Brussels, 20 Jun 2003
While the procedure for 'designating' drugs for orphan diseases has improved dramatically in the EU, further ground must be made to get orphan drugs out of the laboratory and onto the market, the group for emerging biopharmaceutical enterprises (EBE) has said.
Rare diseases are classified as those affecting fewer than five patients in every 10,000 in the EU population. Companies are normally reluctant to develop 'orphan drugs' for these diseases because of their limited market. Since 2000, following EU regulations on 'orphan medicinal products', a variety of incentives have been introduced to encourage pharmaceutical companies to develop new cost effective medicinal products to treat rare diseases.
While agreeing that incentives such as a reduction in registration fees and allowing up to ten years' market exclusivity for approved drugs have led to some improvements to the process, EBE says that the situation still remains problematic. 'So far only 11 products have received market authorisations out of a total of 150 'designations.' This is less than half the rate at which products have been approved in the US. In some EU countries, the approved products still have not been made available to patients,' claims EBE.
Indeed the situation is troubling considering that rare diseases affect some 20 million people across Europe. EBE believes this constitutes a major health problem that must be addressed through policy continuity and coherency between all the stages in the process: 'The OMP [orphan medicinal products] designation is not an end-point in itself but is, rather, the beginning of a long-term commitment to develop and make a medicinal product available to patients with a rare disease. This means that policy continuity should drive the overall process from designation to the placing of the product on the market and patient access,' says EBE.
A need for continuity in policy making is particularly noticeable in the EU, says EBE: Because Member States are responsible for implementing incentives, the situation for processing orphan drugs changes from one country to another. 'The Commission should not merely monitor and list existing national incentives for orphan drug access but also push Member States to implement incentives where they have not done so to date,' urges EBE.
Access to orphan drugs is also hindered by restrictions on usage and issues related to pricing and reimbursement, which is also carried out at national level. Again, EBE notes that the EU could play a leading role in harmonising regulations, particularly in the pre-reimbursement phase to ensure patient access.
Other key challenges that need to be resolved, according to EBE, include ensuring closer coordination between the committees of the European Agency for Evaluation of Medicinal Products (EMEA) responsible for evaluating the orphan drug process, namely the committee for orphan medicinal products (COMP), and the committee for proprietary medicinal products (CPMP). EBE also proposes fostering a continuous dialogue between all stakeholders involved in the process, providing assistance on regulatory requirements to companies developing orphan drug clinical trials, and reassessing how the concept of 'rare' should be measured.
For further information on the orphan drug designation process, please visit the following web address: