In nearly half of the visits made by children to doctors, 70% of medicines prescribed to them have never been tested in pediatric population. There remains no choice but to treat them that way, as there are very few clinical trials that are conducted in children. It is inappropriate to think of children as small adults as they are developing humans with specific age related developmental and physiological differences. The extrapolation of adult data on medicinal products for the child population is inappropriate, which makes age- and development-related research particularly important. The need to develop medicines safe and effective for children (whether a novel agent or an existing agent in need of pharmacokinetic studies) necessitates new drug testing in children.
Presently, the drug dosage for children is calculated by physicians based on their age and weight. If only an adult preparation is available for the particular disease condition it really becomes difficult for the pediatrician to calculate (and assume) the dose that will be effective and completely safe for them. Though it may sound like guess work but due to lack of research in children this is all we have. It is now necessary to understand a how a child’s brain works and develops, as well as the way treatments and medicines are handled over time in a child’s body.
According various surveys, only 100 years ago nearly one in six children died due availability of no proper treatment in the United States of America. Today the infant mortality rates have reduced notably and this change has been attributed to the recent research and clinical trials that have been conducted in children. Clinical research also helps to find treatments for problems that occur only in children and the development of the much needed vaccines.
Although clinical trials are necessary they do carry risks. Clinical trials are carried out in an environment that offers the best possible protection to the study subjects. A number of guidelines for the protection of children in clinical trials have been laid down by various regulatory authorities. “A fundamental concept of research is that it cannot take place without informed consent,” says Murphy. Children however cannot give an informed consent (because consent implies to full understanding of the risks and possible benefits), and therefore parents, as the child’s advocates, must decide if they wish to provide permission in place of informed consent. The child may be asked to assent or dissent depending on the child’s age, psychological state, and other considerations.
Before the start of any clinical trial, the Institutional Review Boards (IRB’s) review the research plans, consent forms and other documentation to ensure that the safety of the study participants is maintained throughout the study. Data and safety monitoring boards are set up that regularly monitor for any safety issues. Medical monitors who are qualified medical professionals are actively involved in clinical trials and review any side effects that occur in a clinical trial.
For parents it is necessary to understand the possible risk and benefits. As a parent they should get familiar with the study team and also find out the resources that are available to help them understand the rights. It is suggested to use the child’s pediatrician as an advisor before enrolling the child in a clinical trial. Parents have the rights to withdraw their child from the study at any point of time if the new treatment is not proving to be helpful or if there are intolerable side effects.
To do research in children is a necessity for the society. It is believed that children who start off less healthy also tend to be less healthy adults. To protect children, improve outcomes, enhancing quality of life and to give them a healthier adulthood, conducting clinical trials in children is important.