OBJECTIVES

: Since 1965, the European Union (EU) has adopted several legal texts aimed at ensuring the free trade of medicines in the EU while warranting public health protection. These texts enabled to build the “Europe of the drug”, materialized by a Community Code for medicinal products for human use. During the 21^stcentury, issues regarding the access to medicines for children and people suffering from rare diseases are remarkable, but legislation has gradually become available. This study investigated the hypothesis of the orphan drugs market access facilitation and acceleration after the EU specific legislation.

METHODS

: We have searched for legal texts regulating the commercialization of orphan drugs. In addition, we analysed numerous activity reports and the database of the European Medicines Agency, but also the recommendations of the European Network for Health Technology Assessment (EUnetHTA).

RESULTS

: We defined two categories of regulations in Europe: (1) specific texts about orphan drugs – Regulation(EC) 141/2000 of the European Parliament and of the Council of 16/12/1999 and Regulation(EC) 847/2000; (2) non-specific measures for orphan drugs but applicable to them – Regulation(EC) 276/2004 for Priority Medicines. Either one or the other category may be used for orphan drugs market access, depending on the industrial strategy. Therefore, since the beginning of the 2000s, there was a significant increase of the orphan drugs availability on the European Union market (3 in 2001, >100 in 2018). Nevertheless, the market authorisation requirements may not be considered as a sufficient condition to commercialization. This raises the issues of health technology assessment (HTA), which concern each EU Member State; perhaps generating unequal patient access to orphan drugs in different countries.

CONCLUSIONS

: A European harmonization of HTA has recently been translated into a draft regulation (31/01/2018), following the EUnetHTA recommendations, which aims to reinforce cooperation between HTAbodies and to reduce inequalities in patient access.