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

2. Discovery research = innovative part
Compound libraries
development
Drug candidate

3. 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

4. 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

5. 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):

6. cost of the drugs - around 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