Seminar PC4: Quality and risk assessment of medicines for children

Linked to EAHP Statements

Section 2 – Selection, Procurement and Distribution: Statements 2.1, 2.2
Section 3 – Production and Compounding: Statements 3.2, 3.3, 3.4, 3.5
Section 5 – Patient Safety and Quality Assurance: Statements 5.1, 5.8

ACPE UAN: 0475-0000-18-018-L04-P. A knowledge based activity.

Abstract

Common incentives allocated to foster research and development mainly favour medicines for adults, as they are more promising for blockbuster drugs and high returns on investments for example in the case of medicines for non-communicable diseases. Many other special patient groups such as those suffering from orphan diseases, the elderly, neonates and children are neglected. It is still not easy to recruit children to participate in clinical trials. Specialties’ profiles of undesirable events become evident mainly after market access and launch. Pharmacological profiles of active pharmaceutical ingredients and of excipients must be assessed on results from basic research. Should developments of dedicated galenic forms for paediatric use be supported, a special focus must be set on excipients, allergies, and intolerances. Alternative research methods are required, e.g. tissue cultures or genetic profiling to anticipate potential drug related problems in children. Governmental programs are needed to improve the perspectives of these special patient groups such as neonates and children to see dedicated specialities for paediatric use which satisfy requests for few undesirable effects, low immunogenicity, or adapted dosing.

Learning objectives

After the session, the participant should be able to:

• Identify special requirement and thresholds for the development of medicines for paediatric use;
• Outline the challenges as related to APIs and excipients for paediatric use;
• Make out opportunities and threats of this class of therapeutic products;
• Assess the suitability of a medicine indicated for children and anticipate risks arising from the paediatric use of these medicines; and,
• Select medicines for a children’s hospital formulary.

Educational need addressed

It is well known that pediatric needs is difficult to satisfy as clinical research with children is an important ethical issue. There is a need to anticipate all harm potentially arising from medicines for children. For this, the hospital pharmacist has to know specific allergens and toxicological threats mediated by active and “non-active” ingredients to properly defend the integrity of children under pharmacotherapy and prevent the use of medicines likely to promote undesirable effects in children as a result of differences in the metabolic capacity as compared to adults.

Keywords: Medicines for pediatric use, excipients, allergies, intolerances, selection