Assessing the Economic Burden of Rare Disease Across Europe: A Health Economic Study of Financial and Quality of Life Impacts
Author(s)
Axelsen K1, Kennedy A2, Feng L3, Poon C4
1Charles River Associates, New York, NY, USA, 2EveryLife Foundation for Rare Diseases, Washington, DC, USA, 3Alexion, AstraZeneca Rare Disease, Boston, MA, USA, 4Charles River Associates, London, UK
Presentation Documents
OBJECTIVES: To collect data showing rare disease total economic burden to patients and the health systems in distint health system environments.
METHODS: Using surveys of people living with 43 distinct rare diseases (PWLRD), and their caregivers, compared to survey respondents without rare diseases and health provider interviews in 2023 and 2024, we gathered information to document the rare disease economic burden in nine European countries with different population sizes, socioeconomics, and health system dynamics.
RESULTS: Among PLWRD and caregivers surveyed (N=545) relative to people without rare disease (N=772), we estimated the per person, per year financial impact of RDs to be $101,800 for a PLWRD, $81,500 in excess of the reference group. Of the PLWRD costs, 58% were direct medical costs, 24% were direct non-medical costs, such as home modification or transportation, and 18% were indirect costs, such as work productivity. Across the nine countries, the population cost of the rare diseases studied was in excess of $1.46 trillion. Direct medical excess costs constituted the largest proportion of costs for people, in most therapeutic areas, particularly in haematology and pulmonary. Higher rates of misdiagnoses and longer times to diagnosis were associated with higher total cost. The availability of an EMA-approved treatment for the rare disease was associated with a shorter time to diagnosis. People living with rare endocrine and metabolic conditions had the longest time to diagnosis. Health-related quality of life (HRQoL) was highest for people with the most and the least spending on medicines; for all other cost categories, the trend is largely linear. Lower cost is related to greater HRQoL.
CONCLUSIONS: These findings established an estimate of the cost of PLWRD and the connection between disease state, speed of diagnosis, quality of life, and cost. The results may also reflect the broader benefit of having medical treatments available for rare diseases.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 12, S2 (December 2024)
Code
EE608
Topic
Patient-Centered Research, Study Approaches
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes, Stated Preference & Patient Satisfaction, Surveys & Expert Panels
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases