The request that rare disease become a priority for public health was advanced by a specific parliamentary intergroup presided over by Senator Paola Binetti, by non-profit Uniamo Fimr (federation of patients with rare diseases) and by Omar during the press conference held last week in the Senate. Other participants included Eurordis and the Telethon Foundation as well as the recipients of the letter, Minister of Public Health Roberto Speranza and Vice-Minister Pierpaolo Sileri. Supporters claim that two million patients have been waiting years for concrete action to be taken on this issue and that several questions can be resolved in a short period of time by implementing the right measures.
The letter, which collected various viewpoints from the world of rare diseases, was articulated in five points.
- Approval and financing of the new Italian national plan for rare diseases. The previous planexpired three years ago and proved highly unsuccessful. The request is not only for a new plan with different objectives, but that the plan be endowed with congruent economic resources.
- Attention to the renowned “100-day procedure”, according to which rapid negotiation, completed within 100 days, is provided for drugs with exceptional therapeutic value and drugs for hospital use. A great notion on paper, but which has had little effect given that it takes, on average, more than 200 days for orphan drugs to be approved.
- The importance of promoting research: according to Carlo Fornario, director of institutional relations for the Telethon Foundation, “A tax incentive scheme that promotes actions in support of clinical and preclinical studies on rare diseases is needed by both the pharmaceutical industry and private investors that want to finance programs dedicated to the launching and realization of such projects”.
- Newborn screening must be implemented and a plan must be made in order to extend the advantages of screening to other diseases. Promotors remind us that Italy has achieved excellence in this field, but there is still so much potential that must be taken advantage of.
- Levels of care (LEA): they seem perfect on paper, but not in practice, as often is the case.
What the institutions say
Vice-minister Sileri, the recipient of these requests, has shown willingness to cooperate and, above all, to quickly carry out the work necessary for the new Italian National Plan for Rare Diseases to be approved, emphasizing the priority of efforts to facilitate diagnosis.
The vice-minister states, “in addition to suffering caused by a disease, [patients are put through] a diagnostic crusade and often without treatment. Above all, we must make efforts to facilitate diagnosis and, thus, improve the diagnostic process.”
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