Did you miss the XIII Foresight Training Course? Or do you wish to look into more in detail some of the topics discussed during the course?
Now you can freely access the highlights prepared by the experts who took part in the discussion that will give you an interesting overview of the topics discussed during the course that was deeply focused on the challenges put in place by the pandemic and the impact on the regulatory field.
Due to these circumstances the European Regulatory System is encouraged and forced to change to be more efficient and closer to the patients’ needs.
In particular, the new EU pharmaceutical strategy, just launched during the pandemic, includes the evaluation from the European Commission and proposal to revise two main pillars that share common key points: The Orphan Medicines Regulation and the Paediatric Regulation.
The Commission underlines the great efforts made in Europe on the availability of drugs for rare diseases thanks to the incentives issued by the regulations: about two thousands of orphan designations granted covering about seven hundreds different indications. 142 authorised orphan medicines have helped up to 6.3 million European patients out of roughly 35 million patients in the EU suffering from rare diseases and increased their quality of life. What emerges is that both regulations have not adequately managed to support Research and Development (R&D) in areas where the need for medicines is greatest and completing the R&D process still remains a challenging issue for an orphan medicinal product.
The course dedicated a specific session exactly on this issue that was analysed and clearly discussed by Viviana Giannuzzi, researcher at Fondazione Gianni Benzi and Violeta Stoyanova-Beninska from the Committee for Orphan Medicinal Products at European Medicines Agency. You can read the highlight here!
The availability of medicines for patients affected from rare diseases still represents a huge unmet medical need and a relevant public health challenge. The purpose of the Orphan Regulation, entered into force in 2000, is to reward R&D of medicines for rare diseases through incentives and to make them available on the market.
Despite the efforts that have been made in Europe, the orphan medicines availability is still challenging and support for research and development is still needed especially in certain therapeutic areas, paediatric and genetic rare diseases.
Discover more about this topic including the future prospective and the actions resulted by the European Commission report here!