1. 1 PRIORITY MEDICINES FOR EUROPE AND THE WORLD: a public health approach to innovation Hans V. Hogerzeil Director, Medicines Policy and Standards WHO, Geneva based on a WHO report by Warren Kaplan Richard Laing

2. 2 Objectives Provide a methodology for identifying pharmaceutical “gaps” from a public health perspective Provide a public-health based pharmaceutical R&D agenda for use by the EU in the 7th Framework Programme Good public policy should spend public funds on areas of greatest public needs

3. 3 Priority Medicines Medicines for priority health care needs of the population which have not yet been developed: "missing essential medicines" Pharmaceutical gap: when treatment for a disease/condition: –does not yet exist OR –will become ineffective soon OR –is available but the formulation is not appropriate for the target patient group

4. 4 Generating a Preliminary List of Diseases and Gaps

5. 5 Pharmaceutical "Gap"

6. 6 Example: no pharmaceutical gap

7. 7 Commonality of interest

8. 8 Global Public Health Threats (1) Antibacterial Resistance Low burden of infectious diseases in Europe removes incentive for R&D Most antibiotics are inexpensive – also removes incentives to create new antibiotics Antibacterials are widely misused, creating resistance Little R&D on antibacterials has consequences for future generations (global spread of drug-resistant bacteria).

9. 9 The rise of antibacterial resistance and the decline in innovation The proportion of MRSA among positive blood cultures of S.aureus in The proportion of MRSA among positive blood cultures of S.aureus in England & Wales, 1989-2002 Antibacterial new molecular entities approved for use in the United States 1983-2002

10. 10 Global Public Health Threats (2) Pandemic Influenza Overdue for a new pandemic Poor uptake of existing vaccines Insufficient current capacity to produce vaccines or antiviral medicines Rates of vaccine distribution per 1000 total population by country

11. 11 Secondary prevention of cardiovascular disease & stroke: lack of suitable formulation Patients with a heart attack or stroke could reduce their risk of a repeat attack by 66% by taking 4 medicines (good evidence) Yet uptake is low <20% The "polypill" in fixed dose combination (aspirin, statin, ACE inhibitor and beta-blocker or thiazide diuretic) deserves further urgent study No real incentive for FDC/R&D as most are generics

12. 12 High burden, preventable diseases with pharmaceutical gaps Smoking-related conditions Public health anti-smoking policies and effective medicines needed Treatment of acute stroke Major basic/clinical research effort needed; most agents ineffective HIV/AIDS HIV formulations for children, HIV vaccine Alcoholic liver disease Reduce prevalence and incidence of alcohol abuse; translate basic science advances into products for trials

13. 13 High burden diseases without bio markers Osteoarthritis New diagnostics, biomarkers and imaging technology will help determine who is likely to get osteoarthritis, and the response to treatment Alzheimer disease More sensitive, reliable and valid tools for detecting changes in normal ageing and the onset of early Alzheimer disease needed. Lack of surrogate markers remains a major barrier in the clinical development of AD drugs

14. 14 High burden diseases where existing therapies could be improved Cancer More capacity (infrastructure and human resources) and coordination to conduct comparative clinical trials Continue to invest in basic research into cancer biology Diabetes Heat stable insulin would be a major advance in public health Gaps in basic biology, stem cell research, transplantation research Depression in adolescents & elderly Existing antidepressants works well for adult depression Gaps in understanding biology of depression and its treatments in these groups

15. 15 Neglected diseases Lack of EU support for translational research for market failure diseases Malaria Lack of experimental models for medicines discovery R&D Tuberculosis More FDCs for 2 nd line treatment of multidrug-resistant TB Diagnostics Leishmaniasis, trypanosomiasis, Buruli ulcer Most of the medicines being used are "old" and often dangerous Post-partum haemorrhage Major cause of maternal mortality in developing countries, heat stable oxytocin would be major advance in public health

16. 16

17. 17 Special needs for women, children, and the elderly All these groups neglected in drug development Complicated by different physiology & metabolism Recent improvements for women and children, but Considerable gaps remain for the elderly, who use the most medicines

18. 18 Other issues Public Private Partnerships may be a vehicle to address market failure; need more "public" Pricing issues are critical to the future of the European pharmaceutical industry; prices should reward innovation Role of patients is unclear but likely to increase Some regulatory barriers to innovation exist and need to be reduced

19. 19 Conclusion (1): Different types of gaps Global public health threats High burden of preventable diseases, but –no suitable formulation –no biomarkers –therapy could be improved Neglected diseases –translational research Special needs for women, children, elderly

20. 20 Conclusion (2) Commonality of interest exists for chronic diseases between Europe and the World Priorities can be set based on evidence, trends and projections and social solidarity Pharmaceutical gaps exist as a result of biological challenges and market failure Highest priorities are antibacterial resistance, influenza, cardiovascular disease and neglected diseases Pricing issues and regulatory barriers to innovation strongly affect the European industry The EU needs to find a way to support translational research for market failure pharmaceutical gaps

21. 21 Priority Medicines Project For further questions, please contact: laingr@who.int +41-22-791-4533 www.who.int/medicines http://mednet3.who.int/prioritymeds/report/index.htm