study design

Applying PRECIS Ratings to Collaboratory Pragmatic Trials

Posted on by Gina Uhlenbrauck

A new article published in the journal Trials provides a look at how the  Pragmatic–Explanatory Continuum Indicator Summary, or PRECIS, rating system can be applied to clinical trials designs in order to examine where a given study sits on the spectrum of explanatory versus pragmatic clinical trials.

The PRECIS-2 criteria are used to rate study designs as more or less “pragmatic” according to multiple domains that include participant eligibility, recruitment methods, setting, organization, analysis methods, primary outcomes, and more. In this context, “pragmatic” refers to trials that are designed to study a therapy or intervention in a “real world” setting similar or identical to the one in which the therapy will actually be used. Pragmatic trials stand in contrast to explanatory trials, which are typically designed to demonstrate the safety and efficacy of an intervention under highly controlled conditions and in carefully selected groups of participants, but which may also be difficult to generalize to larger or more varied populations.

Schematic of PRECIS-2 Wheel used to evaluate where a given trial design resides upon the explanatory-pragmatic spectrum.
PRECIS-2 Wheel.  Kirsty Loudon et al. BMJ 2015;350:bmj.h2147. Copyright 2015 by British Medical Journal Publishing Group. Used by permission.

Clinical trials are almost never wholly “explanatory” or wholly “pragmatic.” Instead, many studies exist somewhere on a spectrum between these two categories. However, understanding how these different attributes apply to trials can help researchers design studies that are optimally fit for purpose, whether that purpose is to describe a biological mechanism (as in an explanatory trial) or to show how effective an intervention is when used across a broad population of patients (as in a pragmatic trial).

In their article in Trials, authors Karin Johnson, Gila Neta, and colleagues  applied PRECIS-2 criteria to 5 pragmatic clinical trials (PCTs) being conducted through the NIH Collaboratory. Each trial was found to rate as “highly pragmatic” across the multiple PRECIS-2 domains, highlighting the tool’s potential usefulness in guiding decisions about study design, but also revealing a number of challenges in applying it and interpreting the results.

Study authors Johnson and Neta will be discussing their findings during the NIH Collaboratory’s Grand Rounds on Friday, January 22, 2016 (an archived version of the presentation will be available the following week).

Johnson KE, Neta G, Dember LM, Coronado GD, Suls J, Chambers DA, Rundell S, Smith DH, Liu B, Taplin S, Stoney CM, Farrell MM, Glasgow RE. Use of PRECIS ratings in the National Institutes of Health (NIH) Health Care Systems Research Collaboratory. Trials. 2016;17(1):32. doi: 10.1186/s13063-016-1158-y. PMID: 26772801. PMCID: PMC4715340.
You can read more about the NIH Collaboratory PCTs featured as part of this project at the following links:

ABATE (Active Bathing to Eliminate Infection)

LIRE (A pragmatic trial of Lumbar Image Reporting with Epidemiology)

PPACT (Collaborative Care for Chronic Pain in Primary Care)

STOP-CRC (Strategies & Opportunities to Stop Colon Cancer in Priority Populations)

TIME (Time to Reduce Mortality in End-Stage Renal Disease)
Additional Resources

An introductory slide set on PCTs (by study author Karin Johnson) is available from the Living Textbook:

Introduction to Pragmatic Clinical Trials
The University of Colorado Denver - Anschutz Medical Campus publishes an electronic textbook on pragmatic trials:

Pragmatic Trials: A workshop Handbook

 

 

 

New Lessons Learned Document Draws on Experiences of NIH Collaboratory Trials

Posted on by Karen Staman

The NIH Collaboratory’s Health Care Systems Interactions Core has published a document entitled Lessons Learned from the NIH Health Care Systems Research Collaboratory Trials. The Principal Investigators of each of the NIH Collaboratory Trials shared their trial-specific experience with the Core to develop the document, which presents problems and solutions for initiation and implementation of pragmatic clinical trials (PCTs). Lessons learned are divided into the following categories: build partnerships, define clinically important questions, assess feasibility, involve stakeholders in study design, consider institutional review board and regulatory issues, and assess potential issues with biostatistics and the analytic plan.

Other tools available from the Health Care Systems Interactions Core include a guidance document entitled Considerations for Training Front-Line Staff and Clinicians on Pragmatic Clinical Trial Procedures and an introduction to PCTs slide set.

Findings from STOP CRC on Pragmatic Trial Recruitment

Posted on by Gina Uhlenbrauck
Gloria Coronado, PhD, and Beverly Green, MD, MPH, Principal Investigators, STOP CRC Trial
Gloria Coronado, PhD, and Beverly Green, MD, MPH, Principal Investigators, STOP CRC Trial

Drs. Beverly Green and Gloria Coronado and colleagues have published an article in Clinical Trials describing the challenges of recruiting participants into large, multisite pragmatic clinical trials—particularly at the health system level. STOP CRC is one of the NIH Collaboratory’s pragmatic clinical trial, which are intended to provide a framework of implementation methods and best practices to enable participation of varied health care systems in clinical research.

STOP CRC is testing a culturally tailored, health care system–based program to improve colorectal cancer screening rates in a community-based collaborative network of federally qualified health centers. The authors observed that recruiting sites to participate in pragmatic trials is time-intensive and involves both preparing materials and organizing face-to-face meetings with staff and clinic leaders. Yet little is known about the characteristics of nonparticipating sites and clinic-level factors that may influence willingness to participate in a pragmatic trial.

“Our findings underscore the importance of assessing and reporting recruitment success at the organizational and/or clinic level in order to know the external validity of the findings and may inform future efforts to select and recruit health systems to participate in pragmatic research.” (Coronado, et al. Clin Trials 2015)

Study Design of LIRE Pragmatic Trial Published

Posted on by Gina Uhlenbrauck
Picture of Jerry Jarvik, MD, MPH
Jerry Jarvik, MD, MPH, Principal Investigator, LIRE Trial

Dr. Jerry Jarvik and colleagues have published an article in Contemporary Clinical Trials describing the design of the Lumbar Imaging With Reporting of Epidemiology (LIRE) pragmatic cluster randomized trial. LIRE is one of the NIH Collaboratory’s pragmatic clinical trial, which are intended to help establish proof of concept for pragmatic trial designs.

LIRE is studying the effect of inserting epidemiologic benchmarks for common imaging findings into lumbar spine imaging reports being delivered to primary care physicians. The primary goal is to measure whether the intervention reduces subsequent spine-related tests and treatments. All outcomes are captured passively through the electronic health record. The authors state that if successful, such a low-cost intervention could potentially be applied to diagnostic tests for other conditions. LIRE has a projected sample size of more than 160,000 patients across an estimated >2000 primary care physicians at 4 health systems. Enrollment will continue through 2016.

“LIRE is a pragmatic cluster randomized trial of a minimal-risk intervention that we believe can serve as a model for future pragmatic trials.”
(Jarvik JG, et al. Contemp Clin Trials 2015)