[Orphan Drugs: Underrated Opportunities for The Developers in Europe]. / Les médicaments orphelins : des opportunités méconnues pour les développeurs en Europe.

In Europe, rules relating to the designation and the protection of orphan drug are derived from regulation (EC) 141/2000 of the European Parliament and Council of 16 December 1999, specified by the implementing Regulation (EC) 847/2000. According to these regulations, obtaining the status of orphan drugs implies, in particular, to demonstrate the absence of any satisfying alternative treatment, or, by default, the significant benefit offered by the concerned drug. In the same sense, medicinal product similar to an original orphan medicinal product but safer, more effective or otherwise clinically superior, will benefit from a derogation to the rules on the 10 years market exclusivity usually provided for these products. This article analyses the concept of significant benefit, namely, the clinically relevant advantage or a major contribution to patient care, in particular in the case of similar drugs, as well as the elements to be provided by the sponsor in order to justify this benefit, and the options under which, where there are few or a lack of clinical data on a concerned orphan medicinal products, the demonstration of the significant benefit can rely on assumptions.

[The adoption of Jardé law modifies the legal framework of clinical research in France]. / L'adoption de la loi Jardé modifie l'encadrement juridique de la recherche clinique en France.

The Jardé law is adopted further to the Public Health Act No. 2004-806 which transposed into French law the Directive 2001/20/EC on clinical trials of medicinal products, made effective by the implementing Decree 2006-477 of April 26, 2006. The main novelty introduced by the Jardé law is to unify all "research organized and practiced on human beings for the development of biological or medical knowledge" and to facilitate its effective conduct, without however excluding from the scope of the law routine care and non-interventional research. The favorable opinion of the French Ethical Research Committee (comité de protection des personnes or "CCP") will now be required before launching any research on human beings, after validation of the risk/benefit ratio of said research. Applicable requirements and procedures - including information and consent - are adapted to each category of clinical research. New provisions are adopted to address special situations, previously forgotten. Finally, if Ethics committees were up until now freely chosen, they will, in two years' time, be randomly assigned. Thus, the Jardé law amends substantially the legal framework of clinical research in France. The question is whether these new national provisions will be compatible with those from the next revision of the so called "clinical trials" directive 2001/20/EC. In any case, the Jarde law will only come into force when all required implementing measures have been adopted.

[Reform Act on safety of drug and health products in France: announcement effect, strengthening or upheaval?]. / La loi de réforme de la sécurité sanitaire du médicament et des produits de santé en France : effet d'annonce, renforcement ou bouleversement ?

In France, the Médiator(®) scandal and the trauma it has created, are the source of Law n° 2011-2012 of 29 December 2011 on strengthening safety of drug and health products, intended by the Ministry of Labour, Employment and Health, adopted by the National Assembly and promulgated in order to restore confidence and enhance safety of drug and health products. This new law affects all stakeholders and health professionals impacts key points in the life cycle of the drug: MA, reimbursement, advertising, promotion, distribution, prescription, dispensing, and pharmacovigilance. It also provides for financial, administrative or criminal penalties, which are intended deterrent. Beyond strengthening constraints on safety of health products, this new law in France foreshadowed a revolution in behaviour, attitudes and overall scenery of the health system and no one today knows exactly the outline.