Skip to main content
PAS 2024 Meeting Main banner
Icon Legend
This session is not in your schedule.
This session is in your schedule. Click again to remove it.
Presentation Icons
Pre-Conference Ticketed Event
Pre-Conference Ticketed Event
APA Award Winner
APA Award Winner
APS Award Winner
APS Award Winner
ASPN Award Winner
ASPN Award Winner
SPR Award Winner
SPR Award Winner
On Demand Presentation
On Demand Presentation
Poster Icons
SPR Award Winner
SPR Award Winner
Back
Evaluation ViewAttendees 65
Hot Topic Symposia

Clinical Research

EHR/Medical Informatics

Epidemiology

Nephrology

Trainee
Session Type: Hot Topic Symposia

Using big ideas and big data to study small numbers of small patients

Sunday, May 5, 2024
3:30 PM – 5:00 PM ET
Location: Metro Toronto Convention Centre: 701 B
CE: Live CME: 1.5

Selected By: ASPN
Disclosure(s):
Rulan Parekh, MD,MS: I do not have any relevant financial / non-financial relationships with any proprietary interests.
Amy Strong, MD MSCE: I do not have any relevant financial / non-financial relationships with any proprietary interests.

Session

Description: Clinical practice in pediatric nephrology is driven largely by extrapolating data from adult trials and expert opinion. While randomized controlled trials (RCTs) remain the gold standard, these may not be feasible in rare diseases and/or with small patient numbers. New approaches may circumvent some of the challenges associated with studying pediatric kidney diseases. This session aims to highlight how innovative methods, such as big data repositories that are easily accessible, cost-efficient and cover large populations can be used to generate high-quality evidence. Associated methodological issues and limitations of conducting big data research in children will be discussed. Additional novel approaches to studying smaller populations and rare diseases (such as N-of-1 trials) will also be discussed.

Learning Objectives:

  • Discuss the history, challenges, and factors influencing the success of clinical trials in pediatric kidney disease and the state of current efforts in the field.
  • Provide an overview of big data sources, types of studies conducted using big data, and advantages and limitations of using big data in clinical research.
  • Describe innovative trial designs (such as N-of-1 trials, clinical trial emulation, cluster randomized trials and cross-over trials) and their application in pediatrics, including pediatric nephrology.

Presentations:

  • 3:30 PM – 3:55 PM ET
    The state of clinical trials in pediatric nephrology

    Speaker: Debbie S. Gipson, MD, MS (she/her/hers) – National Institutes of Health / NIDDK

  • 3:55 PM – 4:20 PM ET
    Overview of using big data in clinical research

    Speaker: Rahul Chanchlani, MD, MSc, FRCPC, FASN (he/him/his) – McMaster Children's Hospital

  • 4:20 PM – 4:45 PM ET
    Innovative trial designs – big ideas for small numbers

    Speaker: Joyce P. Samuel, MD, MS – McGovern Medical School/UT Houston