Advanced therapies for rare and ultra-rare diseases, for cancer, for cardiovascular disease and diabetes are already a reality, and there will be more and more in the future. But in order to reach the patients who need it, it is necessary to face the challenges that these entail in terms of development, production, sustainability, clinical trials and access. Because advanced therapies – from gene therapy to cell therapy, to tissue engineering – can indeed manage patients’ symptoms or even cure them, but they are costly, risky and complex, both for those who develop them and for those who administer them. To meet these challenges, retreAT was born, a project promoted by the Advanced Therapies Observatory, which brings together researchers, clinicians, companies, patient associations and institutions to find concrete solutions to make advanced therapies available and sustainable. It will last one year and will end with a programmatic document to be presented to the institutions.
The “retreAT” project
The challenges that retreAT will face were told by five experts in the field, who, on the stage of the Ara Pacis in Rome, followed one another during the science show of the Advanced Talks on Advanced Therapies (At2) and who will guide the work of the project. Starting from what we know today and the difficulties we encounter when it comes to advanced therapies. “We have 26 advanced therapies in Europe today, but many more will arrive in the future”, said Giulio Pompilio, Scientific Director of IRCCS Monzino Cardiology Center in Milan, former member of the EMA’s Advanced Therapies Committee (CAT) and President of the Scientific Committee OTA. “There are in fact at least 1200 trials on advanced therapies in different stages of development and by 2030 it is estimated that another 40 products may arrive, not only for rare diseases as was the case at the beginning of the development of these treatments. But we have to build the whole ecosystem to welcome them or we risk losing them ”. As already happened – Pompilio recalls – for some advanced therapies withdrawn from the market because they are not very profitable for companies. “We need to rethink the entire production and development chain of advanced therapies, including at the regulatory level, to create a favorable environment for their development in Italy, from the point of view of research and industry”.
The starting point could be a public-private partnership that unites academia and industry, also imagining a new way of doing research and production, as explained by Maria Luisa Nolli, member of the Board of Assobiotec and EuropaBio and Professor of Advanced Biotechnology at the University. of Pavia, and Concetta Quintarelli, head of the Tumor Gene Therapy Unit of the Bambino Gesù Pediatric Hospital in Rome and member of the EMA Committee for Advanced Therapies (CAT). A new way that takes into account the peculiarities of advanced therapies, overcoming the obstacles posed by our country when it comes to research.
Italy can propose itself as a production pole based on the experience gained in the field (some of the first advanced therapies to arrive on the market, such as Strimvelis, were in fact born in our country) by expanding those capacities necessary for the handling of live drugs, from administer intravenously, Nolli says. All this while trying to cut costs. Highly specialized centers capable of conducting clinical trials for such complex drugs are also present in our country, added Quintarelli.
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But alongside very high potential, our country also puts a lot of bureaucratic limits on the start of experiments (just think of the multiplication of ethics committees in the various centers) which lengthen the time and discourage the start of studies. Even when it comes to privacy: “The limits imposed by the privacy regulations compromise the activation of a trial”, recalled Quintarelli. “We ask the patient what he wants, what is really necessary for him, working with the privacy guarantor”.
The issue of sustainability and the role of the centers
That Italy can propose itself as a promoter of the development and production of advanced therapies, not only in their conception phase, is a proposal that also came from Federico Spandonaro, President of the Scientific Committee of the Center for Applied Economic Research in Health ( CREA Sanità), speaking of the sustainability of these treatments. In fact, advanced therapies cost (they can even reach three million euros) and resources in Italy remain limited. And sustainability discussions can not only concern price negotiation, the development of innovative reimbursement models (such as payment by result, i.e. payment based on the results achieved), but must also look at the possibility of making research economically profitable. conducted in Italy.
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Finally, bringing these treatments to patients also means managing their expectations and preparing the centers on the territory, guaranteeing uniformity of treatment. “We must not sell smoke”, concluded Marika Pane, Director of the NeMO Pediatric Clinical Center in Rome. “It is necessary to tell the truth, to explain how these are revolutionary therapies, which can sometimes improve people’s quality of life but not cure. Advanced therapies can save lives, but they are also complicated to manage, they can have very serious effects and if the centers are not prepared to manage them, with multidisciplinary teams, regardless of where the patients come from, it is a problem “.