ORPHAN DRUG PRICING IN FRANCE- INFLUENCE OF MAIN FACTORS
Author(s)
GRAND H1, Samson A2, Aulois-Griot M1
1Université Bordeaux, Bordeaux, France, 2Université Paris Dauphine, Paris, France
Presentation Documents
OBJECTIVES: Orphan drugs (OD) require considerable expenditures, which causes difficulties in their market access. For several years, the price of these new therapies has often been criticized and considered as too high. However few studies about OD pricing mechanisms are available. The aim of the paper is to highlight the main factors that influence OD pricing in France. METHODS: We collected public prices of 37 products designated as OD and approved by the European Medicines Agency until January 2014. Given that an OD can have several indications, our database contains 49 observations. For each observation, we calculated the ex-factory price without tax (defined per daily dose) defined by summary products characteristics. We also collected different characteristics of these products: improvement in actual clinical benefit (IACB), actual clinical benefit (ACB), number of OD indications, number of comparators drugs, number of competing orphan drugs, therapeutic use, target population, inclusion on the list of medicines reimbursed by National Health Insurance. Ordinary least squares are used to analyse the determinants of OD prices. RESULTS: The distribution of OD prices is very heterogeneous, with a minimum of 2.34 euros and a maximum of 2882.08 euros. The average (SD) price is 380.675 euros (687.414). Our main results are that OD prices are significantly lower for OD that do not improve actual clinical benefit (-136%, p=0.024), for OD with only one orphan indication (-80%, p=0.047), and for OD with a high target population (-251%, p=0.000). CONCLUSIONS: There is a need to understand OD pricing mechanisms. Our study shows that innovation and research efforts are encouraged by pricing policy.
Conference/Value in Health Info
2014-11, ISPOR Europe 2014, Amsterdam, The Netherlands
Value in Health, Vol. 17, No. 7 (November 2014)
Code
PSY72
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
Rare and Orphan Diseases