ICH E11 - Clinical Investigation of Medicinal Products in the Paediatric Population

Question 1

What is the most important factor in the early initiation of pediatric studies?

Answer 1

The presence of a serious or life-threatening disease for which the medicinal product represents a potentially important advance in therapy.

Question 2

The Paediatric development program should not ______ adult studies and availability of medicinal products for adult

Answer 2

delay

Question 3

It should not be noted that the most relevant safety data for paediatrics studies ordinarily come from______exposure

Answer 3

adult

Question 4

There is a need for paediatric formulations that permit accurate dosing and enhance patient compliance such as ________

Answer 4

• flavors
• colours
• liquids
• suspensions
• chewable tablets, etc.

Question 5

Since development of paediatric formulations can be difficult and time consuming, it is important to consider the development of these formulations _______.

Answer 5

early in product development

Question 6

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)

Answer 6

safety and tolerability

Question 7

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.

Answer 7

serious or life threatening disease

Question 8

A pharmacokinetic/pharmacodynamic approach combined with safety and other relevant studies could avoid the need for _____..

Answer 8

clinical efficacy studies

Question 9

When would clinical efficacy studies be needed in a paediatric population?

Answer 9

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.

Question 10

Pharmacokinetic studies should be done to support what type of development?

Answer 10

formulation

Question 11

Why do we need to determine different pharmacokinetic parameters in different age groups?

Answer 11

to determine dosing recommendations

Question 12

Measurement of _______such as pain requires different assessment instruments for patients of different ages.

Answer 12

subjective symptoms

Question 13

Normally the paediatric database is limited at time of approval. Therefore, ___________ is particularly important

Answer 13

post marketing surveillance

Question 14

Any classification of the paediatric population into age categories is somewhat _______

Answer 14

arbitrary

Question 15

What are the possible/ examples of age categorizations given in E11?

Answer 15

• 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

Question 16

Study design issues to be considered with neonates include

Answer 16

1 - weight and age
2 - small blood volumes
3 - small number of patients given at the centre
4- difficulties assessing outcomes

Question 17

By 1 or 2 years of age, _______of many drugs on a mg/kg basis may exceed adult values.

Answer 17

clearance

Question 18

Factors useful in measuring the effects of medicinal product on children include?

Answer 18

• weight gain
• school attendance/ performance
• skeletal growth

Question 19

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?

Answer 19

The REB or to be consistent with local legal requirements.

Question 20

Participants of appropriate intellectual maturity should personally sign and date either a separately designed, ______.

Answer 20

written assent form or the written informed consent.

Question 21

Emancipated or mature minors (defined by local laws) may be capable of_____.

Answer 21

Giving autonomous consent

Question 22

Mechanisms should be in place to ensure that a study can be rapidly terminated should _______ be noted.

Answer 22

an unexpected hazard

Question 23

A fundamental principal in paediatric drug development requires that children should not be enrolled in a clinical study unless necessary to achieve ______.

Answer 23

an important pediatric public health need

Question 24

When obtaining child assent, relevant elements of informed consent should be provided that are _______.

Answer 24

Appropriate to the child’s capability to understand.

Question 25

It may be necessary to reasss the assent of a child in recognition of their __________and competency

Answer 25

advancing age and maturity

Question 26

During clinical studies there is a requirement for obtaining adequate informed consent for continued participation from paediatric participants once a child reaches _______.

Answer 26

the age of legal consent

Question 27

_____regulations related to confidentiality and privacy of paediatric participants must be followed.

Answer 27

local

Question 28

A _______, not common regional requirements, is at the cornerstone of efficient paediatric drug development and timely delivery of safe and effective medicines for children.

Answer 28

common scientific approach

Question 29

The neonatal period and preterm newborn infants is defined as __________.

Answer 29

the day of birth through the expected date of delivery plus 27 days.

Question 30

Sometimes, there are difficulties with generating data across paediatric population due to..

Answer 30

a variety of ethical considerations and feasibility issues.

Question 31

What is paediatric extrapolation

Answer 31

An approach to providing evidence in support of a drug/ treatment in paediatric population when it can be assumed that the course of the disease and expected response to a drug would be sufficiently similar in the paediatric and reference population.

Question 32

When a drug is studied in a paediatric population, one should consider all factors which may result in different drug responses, such as ________factors that could impact on the extrapolation of data from one population to the other.

Answer 32

intrinsic and extrinsic

Question 33

What are intrinsic factors?

Answer 33

factors that act within the individual e.g. development, attention

Question 34

What are extrinsic factors?

Answer 34

factors that influence from outside eg. geographical

Question 35

____ can help quantify available information and assist in defining the design of pediatric clinical studies and/or the dosing strategy.

Answer 35

Modelling and simulation

Question 36

Well conducted M&S can inform on the pharmacokinetics, pharmacodynamics, ______ and safety of a drug.

Answer 36

efficacy

Question 37

Three key practical factors to consider in pediatric clinical trials are ____________.

Answer 37

(1) feasibility, (2) outcome assessments, and (3) long-term clinical aspects, including safety

Question 38

Pediatric drug development faces unique feasibility issues, including __________. (3 things)

Answer 38

(1) small number of eligible children for clinical research, (2) limited pediatric specific resources at research centers, and(3) the scarcity of dedicated pediatric trial networks

Question 39

Strategies that foster _________ can facilitate participation, recruitment, and acceptability of a clinical study.

Answer 39

input from children, their caregivers, and the advocacy communities

Question 40

A range of quantitative approaches to characterize the interactions between a drug and an organ system which could predict quantitative outcomes of the drug and/or system’s behavior in future experiments.

Answer 40

Modelling and simulation