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Orphan drugs in Europe

• The European Orphan Drug Regulation
• Availability of orphan drugs in Europe
• Early access to orphan drugs in Europe

The European Orphan Drug Regulation

Efforts have been jointly made at national and European levels by industry and health authorities (EMEA - European Medicines Evaluation Agency), in order to offer the incentives required to stimulate the development of orphan drugs. The goal was to rapidly make available, for rare diseases, drugs with a level of quality equivalent to that required for any other drug.

A policy was implemented much later in Europe than in the USA. The reason lies mainly in the fact that its territory is split-up and its competencies as regard to health are scattered.
Since 1 January 1995, with the new system of EU marketing authorisation that is valid for the whole territory and the free circulation that goes with it, Europe can be considered now as a territory with a population of about 377 million inhabitants, that is a population greater than that of the United States where a common regulation is enforced.

On 16 December 1999, the European Parliament and the Council adopted regulation (CE) N° 141/2000 on orphan drugs.

It was largely inspired by the US regulation and its goals were to :

• encourage the pharmaceutical and biotechnological industry to develop and market orphan drugs ;
• create a Committee of Orphan Medicinal Products (COMP) within the European Medicines Evaluation Agency (EMEA). This committee is responsible for studying the applications for orphan designation and to advise and assist the Commission in discussions on orphan drugs.

In addition, the Commission adopted regulation (CE) N° 847/2000 of 27 April 2000 establishing the provisions of application of criteria for orphan designation and defining the concepts of 'similar medicinal product' and 'clinical superiority'.

According to European regulation n° 141/2000, only drugs for human use can be designated as «orphan drugs». Therefore it does not concern veterinary medicines, medical devices, nutritional supplements and dietary products.

Drugs designated as orphan are entered in the Community register for Orphan Medicinal Products.

The current objectives of the European authorities are to :

• encourage the pharmaceutical and biotechnological industry to carry out research on and development of orphan drugs ;
• have competent small and medium-size enterprises participate in this development in special sectors;
• in parallel, increase the knowledge on these diseases, their environment, improve communication and cross-border collaboration between the various research centres, the institutions, the patients, etc.

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Availability of orphan drugs in Europe

The granting of marketing approval does not mean the drug is available throughout all the European countries (list of marketed orphan drugs in Europe). The marketing approval holder must decide beforehand on its commercialisation status within every country and the drug will then go through numerous steps in each country in order to condition its management, and usually its price.

Drugs which are exclusively used in hospitals are, following positive mention by the Commission, registered on the list of admitted products for the community. Their price is free.

Despite joint efforts, the heterogeneous approaches among countries make patients access to orphan drugs more complex.

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Early access to orphan drugs in Europe

Early access to a drug for patients may be possible before its marketing authorisation is granted to the pharmaceutical industry that develops it, most often during the third phase of the clinical trial and when its safety and efficacy are strongly assumed. Two cases may arise :

• an application for marketing approval has either been or is about to be submitted by the pharmaceutical company developing the drug in the country concerned. Then the company submits a temporary regulatory approval to the administrative authority for a group of patients (Temporary Use Autorization cohort in France and in Italy, or compassionate use authorization in the other European countries) that is valid for a limited time span in the country considered.
• or the physician asks the administrative authorities for a nominative temporary regulatory approval that is valid for a specific patient and for a limited time span in the considered country.

Due to differences in legislation, facility and rapidity of access to orphan drugs is not the same in the 15 European countries, as indicated in the chart below :

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