clinical outcomes

February 7, 2020: Using Nudges to Improve the Delivery of Health Care (Mitesh S. Patel, MD, MBA)

Posted on by Gina Uhlenbrauck

Speaker

Mitesh S. Patel, MD, MBA
Ralph Muller Presidential Assistant Professor
Perelman School of Medicine & Wharton School
University of Pennsylvania
Director, Penn Medicine Nudge Unit

Topic

Using Nudges to Improve the Delivery of Health Care

Keywords

Motivational nudges; Medical decision-making; Behavioral economics; Nudge units; Health behaviors

Key Points

  • Nudges remind, guide, or motivate a decision. In a healthcare setting, medical nudges use principles of behavioral economics and psychology to “steer medical decision-making toward higher value and improved patient outcomes.”
  • A “nudge unit” is a behavioral design team that systematically tests ways to improve decisions and change behavior.
  • It is important that medical nudges be testable and rigorously evaluated. Well-designed nudges have the right fit for the context, are aligned with health system and patient care goals, are scalable, and make a significant impact.
  • Nudges can be used to help clinicians promote evidence-based care, and help patients increase engagement and change health behaviors.

Discussion Themes

Nudges are prevalent in other industries; for example, when asked to add trip insurance to the purchase of an airfare.

In a healthcare setting, effective nudges can help achieve care delivery priorities (for example, improved screening, follow up, adherence) as well as reduce clinician burden.

Understanding the health system’s culture and workflow is essential for the sustainability and effectiveness of nudges.

Read more about nudges in Using Nudges to Improve Value by Increasing Imaging-Based Cancer Screening (Patel et al, Journal of the American College of Radiology, 2019).

Tags
#pctGR, @Collaboratory1, @miteshspatel, @PennNudgeUnit

January 24, 2020: Cardiovascular Trials Over 2 Decades: Progress on Pragmatism? (Justin A. Ezekowitz, MBBCh, MSc)

Posted on by Gina Uhlenbrauck

Speaker

Justin A. Ezekowitz, MBBCh, MSc
Professor, Department of Medicine
Co-Director, Canadian VIGOUR Centre
Director, Cardiovascular Research, University of Alberta
Cardiologist, Mazankowski Alberta Heart Institute

Topic

Cardiovascular Trials Over 2 Decades: Progress on Pragmatism?

Keywords

Pragmatic clinical trials; PRECIS-2; Cardiovascular trials; Enrollment of women

Key Points

  • Pragmatic clinical trials are “designed for the primary purpose of informing decision-makers regarding the comparative balance of benefits, burdens and risks of a biomedical or behavioral health intervention at the individual or population level” (Califf & Sugarman, 2015).
  • This study examined how pragmatic or explanatory cardiovascular (CV) randomized controlled trials are; whether the level of pragmatism in CV trials has changed over 2 decades; and whether the proportion of women enrolled in CV trials has changed over 2 decades.
  • No clinical trial is completely explanatory or pragmatic. In this study, trials that scored higher on pragmatism (using the PRECIS-2 tool) had more sites, a larger sample size, longer follow up, and mortality as a primary endpoint.

Discussion Themes

Randomized controlled trials that were published in general medicine journals scored higher in pragmatism than those published in CV journals. Pragmatism has increased over time in CV trials.

While women account for ~45% of the burden of CV diseases, they are underrepresented in CV randomized controlled trials, with less than one-third of trial participants. There was no difference between pragmatic trials and other trials in terms of women’s enrollment.

Initiatives that focus on patient, clinician, and trial design factors are needed to address the gender gap in trial enrollment.

Read more about the PRECIS-2 tool in the Living Textbook, and Dr. Ezekowitz’s research in Trends in the Explanatory or Pragmatic Nature of Cardiovascular Clinical Trials Over 2 Decades (JAMA Cardiology, 2019).

