Moves to create orphan drugs information exchange network
Common reports on relative effectiveness could reduce delays for access
The European Commission is investigating how to create an information exchange system for orphan medicines involving European Union (EU) institutions and EU member states.
Brussels wants this network established next year and would charge it with spreading knowledge on the scientific assessments of orphan drugs. This would enable the drafting of common reports on the medicines’ relative effectiveness – a ‘well-informed opinion on the place of the product with the authorised therapeutic indication in the therapeutic strategy of the rare condition, to the best of current knowledge,’ explained a Commission note.
The plan follows formal EU guidance approved by EU ministers (called a Recommendation) on the issue. It said such common reports could help speed the pricing of orphan drugs in member states, ‘thereby reducing delays for access to orphan drugs for rare diseases patients’.
But for this to happen, governments would have to ‘share assessment reports on the therapeutic or clinical added value of orphan medicinal products at EU level where the relevant knowledge and expertise is gathered’.
Earlier this year the European Medicines Agency (EMA) held a conference in London to mark the 10th anniversary of the EU’s orphan drug regulation, designed to promote their development.
Delegates heard that since the legislation came into force in April 2000, the agency has received more than 1,100 applications for designations of medicines as orphan drugs, 720 of which were granted – a success rate of 65%. Also, 62 new orphan designated medicines have since been approved in the EU, treating 53 different rare diseases.
An EMA report said many of these were genetic diseases and rare cancers, for which there are no satisfactory treatments, many affecting children and newborn babies. Furthermore, it said recently the number of applications for orphan designation ‘has increased significantly’.
Said EMA: ‘The continued interest in the orphan designation process…indicates more orphan medicines will be coming…’.