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 sixth edition ofOrphan Drug Day, organized by the Rare Diseases Observatory 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 from 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. I believe that this bill, with all that represents its iter and the constructive participation it has generated, may 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 the necessary governmental and health planning resources to support rare disease patients and their caregivers in an increasingly present and continuous way.“.
The first step to take, the senator recalled Annamaria Parente, president of the XII ‘Hygiene and Health’ Commission of the Senate, the same Commission that approved the Consolidated Act in the deliberating session, “is move quickly to have all the implementing decrees, agreements and regulations within the established time and monitor that everything is applied uniformly across all Regions, since one of the aims of this single text is precisely to smooth out territorial inequalities. In particular, in fact, this law provides for both the Regions to 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. The senator focused on this in particular Paola Binetti, rapporteur of the law in the Senate and president of the Rare Diseases Parliamentary Intergroup: “This law – underlined Binetti – gives us a solid basis on which to build and now we have opportunities to seize. 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 Plan for rare diseases., contrary to what happened with the first, which was never funded and in fact remained unimplemented. I will personally speak up for this request “.
The deputy is also on the same wavelength 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, especially financial, but now we have the possibility to have an even greater impact starting from the approved text. One of the first things that could be done, even 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 in order to have more and more adequate funding for patients and families throughout the country “.
The two representatives of the Rare Diseases Alliance also discussed the possible improvements that could be made to the law, Fortunato Nicoletti And Giorgia Tartaglia, who underlined how important it is for rare patients that approved therapies are effectively made available in a short and homogeneous time throughout the territory. “To date – explained the two represented – there are Regions that insert new drugs on the formulary in a few months and others that even exceed the maximum limit of six months provided for by current laws. Everything needs to happen faster 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 therapeutic 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 the reorganization of networks, a topic touched upon by article 9 of the Consolidated Law, and on which the professor focused in particular. Paola Facchin, referent of the Rare Diseases Coordination Center of the Veneto Region, explaining that in recent years a complex system has already been created and care must be taken not to invalidate the work carried out 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.
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