Tags
#pctGR, @Collaboratory1

January 17, 2020: Assessment of the Human Systemic Absorption of Sunscreen Active Ingredients: FDA-Sponsored Randomized Clinical Trial (Murali Matta, MPharm, PhD)

Posted on by Gina Uhlenbrauck

Speaker

Murali Matta, MPharm, PhD
Bioanalytical Lead
Division of Applied Regulatory Science
Office of Clinical Pharmacology
Food and Drug Administration

Topic

Assessment of the Human Systemic Absorption of Sunscreen Active Ingredients: FDA-Sponsored Randomized Clinical Trial

Keywords

FDA; Sunscreen; Randomized controlled trial; Regulatory; Data analysis

Key Points

  • The active ingredients in nonprescription sunscreen products are organic chemicals, some of which have been shown to be absorbed through human skin with detectable levels in the blood or urine.
  • It is important that randomized clinical trials be conducted to better understand the clinical significance of systemic exposure to sunscreen products.
  • In this trial, all active ingredients in all tested products exhibited systemic exposures above the threshold for potentially waiving some nonclinical toxicology studies for sunscreens.

Discussion Themes

Do the observed differences in the sunscreen concentration depend on the application type; for example, spray versus lotion versus cream?

While additional toxicology data are needed, the results of this study do not indicate that individuals should refrain from the use of sunscreen.

Is there opportunity for collaboration with other organizations including private physician-scientists to conduct larger population studies with consumers?

Read more about Dr. Matta’s study at Shedding New Light on Sunscreen Absorption and in a recent JAMA publication.

Tags
#pctGR, @Collaboratory1, @US_FDA

January 10, 2020: Pragmatic Trials of Behavioral Economic Interventions to Increase Colorectal Cancer Screening (Shivan Mehta, MD, MBA)

Posted on by Gina Uhlenbrauck

Speaker

Shivan Mehta, MD, MBA
Assistant Professor of Medicine and Health Policy
University of Pennsylvania

Topic

Pragmatic Trials of Behavioral Economic Interventions to Increase Colorectal Cancer Screening

Keywords

Behavioral economics; Colorectal cancer; Pragmatic clinical trials; Health technology; Communication modality; Informed consent

Key Points

  • Colorectal cancer is the second leading cause of cancer deaths in the United States. Increased rates of screening can reduce mortality from colorectal cancer by 30% to 70%.
  • Use of behavioral economics can help us understand human motivation and behavior related to participating in clinical studies. How the message to patients is framed—and how choices are offered—can alter the response.
  • In the example pragmatic trial, the “choice architecture” for the colorectal cancer screening was designed by the study team in collaboration with health system stakeholders and clinical operations. Changing the framing from opt in to opt out had the effect of increasing participation in screening.

Discussion Themes

In some settings, choice overload can have a negative effect on participation.

When designing embedded pragmatic trials, researchers must be mindful not to increase burden on clinicians’ workflow.

While behavioral economics offers suggestions for how to increase colorectal cancer screening rates, its effectiveness in different contexts needs to be evaluated.

Read more about Dr. Mehta and colleagues’ study in Effect of Sequential or Active Choice for Colorectal Cancer Screening Outreach: A Randomized Clinical Trial (JAMA Network Open, 2019).

Tags
#pctGR, @Collaboratory1, @Shivan_Mehta

December 13, 2019: Reissuance of Funding Opportunity Announcement for HEAL Initiative/PRISM Coming January 2020

Posted on by Gina Uhlenbrauck

The National Center for Complementary and Integrative Health (NCCIH), with other NIH Institutes, Centers, and Offices, intends to reissue Funding Opportunity Announcement (FOA) HEAL Initiative: Pragmatic and Implementation Studies for the Management of Pain To Reduce Opioid Prescribing (PRISM) (UG3/UH3 Clinical Trial Optional).

This RFA solicits applications for phased cooperative research applications to conduct efficient, large-scale pragmatic or implementation trials to improve pain management and reduce the unnecessary use of opioid medications in the health care delivery setting. The re-issuance of the FOA will prioritize the following areas for pragmatic trials to integrate multimodal or multiple interventions that have demonstrated efficacy into health care systems or implement health care system changes to improve adherence to evidence-based guidelines:

  • Pain management in emergency departments, dental clinics, primary care, and hospitals
  • Chronic overlapping pain conditions
  • Pain management in individuals at risk of or with opioid use disorder
  • Pain management in those with co-occurring mental health disorders
  • Noncancer pain management in persons with medical comorbid conditions

The FOA is expected to be published in January 2020 with an expected application due date in March 2020.

