ESSENTIAL MEDICINES FOR CHILDREN
Dr Suzanne Hill
Presented by Adriana Velazquez
HTAI
Rio Janeiro
June 2011
Causes of death in under 5s
injuries
10 3 3 AIDS
3
8 measles
malaria
others
26 11
diarrhoeal diseases
pneumonia
other neonatal causes
17 neonatal severe
infections
19
World Health Statistics 2007
http://www.who.int/child_adolescent_health/data/child/en/index.html
Bryce et al, Lancet 2005; 365: 1147–52
What is the problem?
Children are therapeutic orphans
Lack of appropriate clinical trials
Lack of licensed medicines
Lack of formulations
Lack of information
Initiatives so far concentrated on:
Developed country regulatory structures (eg FDA, EMEA)
Developed country drug information (eg BNF- C)
HIV (eg new public private partnership)
Quantitative study in 14 African countries
Sampling
Adapted methods of HAI/WHO pricing surveys
Limited to capital cities only
12 facilities surveyed: CMS (1) NGO facility (1) Teaching
hospital (1) District hospital (1) Primary health care clinics (3)
Community pharmacies (5)
Questions asked
Are listed medicines included in national EML and STGs
Are listed medicines available for supply in the facility on the
day of the survey and what is the cost to the patient of the
cheapest product available (branded or generic product)
Proportion of medicines available in Public and Private
sector outlets
100
90
% of medicines available
80
70
60
50
40
30
20
10
0
IA
AD
C
RW IA
A
O
DA
A
UN
DA
AL
YA
AL
PI
DR
BI
NG
AN
AN
ER
H
EG
AN
O
AN
M
N
O
M
TC
H
ER
KE
NZ
CO
G
HI
ZA
N
UG
G
NI
ET
SE
TA
M
CA
Teaching hosp District hosp PHC CP
Obvious gaps
Essential medicines list – paediatrics
Formulations eg fixed dose combinations for malaria, TB
Prescribing information
Market incentives for appropriate drug development
International regulatory standards, including quality
International safety monitoring and post-marketing
surveillance
Plan
Work on a global project to make paediatric medicines a
priority:
Develop Model List of Essential Medicines for children and
update 2007-2008
Update treatment guidelines
Develop paediatric prescribing information – a formulary
Collaborate with regulatory authorities to encourage
appropriate drug development and approval processes in all
regulatory authorities
Identify ideal paediatric dosage forms
Develop quality standards for paediatric medicines
Develop a system for enhancing safety monitoring of medicines
in children
Provide guidance on procurement and supply of paediatric
medicines
Paediatric formulation issues
Technical difficulties of manufacturing
Storage and preparation
Impact of various climates
Taste of the medication
Local factors and practice
Specific issues
Liquids
Short shelf lives
Often require refrigeration
Bulky and heavy
(issue for storage and transport)
Powders for suspension
Mixed correctly with sterile fluids
Affected by humidity
Chewable tablet
Tolerated by children two years and older
Limited dose variation
Key issues
Second list of essential medicines for children
Review and confirmation of medicines on the List
Additions proposed (Section 5 of report)
Deletions (Section 5)
New sections:
• Palliative care
• Ear Nose and Throat
• Medicines for use in neonates
Progress
Essential medicines list – paediatrics
Formulations eg fixed dose combinations for malaria, TB
Prescribing information
Market incentives for appropriate drug development
International regulatory standards, including quality
International safety monitoring and post-marketing surveillance
Contribution from industry
Not just liquids..
Experience with innovative dosage forms
Capacity in development
Linkages with academic groups
Finding existing data
Kinetic studies
Adverse event data
Younger age groups
Quality clinical trials
GCP and ethics committee capacity
Registration and reporting
Product development and registration
