patient-centered outcomes research

January 29, 2021: The COVID-19 Citizen Science Study (Gregory M. Marcus, MD, MAS)

Posted on by Gina Uhlenbrauck

Speaker

Gregory M. Marcus, MD, MAS
Professor of Medicine
University of California, San Francisco

Topic

The COVID-19 Citizen Science Study

Keywords

Eureka digital research platform; COVID-19 infections; Mobile health; Risk factors; Citizen science; Geolocation; Participant engagement

Key Points

  • Eureka is an NIH-supported digital research platform built to use mobile health technology to combat the novel coronavirus, focusing on identifying risk factors for infection, transmission, and severity of disease that may inform best practices.
  • Eureka is also intended to serve as a platform for collaborating investigators to answer their own research questions.
  • As a citizen science project, any adult with a smartphone can participate and contribute information. Nearly 50,000 participants have enrolled to date.

Discussion Themes

The COVID-19 Citizen Science website provides data visualizations that show how people answered the survey questions. A study blog through the app is used to translate key information to participants.

Will this project be collaborating with other entities that are potentially overlapping in terms of their COVID-19 applications?

Best practices in mHealth include keeping it simple, avoiding over-explaining, listening to participants, and providing a feedback pathway.

Learn more about the Eureka platform. The smartphone app is available under the name “UCSF Eureka Research.”

Tags

#pctGR, @Collaboratory1  

January 5, 2021: New Video Collection Highlights Advances in Electronic Health Records for Pragmatic Research

Posted on by Damon Seils

We are pleased to announce our new collection of short educational videos that highlight advances in the use of electronic health records (EHRs) in pragmatic clinical trials.

The videos are drawn from our 2020 EHR Workshop Grand Rounds Series, “Advances at the Intersection of Digital Health, Electronic Health Records and Pragmatic Clinical Trials.” The series highlighted advances in digital health, new approaches and evolving standards for EHRs, and implications for researchers conducting pragmatic trials. Experts discuss the evolving regulatory context for EHRs, national policy priorities, and innovative uses of EHRs in several NIH Collaboratory Trials.

Video topics include:

Visit the EHR Workshop Video Modules page for access to all of the new videos.

December 18, 2020: The SAMSON Trial: An N-of-1 Trial of Statin, Placebo, and No Treatment to Assess Patient Symptoms (Matthew Shun-Shin, BMBCh, PhD)

Posted on by Gina Uhlenbrauck

Speaker

Matthew Shun-Shin, BMBCh, PhD
Imperial College London

Topic

The SAMSON Trial: An N-of-1 Trial of Statin, Placebo, and No Treatment to Assess Patient Symptoms

Keywords

Statins; SAMSON; N-of-1 study design; Side effects; Placebo; Nocebo effect

Key Points

  • Studies have shown that more than half of patients abandon statin medications completely within 2 years. Yet, placebo-controlled trials do not show excess withdrawals in the statin arm.
  • The SAMSON trial was a double-blind, three-group, N-of-1 trial in which participants were randomized to receive 12 one-month medication bottles in a computer-generated sequence. Four bottles contained statin tablets, 4 contained placebo tablets, and 4 were empty. The aim was to determine, for an individual participant, to what extent their symptoms were associated with the statin. Participants used a smartphone to rate the severity of their symptoms every day.
  • After 1 year, personalized study results were shared with each participant (n=60). Six months later, the study team evaluated whether participants were able to restart a statin.

Discussion Themes

The most important message from the SAMSON trial is that side effects from statin tablets are very real, but they are mainly caused by the act of taking the tablets, not the statin contained within them.

Because this N-of-1 design incorporated a period with no medication, participants could see as clearly as the study team the powerful magnitude of the “nocebo effect.” This resulted in half the participants successfully restarting statins.

Read more about the SAMSON trial in a letter from study investigators in the New England Journal of Medicine and an interview in Medscape.

Tags

#pctGR, @Collaboratory1

December 11, 2020: PREVENTABLE: Starting a Pragmatic Trial in a Pandemic (Karen Alexander, MD; Schuyler Jones, MD)

Posted on by Gina Uhlenbrauck

Speakers

Karen P. Alexander, MD
Professor of Medicine/Cardiology
Duke University School of Medicine

W. Schuyler Jones, MD
Associate Professor of Medicine/Cardiology
Duke University School of Medicine

Topic

PREVENTABLE: Starting a Pragmatic Trial in a Pandemic

Keywords

Heart disease; Dementia; Cholesterol; Statins; Pragmatic trial; National Heart, Lung, and Blood Institute; National Institute on Aging; Mild cognitive impairment

Key Points

  • More than half of older adults (age 75+) in the United States have cognitive impairment or frailty or both.
  • PREVENTABLE is one of the largest research studies in older adults. The purpose is to learn if taking a statin could help older adults live well for longer by preventing dementia, functional decline, or heart disease.
  • The study medication (statin) is mailed directly to participants’ homes. Study sites are part of the research infrastructure within PCORnet and Veterans Affairs (VA).
  • Pragmatic aspects of the trial include the study question: Do statins work in practice for outcomes that matter most to older adults? Also, enrollment is telehealth-enabled, and consent is electronic (e-Consent).

