Advanced therapies, experts compared today in Rome. The occasion was the Advanced Talks on Advanced Therapies, better known as At2, at the Ara Pacis Auditorium in Rome. Giulio Pompilio, scientific director of Irccs Monzino Cardiology Center in Milan, former member of the EMA Committee for Advanced Therapies, took part in the second edition of the ‘science show’ organized by Ota – Advanced Therapies Observatory, in attendance after the stop imposed by the pandemic. and president of the Ota scientific committee, Maria Luisa Nolli, member of the board of Assobiotec and EuropaBio and professor of advanced biotechnologies at the University of Pavia, 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, Federico Spandonaro, President of the Scientific Committee of the Center for Applied Economic Research in Healthcare (CREA Sanità) and Marika Pane, Director of the Nemo Pediatric Clinical Center in Rome.
At2 objectives: to take stock of the challenges to be faced and the critical issues to be solved in order to carry out the innovative therapies. From the meeting it also emerged that 2023 will be the year of the ‘retreAT’, the first OTA project ever organized in Italy and entirely dedicated to the new frontier of medicine. The chosen name ‘retreAT’ – explains a note – recalls the great ‘retreats’ to which the international scientific community is already accustomed, while At is the acronym for “Advanced therapies”. It is a process that brings together researchers, clinicians, patient associations, companies and institutions, pushing them not only to identify and analyze critical issues starting from different points of view and experiences but, above all, to formulate concrete and immediately applicable proposals.
The ‘retreAT’ is divided into 5 different thematic tables that will address as many different aspects: the public-private partnership, a fundamental element in the long and costly path of clinical development of an advanced therapy; the manufacture of these innovative therapies, a complex process that can become a bottleneck for the arrival of advanced therapies on the market; clinical research, a particularly critical phase in the development of a therapy – especially in the field of rare and ultra-rare diseases – which can last up to 10 years; the sustainability of advanced therapies and access, a highly topical issue in this last year which has seen the withdrawal of important gene therapies from the market, the organization of centers of excellence and the taking in charge of patients, a final and fundamental step for bringing advanced therapies to their goal.
This last table in particular was considered fundamental in order to take charge of the needs of patients and caregivers, to whom it is necessary not only to make revolutionary therapies available but to ensure that they can easily complete their care path without obstacles or differences. due to the place of residence, or also to the economic possibilities of supporting travel and assistance costs, or without interruptions in the paths due to bureaucratic difficulties. A symbolic table also of the attention to the patient that has always characterized the work of the think-tank and of information of the Advanced Therapies Observatory.
“When it comes to advanced therapies, the emphasis almost always and only falls on the issues of economic sustainability – said Francesco Macchia, coordinator of Ota – but we have chosen to tackle several issues at the same time in the firm belief that sustainability cannot be found in simple limitation of expenditure, but in the full enhancement of investments and in the creation of a truly effective and efficient management system that meets the needs of the patient and at the same time constitutes value for the country. For this reason we felt the need to launch the most ambitious policy shaping project on TA in Italy, the ‘retreAT’ “.
“Advanced therapies – recalled Pompilio – are in effect drugs, but they work in a very different way from ‘traditional’ drugs: they are based on the use of DNA, cells or tissues – often genetically manipulated – capable of acting , generally with a single administration, directly on the cause of the disease and not only on the symptoms. The perspectives opened by these new therapies are incredible and can be applied to many rare and genetic diseases, degenerative diseases and cancers, offering new possibilities even where until yesterday there was no cure. A new frontier of medicine that requires continuous scientific research, an in-depth understanding of the potential and limits of these new drugs, and new challenges in terms of the methods of production, regulation and access to treatments. Understanding that must be formed not only in the scientific community, but also in institutions, patients and citizens in general, who are now more than ever called to a conscious participation in care “.
At2 – continues the note – was conceived in 2019 to bring experts and the general public closer to the field of medicine in order to build a shared and widespread knowledge and awareness, essential for correctly addressing the complex choices concerning the arrival of advanced therapies in the life of everyone: experts, patients and citizens.
“To date, little has been done in a systematic way to ensure equal access for all patients who may benefit from these therapies. But, at the same time, this right must be economically sustainable for the system and not be a disincentive for companies that develop, produce and market advanced therapies – added Macchia, who also opened the works of At2 – The first challenge is certainly the determination of the therapeutic value, a complex concept that can be defined in terms of clinical value (efficacy and safety), pharmacological value (mechanism of action), value for the patient (quality of life, acceptability), economic value (utility) and commercial value “.
Challenges that have been dealt with in the consensus document entitled ‘Proposal of measures for determining the value of Advanced Therapies in Italy’. A study based on the Delphi method, recently carried out by Ota in collaboration with CREA Sanità and to which a panel of experts contributed. The issues highlighted in the Consensus document will be a starting point for the new project of the Advanced Therapies Observatory: the retreAT, announced during At2. The Advanced Talks on Advanced Therapies – At2 event was organized with the sponsorship of the Telethon Foundation and with the non-conditioning contribution of Bristol-Myers Squibb, CSL Behring, Miltenyi Biotec, Novartis, Orchard Therapeutics, Pfizer, PTC Therapeutics, Sanofi and Vertex .