There are advanced therapies, there will be more and more in the future, and we need to prepare ourselves to manage them, since they are not simple pills. Thus, if we looked back to look at what has changed in the scenario of gene therapies, cells and tissues used for therapeutic purposes, in the last year we would find a system increasingly prepared to welcome it. Both from industries and from politics and health systems. But with important differences at regional level in Italy: just think that the average access time for therapies (those reimbursed) in the area is over 9 months (wait for the first regional purchase following the price and reimbursement determination), with some Regions where the times are faster (such as Tuscany, Emilia Romagna and Lombardy) and others where the times are higher (Marche, Friuli Venezia Giulia and Sardinia).
The sixth edition of the magazine tells all this Italian report on Advanced Therapeutic Medicinal Products (ATMP), created by ATMP Forum and dedicated to photographing the state of the art, the prospects and challenges that revolve around advanced therapies. The focus is on Italy, but with a look at what is happening in Europe and the rest of the world.
The news on the therapy front
In the last year, the report recalls, on the therapy front there has been the approval, in the USA, of the first topical gene therapy (a gel indicated against epidermolysis bullosa). Or even the first gene therapy against Duchenne disease, also overseas. But it was also the year of the arrival of the most expensive drug in the world, gene therapy against hemophilia B, both in the USA and in Europe. If these were some of the most important innovations at a global level on the advanced therapy front in terms of new treatments – which have not yet changed the number and type of advanced therapies available in Italy, 8 reimbursed and on the market and 6 under evaluation – the last year has also seen an important change on the production front.
Gene therapy in the form of gel for butterfly children approved in the USA by Anna Lisa Bonfranceshi 20 July 2023
New production models and new partnerships
In fact, in September, the Telethon Foundation became responsible for the production and distribution of the drug against the congenital immunodeficiency Ada-Scid, saved after Orchard Therapeutics Plc stopped investing in the field. In this way the Foundation has become the first charity in the world to be responsible for the production and distribution of a drug, effectively embodying that spirit of change that is crossing the world of advanced therapies. And which in recent times has been characterized above all by mergers and acquisitions between small and large companies, he recalled Pier Luigi Canonico, among the members of the ATMP Forum steering committee, as a clear response to the need to combine the skills, experience and resources necessary for the development of advanced therapies. But the last year has also been marked by considerable political interest, added Canonico, marked by the establishment of the National Ethics Committee for trials relating to ATMPs, at AIFA and by the Technical Table on Advanced Therapies at the Ministry of Health.
Telethon saves gene therapy for ‘bubble children’: it is the first charity in the world to produce and distribute a drug by Dario Rubino 12 September 2023
Spending on advanced therapies
The report also contains data relating to Italian spending on advanced therapies, their diffusion across the territory and the timing of their availability. However, data must be read with caution, he recalled Francesco Trotta, Director of the Health Technology Assessment sector at Aifa, presenting them. In fact, rankings cannot be made, or rather they have limited value when it comes to advanced therapies: there are few patients and it is likely that small numbers can easily change the situation. This situation reflects the presence of active centers for the provision of advanced therapies along the peninsula. Thus, having made the necessary premise, Trotta recalled how spending on advanced therapies (largely supported by the fund for innovative drugs) has increased strongly since 2019, when the provision of these treatments came into full swing. In 2022, spending was around 80 million euros, largely attributable to the use of Car-Ts, and represented 3.4 per thousand of the total. “For now, therefore, advanced therapies do not pose sustainability problems – commented Trotta – it will be a different matter in the future”. That is, when new advanced therapies are developed and arrive, as is expected to happen, given the growing number of trials in the field.
Ema, against haemophilia B ok to the most expensive drug in the world 16 December 2022
As regards the average access times in the different Regions and the North-South divide, it should be remembered that “activating just one center can quickly change the dynamics of provision”, underlines Trotta. And with reduced numbers of patients accessing these therapies – around 400 can be estimated by 2022 – this can change the situation.