May 16, 2019: NIH Collaboratory Investigators Author Recommendations for Responding to Guideline or Policy Changes That Affect Ongoing Pragmatic Trials

A new perspective article by NIH Collaboratory investigators describes the unique, unexpected challenges researchers face when clinical practice guidelines and policies change during the conduct of a pragmatic clinical trial (PCT). The article was published online this week in Clinical Trials.

The NIH Collaboratory Demonstration Projects are PCTs that test interventions to address urgent public health problems. They involve hundreds to thousands of participants and generally include usual care as a control arm. During the course of these years-long trials, clinical practice guidelines and policies changed due to new evidence from observational studies, small trials, and shifting expert opinion. Such changes can have profound effects on usual care and, therefore, threaten the ability of the PCTs to address the questions they were designed to answer. Investigators must strike a balance between the primary ethical obligation to protect patients by adhering to new best-practice guidelines and policy and the secondary, yet crucial, obligation to develop high-quality evidence to improve care.

“PCTs are an important means of producing high-quality evidence needed to better inform clinical practice. However, when guidelines or reimbursement policies change during the conduct of a PCT, the ethical obligation to gather information to develop evidence-based practices may conflict with the primary ethical obligation to participants.” — Curtis et al, Clinical Trials, 2019

Based on their aggregate experience with the NIH Collaboratory, the authors provide broad recommendations and strategies for overcoming these challenges, including protecting the well-being of patients; involving stakeholders, health system leaders, and the entity charged with data and safety monitoring; and actively monitoring changes and site-level responses to them. If changes to the standard of care are merited, investigators should provide equal opportunity and support for the recommended changes. Finally, during the design phase, investigators should communicate with the entities charged with creating guidelines to see what is needed and to anticipate possible future changes.

“The ability to appropriately address the tension between modifications to clinical guidelines and the need to generate quality evidence to support those guidelines is a crucial consideration for the fulfilment of a learning health system.” — Curtis et al, Clinical Trials, 2019

May 14, 2019: NIH Announces 3 New Funding Opportunities in Dissemination and Implementation of Evidence-Based Interventions

The NIH published 3 new funding opportunity announcements to support innovative approaches to the implementation of evidence-based interventions and the de-implementation of ineffective interventions. The announcement also encourages studies that advance dissemination and implementation research methods. The 3 funding opportunities are as follows:

 

May 13, 2019: JGIM Issues Call for Papers on Implementation Science and Quality Improvement

The Journal of General Internal Medicine (JGIM) has announced a call for papers that report findings from research at the intersection of implementation science (IS) and quality improvement (QI). Submissions are due September 3, 2019, and should focus on providing information that healthcare delivery systems need about strategies to improve patient and population outcomes. The research should involve multiple settings or systems and apply rigorous scientific methods to test the effectiveness, quality, or utility of QI/IS methods in routine care.

“These articles will illustrate how foundational knowledge and skills from QI and IS help general internal medicine clinicians, educators, and researchers achieve evidence to anchor high quality and reliable care in a learning health system environment.” — JGIM Call for Papers

May 8, 2019: Dr. Greg Simon Receives National Suicide Prevention Award

At the Lifesavers Gala in New York last night, Dr. Greg Simon received the American Foundation for Suicide Prevention (AFSP’s) Research Award for his contributions to suicide prevention. Dr. Simon leads the Suicide Prevention Outreach Trial (SPOT), an NIH Collaboratory Demonstration Project that builds on previous work demonstrating that patients who answer “yes” to thoughts of self-harm on routinely administered PHQ-9 questionnaires at primary care visits are more likely to attempt suicide. For these high-risk patients, SPOT explores different modes of outreach (care management or online skills training versus usual care) to prevent suicide.

