Children often have the same diseases as adults, however many of the approved drugs on the market are not tasted in children. Many rare and serious diseases affecting children have no treatment options and as a result clinicians are forced to use “off label” drugs (drugs not approved to be used in children population).
Undoubtedly clinical research involving kids have plenty of challenges. There are many initiatives from regulatory agencies, which are trying to encourage pharmaceutical companies to include children in clinical trials or to obtain information for the application of marketed drugs in children.
Why is important to have drugs tested for children?
- Paediatricians could be denied access to potential beneficial treatment for children just because there is no data from that population.
- Children are treated with medications based on adults’ data or empirical experience in children. This could increase the safety risk for the young patients.
Controlled clinical trials are the best way to provide children with access to new treatments and at the same time obtain relevant safety data.
What are the most common challenges in clinical research, which lead to exclusion of children?
- The research topic is not relevant for children;
- Laws or regulations that do not allow children to participate;
- There is already some knowledge on the topic;
- The condition is rare in children;
- Limited number of children which does not allow enough data to be generated;
- Not enough data about adults to judge the potential risks for children;
- Children are not homogenous group and absorption, distribution, metabolism and excretion of the drugs depend of age and their current organs development;
Since 2000 in USA and Europe regulatory agencies require pharmaceutical companies to include paediatric data in all new drug application and licence extensions when there is an expectation that the drugs will be used in children.
Another initiative in USA and Europe is the “orphan” drug status which encourages development of drugs for rare diseases; however it is not expected to benefit paediatrics research significantly.
FDA Modernization Act which has similar regulations in Europe gives 6 month extension to drug licences or patents for drugs, which have paediatric data.
There are a number of ethical considerations which have to be observed when conducting research with children:
- Children can be included in clinical trials after it was established that the drug could be beneficial.
- Protocols involving paediatric population should be reviewed by Ethics Committee which includes members knowledgeable in ethical, clinical and psychosocial issues.
- Informed consent is obtained from parent / guardian unless children are in intellectual maturity which allows them to make decision for themselves.
- If the information can be obtained in less vulnerable population, it should be preferred to vulnerable population – for example, if the studies in adolescents that can be consented could be used for younger children, the study should include adolescents and not younger children who cannot consent.
- Studies in handicapped or institutionalised paediatric populations should be limited to diseases found in this specific population or when it is expected the disease and the treatment to be affected in such population – for example, studies cannot include children with disabilities if they can use children without disabilities and will provide adequate data.
- Paediatrics studies have to be conducted by experienced and trained clinician.
- The design of clinical trials should try to minimise the amount of children involved and required procedures without affecting data integrity.
- All measure to be taken to minimise the discomfort and distress that could be caused to the children.
While paediatric clinical trials have their challenges obtaining safety data for children is vital for providing adequate treatment.
Published on 1 June 2018
Author: Olga Peycheva, Director at Solutions OP Ltd. Olga has been working in clinical research since 2005 and has extensive experience in Eastern and Western Europe