To date, 16 thousand advanced gene therapies are being tested. A work that involves 450 companies all over the world and whose results, in the coming years, could represent a solution for over 350,000 sick people who today have no hope of cure. The apparently simple idea is to use genes, at the basis of the functioning of cells, as if they were tiny spare parts capable of replacing defective parts. And so to re-establish, correct or modify the physiological functions compromised in the sick. The reality is instead very complex and the costs can appear high. “The solution is in a change of perspective that leads to consider this expense as an investment and not as a cost,” he explained Americo Cicchetti, Professor of Business Organization of the Catholic University of the Sacred Heart and Director of ALTEMS, speaking at the digital conference “Advanced Therapy: how to reach a sustainable paradigm shift”, organized by FortuneItalia with the Science and Health Parliamentary Intergroup, CittadinanzAttiva and the association # VITA (Value and Innovation of Advanced Therapies, composed of companies engaged in research in this sector), followed by a round table with the Institutions. “Advanced therapies – continues Cicchetti – in fact offer benefits not only in the short term, but above all in the long term. Not only for people’s health and well-being, but also in terms of direct and indirect savings: in the case of curative therapies, there is a radical improvement in the natural history of the disease, with the complete elimination of therapies and treatments that often occur. they would prolong the entire existence of the person. The impact on health is complemented by an equally important impact on the quality of life and productivity at work. This last impact also affects caregivers, who will have the opportunity to work full time and, just like those who were once patients, will be able to start producing income again, with cascading benefits for tax revenues and the pension system ”.
Like military spending
Taking a cue from the 2010 revision of the European System of Accounts (SEC), which redeveloped military spending from current to investment expenditure, the expenditure for advanced therapies could be accounted for in the same way. An innovative approach which to tell the truth has not yet been adopted by any European country but which, according to Mauro Marè, Professor of Finance at the Luiss Business School, would be an advantageous solution both for the patient, who would benefit from highly innovative and effective treatments, and for the National Health Service, which could amortize the cost of therapy over the years. “In the year of the pandemic – said the Undersecretary of Health, the senator Pierpaolo Sileri – we understood the importance of adequate resources in health. Advanced therapies represent a unique potential for treatment in which it is important to invest, both for the health system and for that country. The synergy at the political and technical, national and European level, will be crucial to identify organizational and financing models that guarantee full uniformity in the access to these treatments, in compliance with the principles of equity, universality and sustainability “.
The strengths of advanced therapies
“First of all, they are administered with a single treatment unlike traditional drugs and protocols used for other pathologies, which provide for repeated and regular treatments, with an evident temporal misalignment between current costs, concentrated in the short term, and future benefits, spread over a longer time horizon – points out Antonio Gaudioso, president of CittadinanzAttiva: “They also intervene directly on the causes of the disease they want to cure, not only with the aim of mitigating the symptoms. And this offers new healing perspectives to patients suffering from diseases that, until now, were without a therapeutic solution even in the perspective of prevention “.
In fact, the development of advanced therapies plays a fundamental role in the treatment and prevention of various pathologies: “True gene diseases, oncological ones and those with a long prognosis. A few examples? From haemophilia A and B to Parkinson’s, from spinal muscular atrophy to Duchenne muscular dystrophy, passing through thalassemia and mutations associated with retinal dystrophy. But there are many others still ”, he concludes Angela Ianaro, president of the Intergroup Parliamentary Science and Innovation: “Faced with this revolutionary technology, institutions, patient associations and industry must work together to find courageous and innovative solutions that guarantee universal access to innovation, avoiding accounting conventions and rationing needs arise as an obstacle ”.
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