BioWorld International Correspondent

CORK, Ireland - Bristol-Myers Squibb Co., Pfizer Inc. and the Irish government are the initial backers of a new not-for-profit institute at University College Cork (UCC) that aims to establish a pan-European network specializing in conducting clinical trials of products that have received orphan designation.

The Paris-based European Organisation for Rare Disorders (Eurordis), an alliance of 200 rare disease associations in 16 countries, and the Genetic Interest Group, a London-based coalition of more than 120 medical charities, are the other founding members.

Brendan Buckley, a consultant physician and a member of UCC's department of pharmacology and therapeutics, is director of the organization, called the European Institute for Clinical Trials in Rare Diseases. Until the beginning of May, he was Ireland's representative on the Committee for Orphan Medicinal Products, the scientific body that advises the London-based European Medicines Evaluation Agency on orphan drugs.

Three years on from the establishment of the European Union's orphan drugs regulations, just eight products have received marketing authorizations. A further three, Aldurazyme, Busilvex and Ventavis, have received positive opinions from the Committee on Orphan Medicinal Products, and are currently awaiting authorization decisions, according to EMEA spokesman Harvey Allchurch. But the vast majority of orphan products are at earlier stages of development. The agency has granted a total of 142 orphan designations since the regulations came into force in April 2000.

The fragmented nature of European health care and the small size of European biotechnology firms developing drugs for orphan indications are both hampering faster progress, Buckley told BioWorld International. In addition, language differences and difficulties in obtaining reimbursement for new, high-cost treatments further compound the problem. "You have really very significant barriers within the EU, which are seriously subsegmenting health care in Europe," he said.

In the U.S., because health coverage operates on a federal basis, centers of excellence in particularly rare diseases can develop with sufficient scale and clinical expertise. But the same level of patient mobility does not apply in the EU, so even in the larger member states, treatment centers operate on a substantially smaller scale, Buckley said.

In the absence of any formal pan-European structure, there is a need, Buckley said, for better coordination and integration among existing clinical centers. Part of the center's mission will be educational. Pediatricians dealing with rare genetic disorders often have little clinical trials experience. The institute will, with its partners, seek to develop common methodologies and systems for clinical trials involving small patient cohorts.

The infrastructure underpinning the new institute has already clocked up substantial experience in large-scale clinical trials. It was part of the PROSPER study, which evaluated the use of Pravastatin, marketed by Bristol-Myers Squibb, in reducing the risk of cardiovascular disease and stroke in elderly patients. Buckley said it has developed electronic systems that introduce high levels of efficiencies, allowing patient monitors to follow up to 1,000 subjects.

Its relationship with patient organizations will aid study design and recruitment, Buckley said. "I think, really for the first time, they are being taken in as stakeholders in the trial process."

The institute is finalizing arrangements with several partner centers across Europe, Buckley said, and is in the process of taking on its first assignments.