Seminar Series: Dr. Beth Potter
Generating evidence to improve health care and outcomes for children with rare genetic diseases: opportunities and challenges
Beth Potter, PhD
Professor
School of Epidemiology & Public Health
University of Ottawa
Short Biography:
Beth Potter is a Professor of Epidemiology and Public Health and the University Research Chair in Health Services for Children with Rare Diseases at the University of Ottawa; and an Affiliate Investigator at the Children’s Hospital of Eastern Ontario Research Institute. Dr. Potter completed her PhD in Epidemiology at the University of Western Ontario (Western University) in 2003 and joined the University of Ottawa as a faculty member in 2007. In partnership with patients, caregivers, and health care providers, her research aims to produce evidence to improve health care and outcomes for children with rare genetic diseases, particularly inherited metabolic diseases.
Abstract:
Individuals with rare genetic diseases frequently have high health care needs beginning in early childhood, with important long-term impacts for individual patients, their families, and health care systems. Despite these high needs and the rapid pace of development of potentially disease-modifying therapies, evidence to inform health care for children with rare diseases is sparse and challenging to generate. Addressing important evidence gaps requires collaborative partnerships among patients, clinicians, methodologists, and policy-makers; investment in research infrastructure to support rigorous observational and interventional studies; and an emphasis on patient and caregiver experiences and priorities. Drawing on work within the field of pediatric inherited metabolic diseases and guided by the quadruple aim, example approaches to streamlining evidence generation for rare diseases will be presented, including the development of multi-centre research networks, patient registries, and core outcome sets; and the design of studies to inform improved patient and family experiences with care.
Keywords:
Rare disease, pediatrics, genetic disease, health outcomes, clinical trials
Date: Friday, February 9
Time: 1:30 pm - 2:30 pm
Location: PHFM 3015 (Western Centre for Public Health and Family Medicine) or Zoom (link may be requested at EpiBio@uwo.ca)
