sustaining the study intervention. Cook stressed the importance of educating site personnel about what it means to participate in the trial, including the potential for randomization to a usual care arm.

“We also included nonphysician clinicians in the education about the intervention, because they are often doing the steps being targeted by the intervention,” Gohil added.

How can we apply these lessons in future pragmatic trials? “Doing an embedded pragmatic trial is sort of like having a child: You really need the guidance of people who have gone before you and have real-life experience,” Goertz said.

The panelists proposed the development of more standardized materials for the consent process and other trial elements. They also discussed the need for broader dissemination strategies and new ways to promote implementation tools from trials with successful interventions.

Gohil described her research team’s commitment to developing a dissemination toolkit so people will understand how to approach implementation. She noted that, as soon as the trial was completed but before publication, the research team presented the results to the participating sites.

Greg Simon, the PI for SPOT and a member of the NIH Collaboratory Coordinating Center’s leadership team, stressed the importance of including research partners in dissemination.

“Don’t write the discussion section [of your study results paper] until you’ve talked with your healthcare system partners; they might have something to teach us about what the results mean,” he said.

About the Trials

  • BackInAction, supported by the National Center for Complementary and Integrative Health (NCCIH), is comparing standard and advanced courses of acupuncture with usual care for older adults with chronic low back pain.
  • GGC4H, supported by NCCIH, is testing the feasibility and effectiveness of implementing within healthcare systems the Guiding Good Choices program for caregivers of early adolescents to prevent common behavioral problems, such as substance use.
  • IMPACt-LBP, supported by NCCIH, is evaluating the effect of first-contact patient referral to physical therapists and doctors of chiropractic for low back pain.
  • INSPIRE, supported by the National Institute of Allergy and Infectious Diseases, used personalized clinical decision support to improve judicious antibiotic prescribing for non–critically ill patients hospitalized with abdominal infections or skin and soft tissue infections.
  • SPOT, supported by the National Institute of Mental Health, compared care management and dialectical behavior therapy skills training to usual care for adults at risk of self-harm or suicidal behavior.

This summer, we are sharing highlights from the 2025 Annual Steering Committee Meeting. Access the complete collection of meeting materials.

June 26, 2025: New Podcast Episode Considers How to Make Pragmatic Trials More Pragmatic

Posted on by Sophia Ramirez

Drs. Rich Platt, Greg Simon, and Hayden BosworthIn a new episode of the NIH Pragmatic Trials Collaboratory Podcast, Rich Platt, Hayden Bosworth, and Greg Simon of the NIH Pragmatic Trials Collaboratory discuss their JAMA Viewpoint, “Making Pragmatic Clinical Trials More Pragmatic.”

Listen and subscribe to the podcast on SoundCloud or Apple Podcasts.

In the Viewpoint, the authors propose solutions to the discordance between the results of pragmatic trials and the implementation of those results in healthcare settings.

“I think one of the problems with our evidence generating process is that we may think that our
customers are grant review panels or maybe journal editors,” Platt notes in the podcast. “Those may be our short-term customers, but those are not our ultimate customers. Our ultimate customers are people who have to make decisions about healthcare,” he added.

June 12, 2025: Living Textbook Chapter Covers Consent, Disclosure, and Nondisclosure for Pragmatic Trials

Posted on by Karen Staman

A new chapter of the Living Textbook of Pragmatic Clinical Trials describes regulatory requirements for informed consent, waivers and alterations of consent, mechanisms for notification, and research participants’ perspectives on a variety of approaches to consent and notification—all with a focus on special considerations for pragmatic clinical trials.

For a variety of reasons, the  application of ethical principles and regulations regarding informed consent can be complex for pragmatic clinical trials. For example, pragmatic trials often use novel study designs, including cluster randomization, in which the unit of randomization may be a clinic, hospital, or healthcare system rather than the individual. Some pragmatic trials also use stepped-wedge designs, in which the study intervention is introduced to sites at different times.

The new chapter has 5 sections:

  • Section 1 discusses reasons why the application of ethical principles and regulations regarding informed consent can be complex for pragmatic trials.
  • Section 2 describes the regulatory requirements for informed consent.
  • Section 3 focuses on waivers and alterations of the informed consent process.
  • Section 4 provides examples of mechanisms for notifying participants about the trial when consent is not required.
  • Section 5 presents findings on research partners’ preferences regarding various approaches to research and consent.

The chapter was developed by members of the NIH Pragmatic Trials Collaboratory’s Ethics and Regulatory Core.

May 13, 2025: Video Decision Aid and Clinician Communication Training Boost Advance Care Planning in Oncology Clinics

Posted on by Damon Seils
Headshots of Dr. James Tulsky and Dr. Angelo Volandes
Dr. James Tulsky and Dr. Angelo Volandes, principal investigators for ACP PEACE

A video decision aid for older patients with advanced cancer, coupled with communication skills training for clinicians, led to higher rates of documented advance care planning in oncology clinics, according to the ACP PEACE study.

The results of the study were published in the May issue of JAMA Network Open.

Many older adults with advanced cancer do not discuss treatment preferences or goals of care with their clinicians. Lack of communication about patients’ preferences can lead to patients receiving care that does not match their values.

ACP PEACE, an NIH Collaboratory Trial, was a stepped-wedge, cluster randomized trial testing the delivery of a video decision aid to patients together with goals-of-care communication skills training to oncology clinicians in 3 healthcare systems. The study included 13,800 patients at 29 clinics. Each clinic was randomly assigned to either the intervention or usual care at the beginning of the study. Due to the stepped-wedge design of the trial, all clinics were exposed to the intervention by the end of the study.

