The 4th MonitoRare report has been presented last July 19 in Rome.

The document has been realized by Federazione Italiana Malattie Rare onlus, with the cooperation of the Italian Ministry of Health and represents a detailed overview of the rare disease patients condition in Italy.

MonitoRare offers a general framework with data on epidemiology, access to diagnosis, therapies, assistance, as well as on socio-health, legal and economic organization.

The report highlights the strong points and criticalities of the Italian system of rare diseases.

One of the strong points is related to the access to drugs. At the end of 2017, the number of orphan medicines available in Italy are 94. New authorization from AIFA in the last year are 14. Also,  the consumption of orphan drugs, from 2013 to 2017, increased by 69.3% in absolute terms, and by 66.6% in relative terms, on the total consumption of medicines. The expenditure for orphan drugs rose from € 917 million in 2013 to € 1,599 million in 2016 (+ 74.4%), the number of drugs for rare diseases included in the list of Law n. 648/1996 has grown from 13 in 2012 to 28 in 2017 and 82 people with rare disease used AIFA fund, for a total amount of over 14 millions of euros (€ 7.4 million in 2016, € 1.1 million in 2015).

Another strength is represented by the quality and coverege of the vigilance systems. In fact, the coverage of the regional registries on rare diseases has increased and only 1 ill person in 5 is under 18.

Also the research represents a strong point of our system. The authorized clinical trials on rare diseases increases from 20% in 2013 to 25.5% in 2017. The weight of the biological / biotechnological active ingredients, which represent 37.5% of the total clinical trials on rare diseases, is also increasing. Also significant is the presence of Italian research groups in projects related to rare diseases included in the Orphanet platform, which in 2017 stood at around 19.9%. In addition, there are as many as 268 current research projects on rare diseases conducted by IRCSS in 2017, attesting to 7,6 million euro the resources for health research on rare diseases.

The report also includes data on scientific research, accessibility to therapies, centers of reference and a focus on new LEA, SNE, Registries and European Patient Representation.

The news on rare disease drugs is encouraging and Italy demonstrates an institutional sensitivity, allowing access to pharmacological treatment with various legislative instruments, as well as those available at European and international level to try to meet the patients’ needs as much as possible.

But several critical issues still persist, mainly related to the territorial inhomogeneities in access to health, social-health and social services; the failure to adopt the necessary administrative tools to recognize and guarantee the adequate remuneration of distance consultancy services by the centers of competence; the failure to adopt management and administrative solutions aimed at assessing the feasibility of remuneration methods that consider the complexity of the care management of the person with rare diseases in the hospital and territorial setting; the lack of involvement of the associative representatives of people with rare diseases in the Coordination and Monitoring Organization for the development of ERN.

The complete report is available [here].

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