1. Introduction to Drug Rediscovery John Lisman Attorney-at-law Lisman Legal Life sciences Insert your logo in this area then delete this text box.

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3. Acknowledgements Most of this presentation is based on a study conducted by –Joris Langedijk, Utrecht University –John Lisman, Lisman Legal Life sciences –Pieter Stolk, Escher Project –Aukje Mantel-Teeuwisse, Utrecht University –Marie-Hélène Schutjens, Schutjens De Bruin

4. Acknowledegments c’tnd Under auspices of Utrecht University Financed through a grant of ZonMw 4

5. Agenda What is Drug Rediscovery? Why Drug Rediscovery? Why does Drug Rediscovery not happen? Political and Policy concerns The promotion of Drug Rediscovery Conclusion

6. What is Drug Rediscovery? Drug Rediscovery: –The development and authorisation of new applications of existing, well-known, active substances. Not: if medicinal is still protected by patent or any other IP-right –Probably not by original manufacturer/patent holder

7. What is Drug Rediscovery? (3) 6-TG in IBD Buformin in Lung Cancer Numerous other treatments

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9. Why Drug Rediscovery? Industry perspective –Pipe lines of drug companies are running dry –Development of NCE costs $ 1 billion Scientific perspective –Pharmacological activity in one indication predicts other possible uses –Expected medical advance when better using old molecules is huge

10. Why Drug Rediscovery? (2) Patient perspective –Many unmet medical needs –Many patients depend on new innovative therapies still to be developed Society perspective –Outlook on affordable effective medicines –Generation of new innovative economic activities

11. Why does Drug Rediscovery not happen? Because there is no return on investment to be expected; Because of the perception that no return on investment is possible; Common view is: –In pharmaceutical industry investments need to be protected by IP rights –No investment is made in old off-patent molecules

12. Why does Drug Rediscovery not happen? (2) Because it is expensive to create the necessary dossier for a marketing authorisation Because investments in clinical evidence of new applications of old active substances seem to be a waste of money Because regulatory systems do not allow easy access for new indications for old active substances

13. Why does Drug Rediscovery not happen? (3)

14. But: Physicians want to treat their patients in the best possible way; Patients find themselves entitled to the best possible treatment; Societies are looking for better allocation of scarce resources

15. Practical concerns The unauthorised use of medicinal products does not pose any problems: –Often medicinal products are used off-label –Often unauthorised medicinal products are being used The authorisation process is bypassed, taking away the urgency of Drug Rediscovery –The good becomes the enemy of the better 15

16. Political and Policy concerns Barriers on a higher level –Current drug development paradigm –Patent and other IP is necessary for any investment –Government takes a passive approach –Strict distinction between innovative and generic industry –Drug development is not national, not even regional, but global

17. The promotion of Drug Rediscovery Proposals (initially national) –Regulatory: start dialogue about clinical development of new indication –Legal: create new regulatory pathway for Drug rediscovery –Policy: Limit availability and reimbursement of off-label used medicinal product –Practical: Create a Center for Drug Rediscovery

18. The promotion of Drug Rediscovery (2)

19. Promising developments 19 National attention Drug Rediscovery in the Netherlands National attention in US

20. Promising Developments (2) Example from the Netherlands –6-TG for IBD (presentation prof. Chris Mulder) Example from the US –Buformin for lung cancer (presentation prof. Steven Lehrer) 20

21. Conclusion Drug Rediscovery may prove to be a hugely beneficial approach to innovation in the pharmaceutical industry Drug Rediscovery is an approach to meet unmet medical needs Drug Rediscovery requires efforts from Academia, Governments and Industry together.

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