Objectives and accomplishments of FVBDA Wil Milhous USF College of Public Health

1. Objectives and accomplishments of FVBDAWil MilhousUSF College of Public Health

2. Key References of Academic & PPP Success Stevens AJ, Jensen JJ, Wyller K, Kilgore PC, Chatterjee S, Rohrbaugh ML. The role of public-sector research in the discovery of drugs and vaccines. N Engl J Med. 2011 Feb 10;364(6):535-41.PubMed PMID: 21306239). Kotz, L. Small (molecule) thinking in academia. SciBX 4(22); doi:10.1038/scibx.2011.617. Published online June 2, 2011 Campos KDP, Norman CD, Jadad, AR. Product development public private partnerships for public health: A systematic review using qualitative data. Social Science & Medicine 73 (2011) 986-994.

3. “Optimizing Detection, Prevention, and Treatment of Vector Borne Diseases in Florida” • Multi-institutional/multidisciplinary project: Funded by Florida University System Board of Governors in November, 2010 • UF College of Pharmacy • UF Emerging Pathogens Institute • USF College of Public Health, Department of Global Health • USF College of Pharmacy • Objectives: • Bring together biotech industry, academia, research institutes to facilitate drug, product development in area of vectorborne diseases • Work to introduce FDA’s Critical Path Initiative

4. Implement and continuously refine a structure for the interdisciplinary research activities to include further state wide identification of industrial and academic partners, set-up of efficient communication structures through formal planning meetings.

5. Florida Vector-Borne Diseases Working Group: January 7, 2008 • Jointly sponsored: UF/EPI and USF • Held at Florida Medical Entomology Laboratory, Vero Beach • 65 in attendance: • representation from Academic programs in state involved in work with vector borne diseases • FL DOH, • Representation from key Mosquito Control districts

6. Optimizing Detection, Prevention and Treatment of Vector Borne Diseases in FloridaJan 31, 2011 UF & USF Colleges of Pharmacy USF Center or Drug Discovery and Innovation UF Emerging Pathogens Institute USF Global Health Infectious Diseases Research (GHIDR) Program

7. BioScience Track Florida Health Sciences Gateway Initiative Chair: Steve Bice, MEd Principal, Steve Bice and Associates (Atlanta, GA) Founding Director, CDC’s National Strategic Stockpile Wilbur Milhous, PhD, Professor and Associate Dean College of Public Health, University of South Florida “Product and Process Innovation as Academic Disciplines” Glenn Morris, MD, MPH, TM Director, Emerging Pathogens Institute, University of Florida “Florida Bio-Threats and Emerging Pathogens: Unique Needs and Opportunities” Brennan Klose, PhD Principal, Unither Virology, LLC (Silver Spring, MD) “Development at the Speed of Science” Timothy L. Tellinghuisen, PhD Professor, Scripps Research Institute

8. The challenge of market economy to development of public health interventions Business profit Public health

9. New Florida Cluster Grant: Optimizing Detection, Prevention and Treatment of Vector Borne Diseases in Florida

10. Advertise and Recruit Postdoctoral Fellows Establish Cooperative Research and Development Agreements (CRDAs) and Material Transfer Agreements (MTAs) to support testing and exchange of compounds and reagents within the consortium

11. Product Portfolio Alignment and Needs Assessment. Our Universities have unique grant funded capabilities ranging from: enabling technologiescomputational biologytarget identification and validationlead optimizationADME-Tox, PK/PDcandidate selectionand surrogate markers.

12. Development of Target Product Profiles (TPPs): Using malaria and other neglected diseases as for roadmaps to “drugability,” TTPs will be developed for other vectorborne diseases to include dengue and viral encephalopathies.

13. Reducing Risk with Target Product Profiles Exploratory Discovery Preclinical Clinical Marketing Analysis Lead Optimization Basic Transition Phase 1. . . 2,3,4 NDA FDA-TPPs Target Product Profiles IND/E

14. Malaria Single Exposure Radical Cure Acute uncomplicated malariaSevere and complicated MalariaRadical cure of malaria caused by PV PO.Transmission Blocking Intermittent Preventative Treatment in Pregnant women (IPTp)Chemoprophylaxis

15. Vector Borne Diseases Drugs for Neglected Diseases Initiative • Human African Trypanosomiasis (HAT) • Visceral Leishmaniasis (VL) • ChagasDisease USF-UF Cluster Grant Program • Dengue • Viral Encephalopies

16. Community Engagement Develop and refine a vector borne disease web-site.

17. Educational components: All partners will work together with their PHD and PharmD programs in orphan neglected diseases with emphasis on Pharmacometrics-Drug Discovery & Development- Epidemiological aspects of on vectorborne diseases.

18. Market Consortium Product Portfolio and Target Product Profiles to Industry Partners. Establish CRDA/MTA agreements with commercial partners.

19. R&D ConfoundersThe Research Bermuda Triangle Time Risk $

20. Tour of CDDI/GHIDR facility BSL3 laboratory level 2 insectory ABSL2 animal facility

21. Leveraging the Florida Cluster Grant Program 2010-2011 Bill and Melinda Gates Foundation Funding $1.5 million -Dennis Kyle Advanced Devices that Mimic Human Liver in Malaria Patients $4.1 million -John Adams Long-term Continuous Culture for Vivax Malaria Drug Assays $2.1 million – Tom Unnasch Novel Elimination and Control of River Blindness

22. Leveraging the Florida Cluster Grant Program “Validation of New Antimalarial Leads” RFA for NIAID Partnerships with Product Development Public-Private Partnerships $6.2 M Medicines for Malaria Venture (Geneva, Switzerland) St Jude’s Children’s Research Hospital Rutgers University $2.01 M for USF for 5 Years Early Investigator R01 Roman Manesch Malaria Liver Stage R21-John Adams Vivax Malaria Mutants

23. The Critical Path

24. Regulatory Strategies Discovery Preclinical Clinical Exploratory Sensitivity Specificity Detection Validated Animal Models Basic Biology Transition Patho- genesis Target ID Validation Phase 1. . . 2,3,4 NDA Critical Path Initiative IND/E Leonard Sacks Current FDA CPI Director & Previously at Special Pathogens

25. DDT Qualification Program Goals • Support stakeholders who are trying to establish an acceptable Drug Development Tool. • Provide an organized structure and process for interactions in a consistent and responsive manner. • For animal models, a goal is to reduce use of resources and animals, and ultimately, decrease development time for medical countermeasures.

26. DDT Qualification Program: A New Pathway FDA Consultation & Advice Phase Planning Phase FDA Review Phase Greater Efficiency Letter of Intent (LOI), Briefing Document DDT Available Publicly  Methods & Results Sharing  Dossier Submission; FDA Review 

27. DDT Qualification Program Goals • Support stakeholders who are trying to establish an acceptable Drug Development Tool. • Provide an organized structure and process for interactions in a consistent and responsive manner. • For animal models, a goal is to reduce use of resources and animals, and ultimately, decrease development time for medical countermeasures.

28. Draft Guidance for Industry - Animal Models – Essential Elements to Address Efficacy Under the Animal Rule, published 1/2009 http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM078923.pdf Draft Guidance to Industry - Qualification Process for Drug Development Tools, published 10/2010 http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm121568.htm Drug Development Tools (DDT) Qualification Program, posted 12/2011 http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/default.htm FDA References