Descrizione del progetto
Progettazioni e strumenti innovativi per la valutazione dei farmaci volti a contrastare le malattie rare
La valutazione dell’efficacia e della sicurezza dei nuovi farmaci per le malattie rare, in particolare in condizioni pediatriche, pone diverse sfide, tra cui le dimensioni ridotte dei campioni di pazienti, l’eterogeneità degli stessi e delle malattie che li colpiscono e la conoscenza patologica limitata, che ostacolano l’accesso a un trattamento efficace e restringono le opzioni disponibili. In questo contesto, il progetto INVENTS, finanziato dall’UE, si prefigge di perfezionare le traiettorie patologiche longitudinali basate su modelli, di migliorare i modelli di estrapolazione e di implementare le sperimentazioni in silico, prendendo in considerazione le esigenze dei pazienti, al fine di far progredire il processo decisionale normativo. I risultati offerti dal progetto dovrebbero consentire ai ricercatori, alle autorità di regolamentazione e all’industria farmaceutica europea di sfruttare progettazioni innovative di sperimentazioni cliniche, studi in silico e approcci di analisi dei dati reali. I pazienti affetti da malattie rare potranno in tal modo beneficiare di un accesso più ampio e rapido a trattamenti efficaci e sicuri.
Obiettivo
The evaluation of new medicines for rare diseases (RD) including rare paediatric RDs is challenging for several reasons, among which are the small patient sample sizes, heterogeneity of patients and diseases and heterogeneity in disease knowledge. Due to these difficulties, access to effective treatments and the number of treatment options are often limited in RDs.
INVENTS aims to provide clinical trial trialists, researchers and regulators with a global framework encompassing methods, workflows and evidence assessment tools to be implemented in orphan and paediatric drug development. Our ambition is to significantly improve the evaluation of evidence and regulatory decision-making through the development and validation of: refined longitudinal model-based diseases trajectories and treatment effect, improved extrapolation models, in silico trials (e.g. virtual patient cohorts), optimised model-based clinical trial designs and evidence synthesis methods. These will be evaluated through simulation studies and tested on extensive data from a range of use cases provided by our industrial partners Roche and Novartis and Real World data (RWD) from RD registry. The INVENTS framework will improve consistency and efficiency of the drug evaluation process for RD by augmenting clinical evidence without compromising its scientific integrity and providing regulators assessment credibility criteria.
At the end of this 5 years project, the European industry will be able to exploit novel and improved clinical trial designs, in silico trials and RWD analysis approaches supporting drug development in RD. The European Medicine Agency and European national regulators (including Health Technology Assessment bodies) will be supplied with a general framework allowing better informed decision-making. Most importantly, RD patients will benefit from an increased and faster access to efficacious and safe treatments.
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HORIZON-RIA - HORIZON Research and Innovation ActionsCoordinatore
75654 Paris
Francia