The first agreement (with Novartis) for therapies that allow to reprogram some cells of the patient (the T lymphocytes), in order to make them able, once reinfused, to recognize and target the tumors, is approved by the CPR and CTS of Aifa. Now the agreement will have to be ratified by the AIFA Board and then published in the Official Journal. We talked about it with former DG Aifa Nello Martini.

The agreement between Aifa and Novartis for the new Car-T therapies is ready. An innovative agreement, all based on the payment by result model and which can certainly be a reference also at European level.

The new therapy called tisagenlecleucel (Kymriah) may be arranged for pediatric patients and young adults up to 25 years of age with acute lymphoblastic leukemia (ALL) with B cells that is refractory, in post-transplant recurrence or in second or further recurrence and for adult patients with diffuse large B cell lymphoma (DLBCL) in relapse or refractory after two or more systemic therapy lines.

A real revolution that allows to reprogram some cells of the patient (the T lymphocytes in fact), in order to make them, once reinfused, to recognize and target the tumors. Of all the patients eligible for therapy, the cure rate is 40-50%.

The agreement, which was approved by the CPS and the CTR of Aifa, now to be operational will have to be ratified by the Cd of Aifa (the new ministerial appointments are scheduled for next week after the State-Regions has ratified the guarantee presidency until in September of the President of the Regions Stefano Bonaccini and the confirmation of the board member Davide Caparini ndr.) and then it will have to be published in the Official Gazette.

The total price for a complete therapy will be around 300 thousand euros per patient but what represents the novelty of the agreement is the fact that the State (the therapy will become part of the innovative cancer fund) will pay based on the actual results on the patient . The scheme provides that, after payment of the first bag, the subsequent tranches (at 6 months and 12 months) are paid only if the patient is remitted. In short, you only pay if the therapy works.

A very innovative agreement that follows a model already experimented in the 2000s when Aifa was the Director General Nello Martini whom we heard to make us comment on how this type of agreement can represent the future model to make new innovative therapies sustainable.

“We can say that thanks to the excellent work of Cpr and Cts of Aifa we are facing the negotiation among the most modern and advanced in the world that opens the way to a new negotiation model for innovative therapies such as Car-T and gene therapies “Says Martini, who also explains how this was possible:” Virtually only Italy has a single patient register and network hematologies that certify the RWE result in clinical practice and not in trials. In short, there is clinical appropriateness and you pay only if there is a result and the failures are the responsibility of the company “.

“And all this – he points out – represents an innovative model (the first in Europe) for the new government of high-cost drugs that is not, for example, one based on the calculation of investments supported by companies in research and development and for production costs or that of the Car-T ‘low cost’ that they would like to experiment in Italy ”.