Thanks to the progress of scientific research, today they are available «advanced therapies», such as for example the gene therapywhich they offer opportunities for treatment and recovery for serious illnesses that were once incurableallowing people who suffer from it to have one normal life (or almost). Is access to these treatments really guaranteed to all patients who could benefit from them or not?
This was discussed at «Tempo della Salute», underway in Milan at the Museum of Science and Technology, during a meeting entitled «Advanced therapies – The challenge (to be won) of sustainability», moderated by the editorial manager of Courier Health Luigi Ripamonti, with Marco Alparonevice-president and budget and finance councilor of the Lombardy Region; Patrick Armeniprofessor in Health economics and health technology assessment at Bocconi University in Milan; Concetta Quintarellihead of the Tumor Gene Therapy Research Unit at the Bambino Gesù Children’s Hospital in Rome and member of the Committee for Advanced Therapies of the EMA (European Medicines Agency).
The case of Sma
Spinal Muscular Atrophy (Sma), rare genetic disease which mainly affects the nerve cells of the spinal cord from which direct signals to the muscles originate, progressively compromising the ability to walk and, in the most serious cases, to swallow and breathe.
Until 2017 there were no therapies for SMA, today there are different options, including the genetic one for the treatment of type 1 SMA (the most severe form), which intervenes directly on the genetic defect with a single administration.
Its usefulness and effectiveness, like that of others advanced therapieshowever, depends decisively on the possibility of one timely diagnosis which, in the case of SMA, is made possible by the so-calledextended newborn screening, offered, however, only to children born in some regions,awaiting the signature of the ministerial decree which updates the list of pathologies to be tested with the ENS. A wait that can do the difference between life and deathas underlined once again by a recent case in the news: the death of little Ettore, born a month ago with Sma1 but not diagnosed at birth, because neotal screening for Sma is not carried out in his city, so he did not have could be treated promptly.
Behind the “numbers” there are people
Lombardy is one of the eight Regions who have introduced, at the expense of the regional health service, neonatal screening also extended for SMA, which has been carried out since September for all children born in the region. Marco Alparone, vice-president and budget and finance councilor of the Region, says: «We invested a million euros to do it. The effort we are making is to change our approach: to move from the value of numbers to giving a value to numbers, because behind those numbers there are people’s stories. For the health needs of the community, there is no doubt that we must think about the sustainability of costs, as well as the issue of health outcomes.». In the case of neonatal screening for the early diagnosis of SMA, continues Alparone, it is a «rare pathology, which today finds a therapeutic response in research and innovation. We must team up and think together about direct and indirect costs».
Cost or investment?
The cost of these therapies is sometimes very high: is it sustainable for the Health Service in order to guarantee access to those who can benefit from it?
Economist Patrizio Armeni explained: «Using the two words, cost and investment, when we talk about advanced therapies, makes the difference. The cost is a sacrifice that is usually made in the short term, the investment, however, is made in the long term. With advanced therapies you treat a child only once, who can live and have a good quality of life, even up to 90 years of age. This is an investment and not a cost. One of the mechanisms being worked on to make advanced therapies more sustainable is find solutions to make this expense “spread” over time – continued Armeni –. How you do it? By teaming up, not only in the healthcare sector; another key is the sharing of risk, to ensure that it does not weigh on the community, for example, by making agreements – still little exploited – with producers, pharmaceutical companies”. Again, the economist continues, «investment also means planning. If we think about rare diseases and the fact that we must hope that advanced therapies will become a reality for many people in the future, we must find a path that is sustainable for the community.”
Advanced therapies approved but then “withdrawn” because they were not sustainable
Each drug, before being placed on the market, must follow the established process, in order to guarantee efficacy and safety, to obtain approval from the regulatory body, in Europe Ema, then in Italy Aifa – Agency of the drug, which decides whether it will be reimbursed by the National Health Service, allowing patients to use it for free or by paying a copay.
For the approval of advanced therapies for marketing by EMA, the process is simplified. Concetta Quintarelli explained: «The evaluation is done in Commissions with experts in advanced therapies but, despite the regulatory innovation process, it happens that a third of advanced therapies, which have been evaluated and obtained marketing authorization from the EMAThen they are withdrawn because companies realize they are not sustainable. Patients have waited a long time, they have waited for the development of a drug, its marketing authorization, but then they cannot access it.” What to do? “The pandemic has taught us that we can improve evaluation times and, above all, get in tune with those who are developing the drugs and those who have to use them” said Quintarelli.
The challenge for the future
«In a few years the increase in pathologies that can be treated with advanced therapies will ensure that the costs for the healthcare system are not sustainable – warned Quintarelli -. These therapies cost a lot, but we need to talk about them with regulatory bodies at the beginning, before they are developed, to understand what to do to make these products sustainable. Today most advanced therapies are based on centralized production: if an Italian patient must receive a gene therapy product, it is not produced in Italy, whereby the patient’s cells “leave” from our country to the United States, are genetically modified, the specific therapy for the patient is produced, which then “returns” to Italy to be used. All of this comes with costs. Is this process really necessary? It was until about ten years ago, but today they are there experiences, at a national level, in the production of these therapies. We should go in this direction »she concluded.
November 11, 2023 (modified November 11, 2023 | 4:56 pm)
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