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PRIORITY MEDICINES
FOR EUROPE AND THE
WORLD
A report prepared by WHO for the
Netherlands Government
by
Warren Kaplan
Richard Laing
and
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Saloni Tanna
Eduardo Sabaté
Joyce Wilson
Ann Wilberforce
Marjolein Willemen
Monique Renevier
Lisa Greenough
Kathy Hurst
Special Thanks to:
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Prof. Bert Leufkens (U. Utrecht)
Prof David Henry (U. Newcastle, Australia)
Authors of Background papers
Industry and NGO members
Dutch team of Martijn ten Ham, JanWillem Hartgerink and Bart Wijnberg
Presentation Outline
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Context and Background
Objectives and Methods
Results
Conclusions
Context/Background
• Pammoli, G-10 and EU Commission
Reports
• The Lisbon and Barcelona European
Councils: the “3% solution”
• Framework Programmes FP6
FP7
• European and Developing Countries
Clinical Trials Partnership (EDCTP)
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Objectives of Priority
Medicines Project
• Provide a methodology for identifying
pharmaceutical “gaps” from a public health
perspective, for Europe and the World .
• 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”
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"Priority Medicines"
• Medicines which are needed to meet the
priority health care needs of the population
but which have not yet been developed.
• “pharmaceutical gap”: when treatment for a
disease/condition:
– does not yet exist OR
– will become ineffective soon OR
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– is available but the delivery mechanism or
formulation is not appropriate for the target
patient group.
Generating a Preliminary List of
Diseases and Gaps
Cochrane database of
systematic reviews
Clinical efficacy
Burden of disease ranking
EU10, EU25
The world (including EU25)
Projections
and trends
PRELIMINARY LIST
OF PRIORITY DISEASES AND
GAPS
IN DEPTH REVIEWS OF PRELIMINARY LIST OF DISEASES AND GAPS
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FINAL REPORT
Social
solidarity
Commonality of interest
for Europe and the World
• Considerable commonality of interest
exists for chronic diseases
– Antibacterial resistance & pandemic influenza
– Ischemic heart disease, diabetes, cancer, acute
stroke, alzheimers and other dementias,
osteoarthritis, COPD, & depression
• Social Solidarity needed for:
– HIV/AIDS, Tuberculosis, Neglected Diseases,
Malaria, Maternal Haemorrhage
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Global Public Health Threats
Antibacterial Resistance:
• Infectious diseases: low burden in Europe
removes incentive for R&D
• Most antibiotics are inexpensive- removing
incentives to create new antibiotics
• Antibacterials are widely misused creating
resistance
• Little R&D on antibacterials has consequences
for future generations with the global increase in
the spread of drug-resistant bacteria.
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The Rise of Antibacterial Resistance
and the Decline in Innovation
Antibacterial new molecular entities
approved for use in the United States
1983-2002
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No. of FDA approved antibacterial NMEs
The proportion of MRSA among positive
blood cultures of Staphylococcus Aureus in
England &Wales1989-2002
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0
1983-1987
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1988-1992
1993-1997
1998-2002
Global Public Health Threats
Pandemic Influenza:
• Overdue for a new pandemic
• Uptake of existing vaccines in Europe is poor
compared to Canada, US, Korea and
Australia
• Insufficient capacity to produce vaccines and
antiviral medicines
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Secondary Prevention of
Cardiovascular Disease & Stroke
• 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" using fixed dose combination
(aspirin, statin, ACE inhibitor and betablocker or thiazide diuretic) deserves
further urgent study.
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High burden, preventable diseases
with pharmaceutical gaps
Smoking-related conditions:
• Public health anti-smoking policies are key interventions
• Effective pharmaceutical interventions are needed.
Treatment of acute stroke:
• A major basic and clinical research effort is required as
the current treatment of acute stroke is unsatisfactory.
• Most agents are not effective: associated with increased
risk of adverse events.
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High burden, preventable diseases
with pharmaceutical gaps
HIV/AIDS:
• HIV formulations for children urgently needed
• HIV Vaccine
Alcoholic liver disease:
• Reduce prevalence and incidence of alcohol
abuse
• Translational research to convert basic science
advances into products for clinical trials.
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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
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High burden diseases where existing
therapies could be improved
Cancer :
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More capacity (infrastructure and human resources) and
coordination to conduct comparative clinical trials
Continue to invest in basic research into cancer biology
Diabetes:
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Heat stable insulin would be a major advance in public health
Gaps in basic biology, stem cell research, transplantation
research
Depression in adolescents & elderly:
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Gaps in understanding biology of depression and its
treatments in these groups
"Neglected" diseases
Lack of EU support for translational
research for market failure diseases
Malaria:
• Lack of experimental models for medicines discovery and
development.
Tuberculosis:
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More FDCs for second-line treatment of multidrug-resistant
TB & Diagnostics
Leishmaniasis, trypanosomiasis, Buruli ulcer:
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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 a major advance in public health for women
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Special Needs for Women,
Children, and the Elderly
• All groups neglected in drug development
• Complicated by different physiology &
metabolism
• Recent improvements in situation of women and
children
• Considerable gaps remain for the elderly who
use the most medicines
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Promoting Innovation and
Removing Barriers
• Public Private Partnerships may be a vehicle
to address market failure
• Pricing issues are critical to the future of the
European pharmaceutical industry. Propose
investigating differential pricing based on GNI
per capita and efficacy measures
• EMEA, FDA, Rawlins and Industry have all
proposed similar measures to remove barriers
• Comparative trials provide critical information
on head to head comparisons. Use of European
databases may facilitate such studies
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Role of Patients remains
unclear
• Patients have speeded innovation e.g.
AIDS and Orphan diseases
• Valuable role in treatment guideline
development emerging e.g. NICE
• Patients play important role in ethical &
hospital committees e.g. IRB & DTCs
• Will now be part of CSM in UK
• Future role likely to be important and
growing
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Conclusions
• 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,
smoking and neglected diseases
• Pricing issues and barriers to innovation strongly affect
the European industry
• The EU needs to find a way to support translational
research for market failure pharmaceutical gaps
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Priority Medicines Project
For further questions, please contact:
[email protected]
[email protected]
+41-22-791-4533
http://mednet3.who.int/prioritymeds/report/index.htm
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Differential Pricing: Indicative prices in
US$/annum of highly active antiretrovirals
(HAART) and a new hypothetical regimen in
countries of variable wealth
30000
United Kingdom
France
Italy
25000
15000
Slovenia
Czech Republic
10000
Latvia
5000
Indicative price existing treatment
26076
23225
15000 20000 25000
Per capita GNI $US
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22783
19831
15627
12620
5000
10881
6199
4921
3744
3477
2500
2566
2557
2000
2401
2125
1959
1000
1637
1214
993
500
855
607
441
200
763
Russian Fed.
Kazakhstan
Philippines
Georgia
India
294
267
Mali
0
487
Indicative price $US
Spain
20000
Indicative price new drug