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PRIORITY MEDICINES FOR EUROPE AND THE
WORLD A report prepared by WHO for the
Netherlands Government by
Warren KaplanRichard Laing
and
Saloni Tanna Marjolein WillemenEduardo Sabaté Monique RenevierJoyce Wilson Lisa GreenoughAnn Wilberforce Kathy Hurst
2
Priority Medicines Project
1. Background/Introduction
2. Priority Setting
3. Methods
4. Results/Conclusions
3
Context/Background
• Pammoli, G-10 and EU Commission Reports– Europe was “lagging behind in its ability to generate,
organize, and sustain innovation processes that are increasingly expensive and organizationally complex.”
• 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– is available but the delivery mechanism or
formulation is not appropriate for the target patient group.
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What this Report does not address
• Health system issues such as access or quality of care, or logistical or sociological barriers.
• Underlying risk factors which can be considered a major cause of morbidity or mortality.
• Availability of diagnostics or medical devices • Relationship between trade, pricing, intellectual
property, as this is the subject of the WHO Commission on Intellectual Property Rights, Innovation and Public Health.
(See http://www.who.int/intellectualproperty/en/)
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Priority Medicines Project
1. Background/Introduction
2. Priority Setting
3. Methods
4. Results/Conclusions
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Prioritization must be multifactorial
INTUITION
ANALYSIS
Quality ofAnalysis
Quality ofIntuition
Least precise/explicit Most precise/explicit
MODE: 7 6 5 4 3 2 1
Knowledge Generation
Decision/Policy Making
non-cognitivejudgment
clinicaljudgment
expertconsensusjudgment
descriptivemodels
casecontrolstudy
randomizedcontrolled
trial
laboratoryexperiment
non-cognitivejudgment
clinicaljudgment
expertconsensusjudgment
decisionmodels
A Cognitive Continuum Framework
Source: Adapted from Dr. Kenneth Hammond, Univ. Colorado, USA& NICE (UK)
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Priority Medicines Project
1. Background/Introduction
2. Priority Setting
3. Methods
4. Results/Conclusions
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Generating a Preliminary List of Diseases and Gaps
Burden of disease rankingEU10, EU25
The world (including EU25)
Cochrane database of systematic reviews
Clinical efficacy
FINAL REPORT
Projectionsand trends
Socialsolidarity
PRELIMINARY LISTOF PRIORITY DISEASES AND
GAPS
IN DEPTH REVIEWS OF PRELIMINARY LIST OF DISEASES AND GAPS
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Pharmaceutical "Gap"Treatment of ACUTE Stroke (Outcome: Survival at end of treatment or follow-up, unless noted otherwise)
0
0.2
0.4
0.6
0.8
1
1.2
1.4
1.6
1.8
Rel
ativ
e R
isk
(<1
favo
rs p
lace
bo)
Various excitatory nerve amino acid antagonists
Ion channel,modulators
NMDAantagonists
Fibrinogendepleting agents
Gangliosides
Antiplatelettherapies
Streptokinaseurokinase( 7 days)Glycerol
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Example of an absent pharmaceutical gap
Secondary prevention of occlusive event (Stroke/MI) with antiplatelet therapy
0
0.5
1
1.5
2
2.5
. . . . . . . . . . . .
Rel
ativ
e ri
sk (
<1
favo
urs
pla
ceb
o)
Prior MI
Prior MI
Prior stroke All trials
Aspirin any dose
Dipyridamole
Sulfinpyrazole
Ticlopidine
Suloctidil
Picotamide
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Priority Medicines Project
1. Background/Introduction
2. Priority Setting
3. Methods
4. Results/Conclusions
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"Commonality of interest"EUROPE THE WORLD
?? ??
?? ??
* Includes bladder, breast, cervical,colon, uterine, lungliver, mouth, oesophageal, ovarian, pancreatic,prostate, stomach cancer and leukemias, melanomas,lymphatic cancers and myelomas
** "Cerebrovascular disease"
*** Chagas disease, Dengue, Leishmaniasis, lymphaticfilariasis, Onchocerciasis, Schistosomiasis, Trypanosomiasis
10% 8% 6% 4% 2% 0 2% 4% 6% 8% 10%Antimicrobial Resistance
Pandemic Influenza
Ischaemic Heart Disease
Diabetes Mellitus
Cancer*
Acute Stroke**
HIV/AIDS
Tuberculosis
Neglected Diseases***
Malaria
Alzheimer and other dementias
Osteoarthritis
COPD
Alcohol use disorders
Unipolar depression
Maternal hemorrhage
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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
The proportion of MRSA among positive The proportion of MRSA among positive blood cultures of Staphylococcus Aureus in blood cultures of Staphylococcus Aureus in England &Wales1989-2002
0
5
10
15
20
1983-1987 1988-1992 1993-1997 1998-2002
No. o
f FDA
app
rove
d an
tibac
teria
l NM
Es
Antibacterial new molecular entities approved for use in the United States 1983-2002
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Global Public Health Threats (2)
Pandemic Influenza:
• Overdue for a new pandemic
• Uptake of existing vaccines is poor
• Current capacity to produce either vaccines or antiviral medicines is not sufficient
Rates of vaccine distribution per 1000 total population by country
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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 beta-blocker or thiazide diuretic) deserves further urgent study.
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High burden, preventable diseases
with pharmaceutical gapsSmoking-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 gapsHIV/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 : • 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: • Gaps in understanding biology of depression and its
treatments in these groups
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"Neglected" diseasesLack of EU support for translational research for market failure diseases
Malaria: • Lack of experimental models for medicines discovery and
development.
Tuberculosis: • More FDCs for second-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 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. Reseach prospective price setting.
• 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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Differential Pricing: Indicative prices in US$/annum of highly active antiretrovirals (HAART) and a new hypothetical regimen in countries of variable wealth
0
5000
10000
15000
20000
25000
30000
26
7
29
4
44
1
48
7
60
7
76
3
85
5
99
3
12
14
16
37
19
59
21
25
24
01
25
57
25
66
34
77
37
44
49
21
61
99
10
88
1
12
62
0
15
62
7
19
83
1
22
78
3
23
22
5
26
07
6
Per capita GNI $US
Ind
icati
ve p
rice $
US
Indicative price existing treatment Indicative price new drug
200 500 1000 2000 2500 5000 15000 20000 25000
United Kingdom
France
Italy
Spain
Latvia
Slovenia
Czech Republic
Russian Fed.Kazakhstan
PhilippinesGeorgiaIndiaMali
200 500 1000 2000 2500 5000 15000 20000 25000
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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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ConclusionsPriority Medicines for Europe and the World
• 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 cessation 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:
+41-22-791-4533
http://mednet3.who.int/prioritymeds/report/index.htm
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Identifying gaps (unmet therapeutic needs):a public health perspective
3
Few treatments available, better
formulations and delivery mechanisms
needed
2
Treatable with existing interventions
but obstacles to access exist
1
Treatable with current mix of interventions
Untreatable with existing interventions including incurable chronic
conditions
4
0%
0%
100%
100%Maximum achievable
coverage
Population coverage with current mix of interventions
Combined efficacyof a mix
of allavailable
interventions
31
International organizations and less formal groups have developed methods for prioritizing health research
• The Commission on Health Research for Development (1990)
• The World Development Report (1993 )
• The Ad Hoc Committee on Health Research (1996)
• The Global Forum for Health Research (2000)
• WHO-IFPMA Round Table (2000-2001)
• The UNDP/World Bank/WHO Special Programme for Research and Training in Tropical Diseases (TDR)
• The US National Institutes of Health (NIH) (1998)
