Pharmaceuticals : Commission's Rejection of Medicine Results in Imbroglio

Article excerpt

The European Commission has refused to authorise the placing on the market of a medicine used to treat an orphan disease, in spite of the positive opinion of scientific experts and the member states, which have given their opinion on two occasions. A legal imbroglio has resulted.


Orphacol is a medicine developed to treat a life-threatening orphan disease affecting the liver (there are 90 cases in Europe). The active substance was developed through university-hospital research at the Paris public hospital system (Assistance Publique des Hopitaux). Discovered in 1992 and used as a hospital formulation since then, it has been used to treat 19 patients in Europe. In 2007, the Paris public hospital system transferred its rights to the French laboratory, CTRS.

To receive authorisation to market the medicine, CTRS submitted an application to the European Medicines Agency (EMA) in October 2009.

Under the procedure in force (Regulation 2004/726), the Commission decides whether or not to issue marketing authorisation after obtaining a positive opinion from the EMA and the approval of member states.

In December 2010, the EMA adopted a positive opinion. It even described Orphacol as a medicine of significant public interest. At the Commission's request, the EMA adopted a second opinion, in April 2011, also positive. The member states were then consulted through the standing committee. On two separate occasions - first on 13 October 2011 and again on 8 November 2011 in the appeal committee - they vetoed the Commission's request for rejection of market authorisation on grounds of an incomplete application. The member states' opposition prohibits the Commission from adopting its decision, in this case to reject marketing authorisation, but the executive is nevertheless not obliged to take the opposite decision, ie to issue marketing authorisation. To sum up this Kafkaesque situation, the Commission cannot refuse marketing authorisation but on the other hand is not obliged to issue it either.

In January 2012, the laboratory initiated an action before the EU court system for failure to act. Its case is based on the fact that the Commission has failed to act although, according to the regulation, it is supposed to adopt a final decision within 15 days following the consultation by the standing committee. The EU General Court agreed to apply an accelerated procedure and even advanced the hearing by several weeks, an extremely rare occurrence. At the public hearing, on 24 April, the judges were stunned by this case, as Europolitics socialobserved.


In use for nearly 20 years, Orphacol can be given a waiver from the ordinary market authorisation procedure justified by "well established medical use". Directive 2001/83 exempts the applicant from providing the results of toxicological, pharmacological and clinical tests if it is possible to prove that the medicine has been used in the EU with recognised effectiveness and an acceptable level of safety for at least ten years. …