February 18, 2022: Building a Resource: The Process of Developing a Trans-stakeholder Framework to Enable Pediatric Drug Development (Perdita Taylor-Zapata, MD)

Speaker

Perdita Taylor-Zapata, MD
Best Pharmaceuticals for Children Act (BPCA) Program Lead and NICHD Program Officer
Obstetric and Pediatric Pharmacology and Therapeutics Branch
National Institute of Child Health and Human Development

Keywords

NIH Best Pharmaceuticals for Children Act; Pediatric Trial Network; Trial design; Pediatric drug development

Key Points

  • The current model for pediatric drug development can be slow and neglect neonates and rare pediatric conditions.
  • The NIH Best Pharmaceuticals for Children Act (BPCA) allows the NIH to conduct clinical trials with off-patent drugs in children.
  • Goals of the BPCA program include developing novel trial designs and including diverse and understudied populations.
  • A new framework to enable pediatric drug development could identify resources to assist in drug development, identify areas in need of further research, provide a pathway for integrating approaches, and connect pediatric researchers.
  • The BPCA went through a rigorous systematic approach to develop a comprehensive resource listing for best practices for pediatric drug trials.

Discussion Themes

Most data collected through the opportunistic model presented is PK data to determine dosing so that a more traditional drug trial can be conducted in the future.

With the right infrastructure in place, such as the Pediatric Trials Network, can substantially improve time to conduct trials.

 

Read more about the BPCA and their commitment to diversity in pediatric drug trials.

 

Tags

#pctGR, @Collaboratory1

Grand Rounds September 10: Effect of Salt Substitution on Cardiovascular Events and Death

Speakers:
Bruce Neal, MB ChB, PhD, FRCP, FAHA, FAHMS
Executive Director, George Institute Australia
Professor of Medicine, UNSW Sydney
Honorary Professor, Sydney Medical School, University of Sydney
Professor of Clinical Epidemiology, Imperial College London

Topic: Effect of Salt Substitution on Cardiovascular Events and Death
Date: Friday, September 10, 2021, 1:00-2:00 p.m. ET

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August 20, 2021: Fluvoxamine for Early Treatment of COVID-19: The STOP COVID Clinical Trials (Eric Lenze, MD)

Speaker

Eric Lenze, MD
Wallace and Lucille K. Renard Professor of Psychiatry & Anesthesiology
Washington University School of Medicine, St. Louis

Topic

Fluvoxamine for Early Treatment of COVID-19: The STOP COVID Clinical Trials

Keywords

COVID-19 treatment; STOP COVID; Fluvoxamine; Repurposed therapy; Decentralized clinical trial

Key Points

  • While using Fluvoxamine to treat anxiety in patients with Wolfram Syndrome, Dr. Reiersen from Washington University, St. Louis learned that Fluvoxamine reduced inflammation and interfered with viral function.
  • Dr. Lenze, Dr. Reiersen, and Dr. Mattar began the preliminary pragmatic, non-contact, but high-touch STOP COVID trial in April 2020.
  • Results from the preliminary study showed that 0% of patients experienced clinical deterioration in the Fluvoxamine group while 8.3% of patients in the placebo group deteriorated.
  • The STOP-COVID 2 study was a decentralized, hub-and-spoke clinical trial conducted across the US and 2 provinces of Canada.
  • The Data Safety Monitoring Board(DSMB) for the study recommended an early stop for futility due to a low case rate and difficulty recruiting patients.

Discussion Themes

What outcome variables should we be using in COVID-19 studies? A binary outcome measure may limit the power of the study. When possible, continuous outcomes provide more statistical power and come closer to representing real-life outcomes.

What role should primary care providers play in clinical studies?

Read more about Dr. Lenze’s work with the STOP COVID trials.

