Medicines for use in special populations

When medicines are under development, it is a general rule that the patients entering clinical trials should be reasonably representative of the population that will be later treated by the medicine.

The use of medicines in some populations (e.g. paediatric or the elderly) requires special consideration and should be discussed in the clinical summary of the dossier (Module 2.7 of the CTD).

Medicines for paediatric use

Sponsors should consider whether their medicines are likely to be used in children, and sponsors with paediatric data and appropriate formulations are encouraged to apply for registration of these medicines.

For major applications (see CTD Module 1.12):

• complete the form Module 1.12 Paediatric development program
• include the form in the dossier.

If your registered medicine is likely to be used in children, discuss with the TGA how to:

• make paediatric formulations available
• update the PI document with appropriate information on paediatric use.

The following mechanisms are available to encourage the submission of paediatric data packages:

• the Orphan Drug Program, where registration fees may be waived in conditions with a prevalence of intended users of less than 2,000 per year
• literature based submissions to facilitate submission of modified packages based on existing published data.

Guidelines for generating paediatric data

• Note for guidance on clinical investigation of medicinal products in the paediatric population (CPMP/ICH/2711/95).
• Guideline on the role of pharmacokinetics in the development of medicinal products in the paediatric population (EMEA/SHMP/EWP/147013/2004)
• Guideline on the investigation of medicinal products in the term and preterm neonate (EMEA/536810/2008)
• Guideline on the need for non-clinical testing in juvenile animals of pharmaceuticals for paediatric indications (CHMP/SWP/169215/2005)
• Reflection paper: Formulations of choice for the paediatric population (EMEA/CHMP/PEG/194810/2005).

Medicines for geriatric use

The use of medicines in the geriatric population requires special consideration due to:

• the frequent occurrence of underlying diseases
• concomitant medicinal product therapy
• the consequent risk of medicinal product interaction.

Guidelines for generating geriatric data

The ICH/European Guideline Clinical Investigation of Medicinal Products in Geriatrics is principally directed toward new active substances that are likely to have significant use in the elderly, either because:

• the disease intended to be treated is characteristically a disease of ageing (e.g., Alzheimer's disease), or
• the population to be treated is known to include substantial numbers of geriatric patients (e.g., hypertension).

The guideline also applies to:

• new formulations, and new combinations of established medicines, when there is specific reason to expect that conditions common in the elderly (e.g., renal or hepatic impairment, impaired cardiac function, concomitant illnesses or concomitant medications) are likely, but not addressed in current labelling
• new formulations or new combinations that are likely to alter the geriatric patient's response (with regard to either safety/ tolerability or efficacy) compared with that of the non-geriatric patient in a way different from previous formulations
• new uses that have significant potential applicability to the elderly.

Other relevant guidance

Extrapolation of data from one ethnic population to another

The Note for guidance on ethnic factors in the acceptability of foreign clinical data recommends a framework for evaluating the impact of ethnic factors upon a medicine's effect (i.e. the medicine's efficacy and safety at a particular dosage and dose regimen).