EE65 Aspects Affecting Quality of Life in Patients with Rare Disease and Their Carers: Are Current Utility Instrument Capturing Enough?

Treatments for rare diseases are assessed by payers based on their ability to provide value-for-money, as the collective impact of these drugs represents a substantial part of the medication budget. Their value is often measured through cost-effectiveness assessments that are based on health utilities measured using generic instruments (e.g. EQ-5D) that may not adequately capture the full burden on patients and their caregivers/families.