The announcement is part of the NIH Heal (Helping to End Addiction Long-term) Initiative, which was created in April 2018 in an effort to speed scientific solutions for addressing the national opioid public health crisis.

 

November 22, 2019: It’s Time to Learn From Patients Like Mine (Nigam H. Shah, MBBS, PhD)

Posted on by Gina Uhlenbrauck

Speaker

Nigam H. Shah, MBBS, PhD
Associate Professor of Medicine
Stanford University

Topic

It’s Time to Learn From Patients Like Mine

Keywords

Clinical informatics; Clinical data warehouse; Aggregate patient data; Consult service; Cohort search engine

Key Points

  • The “Green Button” service consists of software, data, and personnel. Multiple datasets are used in the analysis, along with a human filter.
  • The search engine can find matching patients by searching across diagnosis and procedure codes, concepts extracted from clinical notes, laboratory test results, vital signs, as well as visit types and duration of inpatient stays, and then compare their outcomes.
  • Questions that remain include:
    • Does having such a consult service change patient outcomes?
    • How could we enable such consults nationwide?
    • Could we automate such analyses to be “always on”?
    • Could we get such a “curbside consult” from multiple health systems?
    • Could patients benefit from having access to such reports?

Discussion Themes

Could this technology be applied in emergent, critical patient settings where the care is more diagnostic, and where predictive modeling using health system data could be helpful?

What’s missing from the data that would improve accuracy or relevance? For example, social, demographic, and environmental data.

Read more about Stanford’s Green Button clinical informatics consult project.

Tags
#pctGR, @Collaboratory1

November 15, 2019: PCORnet: Health Plan Research Network Data Linkage and Patient Engagement with Patient-Powered Research Networks (Kevin Haynes, PharmD, MSCE)

Posted on by Gina Uhlenbrauck

Speaker

Kevin Haynes, PharmD, MSCE
Principal Scientist
HealthCore

Topic

PCORnet: Health Plan Research Network Data Linkage and Patient Engagement with Patient-Powered Research Networks

Keywords

Data linkages; PCORnet; Patient-powered research networks; Health plan research networks; Computable phenotypes

Key Points

  • One of the biggest challenges facing healthcare today is reducing gaps in evidence necessary to improve health outcomes. Research collaborations between health plans and patient-powered research networks (PPRNs) can help close this gap.
  • PCORnet enables linkages with patient groups through PPRNs, which include participating organizations and leadership teams of patients, advocacy groups, clinicians, academic centers, and practice-based research networks.
  • From the health plan perspective, postal mail outreach to members was more effective than email outreach around engaging patients in research opportunities.

Discussion Themes

When engaging with different patient-powered research networks, are there differences around common conditions compared with rare or stigmatized conditions?

What are participants told about the commercialization of findings, whether in terms of new treatments that might be identified, or the ways in which findings might affect health plans’ willingness to continue to cover certain treatments?

An essential aspect of collaboration is building and maintaining the trust of members in the research networks.

Read more about collaborations between PPRNs and health plans in a recent JAMIA publication and the PCORnet website.

Tags
#pctGR, @Collaboratory1, @KHaynes001

November 8, 2019: Lumbar Imaging with Reporting of Epidemiology: Initial Results and Some Lessons Learned (Jeffrey Jarvik, MD, MPH, Patrick Heagerty, PhD)

Posted on by Gina Uhlenbrauck

Speakers

Jeffrey (Jerry) G. Jarvik MD MPH
Professor, Radiology, Neurological Surgery and Health Services
Adjunct Professor, Pharmacy and Orthopedics & Sports Medicine
University of Washington

Patrick Heagerty, PhD
Professor and Chair
Department of Biostatistics
University of Washington

Topic

Lumbar Imaging with Reporting of Epidemiology: Initial Results and Some Lessons Learned

Keywords

Embedded pragmatic clinical trials; Radiology imaging; LIRE; Stepped-wedge; Cluster randomization; Epidemiology; Back pain

Key Points

  • The LIRE NIH Collaboratory Trial evaluated whether prevalence benchmark data inserted into lumbar spine imaging reports would reduce overall spine-related healthcare utilization for patients referred from primary care.
  • The inserted intervention text urges caution when interpreting the presence of certain findings that are common in normal, pain-free volunteers.
  • While the study team found no decrease in spine-related healthcare utilization for the overall cohort, there was a small but potentially important effect on reducing opioid prescriptions.

