PRIORITY MEDICINES FOR EUROPE AND THE WORLD: a public health approach to innovation

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. 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. 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. Generating a Preliminary List of Diseases and Gaps

5. Pharmaceutical "Gap"

6. Example: no pharmaceutical gap

7. Commonality of interest

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

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

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. 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. High burden diseaseswithout 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. 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. Neglected diseasesLack of EU support for translational research for market failure diseases Malaria • Lack of experimental models for medicines discovery R&D Tuberculosis • More FDCs for 2nd 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

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. 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. 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. 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. 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