Why rare should not mean ignored
In Italy, the new National Plan for Rare Diseases 2023-2026 has just allocated €50 million for the two-year period of 2023-2024. The aim, according to Undersecretary of Health Marcello Gemmato, is to "address the disparities and inequalities in care and assistance, both among regions and within regions" for the approximately two million Italians affected by these conditions.
This progress is an important signal, although much remains to be done. For example, implementing the measures of the comprehensive law on rare diseases (Law 175/2021), approved two years ago and still not issued, as recently highlighted by the representatives of Uniamo, the Italian Federation of Rare Diseases. These measures include support for rare disease research and the development of orphan drugs, as well as a solidarity fund to support the care and assistance of people affected by rare diseases.
Dompé began investing in this field over a decade ago, not only through research but also through awareness-raising initiatives. An example is the video "The rarest ones," produced in collaboration with Uniamo, to effectively give a voice to rare disease patients. The protagonist of the video is Tommaso Galluppi, who was 22 years old at the time and affected by a rare metabolic disorder called hyperphenylalaninemia. The objective of "The rarest ones" is to encourage reflection and sharing as a means to promote a conscious understanding that a rare disease is not a minor illness, and all patients deserve equal attention.