“There are only about a hundred rare diseases with specific therapy available in Italy. We are talking about just over 1%. It remains a dramatic 99%. “He remembers that Annarita Egidi, General Manager of Takeda Italya few days after the XV World Rare Disease Day (Rare Disease Day 2022), which is celebrated on Monday 28 February.
To date over 300 million people around the world are living with rare diseases, remember a company note. The estimate in Italy is over 2 million people. Of these, one in 5 is under the age of 18. 72% of these pathologies have genetic origins, 20% environmental, infectious or allergic; 70% occurs in pediatric age, even when the disease has no genetic origin.
The average time to diagnosis is 4 to 7 years. “The Consolidated Law on rare diseases – underlines Egidi – represents a good goal as the first law dedicated to this sector made up of small specific numbers and large numbers together, but much remains to be done to meet the needs of patients. Now it is important the concreteness, that is the precise definition of the support instruments through the implementing decrees early diagnosis and uniformity of access to care to reduce the risks associated with the evolution of the disease and the immediate availability of new therapy options, a right that cannot be deferred for these patients “. The revision of the European legislation on the pharmaceutical sector, in particular on the unmet needs of patients, including various of these related to rare diseases, according to Takeda is “an opportunity to deal with many of the challenges still open. Another concrete intervention can come from the NRP which provides for specific investments dedicated to the funding of research programs or projects for rare diseases and rare cancers: two loans of 50 million euros awarded with calls to be awarded by the end of 2023 and 2025 ” .
‘We need a permanent table for all the players in the sector’
The arrival of these new resources is positive, Egidi points out, “per strengthen prevention and territorial assistance and improve collaboration between health and social services. Equally important – he adds – is the creation of a permanent discussion table with the ministries, Aifa and the Regions to design a model of constructive collaboration between all the stakeholders involved with a view to a public-private partnership. Collaboration, even with global capacity groups such as Takeda, can enable Italy to compete in the strategic match of attracting new investments in research and innovation “.
The manager recalls that Takeda is strongly committed to treatments: in the next 3 to 5 years, 70% of the product portfolio will consist of drugs for the treatment of rare diseases. To date, there are more than 40 products in the portfolio for 13 rare diseases. In addition, the pipeline consists of 50% molecules with the orphan drug designation.
Takeda Italia set up a new business unit at the end of 2021Rare’, to respond to the needs that patients and their caregivers meet on a daily basis. “Our goal – comments Egidi – is support the entire path of care for people suffering from highly disabling rare diseasessuch as haemophilia, hereditary angioedema, lysosomal storage diseases and primary and secondary immunodeficiencies “.
This new organizational structure, Egidi concludes, “completes our commitment in Italy: the research and development of innovative therapies that make a difference in the lives of patients, early diagnosis, newborn screening, quick access to valuable treatments, ‘home care and telemedicine “.
For the World Day of Rare Diseases Takeda Italia joined the Eurordis campaign for access to medicines, and at a global level it launched the initiative ‘Rethinking Rare‘. Furthermore, on February 28, the company will symbolically illuminate the Roman Bridge in Rieti in blue, where an important Italian plant in Takeda dedicated to plasma processing is located.
“With this initiative – declares Massimiliano Barberis, director of Takeda Manufacturing Italia Spa – we intend to ‘turn on a new light’ on the issue of inclusion in the social fabric for people affected by these diseases. These patients often have symptoms and manifestations that vary according to of the response of each organism and for this reason they are even more difficult to diagnose and treat. Think of the difficulties they experience in trying to understand and treat their pathology. The commitment towards them must include a new social sensitivity “.