“There’s a conspiracy of silence around suicidal thoughts, because it’s awkward to discuss. So we’ve found that we have to incorporate talking about it into our standard care. Our suicide prevention work is a great example of how research and care keep influencing each other to improve our patients’ health. When research springs from clinicians’ and patients’ questions, ‘learning health systems’ can put results into practice much faster than the oft-cited 17-year lag.” — Dr. Greg Simon, from the Kaiser Permanente Washington Health Research Institute Press Release

Dr. Simon and his colleagues are also studying how machine-learning models can be used to predict risk of suicide. The models combine the PHQ-9 mental health questionnaire responses with information from electronic health records, including prior suicide attempts and mental health and substance use diagnoses. In a blog post regarding his research (and recent publication) on machine learning, Dr. Simon compares machine learning to warning lights on cars:

Our paper prompted many questions from clinicians and health system leaders about the practical utility of risk predictions:

“Are machine learning algorithms accurate enough to replace clinicians’ judgment?” our clinical partners asked.

“No,” I answered, “but they are accurate enough to direct clinicians’ attention.”

The AFSP also honored four others, including Anderson Cooper, a CNN and 60-minutes correspondent, and Kate Snow, an NBC news correspondent, for their work raising public awareness of suicide prevention.

Read more about what inspired Dr. Simon to study mental health.

May 3, 2019: Effect of Financial Bonus Sizes, Loss Aversion, and Increased Social Pressure on Physician Pay-for-Performance: A Randomized Trial and Cohort Study (Amol Navathe, MD, PhD)

Speaker

Amol S. Navathe, MD, PhD
Assistant Professor of Medicine and Health Policy
University of Pennsylvania

Topic

Effect of Financial Bonus Sizes, Loss Aversion, and Increased Social Pressure on Physician Pay-for-Performance: A Randomized Trial and Cohort Study

Keywords

Behavioral economics; Performance incentives; Evidence-based quality-of-care measures; Primary care quality; Pay for performance; Value-based medicine

Key Points

  • Pay-for-performance (P4P) programs are increasingly being used by health insurers and healthcare systems to incentivize physicians to practice higher value medicine, yet the evidence for P4P to affect quality and value of care remains mixed.
  • Behavioral economic principles in this study included increased social pressure and loss aversion added to larger bonus sizes to evaluate whether the intervention would lead to higher achievement of evidence-based quality measures.

Discussion Themes

Study findings included that, while a larger bonus size was associated with significantly improved quality for chronic care patients relative to a propensity-matched comparison group, adding increased social pressure and the opportunity for loss aversion did not lead to further quality improvement.

Attrition during the trial contributed some variability to the analysis.

Read more about pay for performance in healthcare in JAMA Network Open (Navathe et al, 2019) and NEJM Catalyst (2018).

Tags

#behavioraleconomics, #pctGR, @Collaboratory1

May 2, 2019: Cost-Effectiveness Analysis of STOP CRC Trial Finds Wide Variation Across Health Centers

A cost-effectiveness analysis of the Strategies and Opportunities to Stop Colorectal Cancer in Priority Populations (STOP CRC) trial, an NIH Collaboratory Demonstration Project, revealed wide variation across participating health centers. The study’s findings reflect the complexity of implementing an intervention in pragmatic research involving community health clinics with diverse patient populations, clinic structures, and resources.

The study was published recently in Preventive Medicine.

The STOP CRC trial tested a program to improve colorectal cancer screening rates in 26 clinics within 8 federal qualified health centers in California and Oregon. Intervention clinics embedded a tool in the electronic health record to identify patients who were overdue for screening and mailed a fecal immunochemical test (FIT) kit to these patients. Screening rates were higher overall in intervention clinics than in clinics that practiced usual care, despite low and highly variable rates of implementation of the program among participating clinics.

In the subsequent cost-effectiveness study, variability in program implementation was likewise a key factor in the results. Intervention delivery costs were highly variable across health centers, and the incremental cost-effectiveness of the intervention was diminished somewhat because usual care clinics generated more colonoscopies than intervention clinics after abnormal FIT results.

The study has implications for the design of implementation strategies in pragmatic trials that are embedded in diverse community health centers.

The STOP CRC trial was supported within the NIH Collaboratory by a cooperative agreement from the National Cancer Institute and received logistical and technical support from the NIH Collaboratory Coordinating Center. Download a study snapshot of the STOP CRC trial, and learn more about the NIH Collaboratory Demonstration Projects.

