OBJECTIVES

In 1999, in response to the lack of treatment for rare diseases, Europe adopted an incentive policy for orphan drugs. Almost 20 years later, some 100 drugs have benefited from economic advantages. But what are the different orphan drugs that have been accepted since then and what factors seem to correlate with the prices obtained?

METHODS

Orphan drugs on the website of the European Medicine Agency and with a price published in the French official journal have been selected. A focused literature review was conducted to identify determinants of price for orphan drugs. The opinions of the French HTA and EMA were used to collect the determinants of each of the orphan drugs (first reimbursed indication). The price was collected using the tariff notices in the Official Journal, the annual cost per patient was calculated according to the doses, survival times, progression free survival, average weight and age found in clinical trials. The correlation analysis was initially performed in univariate using non-parametric tests. Factors with significant correlation (p-value < 0.05) with annual cost per patient were retained for the multivariate analysis (generalized linear model).

RESULTS

From the 293 articles identified, 14 were retained and 34 variables were identified. 98 orphan indications met the inclusion criteria and were retained. In the univariate analysis, 11 were significant with the annual cost per patient. The strongest relationships were obtained for repositioned status, ATC class, molecule type, target population size. The significant multivariate variables (R² = 0.67) were repositioning, ATC class, molecule type, target population size, population size during clinical investigations and marketing date.

CONCLUSIONS

This exploratory analysis makes it possible to identify certain characteristics of drugs that are associated with a low or high price. The profile of drugs in the ATC A class (metabolism) appears to have a significant impact on the treatment price.