clinical trials

January 15, 2021: How CTTI & the Clinical Trials Community Have Risen to Meet the Challenge of the COVID-19 Pandemic (Pamela Tenaerts, MD, MBA; Sara Calvert, PharmD; Leanne Madre, JD, MHA)

Posted on by Gina Uhlenbrauck

Speakers

Pamela Tenaerts, MD, MBA
Executive Director
Clinical Trials Transformation Initiative

Sara Calvert, PharmD
Senior Project Manager
Clinical Trials Transformation Initiative

Leanne Madre, JD, MHA
Directory of Strategy
Clinical Trials Transformation Initiative

Topic

How CTTI & the Clinical Trials Community Have Risen to Meet the Challenge of the COVID-19 Pandemic

Keywords

Clinical Trials Transformation Initiative (CTTI); Best practices; COVID-19 treatment trials; RECOVERY Trial; Digital health technology; Decentralized clinical trials

Key Points

  • During 2020, CTTI conducted a series of intensive surveys, discussions, and collaborations across the clinical trials ecosystem, which shaped the creation of public webinars, a Best Practices for Conducting Trials During the COVID-19 Pandemic playbook, and a searchable, real-time AACT COVID-19 Trials Spreadsheet, among other efforts.

  • CTTI’s efforts helped the research community successfully navigate the challenges associated with adjusting trials that were underway when the pandemic hit, as well as launching new trials for COVID-19 treatments.

  • Among the best practices for conducting clinical trials during the pandemic: keep participants informed, perform ongoing risk-benefit assessment, adjust study startup and enrollment based on current risks, pivot to remote study visits, and switch to remote monitoring.

Discussion Themes

Because of the COVID-19 pandemic, there is more momentum toward digital health technology and decentralized clinical trials.

With the pandemic, we are seeing trial results go straight to practice, bypassing input from the clinical community at large—will this continue after the pandemic?

To what extent do you think the IRB/research oversight sphere might be permanently reshaped by the pandemic’s impact on the conduct of clinical trials? 

Read more about CTTI’s efforts around clinical trials during the COVID-19 pandemic.

Tags

#pctGR, @Collaboratory1

January 14, 2021: NIH Collaboratory COVID-19 Grand Rounds Continues With Clinical Trials Challenges in the COVID-19 Pandemic

Posted on by Damon Seils

Dr. Pamela TenaertsIn this week’s COVID-19 Grand Rounds session, Dr. Pamela Tenaerts of the Clinical Trials Transformation Initiative will present “How CTTI & the Clinical Trials Community Have Risen to Meet the Challenge of the COVID-19 Pandemic.” The Grand Rounds session will be held on Friday, January 15, at 1:00 pm eastern. Join the online meeting.

The NIH Collaboratory Coordinating Center is using its popular Grand Rounds platform to share late-breaking research and promote resources in support of clinical researchers affected by the COVID-19 public health emergency.

For previous COVID-19 Grand Rounds, and more news and resources related to the COVID-19 public health emergency, see the COVID-19 Resources page.

January 11, 2021: Study Snapshots for Four PRISM UH3 NIH Collaboratory Trials Are Available

Posted on by Gina Uhlenbrauck

Want to know more about the goals and challenges of the NIH Collaboratory’s PRISM NIH Collaboratory Trials? New downloadable handouts summarize the study aims, implementation lessons, and recent presentations from these innovative embedded pragmatic clinical trials (ePCTS).

The 2-page study snapshots are accessible from each NIH Collaboratory Trial page and the links below:

  • BackInAction: Pragmatic Trial of Acupuncture for Chronic Low Back Pain in Older Adults
  • FM TIPS: Fibromyalgia TENS in Physical Therapy Study
  • OPTIMUM: Group-based Mindfulness for Patients with Chronic Low Back Pain in the Primary Care Setting
  • NOHARM: Nonpharmacologic Options in Postoperative Hospital-based and Rehabilitation Pain Management

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 4, 2020: The Yale New Haven Health System as an Evidence Generation Ecosystem for Heart Failure (Tariq Ahmad, MD, MPH)

Posted on by Gina Uhlenbrauck

Speaker

Tariq Ahmad, MD, MPH
Director, Advanced Heart Failure Program
Yale School of Medicine and Yale New Haven Health

Topic

The Yale New Haven Health System as an Evidence Generation Ecosystem for Heart Failure

Keywords

Heart failure; Best practice alerts; Electronic health records; Risk prediction; Guideline-directed medical therapy; REVEAL-HF

Key Points

  • The REVEAL-HF study is a pragmatic randomized controlled trial testing an electronic alert system that informs clinicians about the 1-year predicted mortality for their patients with heart failure using validated data from the EHR.
  • It is important to use guideline-directed medical therapy for patients with heart failure. The hypothesis of the trial is that providing prognostic information for a patient with heart failure will lead to improved use of therapies and appropriate referral to subspecialties.

Discussion Themes

How did you get health system leadership and all of the clinicians, IT folks, and others to buy in to implementing your trial?

How can we make risk information valuable and actionable to healthcare providers?

Clinicians bring something to the table that an algorithm does not. It will be interesting to see how clinician behavior is affected by using the prediction models and interacting with the data.

Read more about the REVEAL-HF trial.

Tags

#pctGR, @Collaboratory1

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 13, 2020: Pragmatic and Explanatory Attitudes to RCTs: Using the PRECIS-2 Tool to Describe the Design of the MyTEMP Trial (Ahmed Al-Jaishi, PhD; Amit Garg, MD, PhD, Merrick Zwarenstein, MBBCh, MSc, PhD)

Posted on by Gina Uhlenbrauck

Speakers

Ahmed Al-Jaishi, PhD (candidate)
Health Research Methodology
McMaster University

Amit Garg, MD, PhD
Professor, Department of Medicine
Epidemiology & Biostatistics
Western University, London, Canada
Senior Core Scientist, Site Director, and Program Lead
ICES Western Kidney Dialysis & Transplantation Research Program, London, Canada
Nephrologist, London Health Sciences Centre
Victoria Hospital, London, Canada

Merrick Zwarenstein, MBBCh, MSc, PhD
Professor, Department of Family Medicine
Epidemiology & Biostatistics
Western University, London, Canada
Senior Core Scientist
ICES Western Primary Care & Health Systems Research Program, London, Canada

Topic

Pragmatic and Explanatory Attitudes to RCTs: Using the PRECIS-2 Tool to Describe the Design of the MyTEMP Trial

Keywords

Pragmatic trials; PRECIS-2; Canadian Institutes of Health Research (CIHR); Study design; External validity

Key Points

  • Randomized controlled trial (RCT) intentions (attitudes) can be described as: pragmatic intentions provide evidence for decision-makers to choose between interventions, whereas explanatory intentions test a hypothesis about a mechanism.
  • Pragmatic and explanatory trials are not a dichotomy; a good trial design matches the intention of the trial.
  • Trials can be designed with both internal validity and external validity.
  • Different trial intentions, design choices, conclusions, and recommendations lead to different usefulness for decision-making.

Discussion Themes

Can you comment on the retrospective use of PRECIS-2 to dichotomize trials as pragmatic or explanatory?

What suggestions do you have to improve the labeling of trials as explanatory versus pragmatic, since both types are randomized controlled trials (RCTs)?

Read more about the PRECIS-2 tool and the CONSORT statement for pragmatic trials.

Tags

#pctGR, @Collaboratory1

Podcast November 6, 2020: Lessons from Virtual Trials in Time of a Pandemic (David Boulware, MD, MPH)

Posted on by Gina Uhlenbrauck

This episode of the NIH Collaboratory Grand Rounds podcast follows the conversation between Dr. David Boulware and Dr. Adrian Hernandez as they discuss the complexities of conducting virtual clinical trials during the COVID-19 pandemic.

Click on the recording below to listen to the podcast.

 

Want to hear more? View the full Grand Rounds presentation.

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Read the transcript.

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

October 28, 2020: NIH Collaboratory COVID-19 Grand Rounds Continues With the Minnesota Hydroxychloroquine Experience

Posted on by Damon Seils

Photo of Dr. David BoulwareIn this week’s COVID-19 Grand Rounds session, Dr. David Boulware of the University of Minnesota will present “Lessons From Virtual Trials in the Time of a Pandemic: Minnesota Hydroxychloroquine Experience.” The Grand Rounds session will be held on Friday, October 30, at 1:00 pm eastern. Join the online meeting.

The NIH Collaboratory Coordinating Center is using its popular Grand Rounds platform to share late-breaking research and promote resources in support of clinical researchers affected by the COVID-19 public health emergency.

For previous COVID-19 Grand Rounds, and more news and resources related to the COVID-19 public health emergency, see the COVID-19 Resources page.