EU and US unite on orphan drugs, aim for further harmonisation

And yesterday, 28 further areas where the industry would like to see simplification in the approval and marketing authorisation process came under discussion at the Transatlantic Administrative Simplification Workshop in Brussels.  

The workshop included representatives from EMEA, the FDA, national regulators and authorities and the European and US industry. The aim is to take the first steps towards reducing unnecessary administration and ultimately reducing costs to the end user and industry.

The meeting agreed a priority list of topics for administrative simplification, with work to minimise duplicated or divergent requirements due to begin in early 2008. The results of these discussions will be published by the European Commission and FDA in June 2008.

The move on orphan drug designation illustrates the potential benefits. About 30 million people in the EU and 25 million Americans suffer from more than 6,000 rare diseases, which are defined as affecting fewer than five in 10,000 people in the EU and fewer than 200,000 people in the US. The small number of patients makes it difficult to make a profit from developing drugs to treat them.

The US Orphan Drug Act of1983 and the EU Regulation on Orphan Medicinal Products of 1999 aim to provide regulatory and financial incentives, such as protocol assistance, and marketing exclusivity, to encourage the development of drugs for rare diseases.

But to date developers have had to submit separate applications for orphan designation to the EMEA and to the FDA, using different submission formats. Now the FDA and EMEA have agreed to harmonise the application form, allowing companies to apply to both jurisdictions at the same time with one application.

The common format will also allow EMEA and the FDA to share common experiences and gain an understanding of the similarities and differences of the process of obtaining orphan designation in the two regulatory systems.

The two regulators will continue to conduct independent reviews of submissions to assure they meet the legal and scientific requirements of their respective jurisdictions.