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The new frontiers of medicine

    • Milan
    • 20 June 2016

          Opening discussions at this National Interest session was the observation that new advanced therapies, which are being increasingly adopted today, represent a real revolution in the health sector. They look set to enable a redefinition of the medical as well as social approach to dealing with diseases, especially those defined as rare, which affect around 500 people per million of the population and for which there is largely no treatment available. The new frontiers of personalized medicine pave the way for an approach that can be replicated and, once validated, extended to more common diseases.

          It was noted that Italy is one of the leading countries in the emerging field of Advanced Therapy Medicinal Products (ATMPs), particularly in the areas of cell and gene therapy. Indeed, the first stem cell-based therapy approved by the European Medicines Agency (EMA), and the first ex-vivo gene therapy for the treatment of an ultra-rare disease, adenosine deaminase deficiency (ADA-SCID), recently registered by the EMA, were both Italian discoveries

          The participants remarked that there are a variety of factors which go towards explaining how the country has managed to position itself at the forefront, including: the high standard of its scientists and basic academic research, creativity and the pervasiveness of entrepreneurial skills, and the existence of foundations and charities that promote the overall competitiveness of the system. However, it was stressed that the results achieved seem to be more attributable to entrepreneurship and the motivation of worthy individual organizations than the existence of systemic conditions that facilitate innovation.

          It was suggested that in order to ensure this position is maintained and that the most is made of the competitive advantage obtained, it will be necessary to govern the ecosystem, enhance its infrastructure, and carefully determine the areas requiring reform, while at the same time demonstrating an ability to anticipate needs as well as speed in adapting to changes in conditions. This against a backdrop where the country’s foundations – foremost among them Telethon, which represents a virtuous role model – have managed to fill certain gaps and reestablish a bridge between scientific discovery and growth, and in which the industrial sector has been able to innovate by opening itself up to the outside world and by developing new, more flexible collaborative research models.

          It was generally acknowledged, however, that the experience of the university sphere has been less successful. Indeed, limited levels of public funding for basic research and poor capacity to attract funding from external sources has been accompanied by an isolated university sector that is inadequately integrated with industry, together with an obsolete set of rules of engagement that complicates the timely pooling of skills necessary for development and undermines the future competitiveness of the system. The participants therefore urged that the main focus should be on generating ideas that are the product of unbridled curiosity, which should be structured in such a way as to encourage innovation and attract resources, a crucial prerequisite for the sustainability of the country’s national health system and the overall competitiveness of the country’s economy.

          The watchwords were therefore seen as being integration and collaboration within the ecosystem, which should facilitate the link between basic research, translational research, and clinical research. These elements need to also be accompanied by the creation of a network in which merit is valued, through, for instance, the designation of certain facilities as centers of excellence for having demonstrated an ability to attract resources and generate innovation.

          Finally, it was suggested that the ideas put forward during the discussion should be evaluated on the basis of two further aspects: their impact on economic sustainability and the renewed focus on patient-centered efforts. Innovation is only genuinely so if it is sustainable in the long run. It must hence be evaluated economically according to new models that take into account the specific features of advanced therapies, shedding light on incremental costs and benefits compared to existing treatments. At the same time, the actual effectiveness and the degree of success of new therapies cannot be judged without regard to the feedback of patients, who represent the true ultimate objective of any innovation in this sector and are increasingly involved in determining needs and shaping responses to them.

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