WRITTEN QUESTION E-0848/01

by John Bowis (PPE-DE) to the Commission

(22 March 2001)

Subject: Contradiction between EU orphan drug legislation and national measures resulting in restricted access to innovative therapies

Does the Commission agree that the growing trend across Europe to restrict patient access to new therapies, by limiting reimbursement on the basis of pharmacoeconomic appraisals, runs counter to;

- the principle of centralised authorisation through the EMEA? This is of particular concern for innovative biotechnology therapies for life-threatening, seriously debilitating or rare diseases for which no or limited alternative therapies are available and for which the EMEA has sole responsibility for advising on marketing authorisations. These marketing authorisation decisions are taken on the basis of quality, safety and efficacy criteria;

- the EU Orphan Drug legislation which aims to ensure patient access to innovative therapies by supporting research into, and the development and the availability of orphan medicinal products (Article 9 of Regulation (EC) No 141/2000(1) of the European Parliament and of the Council)?

Can the Commission confirm that it wishes to promote access to innovative therapies, encourage the development of healthcare biotechnology in Europe, address unmet medical needs and ensure a high level of health protection in Europe?

(1) OJ L 18, 22.1.2000, p. 1.

Answer given by Mr Liikanen on behalf of the Commission

(1 June 2001)

The Commission can confirm that it wishes to promote access to innovative therapies, encourage the development of healthcare biotechnology in Europe, address unmet medical needs and ensure a high level of health protection in Europe. As part of its work towards the achievement of this objective it introduced Regulation (EC) No 141/2000 of 16 December 1999, on Orphan Medicinal Products(1), which was adopted by the Council and Parliament in 1999. The Regulation on Orphan Medicinal Products aims to support the development of medicinal products to meet unmet medical needs in the area of rare diseases by offering incentives for companies developing such treatments. These incentives include a 10 year market exclusivity period, fee reductions or waivers for marketing authorisation related activities, advice on issues relating to the design of clinical trials for these products and automatic access to the centralised marketing authorisation procedure which ensures that a medicinal product, once authorised may immediately be placed on the market in all Member States.

A number of complementary measures has also been initiated at a Community level and support for the sharing of information and transnational collaboration on rare diseases is provided within the Community action programme on rare diseases. Specific research opportunities to support research into rare diseases also come within the scope of the Fifth Framework programme. In addition, the Fifth Framework programme also places a high emphasis on biotechnology research.

In accordance with Article 9 of the Regulation on Orphan Medicinal Products, the Commission has published a first inventory of national and Community measures aiming to support research, development and availability of orphan medicinal products, in which the Community measures and complementary national measures are delineated. This may be found at the following web-site address: http://pharmacos. eudra.org/orphanmp/doc/inventory.pdf

Pricing and social security related activities remain national competences and are outside the scope of the Orphan Medicinal Product Regulation.

Council Regulation (EEC) No 2309/93 of 22 July 1993 laying down Community procedures for the authorization and supervision of medicinal products for human and veterinary use

and establishing a European Agency for the Evaluation of Medicinal Products(2) plays an important role in promoting access to these products throughout the Community. This Regulation is currently under review with a view towards strengthening the provisions of this regulation in this regard.

(1) OJ L 18, 22.1.2000.

(2) OJ L 214, 24.8.1993.