(2002; 166 pages)
The EU regulatory system in the international environment
Mr Thomas Lönngren, EMEA
In the European Union regulatory system, there are 15 harmonized member states. In recent years, there has been intense cooperation with the Central and Eastern European countries. Within the framework of ICH, standards are being set through bilateral agreements and discussions with the United States, Japan and others. The EU has also extended its cooperation and attention to other international partners.
The European system has two licensing routes: a centralized procedure, and mutual recognition between national authorities. The EMEA is the focal point in this system. Currently there are a number of initiatives in preparation:
• Review of the European regulatory system in order to have one harmonized standard for new medicines.
• A high-level group on innovation and provision of medicines to improve competitiveness of European companies.
• Regulation and harmonization of all clinical trials in the European Union by 2004.
• Proposal on the development of paediatric medicines.
• Building of a robust electronic regulatory system for the EU.
• Enlargement of the EU.
In recent years, research in genetics and genomics led to the development of many innovative new technologies in the production of medicines, such as gene transfer techniques, cell manipulation, DNA vaccines, therapeutic cancer vaccines and transgenic animals. Such innovations provide many new therapeutic choices and opportunities in terms of prevention, diagnosis, and treatment.
Drug development in this area is likely to generate an unprecedented amount of information for target identification and screening. Some of these data could lead to quicker development of effective medicines and faster solutions for emerging diseases, with shorter and more focused development time. Early-phase pharmacovigilance, postmarketing studies and long-term follow-up should be in place to confirm safety. Classic scientific evaluation of quality, safety and efficacy may need to be complemented by considerations of risk perception, risk management, and societal, economic, traditional, ethical and environmental concerns. Drug regulatory authorities may face pressure to approve new therapies quickly and increasing expectations from patients to be involved in the process.
Regulatory authorities should be prepared for challenges, such as keeping up to date with scientific progress and its potential benefit for public health, keeping the expertise for scientific review, and being flexible in integrating different disciplines and regulatory frameworks to tackle this new challenge. Our rules cannot stand still: regulatory requirements must reflect scientific progress, not define scientific pathways. There should be new ways of ensuring compliance in a changing environment with greater regulatory transparency. In order to take into account different interests, common rules need to be established through international cooperation. This is not an issue for Europe or the United States alone, but is rather a worldwide issue and we, as regulators, need to decide how to handle these new therapies.
In 1995, the EMEA published the first guidelines on gene and cell therapy. The EMEA expert groups are looking at new guidelines and reviewing the existing guidelines. A multidisciplinary team, the
EMEA task force on innovation, is also looking at ways of adapting the current regulatory framework to the innovations. EMEA also aims to take a proactive role in international cooperation with organizations like ICH, FDA, etc.
Last, but not least, here are some perspectives for the future:
• New technologies and therapies have the potential to bring significant benefits to patients, but when will they actually appear?
• What is the relevance of the target diseases selected by industry and which patients will benefit?
• Will the high cost of new therapies limit patient access and range of target diseases?
• Standards should be developed through international cooperation, not through a country-by-country response.