Estimating QALYs for Transformative Therapies for Severe Pediatric Rare Diseases
Author(s)
Moderator: Peter Neumann, ScD, Center for the Evaluation of Value and Risk in Health, Tufts Medical Center, Boston, MA, USA
Speakers: Andrew Lloyd, DPhil, Acaster Lloyd Consulting Ltd, Oxford, Oxfordshire, UK; Jonathan Toft, PharmD, MBA, MedImpact, San Diego, USA; Josie Godfrey, MA, JG Zebra Consulting, Folkestone, Kent, UK; Josh Cohen, PhD, Center for the Evaluation of Value and Risk in Health, Tufts Medical Center, Boston, MA, USA
The objective of this symposium is to discuss estimation issues for QALYs in assessing the value of transformative therapies, with a particular focus on severe, pediatric rare diseases. Firstly the symposium will compare QALY estimates for transformative therapies versus conventional medicines. We will then discuss key methodological challenges in eliciting utility values for rare diseases, including use of trial-based and vignette approaches as well as sourcing values for analogous diseases. Subsequently the panelists will discuss measurement challenges associated with capturing the multidimensional aspects of treatment (e.g. cognitive benefit in additional to motor and respiratory function) from various perspectives such those of the caregiver and parents. The session will conclude with a summary of best practices, and recommendations for health technology assessment agencies and researchers.
Sponsor: Orchard Therapeutics
Conference/Value in Health Info
2023-05, ISPOR 2023, Boston, MA, USA
Code
243
Topic
Patient-Centered Research