Emofilia: Castaman, ‘Terapia genica prevede fase di sperimentazione clinica’
(Adnkronos) - “In relazione alle problematiche della terapia genica, essendo non approvata, prevede una fase di sperimentazione clinica che richiede una serie di adempimenti importanti. Per avere il massimo del risultato in efficacia e sicurezza, la gestione da parte dei centri che utilizzano i farmaci deve essere accurata”, lo ha detto il Direttore del Centro Malattie Emorragiche del Careggi di Firenze, Giancarlo Castaman, a margine dell’evento organizzato a Roma da FedEmo per la XIX Giornata Mondiale dell’Emofilia dal titolo Terapia genica e innovazione terapeutica: i nuovi bisogni nell’assistenza MEC.
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00:00 In relation to the problem of the gene therapy, it is clear that, first of all, being a non-approved therapy,
00:08 it requires a clinical and experimental phase that requires a whole series of extremely important improvements,
00:17 both from a legislative point of view, so approval of ethical committees,
00:22 notification of the system by the Ministry of Health and so on, but it also requires the management of the follow-up of these patients,
00:31 which must be absolutely accurate, tempestuous, repeated over time and also involves interfacing with the external monitoring agencies
00:42 in terms of the effectiveness and safety of the therapy.
00:45 In order to ensure that this requires and can have the maximum effective and safe result,
00:52 they must be managed in an absolutely accurate and precise way by the centers that then use these drugs.
01:01 So it is clear that it is a huge commitment compared to the clinical and monitoring tasks of the therapy
01:08 that usually take place in our centers and that these patients are followed in a very different way from other patients,
01:17 because they must frequently come for check-ups, they must be taken, they must be monitored, so on.
01:23 So these are extremely demanding studies, they require a lot from the center to be able to do it.
01:32 This explains why many times the same pharmaceutical companies, when they want to test an innovative drug,
01:38 try to turn to the centers that are notoriously more likely.
01:43 And so it is a short circuit, because obviously the smaller centers are cut off,
01:48 they have less possibility to promote themselves culturally and to adapt and to understand what it means to enter the clinical trials,
01:55 but we can do little about this because they are the same companies that want to have the maximum
02:00 and therefore try to go where the results are more quickly obtainable.