Paediatrics: an unexplored and rewarding population – the right methods make it easier
Linda Abetz-Webb from Adelphi Values (formerly named Mapi Values) writes for Pharmaphorum about how many treatments with potential value in children have not been tested in paediatric populations. Incentives and penalties to encourage paediatric research are in place, but measuring disease symptoms and impact in children and infants can seem tough. Well designed and insightful patient-reported outcomes (PROs) or caregiver-reported outcomes provide a possible solution.
Linda explores the opportunity surrounding paediatric indications, and the importance of paediatric research despite its apparent complexity. She goes on to discuss the FDA Patient-Reported Outcome guidance on the methods for the development of paediatric PROs, how the methodologies can be tailored appropriately, and provides a selection of important questions to consider when choosing or designing a paediatric patient / parent reported outcome tool for a paediatric trial, with consideration to different age groups and cultures.