Discussion Themes

A characteristic of stepped-wedge study design is that it yields two comparisons: between-group comparisons (clinic A vs clinic B) and within-group comparisons. But temporal trends can have an impact and must be adjusted for in the analysis.

For what type of intervention would a stepped-wedge design be suitable?

The hope is for a wider dissemination about interventions where radiologic testing is done and incidental findings are common.

Read more about the LIRE NIH Collaboratory Trial.

Tags
#pctGR, #PragmaticTrials, @Collaboratory1

October 25, 2019: Real-World Evidence for Drug Effectiveness Evaluation: Addressing the Credibility Gap (Richard Willke, PhD)

Posted on by Gina Uhlenbrauck

Speaker

Richard Willke, PhD
Chief Science Officer
ISPOR

Topic

Real-World Evidence for Drug Effectiveness Evaluation: Addressing the Credibility Gap

Keywords

Real-world evidence; Non-interventional studies; Health economics; ISPOR; Transparency; Reproducibility

Key Points

  • ISPOR is an international, multistakeholder nonprofit dedicated to advancing health economics and outcomes research excellence to improve decision making for health globally.
  • Key characteristics of credible and useful real-world evidence include:
    • Careful data collection or curation
    • Appropriate analytic methods
    • Good procedural practices for transparent study process
    • Replicability and reproducibility
    • Informed interpretation
    • Fit-for-purpose application
  • For transparency, it is recommended that researchers declare their study to be an exploratory (hypothesis evaluation) study and post the study protocol and analysis plan on a public study registration site prior to conducting the study analysis.

Discussion Themes

A draft white paper, Improving Transparency in Non-Interventional Research, is available for comment until November 15, 2019.

Sharing all study implementation parameters and definitions provides clarity on what was actually done and enables reproduction with confidence.

Potential registries for non-interventional real-world evidence studies include:

Read more about ISPOR.

Tags
#pctGR, @Collaboratory1, @ISPORorg

October 4, 2019: Ascertaining Death and Hospitalization Endpoints: The TRANSFORM-HF Experience (Eric Eisenstein, DBA, Kevin Anstrom, PhD)

Posted on by Gina Uhlenbrauck

Speakers

Eric L. Eisenstein, DBA
Associate Professor in Medicine
Duke University School of Medicine

Kevin J. Anstrom, PhD
Professor of Biostatistics and Bioinformatics
Director of Biostatistics, Duke Clinical Research Institute
Duke University School of Medicine

Topic

Ascertaining Death and Hospitalization Endpoints: The TRANSFORM-HF Experience

Keywords

Clinical endpoints: Ascertaining death; Hospitalization; TRANSFORM-HF; National Death Index

Key Points

  • When patient deaths occur outside the care setting, the cause of death may not be reliably documented. For researchers, the challenges of measuring deaths include the lack of a national death data source and incomplete or hard-to-access sources.
  • The death identification and adjudication process differs for explanatory versus pragmatic trials, and has implications for how death endpoints are acquired and measured.
  • The TRANSFORM-HF pragmatic trial is comparing the effects of treatment strategies on long-term outcomes for hospitalized patients with heart failure. The primary study endpoint is all-cause mortality, which is ascertained and verified using a hybrid approach at the clinical site and call center, and includes searching the National Death Index data.

Discussion Themes

What are the tradeoffs in making endpoint ascertainment more simple?

If using a hybrid death data collection strategy, how are discrepancies adjudicated?

Use of call centers that coordinate follow-up patient contact and data collection is a valid approach that ensures a single point of contact for patients or proxies and care providers. This approach should also be supplemented with redundant data sources.

Read more in the Living Textbook about Using Death as an Endpoint and Inpatient Endpoints in Pragmatic Clinical Trials.

Tags
#pctGR, @Collaboratory1, @DCRINews