Measuring Health Related Quality of Life in Pediatric Life Limiting Progressive Diseases: Whose Perspective Are We Measuring?


Moderator: Josie Godfrey, MA, JG Zebra Consulting, Hammersmith, UK
Panelists: Ron Akehurst, DSc, Hon MFPHM, Lumanity, Sheffield, South Yorkshire, UK; Fleur Chandler, MSc, Sanofi and Patient Advisory Board Lead, Duchenne UK, Reading, Berkshire, Great Britain; Jill Carlton, BMedSci, MMedSci, PhD, University of Sheffield, Sheffield, UK

ISSUE: There are methodological challenges in how we measure Health Related Quality of Life in paediatric progressive life-limiting conditions. There are inconsistent approaches to whose perspective is measured: children, proxy or population. Can we reach a consensus on how to capture HRQoL in paediatric progressive life-limiting conditions? Can we account for the inevitable adjustments made by these children over their lifetime? Should we incorporate the value of hope for untreatable life limiting conditions?

OVERVIEW: This panel will discuss different approaches to including HRQoL in paediatric life-limiting conditions in HTA. Which perspective (proxy, patient or population) is most useful to decision-making, how to take into account the impact of disease progression over a lifetime and how best to interpret the results of HRQoL measures in clinical trials.

Josie Godfrey will chair the session and briefly present the challenges faced in incorporating HRQoL of paediatric life-limiting progressive conditions into HTAs.

The panellists will take an international perspective and represent different stakeholder views. Ron Akehurst will take an HTA perspective, sharing views of the strengths and weaknesses of current HTA approaches to incorporating paediatric HRQoL into HTA. Fleur Chandler, a carer and health economist will present a perspective arguing for more consistency and pragmatism in measuring paediatric HRQoL and talk about the work of Project HERCULES in capturing what is important to patients and their families. Jill Carlton, an academic researching HRQoL and burden, will outline her recent work exploring common themes of importance in paediatric progressive life-limiting conditions and highlight emerging research recommendations with reference to differences noted between stakeholder groups. The panel will consider questions about the specific challenges of measuring HRQoL in progressive paediatric conditions and the role of collaboration between patient groups, academics, manufacturers and agencies.




Patient-Centered Research