Fondazione Benzi took part in the joint event including the RE(ACT) Congress – International Congress of Research on Rare and Orphan Diseases (6th edition) and IRDiRC – International Rare Diseases Research Consortium – Conference (4th edition) held online on 13-15 January 2021.

Maddalena Toma, researcher at Fondazione Benzi, submitted an abstract on “Orphan paediatric medicines in Europe”, published on the Congress Brochure. The work was carried out on behalf of TEDDY European Network of Excellence for Paediatric Research  to check the Paediatric Orphan Medicinal Products (OMPs) receiving a Marketing Authorisation in Europe from January 2007, 26 to December 2019, 31 by consulting the TEDDY European Paediatric Medicines Database – EPMD and EuOrphan Database.

The analysis shows that the number of paediatric OMPs in Europe is still low and among them, 41 (around 72%) do not cover children at all ages. Neonates and infants are less represented. As a matter of fact, around 50% of the medicines, for both adult and paediatric rare diseases, have been approved only for adults. They include also therapies for rare cancers.

Therefore, the authors concluded that the therapeutic possibilities and the quality of life of children with rare diseases need to be enhanced.

To this end, two key points should be considered:

• • To give patients an active role and visibility allowing them to work together with the scientific community, the health public bodies and the companies
  • To focus more on research and innovation addressed to the paediatric population that until now has been left behind in the pharmaceutical framework

Discover more about the conference here and stay tuned for additional updates on our activities focused on rare diseases!