BioWorld International Correspondent

VIENNA, Austria - The rules governing clinical trials in Europe will become much stricter after May, when the EU's Clinical Trials Directive comes into force, creating additional challenges for small biotech companies that are already struggling to marshal the resources needed to set up and run trials.

"The Clinical Trials Directive tells you when to get up in the morning, what to eat for breakfast and when to go home in the evening. It is very prescriptive," Francis Crawley, chairman, Ethics Working Party, European Forum for Good Clinical Practice, told delegates at the Cordia-EuropaBio 2003 convention last week.

The directive is intended to harmonize the conduct of clinical trials throughout Europe, including in the 10 accession states that will join the EU, also in May. The new rules have been criticized for focusing on large-scale trials carried out by pharmaceutical companies to gain marketing approvals. As a result, they are too onerous for academics and biotech companies carrying out small-scale trials.

The responsibilities of all parties in a clinical trial - from sponsor companies to clinical investigators, ethics committees and the national bodies with oversight of medicines - are covered by the directive. Crawley warned, "As a sponsor [company], the directive expects a lot of you. The law will weigh heavily on your activities."

And for many countries in Europe it will be the first time there has been a legal framework for ethics, a fact likely to leave ethics committees pondering proposed trials for longer periods of time.

Frank Hulstaert, director of clinical programs at Innogenetics NV, of Ghent, Belgium, said small companies such as his are already at a significant disadvantage to larger pharmaceutical companies in setting up clinical trials, in manufacturing of products, in preclinical and clinical development, and in finding the right skills. The directive will increase those burdens, he said.

"For example, clinical development steps are followed very closely by the financial community - far more than they are in big pharma," he said.

There also is a big gap between academia and the heavy regulation that will be imposed by the directive.

"This can be very difficult if your staff comes from academia," Hulstaert said.

It has been estimated that implementing the directive will increase the cost of clinical trials fourfold, raising the particular concern that it will make it very difficult to justify the cost of trials of orphan drugs.

Such trials are problematic enough already, said Khazal Paradis, vice president, clinical research and therapeutics at Genzyme Corp.

In developing enzyme replacement therapies for the lysosomal enzyme deficiencies Gaucher's, Fabry's and Pompe disease, the afflictions are so rare it is not easy to find patients.

"Then you have the problem of what type of patient - they can be 2 years to 60 years old with a wide range of disabilities," Paradis said. "In addition, these are chronic diseases, so how long should the study be, because of cost, and how do you select endpoints in such heterogeneous populations?"

Paradis concluded, "We are lucky to be Genzyme, because we are financially healthy. But even for ourselves, it is becoming very expensive."

No Comments