ICH E11 - Clinical Investigation of Medicinal Products in the Paediatric Population Flashcards
What is the most important factor in the early initiation of pediatric studies?
The presence of a serious or life-threatening disease for which the medicinal product represents a potentially important advance in therapy.
The Paediatric development program should not ______ adult studies and availability of medicinal products for adult
delay
It should not be noted that the most relevant safety data for paediatrics studies ordinarily come from______exposure
adult
There is a need for paediatric formulations that permit accurate dosing and enhance patient compliance such as ________
- flavors
- colours
- liquids
- suspensions
- chewable tablets, etc.
Since development of paediatric formulations can be difficult and time consuming, it is important to consider the development of these formulations _______.
early in product development
For products exclusively used in paediatric populations, the entire development will be conducted in the paediatric population except for initial _____data (usually obtained from adults)
safety and tolerability
The presence of a ________for which the product represents a potentially important advance in therapy suggests the need for relatively urgent and early initiation of paediatric studies.
serious or life threatening disease
A pharmacokinetic/pharmacodynamic approach combined with safety and other relevant studies could avoid the need for _____..
clinical efficacy studies
When would clinical efficacy studies be needed in a paediatric population?
when novel implications are being sought for medicinal product in paeds or when novel implications of therapy are likely to be different between adults and paeds.
Pharmacokinetic studies should be done to support what type of development?
formulation
Why do we need to determine different pharmacokinetic parameters in different age groups?
to determine dosing recommendations
Measurement of _______such as pain requires different assessment instruments for patients of different ages.
subjective symptoms
Normally the paediatric database is limited at time of approval. Therefore, ___________ is particularly important
post marketing surveillance
Any classification of the paediatric population into age categories is somewhat _______
arbitrary
What are the possible/ examples of age categorizations given in E11?
- Preterm newborns
- newborns 0-27 days
- infant and toddlers (28 days to 23 months)
- children (2-11 years old)
- adolescents (12-16/ depending puberty and region
Study design issues to be considered with neonates include
1 - weight and age
2 - small blood volumes
3 - small number of patients given at the centre
4- difficulties assessing outcomes
By 1 or 2 years of age, _______of many drugs on a mg/kg basis may exceed adult values.
clearance
Factors useful in measuring the effects of medicinal product on children include?
- weight gain
- school attendance/ performance
- skeletal growth
As a rule, a pediatric subject is legally unable to provide informed consent. Where appropriate, participants should assent to enroll in a study. Age of assent is determined by?
The REB or to be consistent with local legal requirements.
Participants of appropriate intellectual maturity should personally sign and date either a separately designed, ______.
written assent form or the written informed consent.
Emancipated or mature minors (defined by local laws) may be capable of_____.
Giving autonomous consent
Mechanisms should be in place to ensure that a study can be rapidly terminated should _______ be noted.
an unexpected hazard
A fundamental principal in paediatric drug development requires that children should not be enrolled in a clinical study unless necessary to achieve ______.
an important pediatric public health need
When obtaining child assent, relevant elements of informed consent should be provided that are _______.
Appropriate to the child’s capability to understand.
