BioWorld International Correspondent

BRUSSELS, Belgium - Orphan drugs, many of them biotechnology-derived products, are genuinely benefiting from EU patronage, according to the latest information from the European Commission.

By mid-February, 75 applications had been received by the European Agency for the Evaluation of Medicinal Products, through the new system set up in 2000 by the EU regulation on orphan drugs. Thirty-four positive opinions had been adopted by the new body set up for this purpose, the Committee for Orphan Medicinal Products, and 17 formal designations had been granted, for potential treatments for rare diseases such as Fabry disease, acute myeloid leukemia and Gaucher disease.

And EU member states also are providing support: Austria and Denmark reduced or waived fees for marketing authorizations for orphan drugs, and Belgium set up a working party to look at possible exemptions from national fees and special consideration during pricing and reimbursement negotiations. The French Ministry of Research is backing orphan products research through national programs covering genomic research, with funding of over EUR30 million (US$27 million) per year. In the Netherlands, a complete national structure for orphan medicinal products is being planned, bringing together patients' support groups, doctors and pharmaceutical companies.

EMEA Clarifies Position On Cell Lines

The European Medicines Evaluation Agency in London has agreed to a new guideline on tumorigenic cells lines of human origin for the production of medicines.

The guideline outlines what the EMEA considers the relevant factors when using this approach. "It is important to define the risk factors attendant on the use of such cell substrates with a view to addressing such concerns in a properly conducted risk assessment for any regulatory submission," it said. It deals with residual cell substrate DNA, genomic and phenotypic stability, and characterization of cell substrates.

But it said that "with respect to novel cell substrates, risk may be difficult to define qualitatively or quantitatively because the endpoints are either theoretical or cannot be measured." Ultimately, it said, potential risk must be evaluated against the intended clinical use of products.

Plants Committee Wary Of Purity Rules

The Scientific Committee on Plants, which advises the European Commission on genetically modified plants, says that the sort of purity requirements that the EU is currently planning are going to be difficult to meet.

The EU is proposing thresholds for what it sees as "acceptable" levels of adventitious contamination of conventional seed with genetically modified seed: thresholds of 0.3 percent for cross-pollinated crops and 0.5 percent for self-pollinated and vegetatively propagated crops are contemplated. But the committee said in a just-published opinion that with the scientific knowledge currently available these limits will be achieved only under ideal seed production conditions.

There will be situations when achieving these thresholds will be problematic, such as with varietal association cultivars, and production of hybrid seed. In addition, it warned that achieving the thresholds will become increasingly difficult as GM crop production increases in Europe, and in due course the thresholds set by the Commission may have to be revised.

McSweeney Appointed EC Center's Director General

The former director of Bio Research Ireland, Barry McSweeney, has been appointed director general of the European Commission's Joint Research Centre, which is responsible for much of the EU's biotechnology research.

McSweeney, a 50-year-old Irishman, formerly was product development manager at American Hospital Supply Corp. in Belgium and Switzerland, and medical director of Biocon Biochemicals in Cork, Ireland. Since last year he has been responsible within the European Commission for research on GMOs and in vitro tissue culture.

European Research Commissioner Philippe Busquin said, "Barry McSweeney's long scientific experience both in the private sector and in the JRC makes him perfectly qualified."

Commission Reorganization Aids Biotechnology

In recognition of the special needs and growing importance of biotechnology-derived medicines, the European Commission has reorganized to give greater prominence to biotechnology.

The Commission unit that previously dealt with all aspects of pharmaceuticals from an industry perspective has been split in two. Frenchman Philippe Brunet remains as head of unit for the regulatory framework and market authorizations for pharmaceuticals, and Abraao Carvalho of Portugal has been made head of a new unit responsible for biotechnology and competitiveness in pharmaceuticals.