Patient involvement in clinical trials with children
50-90% of prescribed treatments have not been tested and approved for children. Therefore the new EU paediatric medicines regulation came into force in 2007 (http://eur-lex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:L:2006:378:0001:0019:en:PDF).
This states that medicine must be tested in the paediatric population, in order to get marketing authorization. One good initiative that aims to expand and promote research on safe and effective medicine for children is the network TEDDY; Task-force in Europe for Drug Development for the Young (www.teddyoung.org). When looking at the topic of clinical research for children it is evident that there are a lot of perceived barriers to this form of clinical research. Some of the difficulties have been described in the literature (78-80):
- Health care decisions made for children have relied on research that has been done in adults. This has resulted in a lack of confidence of the public. This lack of confidence will be a hurdle for investigating medicines in children.
- Children are vulnerable group: cognitively and emotionally immature. They depend on parents to make decisions on their behalf.
- (81) The potential market for children specific medicines is small as most conditions are either not prevalent in children or only prevalent in children in the case of a lot of rare diseases.
- There is a limited number of children eligible for participation in clinical research, and their participation depends primarily on their parents.
As the regulation for clinical research with children has not been enforced for that long, not many studies have been published on the involvement of children as a research subject in clinical trials.
When looking at why parents and their children do take part as a research subject in a clinical trial the foremost reason in children is and should be to help future generations (82).
Furthermore the literature talks of a number of good studies, primarily in cancer (83) in which clinical research studies in children have been successful. The success, the author and others conclude that it is primarily attributed to the trust (78, 82) that had been build between the researchers and the child’s families. Furthermore he suggests that all clinical trials with children be very carefully designed with special attention to appropriate informed consent and low levels of discomfort for the children involved.
Several recommendations have been made in the literature to better paediatric research by involving and informing patients, patient organisations and the general public:
- Better education of the medical community and the public is needed concerning the rationale and risks/benefits of paediatric research (80).
- Negatively biased media coverage about clinical trials involving children needs to be balanced with positive public-awareness campaigns about the societal benefits of clinical trial research (80).
- Researchers must build better relationships with paediatricians and parents by communicating more clearly and openly (19, 78, 84).
- Research should be undertaken to identify the best way to involve parents and children in assessing which outcomes should be measured in clinical trials; Families are invaluable repositories of first hand information about the impact the disease has on the child’s life (85).
- A systematic effort should be made to raise awareness of parents and the public at large about the importance of paediatric research and on how clinical trials in children are carried out (83).
- Patient organisations can help develop training for healthcare professionals to support families in paediatric clinical research (19, 80).
Finally two reports (86) (87) discuss the value of young people’s input in research. The various stakeholders interviewed in these reports share the belief that involving young people as researchers would improve research outcomes and have greater impact on the quality of people's lives. However, they also expressed doubts as to whether such research would impact on decision-making when the general relationship between research and policy is not always a clear one. For this reason some people tended to focus on other ways for children's views to influence government policy e.g. through consultation.