Advanced therapies represent a new and fundamental opportunity for the treatment and prevention of the effects of a variety of pathologies: in some cases they are able to intervene directly on the causes of the diseases, in others the recombined genome is used as a pharmaceutical substance. They use cells, they work with genes. These are therapies that have revolutionized clinical practice in some pathologies and which impose a new paradigm also on the management front of the National Health System. It is therefore necessary, to guarantee patients access and at the same time sustainability, that politicians also have an in-depth and updated knowledge of this new type of therapies. To this end, UNIAMO, the Italian Federation of Rare Diseases Onlus, promoted an institutional training hosted by the Parliamentary Intergroup on Sustainable Innovation in Healthcare. A path, organized in collaboration with LS CUBE, thanks to the non-conditioning support of #VITA (coalition of companies which includes: Bristol-Myers-Squibb, Gilead Sciences, Janssen, Novartis, Pfizer, PTC, Roche and Vertex), which led to the creation of the document “Parliamentary Thermometer of ATMPs” (Advanced Therapy Medicinal Products), presented in the Senate.
A path organized into three training moments of discussion, open, constructive and operational, with subject matter experts, patient associations, trade associations and clinicians for correct and in-depth information on a complex topic in order to produce solutions to address the new challenge of advanced therapies, which imposes a new paradigm. “We are proud of the work carried out – declared Sens. Francesco Zaffini, President of the 10th Commission on Social Affairs, Health, Public and Private Work, Social Security and Daniele Manca, Co-Presidents of the Parliamentary Intergroup on Sustainable Innovation in Healthcare – in which the majority and the opposition have collaborated together to produce technical-operational tools for effective parliamentary activity for the benefit of patients suffering from rare diseases and their caregivers, as well as all the actors of the National and regional Health System”.
by Tiziana Moriconi
The necessary measures
The “Parliamentary ATMP Thermometer” outlines the actions necessary to make Italy competitive also on the new frontiers of advanced therapies, with the aim of guaranteeing the availability of these therapies, intervening on the regulatory framework to encourage research activity, development, production and marketing by research institutions and industry, develop new financial reimbursement models, create conditions that guarantee fair and timely access to ATMPs and identify rapid and effective diagnostic pathways.
“From an economic point of view – stated Americo Cicchetti, Director General of Health Planning of the Ministry of Health – ATMPs are characterized by being therapies with high investment costs, but also notable benefits in clinical, therapeutic, social and economic terms for healthcare systems and the health of patients, capable of producing a clear temporal misalignment between actual costs, concentrated in the short term (as they intervene directly on the disease and do not treat the symptoms), and future benefits, spread over a longer time horizon. These characteristics differentiate them significantly from traditional drugs and for this reason it is important to understand whether it is possible to imagine different accounting models for the expenditure associated with them. It is clear that disruptive innovations such as ATMPs require serious reflection on the need to adapt regulatory, economic and regulatory frameworks”.
The need in Italy
“In Italy – explained Annalisa Scopinaro, President of UNIAMO – there are between 2 and 3.5 million rare disease patients and diagnosis can take up to 4 years. Today and in the near future, advanced therapies are a new frontier for the treatment of many rare diseases: it is estimated that over 350,000 patients will be treated with these innovative therapies by 2030. We are talking about rare and orphan diseases, including some oncology. For only about 5% of these severe and often disabling pathologies, there is a therapy.” Advanced therapies have an extraordinary impact on the clinical history of people with rare diseases, radically changing their evolution, with obvious benefits in terms of health but also with repercussions on caregivers and families, as well as on the health system in terms of direct savings and indirect.