Podcast April 16: Development of Harmonized Outcome Measures for Use in Research and Clinical Practice

April 22, 2019: TiME Trial Confirms Feasibility of Embedding Large Pragmatic Trials in Clinical Care

Laura Dember

The primary results of the Time to Reduce Mortality in End-Stage Renal Disease (TiME) trial, an NIH Collaboratory Demonstration Project, were published online this month in the Journal of the American Society of Nephrology. The study confirmed the feasibility of embedding a large pragmatic clinical trial in clinical care delivery.

Although maintenance hemodialysis has long been a staple of care for patients with end-stage renal disease, there are limited data from clinical trials to inform optimal approaches, including the optimal duration of hemodialysis sessions. The TiME trial investigators, in partnership with 2 large dialysis provider organizations, evaluated the effects of a longer hemodialysis session duration on mortality and hospitalization rate among more than 7000 patients receiving care in 266 dialysis facilities.

The TiME trial was discontinued early (median follow-up, 1.1 years) because there was an insufficient difference in mean hemodialysis session duration between the intervention group and the usual care group. The investigators observed no reduction in mortality or hospitalization rate in either group.

Despite ending early, the trial met important objectives for informing the implementation of large pragmatic clinical trials embedded in health care systems. In a large multicenter study with no onsite research personnel, the investigators quickly and efficiently enrolled a large number of participants using an opt-out consent approach. The study data were obtained entirely from the electronic health and administrative records of the partnering dialysis provider organizations and were generated from routine clinical care delivery.

“The TiME trial provides an important foundation for future pragmatic trials in dialysis as well as in other settings,” said Dr. Laura M. Dember of the University of Pennsylvania Perelman School of Medicine, the principal investigator of the TiME trial.

The TiME trial was supported within the NIH Collaboratory by a cooperative agreement from the National Institute of Diabetes and Digestive and Kidney Diseases and received logistical and technical support from the NIH Collaboratory Coordinating Center. Download a study snapshot about the TiME trial, and learn more about the NIH Collaboratory Demonstration Projects.

April 18, 2019: New Commentary Highlights Value of Pragmatic Trials for Learning Health Systems

In an eGEMs commentary published this month, Leah Tuzzio and Dr. Eric Larson of the NIH Collaboratory’s Health Care Systems Interactions Core discuss the value and impact of embedded pragmatic clinical trials for learning health systems.

Pragmatic trials embedded in healthcare systems are designed to align with the care delivery goals of the health system to produce better health outcomes. The commentary highlights the NIH Collaboratory’s pragmatic trials as “the best-case examples to learn about the challenges of conducting research and of dissemination, implementation and sustainability of research results in real-world settings.”

Designing and implementing an embedded pragmatic clinical trial “requires a bidirectional flow of information and cooperative problem solving between investigator teams and clinical teams,” an important feature of learning health systems. In implementing the trial, the clinical and research teams not only generate useful data, but also demonstrate how the trial results can be incorporated into evidence-based clinical practice.

The commentary is part of a special collection of eGEMs articles commemorating 25 years of the Health Care Systems Research Network.

eGEMs is AcademyHealth’s peer-reviewed, open-access journal for electronic health data and methods. At AcademyHealth’s 2019 Annual Research Meeting in Washington, DC, the NIH Collaboratory will offer a full-day pre-conference seminar on the essentials of embedded pragmatic clinical trials. Registration for the seminar is open now.

April 15, 2019: Registration Now Open for Workshop on the Design & Analysis of Embedded Pragmatic Clinical Trials (ePCT)

The NIH Health Care Systems Research Collaboratory is hosting a one-day workshop on the Design & Analysis of Embedded Pragmatic Clinical Trials (ePCTs) on May 2, 2019, in the Lister Hill Auditorium on the NIH Campus.

The workshop will include a series of moderated discussions that focus on issues of measuring trial outcomes from available data sources, potential randomization strategies, specific ePCT design considerations, and unique challenges associated with ePCTs. Panel discussions will utilize case examples from the Collaboratory repertoire and beyond to illustrate how clinical investigators and biostatisticians work to address research questions posed by specific trials.

The Workshop Website provides information on meeting logistics, agenda, and registration. There is also an option to attend the workshop remotely via the NIH Videoconference Center, and those details are also available at the Workshop Website.