Rare disease use of clinical trial simulation for the chioce and optimization of study designs/Priomedchild Call/ER
Lead Research Organisation:
University of Manchester
Department Name: UNLISTED
Abstract
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Technical Summary
Background: Rare diseases are defined based on their low incidence, less than one in 2,000. Whereas randomized controlled trials (RCTs) are currently the gold standard for drug evaluation, there is still no general validated approach for the assessment of (orphan) drugs in this field.|Objectives of the CRESim (Child-Rare-Euro-Simulation) project: To develop a platform performing trial modelling and simulation in order to identify optimal trial designs in children for the evaluation of (orphan) drugs tailored to different types of rare diseases.|Methods: In silico approach using RCT modelling and Monte Carlo simulation, implemented for different categories of rare diseases in pediatrics. Several RCTs with different experimental designs will be modelled and simulated, and the different designs will be compared in terms of trial duration and precision of the estimation of the treatment effect.|Expected Outcomes: Improvement and optimization of the development of new orphan drugs.|Dissemination of results: Dissemination will target those involved in drug development (i.e pharmacologists, therapeutic specialists, methodologists and statisticians), and in drug registration (public health decision makers), and registration authorities (EMEA and national agencies).
People |
ORCID iD |
Leon Aarons (Principal Investigator) |
Publications
Wendling T
(2016)
Predicting survival of pancreatic cancer patients treated with gemcitabine using longitudinal tumour size data.
in Cancer chemotherapy and pharmacology
Description | IMI |
Amount | € 600,000 (EUR) |
Organisation | European Commission |
Department | Seventh Framework Programme (FP7) |
Sector | Public |
Country | European Union (EU) |
Start | 06/2012 |
End | 04/2017 |
Description | Cystic Fibrosis |
Organisation | Erasmus MC |
Country | Netherlands |
Sector | Hospitals |
PI Contribution | Jointly we have developed a KPD model for DNase alpha. |
Collaborator Contribution | They performed the literature revied and provided some data for model validation. |
Impact | KPD model for DNAse alpha |
Start Year | 2011 |
Description | Dravet Syndrome |
Organisation | University of Lyon |
Department | Department of Clinical Pharmacology |
Country | France |
Sector | Academic/University |
PI Contribution | Jointly we have developed a minimum PBPK to desribe the PK of clobazam, stiripentol and valproate |
Collaborator Contribution | They have reviewed the model with some imputs into the structural model |
Impact | A minimal PKPK model for clobazam, stiripentol and valproate. |
Start Year | 2011 |
Description | Lymphoma |
Organisation | University of Lyon |
Department | Department of Clinical Pharmacology |
Country | France |
Sector | Academic/University |
PI Contribution | A framework for the development of minimal PBPK model for methotrexate and mercaptopurine |
Collaborator Contribution | Feedback on the structural model |
Impact | A minimal PBPK model for Methotrexate and Mercaptopurine |
Start Year | 2012 |