[PHP6] Quantitative analysis of the influence of disease and product characteristics on orphan drug prices in Europe

[PHP6] Quantitative analysis of the influence of disease and product characteristics on orphan drug prices in Europe

2010 Value in health

Aballea, S. | Toumi, M. | Vataire, A.L. | Millier, A. | Lamure, M. | Volume: 13, Issue: 3, Pages: A82, poster, analysis, Disease, Price, Europe,

OBJECTIVES: Specific regulation and incentives to develop new orphan drugs in US
and Europe, as well as increased revenues associated with sales of orphan drugs have
boosted the interest of pharmaceutical companies in this field. Pricing is a key success
factor for manufacturers as sales volumes are very limited. We investigated the determinants
of orphan product prices in five European countries: France, Italy, Germany,
Spain and UK, using regression analysis. METHODS: Products with orphan designation
approved by EMEA were identified up to June 2009. Yearly prices per product
were derived from public prices, WHO defined daily dose, and treatment duration
based on guidelines or summary product characteristics. The analysis included diseaserelated
variables: prevalence, disease area, prognosis, age and vulnerability of target
population, seriousness, number of available treatments and course of illness, and
drug-related parameters: year of approval, trial size, number of trials, comparator in
trial, ATC code, evidence of benefit. A generalized linear model was used to identify
price predictors for the 5 countries. RESULTS: Fifty-one products were identified of
which respectively 37, 35, 28, 36 and 39 were available in France, Germany, Italy,
Spain and UK respectively. Available products did not overlap across countries. Prices
were highest in Italy and lowest in UK. No significant correlation was found between
any of the variables and price. Low prevalence and low number of available treatment
alternatives seemed associated with high prices, but these trends were not statistically
significant. CONCLUSIONS: Pricing of orphan drugs is a complex process, with no
identifiable objective price determinant. Patient group pressure, low overall impact on
health care expenditure budget, influence of opinion leaders and the ability of leveraging
the importance of unmet need in the target population might play an important
role. This analysis generated hypotheses for further research into drivers of prices for
orphan drugs.