Unlicensed Medicines in Paediatrics
Is there a problem ?
Scottish Neonatal and
Paediatric Pharmacist Group
National Paediatric PreRegistration Pharmacist
Study Day January 2011
AIM
To discuss the issues relating to the
use of unlicensed medicines in
paediatric patients and facilitate
discussion of the implications
in practice
Objectives
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describe the licensing
process
identify problems of
clinical trials in children
examine the extent of
the problem
discuss seamless care
issues
pose questions for
discussion!!!
Kids’ medicine
‘danger’ shock
SOME children’s medicines have not been approved for
consumption by youngsters according to a report
published yesterday.
It warns that most medicines either prescribed or sold direct
are safe but a minority could do harm in the wrong doses.
They are licensed , but only for adults.
Extent of unlicensed and off-label prescribing in
children
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90%
70%
care
67%
11%
GP
of babies in neonatal intensive care
of children in paediatric intensive
of children in hospital across Europe
of children treated at home by their
Must use a licensed product when it is
suitable
Unlicensed and Off-label Medicine Definitions
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Unlicensed
• Extemporaneous
dispensing
• Pharmaceutical
‘specials’
• Importing medicines
licensed in other
countries
• ‘Named patient’
supplies
• Chemicals not licensed
at all for human use
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Off label
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Dose
Age of patient
Indication
Route of administration
Contra-indications
Risk Assessment of Options
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Licensed products
Route of administration
Confidence in manufacturer
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Import licensed medicine from QC approved country
Pharmaceutical Special (Hospital / Commercial)
Extemporaneous
Borderline substance
Labelling language/ PIL language
Finished product testing
• Certificate of Analysis
• Certificate of Conformity
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QC Approved importers/ countries
• (e.g. within EU, N America, Australia, Japan, Switzerland,
New Zealand)
The licensing process
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To market a drug in the UK a
pharmaceutical company must
obtain a licence from the
licensing authority (health
ministers guided by the MHRA
or EMA)
The aim of a licence is to
ensure that all medicines in
the UK meet acceptable
standards of safety, quality
and efficacy
Extent of a product licence
It allows a company to
market a drug for;
 specified conditions
 in an agreed dose
range
 by a particular route
 using a tested
formulation
Based on clinical trial
data
EMA / MHRA Product Licence
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Paediatric Investigation Plan (PIP)
• Paediatric Regulation to submit a
development plan for new medicines
• Specific class waivers
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Paediatric-Use Marketing
Authorisation (PUMA)
• Already authorise medicine
• 10 years market protection
Paediatric clinical trials
“By an odd and unfortunate twist of fate, infants and children are
becoming therapeutic orphans”
Shirkey H 1968
Lack of Clinical trials
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80% of new drugs had no
info in paeds
WHY ?
ethics
long term effects
micro-analytical techniques
return for investment
Directive
89/341/EEC
all doctors can prescribe:
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there is no statutory requirement to
disclose to a patient when a drug is
unlicensed
all unlicensed prescribing /
dispensing should be done
knowingly(?)
the decision to prescribe unlicensed
medicines
• should be in the best interest of the
patient
• in accordance with a respectable
body of professional opinion
Practical Implications
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Responsibility
Liability
Patient Information
Inappropriate
prescribing?
Medication Errors
• Differing strengths
• Differing
manufacturer’s
Practical Issues
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Barrier to seamless
care
Effective
communication
systems
Unlicensed
manufacturer’s /
suppliers cannot
advertise products
Dispensing Problems
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Lack of familiarity
Calculations
Lack of suitable
products- risk
assessment of options
Only able to order
from specific specials
suppliers
Medication Errors
Professional check
Cost
How Extensive is the problem?
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Over to you some audience
participation please
Consider the licensing status of the following medicines for
children and neonates
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Buccal midazolam for seizures
Paraldehyde for seizures (rectal)
Spironolactone for liver ascites
Paracetamol for pyrexia
Phenobarbitone for epilepsy
EMLA cream for topical anaesthesia
Melatonin for sleep disorders in children
Ibuprofen for pyrexia