BioWorld International Correspondent

BRUSSELS, Belgium - After two years' debate, the European Union has reached agreement on a new regulatory framework for pharmaceuticals, geared particularly to the needs of high-technology medicines.

The European Parliament gave its backing on Dec. 17 to a complex series of compromises balancing faster authorization procedures against better patient protection and taking into account the interests of research-based firms against generic copyists. The European ministers and officials also indicated their accord with the package, and the new rules could be in effect across 25 EU member states by mid-2005.

For the biotechnology industry, key changes - among hundreds of modifications included in the new rules - should mean a higher degree of scientific expertise in the assessment of new products, faster approval times and some increased intellectual property protection.

All high-technology medicinal products, and particularly those derived from biotechnology, will still use the centralized EU authorization procedure operated by the European Agency for the Evaluation of Medicinal Products "in order to maintain the high level of scientific evaluation of these medicinal products in the European Union and thus to preserve the confidence of patients and the medical professions in the evaluation," the new rules state. "This is particularly important in the context of the emergence of new therapies, such as gene therapy and associated cell therapies, and xenogenic somatic therapy. This approach should be maintained, particularly with a view to ensuring the effective operation of the internal market in the pharmaceutical sector."

But the quality of the assessment is to be boosted by widening the scope for bringing experts into the agency - largely to meet the long-standing concerns from the industry that regulators know significantly less about biotechnology products than the companies developing them. The new rules also say that "the structure and operation of the various bodies making up the agency should be designed in such a way as to take into account the need constantly to renew scientific expertise," and the field of activity of its scientific committees "should be enlarged and their operating methods and composition modernized."

Scientific advice for future applicants seeking marketing authorization should be provided more generally and in greater depth. Similarly, structures allowing the development of advice for companies, in particular small and medium-sized enterprises, should be put in place. The committees should be able to delegate some of their evaluation duties to standing working parties open to experts from the scientific world appointed for that purpose, and re-examination procedures should be amended to provide a better guarantee for applicants' rights.

The scientific committees will be allowed to co-opt additional members "chosen on the basis of their specific scientific competence" to complement the national representatives. And the committees will be reinforced so the EU can play a bigger role in international scientific dialogue on harmonization and technical cooperation.

But to cover environmental risks that "may arise from medicinal products containing or consisting of genetically modified organisms," an environmental risk-assessment procedure will have to be conducted in parallel with the evaluation of the quality, safety and efficacy of the product.

Accelerated assessment procedures are to be set up for medicines "of major therapeutic interest," cutting review time by at least two months - 30 days faster than the U.S. system for priority review. A new conditional marketing authorization will allow for a one-year authorization to be granted, provided that there is an important expected health benefit for the patients concerned and that the company agrees to carry out additional monitoring and clinical studies. And new provisions for compassionate use will allow the supply of medicines "for compassionate reasons to a group of patients with a chronically or seriously debilitating disease or whose disease is considered to be life-threatening, and who cannot be treated satisfactorily by an authorized medicinal product."

Additional protection will be provided for approved products: it will not be possible to market generic copies of a product anywhere in the EU until 10 years have elapsed from its first EU authorization, and that can be extended by an additional year if a further innovative indication for the medicine is authorized.

There also will be clearer rules to cover copying, with new and tighter definitions of generic medicines, particularly of "biosimilars." In recognition of the complexity of biotechnology products, biosimilars will be governed by a more rigorous testing regime than generics of chemical-based products.

Erkki Liikanen, the European commissioner responsible for the pharmaceutical industry, greeted the vote as "an important day for European patients and the European pharmaceutical industry." He said it will "ensure a high level of protection for public health, while improving access to medicines and boosting the competitiveness of the European pharmaceutical industry."

There was a muted reaction from the research-based industry. The European Federation of Pharmaceutical Industries and Associations was somewhat disappointed, but said the reform is "a reasonable balance" that "will encourage pharmaceutical research, which is vital to find innovative treatments and cures for the benefit of patients, and provide more rapid access of patients to innovative medicinal products."

The biotechnology association, EuropaBio, welcomed agreement on "the first legal rules in the world for authorizing biosimilar products," since "the next few years will see more biotech medicines coming off patent, and a clear set of rules to handle the authorization for these products is in urgent need." But now the agency must produce adequate guidelines for practical implementation, EuropaBio stressed.

Elsewhere, however, EuropaBio said it was disappointed by what it saw as weakening of earlier proposals for data protection of innovative products. "This is not a good message that Europe is sending to its companies who would like to have a European legislative environment that stimulates research," said Chris Van Den Broucke, member of the EuropaBio health care board.

The U.S.-based Biotechnology Industry Organization said it supported the EU initiative on biosimilars.

The next stage in the process is for EU ministers to give their formal agreement - probably in January. That would mean the changes in the quality of the agency's product-assessment procedures and staffing could come into effect as soon as February. The other changes would come into force 18 months later.

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