Orphan drugs cost likely to be sustainable to European health budgets in the next 10 years, new research shows

Despite an increase in the number of approved orphan drugs in Europe, the overall growth in cost of these medicines as a proportion of total pharmaceutical expenditure is likely to be sustainable over the next decade, according to the results of a new analysis published in the Orphanet Journal of Rare Diseases.

Currently perceived as a strain on resources, the budget impact of orphan drugs is projected to flatten from 2016 onwards. It is predicted to stabilize at around 4% to 5% of the total pharmaceutical spending in Europe by 2020, suggesting that affordability issues should not be a barrier to access to treatments for rare diseases.

Concerns about the cost of orphan medicines are delaying the acceptance and uptake of these medicines and have led to uneven access across Europe. Policy makers and health care managers are concerned that the future growth in orphan drug costs may be unsustainable to health care systems.