Masterstudiengang "Drug Regulatory Affairs"


Legal Framework for Compassionate Use in the European Union - Comparison of the Implementation of Compassionate Use Programmes by Means of Selected European Member States

Dr. Dagmar Theis (Abschlußjahr: 2007)

Language: English

The marketing authorisation procedures have been developed gradually to protect patients from harmful medicinal products. According to Article 6 (1) of Directive 2001/83/EC it is not allowed to place a medicinal product on the market of a Member State unless a marketing authorisation has been granted. This legislation is useful for most of the patients, but there are still patients suffering from a chronically or seriously debilitating disease or a life-threatening disease who cannot be treated satisfactorily by an authorised drug. There are some possibilities in receiving a medicinal product without marketing authorisation. For example, the drug can be prescribed by a physician for an indication not listed in the Summary of Product Characteristic (i.e. off-label use). Another possibility is the use of an investigational drug in a clinical trial. In this case, the patients have to meet specific inclusion and exclusion criteria laid down in a clinical trial protocol. However, there are still patients who cannot enrol in one of the two above mentioned programmes. For these patients, Article 5 of Directive 2001/83/EC offers the use of an unlicensed drug on a single, named patient basis. Based on this article the European member states have implemented different programmes for a single patient but also for a group of patients, although this does not fall under the scope of Article 5 of Directive 2001/83/EC. For example, France has the earliest and best defined implementation of compassionate use programmes in the EU, providing medicinal products without marketing authorisation to a single patient or to cohort. This is called a “Temporary Authorisations for Use”. In other European countries the terminology and the legal framework differ and a great variability can be found in the EU. Regulation (EC) 726/2004, Article 83 introduced the term compassionate use to all European countries and clarifies that compassionate use is intended for the treatment of a group of patients. The objective of Article 83 is to improve the equality of access to an unapproved drug of all European patients and to harmonize the set up of a compassionate use programme within the EU. In addition, the Committee for medicinal Products for Human Use (CHMP) drafted a guideline on compassionate use to give guidance on the use of Article 83 of Regulation (EC) 726/2004. The aim of this master thesis is the description and comparison of different compassionate use programmes in the EU. Comparing compassionate use programmes in some selected countries, demonstrates the variability within the EU in regards to the patients targeted, the liability and application procedures and shows the need for a centralised procedure given by Article 83 of Regulation (EC) 726/2004.