“While solidarity with rare patients and the celebration of research is being broadcast on state networks, in the Senate a serious attack is taking place precisely against the drugs developed to give therapy to those patients. L’attacco – denuncia l’Osservatorio delle Malattie Rare – ha la forma di un amendment, the no. 2.1623, presented by Sen. Marcello Gualdani (NCD). Stand in the coming days the Senate were to say 'yes' to the approved drugs for the treatment of rare diseases, from next year, they will return to pay the 'Pay Back', a sort of 'fine' on the overrun of the already tight hospital budgets. Too bad that in rare diseases the only way not to go overboard is to avoid making new diagnoses and/or giving therapies to new patients”.
“Questo emendamento – prosegue l’Osservatorio – va nella direzione opposta a quanto deciso appena un anno fa proprio nella legge di Stability, from the same Parliament and from the same Minister: a schizophrenic attitude that puts the treatment of thousands of patients at risk. The effects of this turnaround would be harmful for both patients and companies who, after years of investment in research, come to bring them life-saving therapies. An incomprehensible measure, which would bring no savings to the NHS, but which only serves to place the burden of new patients treated, and progressively, on the few companies that produce orphan drugs rather than on the wider pharmaceutical sector. The Pay Back could penalize them to the point of pushing them to change course".
“This attempt to eliminate protection for orphan drugs must be read, together with the ongoing attempt to eliminate the 20-year fund for patients with Cystic fibrosis, as the last clear sign of disinterest in rare patients. At this point – concludes the Observatory – all that remains is to appeal to Minister Lorenzin and to the Senate Budget Commission which will have to vote on the amendments, so that they all become operational guarantors of rare disease patients".
