More than half of all medicines intended for children already on the market today are actually not licensed or suitable for paediatric use, according to the FDA. This means a big proportion of prescribing for children has not been tested and thus children are at risk of receiving the wrong dose. The main reason for this is because patient recruitment is still the biggest challenge in paediatric trials for both European and American pharmaceutical companies. This difficulty causes delays, high costs, poor data or failure to complete drug trials.
According to the experts, paediatric patient recruitment remains the main challenge -- in addition to ethical, regulatory, economic and practical difficulties -- because sponsors tend to simply transfer clinical trial designs from adult to children studies. However, children are a unique population that have many psychological differences from grownups and that need to be approached in a different way according to age groups and culture. Due to all of this, paediatric studies are actually harder to conduct and take longer than adult ones.
The recruitment process faces challenges at different stages, for example at identifying eligible paediatric patients, explaining the study to them and their parent, obtaining consent, and retaining them. It goes without saying that minimizing the risk/benefit ratio and maintaining ethical standards to get good data are of great importance and a challenge in itself. However, having in mind a few practices can improve young patient recruitment and lead to better scientific results.
Skilled, open communication with the child and the parent, as well as trust and transparency are crucial in order to overcome this problem. As such, investigators need to make sure parents receive the necessary information about clinical research in general and about the specific study, as well as the benefits and challenges involved. Studies have shown that paediatric patients prefer to have access to simple and easy to use trial documents, and that they are motivated to enter a trial to learn more about their condition, help themselves and others in the same situation. Parents focus on the benefit of the child and feel reassured that their child's health to be closely monitored.
A good strategy could be to have a doctor spokesperson to increase credibility and receptivity, and to communicate the information in the kid's age-appropriate language to make him or her comfortable. Children respond better when both their parents and doctors speak to them together. It is also important to prepare answers to questions children might have and to provide parents with tips on how to talk to their children about clinical trials.
Doctors need to evaluate preferences, cultures and customs of parents and paediatric patients they intend to enrol to make sure they motivate them to participate. Research has shown that it is better for investigators to focus more on the benefits the trial will have on the child's health rather than on the altruistic benefits.
It might also be a good idea to use digital communication, such as e-mails and social media, to stay in touch with younger parents and inform them about a particular study. Face-to-face communication could also be effective with other families. Child-friendly communications and child-friendly site surroundings across the whole process can accelerate engagement.
Also, it is important to bear in mind that study visits need to be flexible and scheduled creatively as most parents work and children go to school. Time and travel commitments should therefore be taken into account to recruit and retain subjects. For example, it might be a good idea to start enrolment during the summer months to assure children will have more time to attend trials. Early morning and evening meetings could work for parents who work to attend meetings and drive their kids to sites.
Trials in young people are problematic, but it is possible to conduct successful ones if these considerations are taken into account. Remembering that children are not small adults and that they need to be addressed in a particular way is key to speed up enrolment.