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Archive | Pediatric Pharmacoepidemiology

Making Real-World Evidence (RWE) Work for Kids

The increased attention given to Real-World Evidence is a welcome development

The National Academies of Science held a workshop last October, “Real-World Evidence Generation and Evaluation of Therapeutics—A Workshop”, bringing together key thinkers to examine opportunities and challenges for incorporating real-world evidence into evaluation of medical products. Regulatory experts and FDA scientists followed-up with a cogent discussion of RWE and its use in the regulatory setting published in the New England Journal of Medicine, “Real-World Evidence — What Is It and What Can It Tell Us?” They reported that “the FDA is developing guidance on the use of ‘real-world evidence’ — health care information from atypical sources, including electronic health records, billing databases, and product and disease registries — to assess the safety and effectiveness of drugs and devices.”

Real-world evidence and the regulatory process

NAS workshop reviews RWE

Kids stand to benefit

Real-world evidence is of special importance to children because we can’t always conduct randomized clinical trials in pediatric populations. Real-world evidence, from large databases, electronic health records, and similar sources, may be the only evidence we have about medications or devices used by children. All the more reason to make sure that real-world evidence meets the unique needs we have when studying children: adequate sample sizes for all age sub-groups, critical data such as birth date, birth weight, and gestational age, ability to link to parental (and even sibling) data, and linkage with school performance metrics.

As efforts increase to address the challenge of developing high-quality RWE, we must consider and address the special considerations and challenges in using RWE to answer questions about the effects of medications in children. Asking these questions now will ensure that advances in the use of RWE will benefit children as well as other sectors of the population.

See Tamar Lasky’s editorial in the journal Drugs – Real World Outcomes, “In the Real-World, Kids Use Medications and Devices”.

Pediatric Pharmacoepidemiology at ICPE 2017 Annual Meeting

For the third year, the ISPE annual meeting (ICPE 2017 Montreal) will offer the pre-conference course in Pediatric Pharmacoepidemiology.

Mark your calendars for Sunday, August 27, 2017 (register here).

Brief Overview of Course

The increasing use of medications by children and the history of excluding children from clinical trials have created the need for pediatric pharmacoepidemiology, a sub-specialty within pharmacoepidemiology. Unique challenges in studying children, accessing data, defining outcomes, and designing studies require specialized methodologic skills and operational approaches. This half-day course will introduce participants who have a good understanding of pharmacoepidemiology to the specialized methodologic and operational approaches used to study medications in children.

Pediatric Pharmacoepidemiology

Age is not a simple variable when studying children

Educational Objectives

The course will be taught in an interactive format with ample opportunities for questions and discussion. Regulatory issues around use of medications in children will be highlighted throughout the course. Participants will gain an understanding of key issues in pediatric pharmacoepidemiology including:

  • An overview of pediatric pharmacoepidemiology and how it differs from pharmacoepidemiology in older age groups
  • Study design and databases to monitor safety
  • Defining, measuring and validating outcomes in pediatric pharmacoepidemiology
  • Variables critical to pediatric pharmacoepidemiology such as month of birth, seasonality, growth and development

Target Audience

ICPE attendees interested in gaining a basic understanding of the need for and approaches to pediatric pharmacoepidemiology.

Course Faculty/Presentations

  • Tamar Lasky, PhD Owner, MIE Resources Why Pediatric Pharmacoepidemiology? Age Sub-groups in Children: Methodologic Considerations
  • Alan Kinlaw, PhD Post-doctoral Research Fellow, Sheps Center for Health Services Research, University of North Carolina at Chapel Hill Leveraging granular birthdate information for studies of young children
  • Rachel E. Sobel, DrPH Senior Director, Epidemiology, Worldwide Research and Development / Pfizer Inc. Monitoring Drug Safety: Study Design, Data Sources and Case Study
  • Timothy Beukelman, MD, MSCE Associate Professor of Pediatrics, Division of Pediatric Rheumatology, Department of Pediatrics, University of Alabama at Birmingham The Need for Validation in Pediatric Outcomes
  • Daniel B. Horton, MD, MSCE Assistant Professor of Pediatrics and Epidemiology, Rutgers University Growth and Development: Variables of Particular Importance in Pediatric Pharmacoepidemiology

 

 

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