Acceptability of Formulations: Testing Strategies
摘要
In recent years, acceptability of formulations by pediatric patient populations has come to be considered as a key factor for patient adherence. Subsequently, due to the related impact of acceptability on efficacy and safety, the latest guidelines and recommendations from regulators and health agencies (EMA, FDA, WHO) highlight the importance of this key factor which must be considered in the design of medicines appropriate for pediatric populations. Acceptability is defined by the EMA as the overall ability and willingness of the patient to use and their caregiver to administer the medicine as intended. This multifaceted concept is likely to be driven by both the different product features and the patient’s characteristics. From newborns to 17-year-olds, the pediatric patient population is particularly heterogeneous with regard to their perceptions, cognitive and physical capabilities. The assessment of patient acceptability, for these different sub-populations, requires a cross-functional effort to cover their different needs during the entire development process, from the early phases of formulation to the final marketing authorization. In this chapter, EMA recommendations are presented as a guide to address the various expectations. Ongoing efforts to investigate patient acceptability for oral dosage forms are summarized. Aspects such as shape and size, formulation type (e.g., tablets, granules, liquid preparations), palatability, and swallowability are covered. For various dosage forms, the method of administration, packaging configurations, and their overall usability will be discussed. Based on the literature, an overview of methods for assessing acceptability is provided and discussed. The chapter also identifies potential gaps in the field and elaborates on challenges faced by the industry to achieve adequate acceptability for a pediatric dosage form.