A methodological framework for drug development in rare diseases. The CRESim program: Epilogue and perspectives.

Thérapie
Patrice NonyCRESim, Epi-CRESim research groups

Abstract

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.

References

Nov 1, 1996·Health Economics·K Claxton, J Posnett
Apr 29, 1998·Trends in Pharmacological Sciences·P NonyJ P Boissel
Jun 6, 2000·European Journal of Pharmaceutical Sciences : Official Journal of the European Federation for Pharmaceutical Sciences·S B DuffullL Aarons
Feb 5, 2002·Clinical Pharmacokinetics·Jean-Pierre Boissel, Patrice Nony
May 31, 2002·Clinical Pharmacokinetics·Patrice Nony, Jean-Pierre Boissel
Jan 10, 2003·Journal of Pharmacokinetics and Pharmacodynamics·Sylvie ChabaudUNKNOWN HERapeutic MOdeling and Simulation Group
Sep 23, 2003·Health Technology Assessment : HTA·J ChilcottP Tappenden
Jan 25, 2006·Clinical Pharmacokinetics·Alexia BlesiusPatrice Nony
Sep 19, 2006·Contemporary Clinical Trials·Ismail AbbasJosep Casanovas
Jan 18, 2008·Progress in Biophysics and Molecular Biology·Jean-Pierre BoisselMarie-Aimée Dronne
Jul 30, 2008·Biometrical Journal. Biometrische Zeitschrift·Thomas A GerdsMartin Schumacher
Feb 19, 2009·Emerging Themes in Epidemiology·Hao WangPatrice Nony
Jul 7, 2009·Contemporary Clinical Trials·Agathe BajardPatrice Nony
Jul 9, 2010·Clinical Pharmacology and Therapeutics·N HolfordB A Ploeger
Jan 25, 2011·Health Policy·Steve MorganDevon Greyson
Nov 22, 2011·Best Practice & Research. Clinical Anaesthesiology·Richard B ColquittRobert H Thiele
Dec 12, 2012·British Journal of Clinical Pharmacology·Johan G C van HasseltAlwin D R Huitema
Mar 28, 2013·Orphanet Journal of Rare Diseases·Catherine CornuRima Nabbout
Mar 22, 2014·Journal of Pharmacokinetics and Pharmacodynamics·Kayode OgungbenroUNKNOWN CRESim & Epi-CRESim Project Groups
Mar 22, 2014·Journal of Pharmacokinetics and Pharmacodynamics·Kayode OgungbenroUNKNOWN CRESim & Epi-CRESim Project Groups
Jul 11, 2014·European Journal of Pharmaceutical Sciences : Official Journal of the European Federation for Pharmaceutical Sciences·Kayode OgungbenroUNKNOWN CRESim & Epi-CRESim Project Groups
Jul 24, 2014·Pharmaceutical Research·Kayode OgungbenroUNKNOWN CRESim & Epi-CRESim Project Groups
Jan 24, 2015·British Journal of Clinical Pharmacology·Kayode OgungbenroUNKNOWN CRESim & Epi-CRESim Project Groups
Mar 3, 2015·JAMA Neurology·E Ray DorseyKarl Kieburtz
Mar 10, 2015·Journal of Theoretical Biology·P KurbatovaUNKNOWN CRESim Working Group
Jan 1, 2014·Orphanet Journal of Rare Diseases·Patrice NonyUNKNOWN Epi-CRESim
May 23, 2015·Pharmaceutical Research·D TeutonicoO Della Pasqua
Jul 19, 2015·Journal of Clinical Epidemiology·Agathe BajardUNKNOWN CRESim & Epi-CRESim study groups
Mar 2, 2016·Journal of Health Economics·Joseph A DiMasiRonald W Hansen
Jul 25, 2019·Journal of Pharmacokinetics and Pharmacodynamics·Mariam A AhmedReena V Kartha

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