European Patients Academy on Therapeutic Innovation Special Populations

European Patients’ Academy on Therapeutic Innovation Special Populations

What groups are defined as special populations? European Patients’ Academy on Therapeutic Innovation § Some groups in the general population may require special study within the clinical development process: Ø elderly Ø patients with impaired excretion Ø pregnant women Ø breast feeding women Ø children Ø ethnic sub-groups § These groups may have unique benefit-risk considerations or they may need to use a different dose or treatment schedule. 2

Elderly people European Patients’ Academy on Therapeutic Innovation § The use of medicines in this population requires special consideration due to: Ø the frequent occurrence of underlying diseases Ø use of other medicines at the same time and the consequent risk of medicines interaction 3

Elderly people European Patients’ Academy on Therapeutic Innovation § Not all potential differences that can occur in the elderly population can be predicted from non-elderly populations. This might include differences in: Ø pharmacokinetics - what the body does to the medicine Ø pharmacodynamics - what the medicine does to the body Ø interactions between the medicine and existing diseases Ø interactions between the tested medicine and other medicines the patient might be taking Ø clinical response - the effect of the medicine on the disease 4

Patients with impaired excretion European Patients’ Academy on Therapeutic Innovation § People who may have difficulty removing the medicine from their body due to kidney or liver problems. § Specific pharmacokinetic studies are needed to observe the effects of the medicine in these patients. § These studies should include elderly patients, or younger patients with impaired excretion. 5

Pregnant women European Patients’ Academy on Therapeutic Innovation § In general, pregnant women should be excluded from clinical trials where the medicine is not intended for use in pregnancy. § If a woman becomes pregnant whilst receiving a medicine, treatment should be stopped (if can be done safely). 6

Pregnant women European Patients’ Academy on Therapeutic Innovation § Special attention must be paid to reproductive toxicity studies before pregnant women are included in clinical trials. § For clinical trials that include pregnant women because the medicine is intended for use during pregnancy, it is very important to follow up the: Ø pregnancy, Ø foetus, and Ø child 7

Breastfeeding women European Patients’ Academy on Therapeutic Innovation § In some cases the medicine (or its metabolites) will be excreted into human milk and this should be examined. § When breastfeeding women are taking part in clinical trials, their children should be monitored for the effects of the medicine. 8

Children (paediatrics) European Patients’ Academy on Therapeutic Innovation § Children represent an especially vulnerable population, thus, clinical trials involving children should be carried out under conditions that give the best possible protection for each age category. § Age categories are defined as: Ø preterm new born infants (born before 37 weeks gestation) Ø term new born infants (0 to 27 days) Ø infants and toddlers (28 days to 23 months) Ø children (2 to 11 years) Ø adolescents (12 to 16/18 years, depending on region) 9

Children (paediatrics) European Patients’ Academy on Therapeutic Innovation § Safety data from adults should be available and assessed before children are included in clinical trials. § It is usually appropriate to begin with older children before extending the trial to younger children, and then to infants. § The organisation developing a medicine should submit the mandatory paediatric investigation plan (PIP) early enough to ensure that the medicine can be appropriately developed for children. 10

Ethnic groups/regions European Patients’ Academy on Therapeutic Innovation § Differences in ethnic factors could alter the efficacy or safety of the medicine in the population. § A limited amount of clinical data may need to be generated in ‘bridging studies’. Understanding a medicine’s characteristics will determine what sort of bridging studies are needed in ethnic sub-groups or different regions. § Pharmacokinetics, pharmacodynamics and therapeutic effects could be impacted by ethnic factors. § The type of medicine concerned, its indication, as well as the age and/or sex of a patient can influence how ethnic factors change a medicine’s effect. 11