Globally, 17-23% of children are chronically ill and need continuous medication. Depending on the age group and disease area, up to 95% of medication is not approved for use in children (off-label). In disease areas such as cancer, epilepsy, arthritis, rheumatology, diabetes type 1 or rare diseases, there is hardly any clinical data for children – not to mention Real-world-Data (RWD). Hence, paediatric treatment is based on experience and conservative approximation – adjusting the dosage of medicines approved for adults – and the knowledge gained from experience remains local, as every clinic develops its own standard operating procedures (SOPs) and sharing only happens on premise.
The lack of data in paediatric care is a legacy problem: pharmaceutical companies do not generally conduct clinical trials in paediatric population, regulatory bodies allow the use of off-label medication, and clinicians are forced – for the sake of their patients – to use these medication. We believe that global granular real-world data RWD and patient reported outcome measures (PROMs) in paediatric treatment with off-label medication would help to identify appropriate dosage for each child, track the efficacy and side effects of treatments, and study drug-to-drug interactions, providing critical evidence for the safe use of off-label medication. Whereas the use of PROMs and RWD is becoming more and more established in some areas of treatment in adult medicine (please see, among others, the Emilyn app for MS, Cara.Care app for gastroenterology or the Woom app for fertility), its use in paediatric care has not yet been considered, even though the lack of clinical data in paediatrics makes PROMs and RWD even more valuable.
International regulatory bodies (FDA and EMA) support the use of RWD for clinical research, drug development and also post-market surveillance. Therefore, we believe that our project fits to the global movement towards adding RWD to clinical data and closes a much-needed gap in paediatric care.
Current solutions for collecting PROMs do not solve the problem in paediatrics:
1. Most initiatives focus on diseases that affect a broad range of the population. Luckily, paediatric patients represent a small fraction of chronically ill patients, but it means that there are very few projects focusing on chronically ill children. Nonetheless, there are many disease areas where children need to take medication for their entire lifetime and where a granular feedback-loop and data aggregation can help them to get a more adequate treatment. Currently, parents of chronically ill children keep diaries mostly on paper and provide (partial) treatment feedback during their doctor appointments – and even the most detailed feedback is not shared or analysed.
2. There are solutions targeted at parents to guide them through their (healthy) child’s development. However, these solutions are not suitable for the vulnerable population of parents of chronically ill children. A right balance between a “medical” and “engaging” solution is needed.