Discussion Themes

How did the study team work with IT to make the EHR system changes (eg, tools and workflow) needed to conduct the research?

Are older participants able to interact well with the technology used in the trial?

Are there special considerations embedded within the study to encourage the retention of study participants?

Read more about the PREVENTABLE study.

Tags

#pctGR, @Collaboratory1

December 8, 2020: PCORI to Issue Funding Announcement for Pragmatic Clinical Studies to Evaluate Patient-Centered Outcomes

Posted on by Damon Seils

The Patient-Centered Outcomes Research Institute (PCORI) will issue a funding announcement in January for pragmatic clinical studies to evaluate patient-centered outcomes. A total of $90 million will be available in the funding cycle to support individual awards of up to $10 million in direct costs with a maximum project duration of 5 years.

More from the preannouncement:

PCORI seeks to fund clinical trials, large simple trials, or large-scale observational studies that compare two or more alternatives for addressing prevention, diagnosis, treatment, or management of a disease or symptom; improving healthcare system-level approaches to managing care; or eliminating health or healthcare disparities. Randomized study designs are strongly encouraged but not required.

Proposed studies must address critical clinical choices faced by patients, their caregivers, clinicians, or delivery systems. They must involve broadly representative patient populations and be large enough to provide precise estimates of hypothesized effectiveness differences and to support evaluation of potential differences in treatment effectiveness in patient subgroups.

The PCORI funding announcement will open on Tuesday, January 5, 2021, with more information about the funding opportunity.

November 20, 2020: Drug Development in Kidney Disease: Proceedings from a Multi-Stakeholder Panel (Daniel Edmonston, MD)

Posted on by Gina Uhlenbrauck

Speaker

Daniel Edmonston, MD
Medical Instructor
Duke University School of Medicine

Topic

Drug Development in Kidney Disease: Proceedings from a Multi-Stakeholder Panel

Keywords

Chronic kidney disease (CKD); Dialysis; Evidence-based medicine; Electronic health records; Think Tank

Key Points

  • Only 5 percent of treatment recommendations for kidney disease reach a Grade A level of evidence.
  • In 2019, the DCRI convened a panel to address the urgent need for evidence-based treatments for kidney disease. “Accelerating Drug Development for Chronic Kidney Disease and End-Stage Renal Disease” included stakeholders from regulatory agencies, kidney societies, patient advocacy organizations, academia, and industry.
  • Key discussions explored the uses of interconnected data and site research networks, pragmatic and adaptive trial designs, robust surrogate endpoints, real-world data, and methods to improve the generalizability of trial results and uptake of approved drugs for kidney-related diseases.

Discussion Themes

Since there is more than one therapeutic agent showing promise for CKD, how should evidence be generated to understand the right combination of agents?

Could the principles laid out in the FDA Patient Focused Drug Development guidance be applied in these trials to ensure the patient perspective is included?

What are your thoughts on whether to focus on enrolling early CKD patients—some of whom will not progress—or patients later in the course of disease, when disease modification may be more challenging?

Read more about the Think Tank in a special report in the American Journal of Kidney Disease.

Tags

#pctGR, @Collaboratory1

November 6, 2020: Appendectomy Versus Antibiotics for Appendicitis–Early Results from the CODA Trial (David R. Flum, MD, MPH)

Posted on by Gina Uhlenbrauck

Speaker

David R. Flum
Professor, Surgery, Health Services, and Pharmacy
Associate Chair for Research, Department of Surgery
University of Washington

Topic

Appendectomy Versus Antibiotics for Appendicitis–Early Results from the CODA Trial

Keywords

Appendectomy; Antibiotic therapy; CODA Collaborative; Comparative effectiveness research; Patient-centered outcomes research; Pragmatic clinical trial; Noninferiority; PCORI

Key Points

  • The CODA (Comparing Outcomes of Drugs and Appendectomy) Collaborative involved 25 sites across 14 states. The Co-PIs included surgeons and emergency medicine physicians.
  • The CODA pragmatic trial aimed to inform the health decision for appendicitis treatment by asking two research questions:
    • Are antibiotics as effective as appendectomy for appendicitis?
    • Which patients are most likely to have a successful outcome with antibiotics?
  • Instead of appendectomy, the intervention group received antibiotics intravenously for 24 hours followed by pills for a  total of 10 days. The primary outcome in the trial was 30-day health status, as assessed with the European Quality of Life-5 Dimensions questionnaire.
  • The patients who received antibiotics cared about outcomes such as “Am I going to feel better,” followed by “Is it safe,” and “Will I return to work sooner?”

