CAR-T, A Lifesaving Therapy: Expanding Indications and Challenges Ahead

CAR-T, lifesaving therapy: today there are new indications in the clinic with great expectations of healing for a wider range of hematologic cancer patients and also prospects in solid tumors. This comes as the three years defining 'innovation' (and coverage by the related national fund) expire, and new indications are added, in earlier stages of disease (for example in myeloma and in the treatment of adult patients with diffuse large B-cell lymphoma and high-grade B-cell lymphoma refractory), in which CAR-T significantly improves prognosis. At the same time, the issue of cost sustainability and the reorganization of authorized centers arises. In Italy, there are 45, but each with different organizational solutions: hub and spoke in Lombardy, mono hub in Emilia Romagna, networked in Tuscany, no center in Sardinia, while the governance model of Friuli Venezia Giulia emerges, which has identified a dedicated fund of 2.5 million euros per year to ensure the need and a quick and certain access for eligible patients. The goal: to shed light on the realities of the various territories and identify the needs and urgencies of CAR-T treatments. Ultimately, to propose more appropriate and sustainable models of this lifesaving innovation and to highlight the best experiences developed in the Regions. Francesca Futura Bernardi, pharmaceutical sector manager of the Campania Region - in a table proposed by the Innovation Observatory of Motore Sanità among clinicians, public health experts, institutional figures engaged in legislative activity, which closed the Road map among the Regions inaugurated a year ago and unfolded through events and in-depth analysis, in person and remotely, along the Boot, in a webinar held recently with the non-conditioning contribution of Gilead / Kite - reminded that the regional hematological network is being completed in which to the two initial CAR-T centers (polyclinic and Santobono for pediatrics) two more for adults have been added (at Moscati of Avellino and at Cardarelli of Naples, the latter in the process of certification). All nodes from which emerges a winning model implemented in Friuli Venezia Giulia that has established a fund dedicated to CAR-T procedures, allowing an approach in which the procedure is not the last resort but guaranteed to the patient when needed. A reality experienced firsthand by Joseph Polimeniex commissioner in Campania and for some years general director ARCS Friuli-Venezia Giulia: "There is still room for improvement maybe in the analysis of needs - he said - but we have allocated 2.5 million annually for CAR-T, supra-company, regional funds. We aim to estimate the target and need precisely. In my opinion, the silo and spending cap system, now outdated, should be overcome. Allocating impact budgets and meeting the indicated demands is the way forward since we are facing therapeutic options that increase quality and quantity of life. Today we talk about CAR-T, tomorrow it will be Artificial Intelligence." Gaspare Guglielmi, manager at the Cardarelli center in Naples, recalls a study by Tor Vergata and Cattolica in which it emerges that innovative therapies, including CAR-T, "are underutilized in Italy compared to the patients who could use them both for costs (if we wanted to treat everyone it would take 4 billion), and for structural, organizational, and personnel deficiencies of the networks". Guglielmi then referred to the Hcv model, in which years ago despite a very high number of eligible patients and limited resources, it was still possible to treat everyone. Gianni Amunni coordinator of the scientific Ispro emphasized how the theme of innovative lifesaving CAR-T therapy should be on the political agenda considering the biotechnological impact, the new indications, the lifesaving efficacy on patients who have no alternative treatments. On the other hand, the intervention of Claudio Zanon, scientific director of Motore Sanità, who recalled that the fund for innovations is not fully spent by all Regions and the organizational management part significantly affects sustainability.