It was discussed today at a conference organized by ISS and Assobiotec-Federchimica. More than 1,900 clinical trials worldwide, of which 112 in phase 3 (99 carried out by the bio-pharmaceutical industry, 13 by academia, governments or other institutions). Key role for Italy.
20 MAY –
Advanced Therapies are the future of medicine. From drugs for a narrow niche of patients, they will soon be standard of care for an ever-increasing number of people. How to prepare healthcare systems to welcome the fruit of innovation represented by Advanced Therapies to ensure fairness and equal treatment in the right to treatment for Italian and European patients was the theme at the center of the event: “Advanced therapies: from sustainability to organizational models in the area” organized by the Istituto Superiore di Sanità and Assobiotec, the Federchimica Association for the development of biotechnology, which was held today in Rome at the ISS headquarters.
The latest report from Alliance Regenerative Medicines talks about more than 1,900 trials in the world, of which 112 are already in phase 3 (99 carried out by the bio-pharmaceutical industry, 13 by academia, governments or other institutions). And 360 clinical studies in Europe with around fifty in the pre-authorization phase. Italy has always had a key role in the research and development of Advanced Therapies: just to give an example, among the first Advanced Therapies approved by EMA, 4 were the result of Italian research. And today the commitment continues with important economic investments and 23 clinical trials underway in 2022 in areas with unmet clinical needs such as rare diseases, neurodegenerative diseases or tumors. During the various sessions of the event, the state of the art, the possibilities and challenges represented by these therapies were discussed, but also the possible solutions for their financial sustainability and the organizational aspects for their distribution across the territory through the provider centres.
“The ISS willingly accepted Assobiotec’s invitation to jointly organize this meeting because we believe that the topic of transferring treatments, and in this case advanced therapies, from the laboratory to the patient’s bed is extremely important – states the president Rocco Bellantone -. Indeed, although advanced therapies represent an important hope for a cure for some previously incurable diseases, there are still significant obstacles to the real possibility of use by patients. I hope that the joint discussion of the main critical issues can contribute to promoting an effective, timely, fair and sustainable transfer of the promises of these therapies to the bedside of patients who need them”.
“Advanced therapies constitute an epochal change in the concept of care which becomes patient-specific and which brings great benefits from a clinical and therapeutic point of view. For the first time in the history of medicine, personalized drugs based on biological material have been developed which aim to cure the patient in a single administration. – explains Fabrizio Greco, President of Assobiotec – In the next 10 years, many new therapies will arrive in clinical practice, intended for niche pathologies, but certainly less rare. It is estimated that up to 60 new gene and cell therapies could be launched globally by 2030, potentially affecting a total of 350,000 patients.”
“With reference to the economic impacts in Italy – continues Greco – recent analyzes report, for the year 2023, an expense of between 132 and 264 million euros, up to an expense borne by the National Health Systems between 905 and 1,810 million euros for the year 2027. The need to prepare healthcare systems to welcome the fruit of this innovation which is proceeding at an unprecedented pace in history is therefore clear and evident. A permanent discussion table is needed between all the players in the system Health to build a new organizational model capable of guaranteeing equity of access and care. For this we thank the Istituto Superiore di Sanità which, as has happened several times in the past, accepted our new invitation to sit at a table and discuss together. how to face the challenge of innovation and how to accompany the unstoppable progress of science for the benefit of patients and the system”
What are Advanced Therapies (ATMPs)
Advanced therapies, which include cell and gene therapies, tissue engineering and combined products, and which in Europe are called ATMPs (Advanced Therapy Medicinal Products) are the protagonists of a revolution in the medical field. For the first time in the history of medicine it has been possible to develop drugs based on biological material, personalized and which aim to cure the patient in a single administration.
Unlike small chemically synthesized molecules and biotechnological macromolecules such as monoclonal antibodies and recombinant proteins, ATMPs are made up of cells or tissues, possibly engineered, or nucleic acids. These drugs, innovative by definition, are demonstrating their effectiveness not so much in treating the symptoms, but the disease itself, intervening directly on the causes and offering new prospects for healing pathologies that until now had no therapeutic solution.
The characteristics of ATMPs can be summarized as follows:
▪ significantly transform the clinical history of patients suffering from diseases that do not have a therapeutic alternative, satisfying an unsatisfied clinical need and are able to re-establish, correct or modify physiological functions compromised in humans, even with the correction of genetically acquired mutations and the addition of copies of functioning genes.
▪ they are developed to have a significant clinical benefit in pathology areas with high medical need, on serious and disabling diseases often with unfortunate outcomes, rare diseases and with little or no therapeutic options available;
▪ they are one-shot, that is, they are administered in a single treatment, unlike traditional drugs and protocols, which involve repeated and regular treatments, with a clear temporal misalignment between current costs, concentrated in the short term, and future, widespread benefits over a longer time horizon;
▪ they have high investment costs, they are personalized therapies with high complexity and production, but which present significant future benefits in clinical, therapeutic, social and economic terms for healthcare systems and the health of patients (direct, indirect and social costs avoided, recovery in productivity, greater tax revenue, etc.);
▪ they are administered only in qualified and specialized centers and arise from extremely innovative and complex platforms
In Europe, 25 advanced therapies have been approved, 18 of which are currently on the market. Of these 18 therapies, Italy has granted reimbursement to 8 with significant differences in numerical terms at a European level.
May 20, 2024
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