Using HRQoL Data From Children and Youth to Strengthen HTA: What Are the Barriers? How Can We Improve Current Practice?

Speaker(s)

Moderator: Nancy Joy Devlin, PhD, School of Population and Global Health, University of Melbourne, Melbourne, Australia
Panelists: Fleur Chandler, MSc, Sanofi and Patient Advisory Board Lead, Duchenne UK, Middlesex, UK; Wendy J Ungar, MSc, PhD, Child Health Evaluative Sciences, The Hospital for Sick Children Research Institute, Toronto, ON, Canada; Koonal Shah, PhD, Science Policy and Research Programme, National Institute for Health and Care Excellence (NICE), London, England, UK; Saskia Knies, PhD, Zorginstituut Nederland, Diemen, Netherlands

Presentation Documents

Notwithstanding the increased availability of instruments for measuring and valuing children and adolescents’ HRQoL, the use of PRO instruments in the economic evaluation of health care technologies in pediatrics continues to show substantial gaps and weaknesses. Recent reviews of pediatric technology assessments submitted to England’s NICE and Australia’s PBAC found limited use of child-specific measures to generate health state utilities, considerable variation in the methods used to generate utilities, and frequent use of adult instruments and health state utilities. Use of poor-quality evidence in decisions regarding children’s health care could potentially lead to non-optimal resource allocation and disadvantage children.

The aim of this symposium is to examine the barriers to using existing age-appropriate tools for HTA in children and adolescents, to identify methods gaps that need to be addressed, and to explore ways that pediatric evidence-based decision-making can be strengthened. The symposium will present a range of perspectives, with speakers from HTA, industry, and academia.

Prof. Devlin will introduce the speakers and provide background for their presentations by describing some of challenges and recent advances in the pediatric PRO and HTA field. Dr. Shah will then talk about the issues faced by decision-makers in organisations such as NICE who have to deal with the reality of basing decisions on sometimes less-than-optimal evidence, and ways of addressing those difficulties, while Dr. Knies will describe efforts made by the National Health Care Institute of the Netherlands to extend formal product appraisal to pediatric health care. Dr. Chandler will provide an industry perspective on the challenges involved in assembling robust evidence in support of pediatric health-care products, and Prof. Ungar will explore ways in which the field could advance from a methodological perspective. Prof. Devlin will lead a Q&A session with the panel of speakers; questions and comments from the audience are welcomed.

Code

116

Topic

Health Technology Assessment