Pediatric patients are not tiny adults – but they aren’t Martians either.* They are children, and when involving them in a trial, we need to take into account all of the physiological, social, cultural, and developmental differences that make them unique.
When planning a clinical trial that involves children, keep the following in mind.
- Tests must be age appropriate. Researchers often try to retrofit adult trials for children by merely shifting the age range specified in the protocol. But they don’t always consider the practicalities of the testing instruments - how a child will complete required tasks. Imagine for example, asking a toddler to complete a six-minute walk test. It’s a standard test for adult patients, but would be ridiculous for a toddler. I've not known one yet who will walk with purpose in a straight line for six long minutes.
- Train staff in distraction techniques. For young patients, fear of needle sticks and other discomforts can be one of the biggest barriers to trial participation. Having staff that are trained in distraction techniques will minimize their distress, and help your team win favor with patients and their parents.
- Be thoughtful about personal information. Traditional trial questions about alcohol and drug use, birth control and sex may be easy for an adult to answer, but they will make a teen-aged trial participant clam up. If you want to enroll adolescents into a trial, you need to be sensitive to the delicacy of these questions, and have staffers who are trained in how to communicate with young patients about these topics.
- Factor the patient's age and natural growth and development rates into test results. For vital signs and lab tests, be sure to compare results to age appropriate norms - blood pressure or heart rate ranges in children are not the same as for adults and change as children age. Changes that occur over time need to be put into context. In adults, for example, significant weight gain is noteworthy, but in children? Depending on the age of the patient, they will almost certainly gain weight and height over the course of a long study.
- Remember that even among children, one size does not fit all. Take into account the vast developmental, physical and psychological differences among different aged children in your study and set goals accordingly. Even when only a limited age range is studied, there are vast differences between a two- and a six-year-old, or a 10- and 16-year old, or between boys and girls of similar ages. These children will have different cognitive skills, developmental needs, and emotional maturity that you need to account for when selecting outcome measures, testing instruments, norms for comparison etc. in order to produce meaningful and high quality results.
- Factor school commitments into scheduling. When a child participates in a trial, parents have to take off work, siblings get dragged along for the ride, and everyone has to work around school and extracurricular activities. Researchers need to be sensitive to these challenges and offer a schedule that works for families if they want them to stay engaged.
- Don’t forget siblings. In many cases, when a patient arrives at a trial, parents may have one or more siblings in tow. Those kids need more than a stack of old Highlights magazines to keep them occupied. The trial site should offer play spaces and access to technology, and provide other entertainments to keep youngsters occupied. If you don’t, parents and kids will quickly come to dread these visits, and patients will drop out of the trial.
- Provide support materials. You want to create a memorable identity for the trial that will resonate with parents and children. Such materials might include study visit guides to help parents plan, wall chart stickers so patients can track their progress, and a study portal where parents and kids can get more information about the study and the disease. Anything you can do to engage families in the study process will make them more likely to enroll and stick with the trial.
* with thanks to Edmund Capparelli at the University of California, San Diego