The pharmaceutical R&D process Discovery Development Use 10-12 years, costly ($30 Mn – $1300Mn)

Discovery research = innovative part Compound libraries development Drug candidate

Risks in research and development of pharmaceutical drugs Reasons for failure include: Lack of bioavailability Toxicity Lack of efficacy But also: Patent position Market prospect / competitors Mergers/acquisitions development

Therapeutic innovation : many different ways to innovate including new or better uses of existing molecules / treatments Existing compounds: Library screening & compound mining New compounds through medicinal chemistry New uses of existing compounds New indications of existing drugs Improved formulations of existing drugs Completing registration dossier Geographical extension Combination treatments Simplifying use Changing guidelines EML

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):

cost of the drugs - around 10 000 US$/year for life long Tx - put treatment beyond reach for the millions of people living with the virus in developing countries. Most experts also maintained that the treatment was too complex to be implemented in developing countries and essentially these patients were written off. Treatment versus prevention debate Why so expensive: monopolies through patents; single producers for the first generation of ARV’s few words on what is a patent: public policy incentive to stimulate innovation by providing a temporary monopoly to produce and sell the “invention”, in exchange for publication; monopoly has to be applied for in each country where protection is sought; it allows the patent owner to prohibit others from using/selling the invention 3 main criteria: novel, inventive, indusctrial applicability