“There is little recourse to Law 648/96 for rapid access to orphan drugs for pathologies that have no cures available (Early Access Program)”. The criticality could be overcome “by integrating the cost of these drugs into the Fund for the innovative ones”. This was stated by Valeria Viola, Institutional and Regulatory Affairs Consultant Pharma Value, at the presentation, this morning in the Senate, of the VI Annual Report of the Orphan Medicines Observatory (Ossfor), illustrating the proposal made in the document to remedy the problem.
In Italy – it was recalled during the event – one of the ways to be able to get the drug to patients who need it early is to be included in the list established pursuant to Law 648/96. In 2021, a low recourse to Law 648 was found as an early access tool for pathologies with a high ‘unmet medical need’ (unmet clinical needs). In fact, there were only 4 requests for inclusion on the 648/96 list for orphan drugs in 2020, one of which obtained a favorable opinion and 3 unfavorable, while in 2021 there were 6, of which 5 with an unfavorable opinion and one remained under investigation .
To remedy this scarce recourse to Law 648, the Ossfor report contains a proposal which provides for “a revision of the Fund for innovative drugs, associated with drugs on the 648/96 list in their first authorized indication – explains Viola – This fund could be large enough to contain the costs related to the drugs that would be allocated there, so that there can be ‘early access’.The expenditure – he continues – would represent 9% of the total, around 91 million euros out of a billion euros for 2022. This estimate, among other things, does not consider the negotiation envisaged by the new Ministerial Decree for negotiation criteria.To integrate this type of drug into the Fund for the innovative, the regulatory change should include the assessment of innovativeness immediately after the positive recommendation of the EMA’s CHMP ( the specific commission of the European Medicines Agency, ed.). This would allow patients who need them to have access to these medicines in time shorter and with an economic coverage already foreseen by the system”.
From the point of view of the delivery of innovative medicines, the role of hospital pharmacist “must be expanded” according to Tiziana Corsetti, director of UOC Pharmacy, Bambino Gesù Children’s Hospital in Rome and head of the scientific area of rare diseases Sifo, the Italian Society of Hospital Pharmacy and Services pharmaceuticals from healthcare companies.
“The galenic – says the pharmacist – prepared in the hospital for off-label use (unauthorized, ed) for use in patients with rare diseases is also a reduction in expense. Based on an analysis by HTA (HealthKit technology assessmenti, ed) on the direct distribution of off-label drugs from the prescribing center in pediatric patients with rare diseases, preparation in hospital reduces drug dispensing times because the hospital pharmacist, knowing the patient, also prepares the most suitable pharmaceutical formulation , often liquid, in a safe context, because it is sterile, and ready to use.This practice could therefore contribute to optimizing direct costs, but the patient and caregiver must be taken into account who, in half of the cases, would prefer to have the drug in the Local Health Authority close to home. One solution – concludes Corsetti – could be to take it directly to the patient’s home”.