Discussion Themes

Antibiotics for appendicitis can be a good choice for some but not all. Decision-makers must weigh patient characteristics, preferences, and circumstances.

Can you comment on the relative “disappearance” of appendicitis during the COVID-19 pandemic? 

Based on your results, how would you counsel a patient (or parent) in the emergency department with a diagnosis of appendicitis?

Will there eventually be a biomarker that’s predictive for appendectomy?

Read more about CODA results in the New England Journal of Medicine.

Tags

#pctGR, @Collaboratory1

November 5, 2020: NIH HEAL Initiative Common Data Elements Published in the Living Textbook

Posted on by Karen Staman

Logo for the NIH HEAL InitiativeThe Helping to End Addiction Long-Term Initiative℠, or NIH HEAL Initiative℠, has created common data elements (CDEs) to facilitate cross-study comparisons for pain, improve interoperability of findings for patient-reported outcomes, and compare results across trials. The core CDEs were published recently in the NIH Collaboratory’s Living Textbook. They include CDEs for adult acute pain, adult chronic pain, and pediatric acute and chronic pain, and represent patient-reported outcomes that HEAL Initiative clinical trials are required to collect.

“Having common measures to capture the patient’s perspective regarding pain will enable researchers to compare meaningful data across pain conditions, in diverse populations, and in multiple research studies, including in pragmatic clinical research. This should greatly increase our understanding of pain and how best to treat it.”

      —Emily O’Brien, PhD, co–principal investigator of the NIH Collaboratory Patient-Centered Outcomes Core

The NIH Collaboratory serves as the resource coordinating center for 6 NIH Collaboratory Trials funded through the Pragmatic and Implementation Studies for the Management of Pain to Reduce Opioid Prescribing (PRISM) program, a component of the NIH HEAL Initiative:

Because the PRISM studies are pragmatic clinical trials embedded in healthcare systems, it may not be feasible for investigators to collect all the required CDEs, as the trials need to be incorporated into clinical workflow with as little burden as possible. The NIH Collaboratory’s Patient-Centered Outcomes Core Working Group will work with the NIH Collaboratory Trials to understand and document which patient-reported outcome measures are collected and the perceived burden associated with collecting them.

Learn more in the Choosing PRO Measures chapter of the Living Textbook.

October 2, 2020: Driving Toward Health Impact: Our Journey and Path Forward (Nakela Cook, MD, MPH)

Posted on by Gina Uhlenbrauck

Speaker

Nakela L. Cook, MD, MPH
Executive Director
Patient-Centered Outcomes Research Institute (PCORI)

Topic

Driving Toward Health Impact: Our Journey and Path Forward

Keywords

PCORI; Health outcomes; Health disparities; Patient-centered research; Comparative clinical effectiveness; COVID-19; Maternal health

Key Points

  • Despite improvements in health, disparities and variation in care remain—especially underscored by the unprecedented COVID-19 pandemic.
  • PCORI funds studies that can help patients and those who care for them make better informed healthcare choices.
  • PCORI has funded the HERO Registry to assess COVID-19 exposure responses and outcomes within the community of healthcare workers. Other research support is targeted toward vulnerable populations, healthcare delivery, and emerging health trends.
  • With PCORI 2.0, new funding will be available for large-scale, high-impact comparative effectiveness trials. Each study will include an initial phase to determine the feasibility and viability of the study and maximize the likelihood of success during the full-scale phase.

Discussion Themes

What are your observations regarding COVID-19’s impact on health, healthcare, and research that you think will be critical for PCORI's strategy? What will bring about the necessary innovations to the system?

Addressing maternal health disparities is a high priority in PCORI 2.0. The next phase will also expand PCORI’s role in collecting and generating relevant evidence and focusing on a deliberate and transparent process for implementation.

How will PCORI broaden its reach to reduce outcome disparities among people with disabilities?

Read more about PCORI.

Tags

#pctGR, @Collaboratory1

September 30, 2020: PCORI’s Nakela Cook to Offer a Look Ahead in PCT Grand Rounds

Posted on by Damon Seils

Photograph of Dr. Nakela CookDr. Nakela Cook, executive director of the Patient-Centered Outcomes Research Institute (PCORI), will present “Driving Toward Health Impact: Our Journey and Path Forward” during the NIH Collaboratory’s PCT Grand Rounds.

The Grand Rounds session will be held on Friday, October 2, at 1:00 pm eastern. Join the online meeting.

Since 2010, PCORI has funded more than $2 billion in research to help patients, caregivers, and clinicians make informed healthcare decisions and to improve healthcare delivery and outcomes.