Budget impact analysis of drugs for ultra-orphan non-oncological diseases in Europe

Abstract

Background/aim: Ultra-orphan diseases (UODs) have been defined by a prevalence of less than 1 per 50,000 persons. However, little is known about budget impact of ultra-orphan drugs. Methods: For analysis, the budget impact analysis (BIA) had a time horizon of 10 years (2012–2021) and a pan-European payer’s perspective, based on prevalence data for UODs for which patented drugs are available and/or for which drugs are in clinical development. Results: A total of 18 drugs under patent protection or orphan drug designation for non-oncological UODs were identified. Furthermore, 29 ultra-orphan drugs for non-oncological diseases under development that have the potential of reaching the market by 2021 were found. Total budget impact over 10 years was estimated to be €15,660 and €4965 million for approved and pipeline ultra-orphan drugs, respectively (total: €20,625 million). Conclusion: The analysis does not support concerns regarding an uncontrolled growth in expenditures for drugs for UODs.

Keywords:

• budget impact analysis
• drugs
• Europe
• non-oncological diseases
• ultra-orphan

Financial & competing interests disclosure

The study was supported by two biopharmaceutical firms, Alexion, Cheshire, CT, and BioMarin, San Rafael, CA, under an unrestricted educational grant policy. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Key issues

• There is an inverse relationship between disease prevalence and annual per-patient orphan drug costs.

• Health economists have expressed concern that drugs for orphan disorders may impose substantial and increasing costs to the health care system.

• The present study addressed the budget impact of drugs for ultra-orphan non-oncological diseases in Europe.

• The analysis based on patent expiries and likely new market entrants over a ten-year period predicted a moderately increasing rate of ultra-orphan drug expenditures through year 2012.

• While the analysis, which was enhanced by extensive sensitivity analyses, did not support popular concerns about un uncontrolled growth in ultra-orphan drug spending, continuous monitoring of ultra-orphan drug expenditures was recommended.

Notes

¹In the present context, we use the term ‘conservative’ to characterize all those assumptions potentially leading to a higher sales forecast. Whenever we had a choice between plausible assumptions, we selected the ‘conservative’ one.