German biotech calls for fair treatment of orphan drugs

Orphan drugs are a rapidly expanding subsector of the pharmaceuticals industry. Martina Ochel, chief executive of Genzyme and acting chairman of vfa bio, the biotechnology branch of the German pharmaceutical association the VFA, noted last week that more and more pharma companies, notably in biotech, are working on developing new drugs for rare diseases.

In the European Union, a disease is classified as rare if it afflicts at most five in 10,000 people. Orphan drug development has been supported and regulated by the EU since 2000, since when 60 drugs have become available to EU patients. And plenty more are in the pipeline, noted the VFA: around 800 further drug development projects have achieved orphan drug status from the European Commission. That lowers approval costs and confers 10 years’ marketing exclusivity on a drug. EU states are now also developing complementary national action plans to tackle rare diseases.

In Germany, orphan drugs have been exempted from the new vetting procedure introduced by the government to clamp down on drug costs for the national health system (The Pharma Letter October 8, 2010). Under the new rules, instituted at the beginning of the year, manufacturers will henceforth have to prove that a newly-approved drug is an improvement on existing treatments.