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The results of a new analysis published in the Orphanet Journal of Rare Diseases suggest that 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 be sustainable over the next decade.
Currently perceived as a strain on resources, the budget impact of orphan drugs is projected to flatten from 2016 onwards.
It is predicted to stabilise at around 4-5% of the total pharmaceutical spending in Europe by 2020, suggesting that affordability 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 healthcare managers are concerned that the future growth in orphan drug costs may be unsustainable to healthcare systems.
This fuels questions as to whether the European Orphan Drug Regulation, enacted in 2000, which created regulatory pathways and incentives for manufacturers of orphan drugs in Europe, needs to be revised.
“Orphan diseases have been ignored for a long time; prior to the introduction of the European legislation few products had been developed specifically for these patients,” said Adam Hutchings, partner at health economics consultancy Global Market Access Solutions, and one of the study authors.
“We hope these findings can further support the sustainability of the orphan drug legislation and provide a useful insight to aid healthcare policy makers when planning for the funding of orphan medicines.”
The study, titled Estimating the budget impact of orphan medicines in Europe: 2010 – 2020, suggests that despite an increase in the number of approved orphan drugs, the fast growth in expenditure seen in the past decade is unlikely to continue in the next ten years due to four key trends:
Furthermore, results from this analysis have shown that the share of the total pharmaceutical market represented by orphan drugs is predicted to increase from 3.3% in 2010 to a peak of 4.6% in 2016 after which it is expected to level off through 2020, as growth falls into line with that in the wider pharmaceutical market.
“The majority of patients who rely on orphan drugs are coping with very rare diseases for which no alternative treatment exists,” said Yann Le Cam, Chief Executive of EURORDIS, the Patient’s European Organisation for Rare Diseases
“Provision of treatments for these conditions is part of the European ethos of allocating resources to those who need them most.
“This study provides further argument that access to the treatments for rare diseases should not be limited based on the their unsustainable burden on health systems, which in reality may be smaller than originally anticipated and currently feared by national competent authorities.”