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Explore the pharmaceutical R&D process, from discovery to lead optimization, and discover methods to innovate efficiently while managing risks. Learn how compound mining and literature reviews lead to promising drug candidates like fexinidazole for sleeping sickness.
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10-12 years, costly ($30 Mn – $1300Mn) The pharmaceutical R&D process
Risks in research and development of pharmaceutical drugs (Preziosi, P. 2004, Nature Reviews in Drug Discovery 3, 521-526)
The pharmaceutical R&D processmany different ways to innovate • Innovation in discovery: identifying new molecules, new drug candidates • - Basic research to understand critical pathways that can be blocked • - Development of test systems to model these pathways • - Screening of large number of molecules (=libraries) to identify « hits » • - Hit-to-lead characterisation and selection: druggability • - Lead-optimisation: more potent, better absorbed, less toxic,
The pharmaceutical R&D processmany different ways to innovate • new or better uses of existing molecules / treatments
Compound mining:creative utilization of existing knowledge Nitroimidazoles: rediscovered drug candidates for protozoal diseases Extensive literature / patent review, including personal consultations with researchers (previously) active on this class of compounds to uncover possible compounds of interest >600 compounds identified from >15 sources; assessed in in vitro / in vivo disease models; several series with promising activity identified 1 new drug candidate in clinical development for sleeping sickness: fexinidazole (off-patent):
The pharmaceutical R&D processmany different ways to innovate