“I am very happy that the European reference networks (Ern) are finally part of the Consolidated Law on rare diseases.” Thus Maurizio Scarpa, coordinator of Metabern, the network dedicated to hereditary metabolic diseases, in his speech at the round table ‘Community regulation on orphan drugs , what prospects for the future ‘which took place within the VI Edition of the Orphan Drug Day, an event promoted by the Observatory for rare diseases, strictly online.
“The Erns – continues Scarpa – are networks that bring together the best institutes of excellence recognized by all the ministries of the 27 member states in the organizations. Just to talk about the metabolic, we currently have about 400 clinical trials carried out or in progress”. Initially set up only for clinical practice, Erns have become important elements for research which is very difficult to carry out within national borders, given the small number of these pathologies.
According to the expert, “ERNs can be a test bed for the testing of new drugs – explains Scarpa – but also for the repositioning of drugs that are available and tested for other diseases, but which have not had the results. expected and therefore can be tested on people in some conditions without going through the first stages which are extremely expensive for a drug. To do all this we have also created a program, ‘Erica’, which gathers all the Erns around a single purpose, that is to facilitate research and the interaction between public and private so that we ourselves can be promoters of trials and drugs. “.
Italy represents “a top country because we are a country very sensitive to the activation of orphan drugs as soon as they are available and with Covid we have seen how important this aspect is. Aifa itself has managed to allow the use of drugs at home in a period in which it was not possible to go to the hospital for administration. However, we had the opportunity to assist patients in such a dramatic moment “, concludes the expert.