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Therapies
Sous presse. Epreuves corrigées par l'auteur. Disponible en ligne depuis le dimanche 8 mars 2020
Doi : 10.1016/j.therap.2020.02.005
Received : 16 September 2019 ;  accepted : 15 November 2019
A methodological framework for drug development in rare diseases. The CRESim program: Epilogue and perspectives
 

Patrice Nony a, b, , Behrouz Kassai a, b, c, Catherine Cornu b, c

for the CRESim, Epi-CRESim research groups1

  Members of the CRESim and Epi-CRESim project groups: Leon Aarons, Corinne Alberti, Agathe Bajard, Pascal Benquet, Yves Bertrand, Frank Bretz, Daan Caudri, Charlotte Castellan, Sylvie Chabaud, Catherine Chiron, Catherine Cornu, Frank Dufour, Nathalie Eymard, Roland Fisch, Renzo Guerrini, Vincent Jullien, Behrouz Kassai, Polina Kurbatova, Salma Malik, Rima Nabbout, Patrice Nony, Kayode Ogungbenro, David Pérol, Gérard Pons, Anna Rosati, Harm Tiddens, Fabrice Wendling.

a Service hospitalo-universitaire de pharmacotoxicologie (SHUPT), hospices civils de Lyon, 69424 Lyon, France 
b Laboratoire de biométrie et biologie évolutive (LBBE), UMR 5558 CNRS, University Lyon 1, 69376 Lyon, France 
c EPICIME-CIC 1407 de Lyon, hospices civils de Lyon, Inserm, 69677 Bron, France 

Corresponding author. Service hospitalo-universitaire de pharmacotoxicologie, hospices civils de Lyon, 69424 Lyon, France.Service hospital-universitaire de pharmacotoxicologie, hospices civils de LyonLyon69424France
Summary

Based on the ‘European Child-Rare-Euro-Simulation’ (CRESim) project, this article proposes a generalizable strategy utilizing datasets analysis in combination with modeling and simulation, in order to optimize the clinical drug development applied in the field of rare diseases. The global process includes: (i) the simulation of a realistic virtual population of patients (modeled from a real dataset of patients), (ii) the modeling of disease pathophysiological components and of pharmacokinetic-pharmacodynamic relations of the drug(s) of interest, (iii) the modeling of several randomized controlled clinical trials (RCTs) designs and (iv) the analysis of the results (multi-dimensional approach for RCTs durations and precision of the estimation of the treatment effect). However, whereas modeling and numerical simulation may provide supplementary tools for drug development, they cannot be considered as a substitute for RCTs performed in ‘real’ patients.

The full text of this article is available in PDF format.

Keywords : Integrative modeling, Numerical simulation, Randomized controlled clinical trials, Drug development, Rare diseases



 This article has been written as part of the Child-Rare-Euro-Simulation (CRESim) and Epilepsy-CRESim (Epi-CRESim) projects.

1  Members of the CRESim and Epi-CRESim project groups: Leon Aarons, Corinne Alberti, Agathe Bajard, Pascal Benquet, Yves Bertrand, Frank Bretz, Daan Caudri, Charlotte Castellan, Sylvie Chabaud, Catherine Chiron, Catherine Cornu, Frank Dufour, Nathalie Eymard, Roland Fisch, Renzo Guerrini, Vincent Jullien, Behrouz Kassai, Polina Kurbatova, Salma Malik, Rima Nabbout, Patrice Nony, Kayode Ogungbenro, David Pérol, Gérard Pons, Anna Rosati, Harm Tiddens, Fabrice Wendling.


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