Institutions and civic representatives, but also the private and research world, express words of great satisfaction
A new law that represents a real regulatory point of reference for the entire Italian community of patients with rare diseases, which was created not only to improve the diagnosis and treatment of these pathologies and to guarantee equitable and uniform assistance to the people affected by them, but also to promote scientific research in the sector and the development of orphan drugs. This is what the so-called Single Text for Rare Diseases, which was finally approved yesterday, in the Senate, represents: it is an important milestone of civilization for our country, as evidenced by theunanimous satisfaction expressed by representatives of politics and institutions, and by the main exponents of medical science, the pharmaceutical industry and patient associations.
“The approval of the text is certainly not a point of arrival but, on the contrary, the finger pressed on the start of the timer to mark the timing of the definition and issuance of the relative implementing decrees, on which I put my personal commitment “, underlined the Undersecretary of Health, Pierpaolo Sileri. “The Technical Roundtable on rare diseases, set up on my impulse at the Ministry of Health, which has already identified the priorities for action and possible intervention proposals to work on, will also help. The Consolidated Law on Diseases rare – recalls Sileri – had a genesis and development on average quickly and is the result of a fruitful inter-parliamentary, inter-institutional collaboration, which has seen the active and continuous contribution of the parties directly concerned, first of all the patient associations, which have been listened to and involved in the definition of this law “.
The approval of the Consolidated Law is “a sign of great civilization, which aligns our country with other countries in the world; a tangible political response to the more faded needs of rare patients, which can be summed up in the need for diagnosis, treatment and research “, commented the prof. Bruno Dallapiccola, Scientific Director ofBambino Gesù Pediatric Hospital From Rome. “The most qualifying points of the recently approved document are the support for research, the production of orphan drugs, the updating of the Essential Levels of Assistance (LEA) with innovative diagnostic tests, the provision of therapies and services, finally overcoming regional differences. Today, 95% of rare diseases do not have effective therapies and over 80% of rare and ultra-rare diseases are genetically determined: for this reason, being able to invest in research means identifying the molecular defect underlying these diseases and finding the way to counter it. . Rare patients need innovative treatments and only with research will we be able to give tangible answers”.
The Telethon Foundation also insists on the topic of research. “We are really happy that the institutions of our country have wanted to send out a signal of concrete interest for patients and families facing a rare genetic disease and for the research dedicated to them.. Precisely because of their rarity, these pathologies tend to be neglected by public and private funding, which is why there are realities such as our Foundation, which open ways to increase knowledge and give life to new treatments “, he said. Francesca Pasinelli, General Manager of Telethon Foundation. “We are all seeing how much thanks to scientific research it is possible to illuminate the vision of the future with hope even in complex periods like the present one. This is why it is an important sign that the institutions have wanted to give support to those who, during a pandemic, are even more fragile and vulnerable. We are confident that this regulatory transition can be a harbinger of a further strengthening of our system with the support of those realities that strive every day to give concrete answers to those living with a rare genetic disease, contributing in general to knowledge and scientific progress ” .
Great enthusiasm also comes from representatives of civic organizations, primarily Cittadinanzattiva: “we are satisfied with the approval of the Consolidated Law on rare diseases, a very important text that provides, among other things, the updating of the list of rare diseases, establishment of an economic fund dedicated to patients and their families and caregivers, the strengthening of screening. It is therefore about an important step forward to improve citizens’ right to access to treatment and prevention“, he has declared Anna Lisa Mandorino, general secretary of Active citizenship. “Now we will work, together with the many organizations of our CnAMC (National Coordination of Chronic and Rare Patient Associations) so that what is foreseen is applied concretely and uniformly throughout the national territory”.
A ‘just law’: this is how the members of Parliament describe this Consolidated Law in different words. “This is a law of parliamentary initiative that we have been working on for over three years, approved with a transversal and very wide consensus of the political forces. It is an important law because we finally define rare diseases in an organic way and we recognize the rights of all the people who are affected by them.“, He declared in a video, published on his social channels, Lisa Noja, group leader of Italia Viva in the Social Affairs Commission in the Chamber. “We must now get to work to ensure that this law has solid legs to walk on, starting from the approval of the acts that are necessary to implement the rules contained therein, including the establishment of the Solidarity Fund for rare patients, the approval of the second National Plan on rare diseases and the establishment of the National Committee for rare diseases. This law gives an important answer to the rare sick, over 2 million citizens who every day have to deal with a sneaky enemy who enters their lives, never abandons them and makes everyday life difficult for them and their families. With this law we tell the citizens who have a rare disease that in the face of this great effort they are not alone, that the institutions are close to them; and the nice thing is that we gave this answer all together, in a great united effort by Parliament ”.
“Today we have given a better life expectancy to many children, to two million sick people, their families and caregivers, but also to research institutions which will be granted a tax credit on the expenses incurred”, he highlighted Paolo Russo, Forza Italia parliamentarian. “I am proud to have contributed to writing a beautiful page in the history of Parliament and, above all, to have presented, on 23 March 2018, that is just 19 days after the elections, the bill then merged into the Consolidated Law which received the final go-ahead “.
The Consolidated Law on rare diseases is “concrete help to patients and their families and an example of good politics. Now we need to implement the regulations in all regions, put the existing one into a system, update the plan for rare and diseases every three years “, he said. Annamaria Parente, president of the Hygiene and Health Commission of Palazzo Madama. “The text is a new beginning: the fairness of a health service is measured by how we treat rare diseases”.
“Italy finally equips itself with a tool capable of giving concrete answers to thousands of patients and families affected by rare genetic diseases, ”he said Vito De Filippo, member of the Social Affairs Commission of the Chamber. “On the fundamental research front, the establishment of a fund for preclinical and clinical studies has been introduced; in addition, a National Plan for rare diseases will be approved every three years. I now look forward to a quick implementation of the new rules for all regions ”.
“Today, thanks to the tenacity of Senator Paola Binetti, rapporteur of the Consolidated Law on rare diseases, our Parliament has finally endowed itself with a law of civilization which restores dignity to people with rare diseases and to their families ”, he explained in a note Lorenzo Cesa, UDC national secretary. “Therefore, a long battle ends today which was strongly supported by the whole party: now, however, the implementing decrees must be approved”.
“The approval of the Consolidated Law on rare diseases in the Hygiene and Health Commission of the Senate is a great satisfaction”, declared Daniela sbrollini, Senator and Head of Culture of Italia Viva. “An important step forward to respond to the concrete needs of the sick, finally removing any inequality or discrimination. Good politics and reformism exist and put an end to the indifference of the system, improving the lives of the most vulnerable and their families ”.
“It is good news that the bill on rare diseases has been unanimously approved in the Senate”, commented on Facebook the Minister of Health. Roberto Speranza. “Among other things, it provides for the updating of the list of diseases, the strengthening of screening activities and the establishment of a solidarity fund for patients and families. The National Health Service must take care of everyone, even those suffering from the rarest diseases”.
And finally, applause also comes from the pharmaceutical world, which plays a crucial role in the research and production of therapies for rare patients. “With the passage of this law, the rare patients are less ‘orphaned’, and are given due consideration“, he has declared Massimo Scaccabarozzi, president of Farmindustria. “There are several positive aspects: from faster access to drugs, to measures that favor research in Italy for new therapies. A leading role will be played by the implementing decrees – argues the leader of the pharmaceutical industry – which should be issued as soon as possible to make the regulations operational, thus overcoming the differences between the various Regions in terms of homogeneity of access to treatment, often the only available for the sick “.
Read also: “What does the Consolidated Law on Rare Diseases provide? Here is the summary of the approved law “
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