This International Clinical Trials Day, a day celebrated around the world to mark when James Lind, a Scottish doctor, started the first randomised clinical trial aboard a ship on May 20, 1747, we wanted to highlight a topic close to heart – paediatric clinical trials.
Clinical trials are an important step in discovering new medicines, however, only a minority of drugs and vaccines we use to treat children are actually tested on them. At Aurora, we recognise the importance of Dr Seuss’s words; children are not ‘little adults’. We understand the challenges faced by families and our clients when it comes to developing treatments and clinical trials for paediatrics. With this in mind, we developed Kids in Trials© or as we’d like to call it KiT©, a protocol where children and their close connections are at the heart of paediatric clinical trials.
Historically, once a treatment becomes available on the market for adults, it is possible to use it in children off-label. Prescriptions are often based on extrapolation from trials conducted in adults due to the lack of pediatric trial data. Children are given only approximate doses of potentially effective medications. The World Health Organisation (WHO) reports that off-label use ranges between 50 and 90 percent in the paediatric population in most countries.
The complex physiological, developmental, psychological and pharmacological characteristics of children vary from adults, and moreover, paediatrics is not just a single population, but multiple based on age. Ignorance of these differences has led to medicine-related tragedies in the past. The need to conduct studies to find out how to appropriately use medicines in children of different ages is apparent. However, companies are reticent to conduct trials in children due to cost and the greater risk of liability.
In the last 10 years, a lot has happened
US and European regulators mandated pharmaceutical industries to evaluate the safety and efficacy of medicines used by children in all appropriate paediatric age groups. This ensures that product labels contain the known paediatric data, to develop paediatric appropriate formulations and provide a Paediatric Investigation Plan to test children at the time of medicine application submission. In addition to this, both regulations also provided a six-month extension of patent protection to encourage companies to conduct paediatric trials.
These regulations have done wonders. The proportion of clinical trials conducted in Europe increased by 50 percent between 2007-2016, from 8.25 to 12.4 percent. The ten year report of the European Medical Association (EMA) showed more medicines approved for children, better and more information for prescribers and patients, more regulatory support for paediatric matters and more importantly better paediatric research and development.
However, even with all the regulations and incentives in place, pediatric drug development still poses particular challenges and requires a distinct approach.
Through our unique and exclusive collaboration with the Patient Intelligence Panel (PIPHealth), we involve patients and their families as partners from the start, to build and co-create clinical trials to address the challenges. Here are three insights:
- Trial design: You cannot use or adapt the clinical trial design and study assessments from adult studies to paediatric ones. It is not that simple; it needs to be centred around the child’s needs and perspective. Tools needed for quantitative and qualitative assessment also need to be tailored and validated for the paediatric population.
- Enrolment and retention: More than often, communication provided by pharmaceutical companies at the stage of enrolment are age-inappropriate for both study investigators and potential participants. The ‘family’ needs to be considered as the participant; not just the child; the information needs to be tailored for each audience and updated throughout the study to keep participants informed, motivated and engaged.
- Informed consent: The information provided must be tailored to the research, taking into consideration the cognitive and emotional maturity of the child, the family, the participant’s priorities and well-being, and focussed on the issues that are of concern to potential participants and to help them achieve understanding of a trial.
Co-created tools from KiT© will be relevant, concise and accessible and of an age-appropriate format to provide clarity for children and to aid their ability to make informed decisions about participation in a specific clinical trial. So this Clinical Trials Day, we’re with Dr Seuss. ‘A person is a person, no matter how small’ and we will do our best to make a difference in each of their lives.
If you’d like to hear more about KiT© and find out about our approach to clinical trials, give Sue Neilen a call (0207 148 3055) to see how we can help.