Logo for the ACP PEACE Demonstration Project

The intervention included an evidence-based, patient-facing video decision aid available in 25 languages. It also included a communication training program to improve clinicians’ skills in delivering serious news, responding to emotion, and eliciting patients’ goals. The primary outcome was documentation of advance care planning in the electronic health record, which could include documentation of a goals-of-care conversation, palliative care, hospice, or limitation of life-sustaining treatments.

The proportion of patients with documentation of advance care planning was greater with the ACP PEACE intervention than with usual care. The difference was attributable to a greater proportion of patients in the intervention phase having a goals-of-care conversation. There were no significant differences between the intervention and usual care for the documentation of palliative care, hospice, or limitation of life-sustaining treatments.

Read the full article.

“By focusing concurrently on both clinicians and patients—giving clinicians the skills to have these difficult conversations and preparing patients to engage with them—we were able to increase the number of documented goals-of-care conversations,” said James Tulsky, the Poorvu Jaffe Chair in Supportive Oncology at the Dana-Farber Cancer Institute and a co–principal investigator for ACP PEACE.

“If we are going to achieve shared decision-making, we need to bring everyone onto the same page,” Tulsky said.

Angelo Volandes, the Anna Gundlach Huber Professor in Medicine at the Geisel School of Medicine at Dartmouth and a co–principal investigator for ACP PEACE, highlighted the logistical challenge of conducting a large pragmatic clinical trial across 3 major healthcare systems during the COVID-19 pandemic.

“What made it work was a shared commitment—from frontline clinicians to healthcare system leaders—to ensure that older adults with advanced cancer had the opportunity to reflect on their values and have those conversations documented,” Volandes said.

“This trial wasn’t just about changing documentation rates, it was about changing the culture of communication, at scale,” he added.

ACP PEACE is supported within the NIH Pragmatic Trials Collaboratory by a cooperative agreement from the National Institute on Aging.

Learn more about ACP PEACE.

April 22, 2025: New Report Highlights Value of Informing Participants About Research Conducted Under a Waiver of Consent

Posted on by Karen Staman

Even in minimal-risk studies that do not use the standard consent process, there may be value in informing participants about the research. Such notifications should be considered the default for clinical trials conducted under a waiver of informed consent, argue the authors of a new report from the NIH Pragmatic Trials Collaboratory.

The open-access article was published online ahead of print this week in Learning Health Systems.

Pragmatic clinical trials conducted in the context of routine healthcare often meet the regulatory criteria for a waiver or alteration of the standard informed consent process. In such cases, researchers and reviewers might assume there is no reason to communicate information about the study to participants. However, providing information to participants, even in minimal-risk research conducted with a waiver of consent, can promote important ethical values.

Experts from the NIH Collaboratory’s Ethics and Regulatory Core teamed up with investigators from several of the NIH Collaboratory Trials to describe methods of informing participants in minimal-risk research.

The investigators used a variety of notification approaches in their studies, including letters and email campaigns, posters in waiting rooms and other common areas, conversations with clinicians, and presentations at staff meetings. The amount of information provided to participants ranged from a general statement that research was being conducted at the institution to detailed information about the study in question.

“When a study is approved with a waiver of research consent, investigators and review committees should consider on a case-by-case basis what information, if any, to disclose to participants, and how it will be disclosed,” the authors wrote. The costs, benefits, and feasibility of these approaches vary from study to study.

Communicating information to participants can promote several important goals:

  • The ethical principle of respect for persons
  • Participants’ understanding of the study and of research in general
  • Participants’ understanding of their contributions to the research
  • Participants’ ability to voice and discuss any concerns about the study
  • Participant engagement in research
  • Trust in research and researchers

“Providing information to the participants should thus be the default for trials conducted under a waiver of research consent,” the authors wrote.

Read the full report.

March 20, 2025: iPATH Team Explores Integration of Artificial Intelligence Into Analysis of Qualitative Data

Posted on by Damon Seils
Headshot of Dr. Sara Singer
Dr. Sara Singer, principal investigator for iPATH

Researchers from iPATH, an NIH Collaboratory Trial, described key considerations for integrating artificial intelligence tools into analyses of qualitative data.

The report was posted this month on the AcademyHealth Blog.

The iPATH trial, led by principal investigator Sara Singer at Stanford University, will test the implementation of a practice transformation strategy for type 2 diabetes in federally qualified health centers in California, Massachusetts, Ohio, and Puerto Rico. In the first phase of the project, the study team is refining the strategy by conducting case studies with 12 health centers to identify organizational conditions and processes that promote or impede the effectiveness of diabetes care.

Interviews for the 12 case studies generated 170 hours of qualitative data plus related materials. The study team explored how rapidly evolving artificial intelligence tools, such as large language models, might enhance researchers’ handling of large qualitative datasets, including labor-intensive and time-consuming processes of transcription, coding, and analysis.

Read the full report.

iPATH is supported by a grant award from the National Institute on Minority Health and Health Disparities. Learn more about iPATH.

March 12, 2025: In This Week’s PCT Grand Rounds, Spillover as a Potential Source of Bias in Pragmatic Trials

Posted on by Damon Seils
Headshot of Sean Mann
Sean Mann

In this Friday’s PCT Grand Rounds, Sean Mann of RAND will present “Spillover Due to Constraints on Care Delivery: A Potential Source of Bias in Pragmatic Clinical Trials.”

The Grand Rounds session will be held on Friday, March 14, 2025, at 1:00 pm eastern.

Mann is a senior policy analyst at RAND.

Join the online meeting.