Rare diseases, VI Orphan Drug Day dedicated to new regulations for 2 million patients

The Consolidated Law on rare diseases is great news for Italy and for the 2 million people with rare diseases and cancers in our country. After its approval by the Parliament, on 3 November, the effects of the revision process of the European regulation on orphan drugs will fall on our country. Effects that certainly respond to needs but which can also involve difficulties or, according to the decisions that will be taken, even real dangers to be prepared for. This is what emerged this morning on the occasion of the 6th edition of the Orphan Drug Day, organized by the Observatory for rare diseases with the unconditional contribution of Chiesi Global Rare Diseases Italia, Janssen, Kyowa Kirin, PTC Therapeutics, Recordati Rare Diseases, Sanofi Genzyme, Takeda and Vertex. At the opening of the proceedings, the message of the Undersecretary for Health Pierpaolo Sileri: “This event falls favorably to now introduce the discussion on how to make concrete the provisions envisaged in the law and to therefore give effective and rapid responses to rare patients and their families. this bill, with everything that represents its process and the constructive participation it has generated, can constitute a model to be followed in the ordinary. A paradigm for other similar issues. We now have a regulatory framework that will also allow us to allocate necessary governmental and health planning resources to support rare disease patients and their caregivers in an ever more present and continuous way “. The first step to take, said Senator Annamaria Parente, president of the XII ‘Hygiene and Health’ Commission of the Senate, the same Commission that approved the Consolidated Act in the deliberative session, “is to move quickly to have all the decrees, agreements and implementing regulations within the established time and monitoring that everything is applied uniformly across all Regions, since one of the purposes of this Consolidated Law is precisely to smooth out territorial inequalities. In particular, in fact, this law provides for both Regions guarantee faster access times to nationally approved drugs, and greater uniformity of access to pharmacological and non-pharmacological treatments, which people with rare diseases need “. Among the various deadlines that are set by law, one of the first concerns the National Rare Diseases Plan which must be the subject of a decree within three months and therefore before the next World Day of Rare Diseases, at the end of February. Senator Paola Binetti, rapporteur of the law in the Senate and president of the Rare Diseases Parliamentary Intergroup, focused on this in particular: “This law – underlined Binetti – gives us a solid basis on which to build and now we have suffered opportunities to The first is undoubtedly the Budget Law for 2022. I hope that the great convergence that has taken place on the Consolidated Law will also be repeated when it comes to voting in favor of an allocation of funds for the next National Disease Plan rare, contrary to what happened with the first, which was never funded and in fact remained unfulfilled. I will personally speak up for this request “. On the same wavelength also the deputy Fabiola Bologna, who was rapporteur in the Chamber of the Consolidated Law: “To be able to approve this law – he said – it was necessary to find some mediation, above all financial, but now we have the possibility to affect again more starting from the approved text. One of the first things that could be done, also in the Budget Law, is to increase the Solidarity Fund that is being set up: for now there is only one million euros but it is possible to ask to increase it for have increasingly adequate funding for patients and families across the country “. The two representatives of the Rare Diseases Alliance, Fortunato Nicoletti and Giorgia Tartaglia, also focused on the possible improvements that could be made to the law, underlining how important it is for rare patients that the approved therapies are effectively made available in a short time and homogeneous throughout the territory. “To date – explained the two representatives – there are Regions that insert the new drugs on the formulary in a few months and others that also exceed the maximum limit of six months provided for by current laws. Everything needs to happen more quickly and that there are no regional disparities. Not to mention the drugs in Band C, which many regions leave to the patients even when they are included in the treatment plan, a problem to be overcome “. The associations also expressed satisfaction with the fact that the Consolidated Law provides for a specific action by the Ministry of Health to implement information and training actions on rare diseases. The topics covered also include that of the reorganization of the networks, a topic touched upon by article 9 of the Consolidated Law, and on which Professor Paola Facchin, referent of the Coordination Center for rare diseases of the Veneto Region, focused in particular, explaining that in these years a complex system has already been created and care must be taken not to invalidate the work done and instead try to enhance what already exists and works. Finally, during the discussion, particular attention was also given to the issue of tax incentives for research and production of drugs for rare diseases, which will have to be subject to subsequent regulation: an issue that also served to introduce the second major topic of the day, the one relating to the forthcoming revision of the European Regulation on orphan drugs.