The project "Integration of the pharmacokinetics-dynamics models to the multiple-criteria decision analysis (MCDA) for the perspective risk-benefit assessment of the iron oral chelators in paediatric patients with transfusion dependent haemoglobinopathies" aims to prove the usefulness and benefits of the modelling approach, multiple criteria decision analysis (MCDA), integrated with pharmacokinetics-dynamics models (PK/PD) for the risk-benefit assessment of medicines for rare diseases. To this end, a case study on the use of Deferiprone (DFP), an oral chelator, is carried out. The analysis is based on efficacy (ferritin) and safety (neutropenia and other adverse events) endpoints. It aims to predict the efficacy and safety profile of DFP. The model will be validated with literature data and with an elicitation process involving international clinical and regulatory experts.

ROLE OF THE FOUNDATION
The Foundation deals with the methodological and regulatory aspects of the research. It is involved in the literature search to collect existing data on the DFP efficacy and safety. Furthermore, the Foundation cooperates with the project Scientific Coordinator in the MCDA integration with a PK/PD model to support the relationship between neutropenia and ferritin levels in the DFP case study. The Foundation arranges regular meetings with the experts and it is in charge of the administrative task of the project.

FUNDED BY: Art.6 della Legge regionale del 14 Aprile 2018 n.15 - Interventi finanziari in favore della ricerca per la cura delle malattie rare

DURATION: Nov 2020 - Jun 2022

PARTNERS:

• Italy, United Kingdom