Tags

#pctGR, @Collaboratory1

August 13, 2021: Got anything for this cough? Outpatient Treatment Trials in the Time of COVID (Davey Smith MD)

Speaker

Davey Smith, MD
Protocol Co-Chair ACTIV-2
Professor of Medicine, UC San Diego

Topic

Got anything for this cough? Outpatient Treatment Trials in the Time of COVID

Keywords

COVID-19 treatment; Adaptive Platform Trial; ACTIV-2 Trial; Antiviral therapy

Key Points

  • An Adaptive Platform Trial is a trial in which multiple drugs can be tested at the same time to increase efficiency and transition seamlessly from a Phase II to a Phase III trial.

  • An interim analysis determines if a drug is moved on to trial Phase III.

  • The trials adapt as researchers learn more about COVID-19 and potential treatments.

  • Monoclonal antibody therapies showed promise initially, but COVID-19 variants have lessened their efficacy.

Discussion Themes

The ACTIV-2 trial initially focused on using antivirals, specifically monoclonal antibodies, to achieve a reduced viral load as an endpoint measure.  Changes were made to this endpoint measure as it became clear that the FDA would issue an Emergency Use Authorization only for a decrease in hospitalization and death.  

To adjust to the rapidly changing COVID-19 landscape of changing therapeutics, vaccines, and variants, the ACTIV-2 team remained nimble and flexible with the ability to act quickly when presented with new situations and information.  

Read more about the Active-2 Trial.

Tags

#pctGR, @Collaboratory1, @DaveySmithMD

Grand Rounds August 20: Fluvoxamine for Early Treatment of COVID-19: The STOP COVID Clinical Trials

Speaker:
Eric Lenze, MD
Wallace and Lucille K. Renard Professor of Psychiatry
Washington University School of Medicine, St. Louis

Topic: Fluvoxamine for Early Treatment of COVID-19: The STOP COVID Clinical Trials
Date: Friday, August 20, 2021, 1:00-2:00 p.m. ET

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Grand Rounds September 24: Enabling Patient-Reported Outcome Measures in Clinical Trials, Exemplified by Cardiovascular Trials

Speakers:
Theresa Coles, PhD
Assistant Professor
Department of Population Health Sciences
Duke University School of Medicine

Kevin Weinfurt, PhD
Professor and Vice Chair of Research
Department of Population Health Sciences
Duke University School of Medicine

Topic: Enabling Patient-Reported Outcome Measures in Clinical Trials, Exemplified by Cardiovascular Trials
Date: Friday, September 24, 2021, 1:00-2:00 p.m. ET

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January 24, 2020: Special Grand Rounds Series Begins January 31st With ‘Pragmatic Clinical Trials: How Do I Start?’

Join us Friday, January 31st, for “Pragmatic Clinical Trials: How Do I Start?”—the first in our special 5-part Grand Rounds series focused on the Living Textbook. NIH Collaboratory investigators Dr. Greg Simon and Dr. Lesley Curtis will offer guidance on what researchers need to begin an embedded pragmatic clinical trial, including:

  • Developing your research question
  • Establishing close partnerships with participating healthcare system leaders and staff
  • Putting together your trial team

Visit our events calendar here for details about how to join this webinar. See below for the full schedule of sessions and a special message from Dr. Kevin Weinfurt.

Full Schedule of sessions

Title Speakers Date
Pragmatic Clinical Trials:
How Do I Start?
Greg Simon, MD, MPH, KPWHRI

Lesley Curtis, PhD, Duke University

January 31, 2020
Preparing for the Unknown: Conducting Pragmatic Research in Real-World Contexts Jerry Jarvik, MD, MPH, University of Washington

Vince Mor, PhD, Brown University

Leah Tuzzio, MPH, KPWHRI

February 28, 2020
Tips for Putting Together a Successful PCT Grant Application Wendy Weber, ND, PhD, MPH, NCCIH March 27, 2020
Choosing What to Measure
and Making It Happen: Your Keys to Pragmatic Trial Success
Rachel Richesson, PhD, MPH, Duke University

