Tag Archives: pragmatic clinical trials

PCORnet: “Not your father’s clinical trial network”


“PCORnet: Turning a Dream Into Reality,” an editorial published online this week in the Journal of the American Medical Informatics Association, details the promises of the National Patient-Centered Clinical Research Network (PCORnet):

“Providing accurate answers based on the highest levels of scientific evidence for the majority of unresolved clinical questions is a revolutionary dream shared by patients, providers, payers, health plans, researchers, and policy makers alike. PCORnet, the National Patient-Centered Clinical Research Network, promises a transformative platform that will turn this revolutionary dream into reality.”

The authors describe PCORnet’s patient-centered vision and how its transformative clinical research will be carried out through a national research network involving both Clinical Data Research Networks and Patient-Powered Research Networks. This “network of networks” is designed to include a large, highly representative population. PCORnet will first work to establish data architecture and standards and address key policy questions, followed by the conduct of its first pragmatic clinical trial slated to begin in September 2015.

Editorial authors include NIH Director Dr. Francis Collins and PCORnet Steering Committee members Kathy L. Hudson, PhD, and Josephine P. Briggs, MD.


Grand Rounds (4-4-2014): Defining Denominator Populations Within and Across Complex Health Systems

Update:

Archived video and slides from the April 4 Grand Rounds are now available on the NIH Collaboratory Grand Rounds webpage.


This Friday’s NIH Collaboratory Grand Rounds (“Defining Denominator Populations Within and Across Complex Health Systems”) will be presented by Greg Simon, MD, MPH, of the Group Health Research Institute. Dr. Simon is the principal investigator for the NIH Collaboratory Demonstration Project “Pragmatic Trial of Population-Based Programs to Prevent Suicide Attempt” (detailed description available here).

The Grand Rounds presentation will take place from 1:00-2:00 PM Eastern time on Friday, April 4. Details are available here. Archived video and slide sets from the presentation will be available early the following week; links to archived material will be provided in an update to this post.


LIRE Pragmatic Clinical Trial Begins Randomization



One of the NIH Collaboratory’s initial Demonstration Projects, the Lumbar Image Reporting with Epidemiology (LIRE) study, has begun randomization in early April. The LIRE trial is designed to test whether inserting additional epidemiological information into the lumbar spine imaging reports of patients being treated for lower back pain can help both doctors and patients to better understand and interpret the reports. This in turn could help doctors avoid subjecting patients to unnecessary tests and procedures.

LIRE is a cluster randomized trial, which means that instead of randomizing individual patients, whole clinics (one at the Henry Ford Health System in Detroit; one at Group Health Cooperative in Seattle, with more to follow) are randomly assigned to provide either the experimental treatment or the control treatment to patients.

Cluster-randomized trials offer a number of advantages, including the avoidance of certain kinds of bias that can effect the outcome of a study, but they also raise special issues that can require careful consideration.

The principal investigator of the LIRE trial is Dr. Jeffrey Jarvik  of the University of Washington. You can read more about the LIRE trial here.


STOP CRC Study Featured on NPR Health Blog


The Strategies and Opportunities to Stop Colon Cancer in Priority Populations (STOP CRC) study was recently featured on National Public Radio’s Shots Blog, which is devoted to coverage and discussion of health and healthcare issues.

Picture of U.S. stop sign.
Public-domain image via Wikimedia Commons.

STOP CRC, which is exploring innovative approaches for increasing colon cancer screening rates among low-income and minority populations, is one of the seven UH2 pilot projects supported by the NIH Collaboratory. The demonstration phase of the project will be conducted within a pair of health centers that are part of OCHIN, a community-based network of Federally Qualified Health Centers.

More information about about STOP CRC is available here.


Changes to Informed Consent in the Era of Learning Healthcare Systems


In 2007, a seminal report from the Institute of Medicine (IOM) threw a sharp spotlight on a series of problems facing the broader U.S. healthcare system:

Evidence on what is effective, and under what Cover page of IOM report - The Learning Healthcare Systemcircumstances, is often lacking, poorly communicated to decision makers, or inadequately applied, and despite significant expenditures on health care for Americans, these investments have not translated to better health.

—The Learning Healthcare System (IOM Workshop Summary), 2007

The IOM report called for a new approach to closing the existing gaps in patient care, one that would create a system in which patient care, quality improvement efforts, and clinical research would exist as integrated components within a virtuous cycle of feedback—the “learning healthcare system.” (The original report, plus a series of related reports [PDF], can be read for free online at the National Academies Press website.)

In the years since The Learning Healthcare System was first published, researchers, healthcare providers, health systems, and governmental and regulatory agencies have all struggled with the monumental task of building such a system. One aspect that has presented a particularly complex set of challenges centers on the issue of informed consent. The principle of informed consent—the idea that all patients have the right to make a fully informed decision, free from coercion or other undue pressure, about whether or not to participate in research—is a foundational tenet of clinical research ethics. However, some of the tools that are widely considered to be critical to the success of achieving a workable learning healthcare system, such comparative effectiveness studies and cluster-randomized trials, are difficult or even impossible to conduct under existing models of individual informed consent.

Recent efforts from the Ottawa Consensus Statement Group and a series of articles published in an issue of the Hastings Center Report have explored explored informed consent in such circumstances. These are now joined by a pair of articles published in the February 20, 2014 issue of the New England Journal of Medicine. In the first, Faden and colleagues outline a case for streamlining or even dispensing with individual informed consent in certain kinds of randomized comparative-effectiveness or quality improvement studies that present a minimal risk of harm to patients [1]. The authors also describe the larger framework that would provide transparent and accountable oversight of such studies, as well as overseeing the integration of findings from such research into the patient-care process.

The second article, by Kim and Miller, presents a different vision for informed consent,  the “Integrated Consent Model” [2]. Unlike Faden and colleagues, the authors argue for preserving the element of informed consent for all randomized pragmatic research, even in circumstances considered to pose minimal risk to patients. Kim and Miller suggest that such a model, in which the prospect of participating in research is offered as part of the general clinical discussion about treatment options and documented by the physician, will satisfy ethical imperatives for informing patients while remaining sufficiently streamlined to meet the demands of pragmatic clinical research. They also argue that an “integrated consent” approach could be accommodated under existing regulations.

Regardless of whether either or both of these perspectives can be incorporated into the emerging learning healthcare system, the larger questions surrounding informed consent are  garnering significant interest, as evidenced by the response to the Department of Health and Human Service’s 2011 call for public comment on a proposal to modify rules governing clinical research. And with the proliferation of new technologies and trial designs that use data extracted directly from patient’s electronic health records (such as the demonstration projects coordinated through the NIH Collaboratory) and the emergence of innovative networks devoted to pragmatic patient-centered research, the need for a solution both protects patients and enables vitally needed research will only continue to grow.

For additional perspective on the recent publications in the New England Journal of Medicine, see "Research Permissions–Angels on the Head of a Pin, or the Key Issue to Decipher?" by NIH Collaboratory PI Dr. Rob Califf.

References


1. Faden RR, Beauchamp TL, Kass NE. Informed consent, comparative effectiveness, and learning health care. N Engl J Med 2014;340:766-768. 10.1056/NEJMhle1313674.

2. Kim SYH, Miller FG. Informed consent for pragmatic trials — The integrated consent model. N Engl J Med 2014; 370:769-772. doi: 10.1056/NEJMhle1312508.