The «advanced therapies», like the gene and cell therapiesoffer new opportunities for treatment and recovery for serious illnesses that were once incurable. Theirs costhowever, it can be a lot highfor which a sustainability problem economic for the National Health Service. But how to guarantee fairness and equal treatment to all the patients who need these innovative treatments? This was discussed during a conference, «Advanced Therapies: from sustainability to organizational models in the area», organized by the Higher Institute of Health and Assobiotec – National Association of Federchimica for the development of biotechnology.
What are advanced therapies
Advanced therapies, called ATMPs (Advanced Therapy Medicinal Products), are made up of cells or tissues, possibly engineered, or nucleic acids. They include the cell therapies, gene therapies, tissue engineering, combined productsthe. They are protagonists of an epochal revolution since for the first time in the history of medicine it has been possible to develop drugs based on biological material, personalized and who aspire to heal the patient with a single administration. By intervening directly on the causes of the disease, in fact, they offer new ones healing prospects for pathologies that until now had no therapeutic solutions.
Their main characteristics are:
▪ tsignificantly transform the clinical history of patients affected by diseases that have no therapeutic alternative, and are able to restore, correct or modify compromised physiological functionsalso with the correction of acquired mutations on a genetic basis and the addition of copies of functioning genes;
▪ I am developed to have a relevant clinical benefit for serious and disabling diseases often with unfortunate outcomes, rare diseases and with little or no therapeutic options available;
▪ are administered in a single treatment (one-shot), unlike traditional drugs and protocols, which require repeated and regular treatments;
▪ have high investment costsi, since these are highly complex and productive personalized therapies, but which present notable benefits futures in clinical, therapeutic, social and economic terms for health systems and patient health;
▪ they are therapies administered only in qualified and specialized centres and arise from extremely innovative and complex platforms.
They have been approved in Europe 25 advanced therapies, 18 of which are currently on the market. Of these 18 therapies, Italy has granted reimbursement to eight.
Key role for Italy
According to a recent report by Alliance Regenerative Medicines, there are more than 1,990 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). In Europe there are 360 clinical trials with around fifty in the pre-authorisation phase. Italy has always had a key role in the research and development of advanced therapies: among the first advanced therapies approved by EMA, the European Medicines Agency, four are the result of Italian research. And today the commitment continues with important economic investments and 23 clinical studies underway in 2022 in medical areas with unmet clinical needs such as rare diseases, neurodegenerative diseases, tumors.
Right to care and sustainability
«The theme of transfer of carein this case advanced therapies, from the laboratory to the patient’s bedside is fundamental and, although these therapies represent an important hope of cure pfor some previously incurable diseases, there are some obstacles to the real possibility of use by patients» says the president of the Higher Institute of Health Rocco Bellantone, according to whom «the adoption of innovative models» is necessary to ensure that this transfer is «effective, timely, fair and sustainable».
Robert Giovanni Nisticò, new president of Aifa, the Italian Medicines Agency, reports: «Cwe are focusing on the need to ensure equitable and rapid access for patients and maintain the sustainability of the healthcare system. We must think about new schemes, because complex problems require complex solutions.”
60 new gene and cell therapies by 2030
A very current topic also because, as the president of Assobiotec Fabrizio Greco reports, «it is estimated that Up to 60 new gene and cell therapies could be launched globally by 2030which could concern them overall 350 thousand patients. With reference to the economic impacts in Italy, recent analyzes report for the year 2023 an expenditure of between 132 and 264 million eurosup to an expense borne by the national health systems between 905 and 1,810 million euros for the year 2027». Hence the need, according to Greco, for «a permanent discussion table between all the actors of the Health System to build a new organizational model capable of guaranteeing equity of access and care. Last year, a technical table on advanced therapies was established at the Ministry of Health: the hope is that work will start soon” concludes the president of Assobiotec.
