Policy continuity needed to speed up orphan drug development

While the procedure for designating drugs for orphan diseases has improved dramatically in the EU, further progress must be made to get orphan drugs out of the laboratory and onto the market, the Emerging Biopharmaceutical Enterprises industry group 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. However, since 2000, a variety of incentives have been introduced to encourage pharmaceutical firms to develop new cost effective medicinal products to treat these rare diseases.

While acknowledging that incentives such as a reduction in registration fees and allowing up to 10 years' market exclusivity for approved drugs have led to some improvements to the process, the 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 USA," said the group, which operates as a division of the European Federation of Pharmaceutical Industries and Associations. In some EU countries, the approved products still have not been made available to patients, claims the EBE.​

"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,"' continued the EBE.​

A need for continuity in policy making is particularly noticeable in the EU: 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, urged the industry group.

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, the EBE suggested 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 Medicines Evaluation Agency responsible for evaluating the orphan drug process, namely the committee for orphan medicinal products, and the committee for proprietary medicinal products. It 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 the definition of a rare disease.