Emily O’Brien, PhD, FAHA, Duke University

May 1, 2020
Demystifying Biostatistical Concepts for Embedded Pragmatic Clinical Trials Liz Turner, PhD, Duke University

Patrick Heagerty, PhD, University of Washington

David Murray, PhD, NIH

June 19, 2020

 

December 20, 2019: Embedded Pragmatic Clinical Trials: From Idea to Funding to Implementation: A Living Textbook Grand Rounds Series

Coming in 2020, the NIH Collaboratory will host a 5-part Grand Rounds Series focused on the Living Textbook. The series will feature Collaboratory expert speakers guiding the audience through the life cycle of an ePCT by way of the textbook—from idea to funding to implementation.

The series begins January 31 with “Pragmatic Clinical Trials: How Do I Start?” and will end June 19 with “Demystifying Biostatistical Concepts for Embedded Pragmatic Clinical Trials.”

See below for the full schedule of sessions and a special message from Dr. Kevin Weinfurt.

Full Schedule of sessions

Title Speakers Date
Pragmatic Clinical Trials:
How Do I Start?
Greg Simon, MD, MPH, KPWHRI

Lesley Curtis, PhD, Duke University

January 31, 2020
Preparing for the Unknown: Conducting Pragmatic Research in Real-World Contexts Jerry Jarvik, MD, MPH, University of Washington

Vince Mor, PhD, Brown University

Leah Tuzzio, MPH, KPWHRI

February 28, 2020
Tips for Putting Together a Successful PCT Grant Application Wendy Weber, ND, PhD, MPH, NCCIH March 27, 2020
Choosing What to Measure
and Making It Happen: Your Keys to Pragmatic Trial Success
Rachel Richesson, PhD, MPH, Duke University

Emily O’Brien, PhD, FAHA, Duke University

May 1, 2020
Demystifying Biostatistical Concepts for Embedded Pragmatic Clinical Trials Liz Turner, PhD, Duke University

Patrick Heagerty, PhD, University of Washington

David Murray, PhD, NIH

June 19, 2020

 

We hope you will join us to learn more about the fundamentals for designing and launching a successful ePCT. Please download the flyer for the Living Textbook Grand Rounds series and share with your colleagues and institution.

December 18, 2019: National Institute on Aging IMPACT Collaboratory Launches Grand Rounds Series and New Website

The National Institute on Aging (NIA) IMbedded Pragmatic Alzheimer’s disease (AD) and AD Related Dementias (AD/ADRD) Clinical Trials (IMPACT) Collaboratory has launched a new website highlighting the project.

The IMPACT Collaboratory recently invited applications for pilot grant funding for projects that aim to generate preliminary data necessary to design and conduct full-scale embedded pragmatic clinical trials of nonpharmacologic interventions for persons living with Alzheimer’s disease and related dementias and/or their caregivers. Pilot grants will help IMPACT Collaboratory’s mission to build the nation’s capacity to conduct pragmatic clinical trials of interventions embedded within health care systems for people living with dementia and their caregivers.

Since the IMPACT Collaboratory started this September, the program has been hard at work launching its new website, as well as a series of specialized Grand Rounds focused on the topic of pragmatic research in the area of Alzheimer’s disease and related dementias.

IMPACT Collaboratory Grand Rounds occur the 3rd Thursday of each month at 12 noon ET. Join the next webinar on Thursday, December 19, “Stepped Wedge Cluster Trials: What, How, And When?,” by Monica Taljaard, PhD, Senior Scientist, Ottawa Hospital Research Institute and Associate Professor, University of Ottawa from the IMPACT Collaboratory Design and Statistics Core.

Following in the footsteps of the NIH Collaboratory, the IMPACT Collaboratory has also launched a podcast series to correspond with its Grand Rounds Series. Both the Grand Rounds webinar recordings and accompanying podcasts for all the sessions are available on the website.

Those interested in getting updates about the IMPACT Collaboratory can also join the program’s mailing list .

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

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.