Parents are often reluctant to enroll their child in a research study, particularly if the study doesn't offer a direct benefit to their child. Some parents distrust medical research and believe that clinical trials are unsafe, and they worry their child may suffer from bad side effects or be treated like a guinea pig. For children, participation may mean a disruption to their daily activities.
In pediatric trials, the burden of participation is really on the entire family. Parents must be present to assist their child in completing diary entries and getting to and from multiple study visits, which may require the family to travel long distances. Logistically, meeting study requirements can be quite a juggling act for parents. Will the study center have overnight accommodations for patients and their families? Will they need to get childcare for siblings? What about time off away from their jobs? Parents often fear they will not be able to make it all work.
All of these concerns can derail a trial before it even gets started. However, concerns can be overcome throughout each stage of the trial through effective communication and following a few simple principles—educate and inform, establish ongoing engagement, and convey appreciation. These three principles are a framework for minimizing risks and promoting a positive trial experience in the pediatric patient population.
Principle 1 – Educate and Inform
Parents and Children. Most parents are unaware that many of the FDA-approved drugs that children are given have only been tested in adults and are prescribed off-label for children without an adequate understanding of the appropriate dose, safety, or efficacy. As an industry, we need to do a better job of educating and communicating with parents about the need for more research in children.
Making sure parents fully understand why the medication is being investigated, why their child is being considered, and the health benefits and risks will start to ease some of their worries. Once enrolled in a study, parents need to be reassured that their child will receive optimal care. Researchers follow strict safety and regulatory guidelines to ensure children are not compromised during a study. Medications are monitored very closely to assess any potential risks. If a side effect does happen to occur, the child’s medication will be adjusted, or the researcher may even decide the patient should exit the trial. For any reason and at any time, it’s important for children to know that they can choose to opt out of the study—the child and the parents are in control.
Children should be active participants and truly understand what it’s like to be in a clinical trial. Therefore, educational materials should be customized based on the patient’s age group, which may include age-appropriate recruitment videos and discussion guides that supplement the Informed Consent Form, known as ICFs. All studies require informed consent and ICFs are often long and difficult to understand. Educating both parents and patients can help them make informed decisions and improve the overall trial experience.
Site Staff: Study-specific site training is very important. The entire staff needs to be educated about why they are doing the study, so they can explain it to parents. Equipping the staff with a pre-established, consistent message will help to build confidence and trust.
Study Awareness: Once parents find out about a potential trial, the first thing they usually do is Google it. Having a study website gives it credibility and is a great tool to educate not only parents but also families and advocacy groups.
Depending on the patient setting, various strategies and tools are used to educate and inform all stakeholders about a trial. In rare disease studies, we communicate directly with advocacy groups and share information. These groups are made up of parents who are very well connected to the community. It is important to educate them about trial opportunities and build relationships, so referral pathways are established.
The inpatient setting is a bit different. Potential study participants need to be identified upon onset into the hospital. Staff may wear small, study-branded buttons, put up study information notices on bulletin boards and their computer desktops. These tools help keep the study front of mind, so a potential patient can be quickly identified. For parents of preemies and neonates, educational videos are available to help them assess whether to put their infant in a clinical trial.
Principle 2 – Ongoing Engagement
Pediatric patients come in all sizes and have different needs based on their age group, indication, and patient setting. That’s why age-appropriate tools are important to help educate children, so they can feel more comfortable and involved in the study.
For preschool and kindergarten children, this means simple tools that involve play-based learning that provides guidance and helps them interpret their feelings and emotions. Preadolescent children are early readers and are becoming more independent and opinionated on what they will read and not read, so materials need to keep their interest. Digital, video content, and particularly comics can also be powerful tools.
As children reach adolescence, they require more direct, clear, honest, peer-to-peer communication. They seek out like-minded peers and support using social media. This age group is very independent and responds well to digital technology.
For parents and caregivers, mobile technology tools can help them stay connected while also easing the burden of clinical trial participation.
From a retention standpoint, different engagement tools improve the trial experience:
- Text and email messaging
- Mobile apps
- Lost to follow-up services
- Patient, parent, and kid portals
- Patient and parent surveys
When appropriate, families can also be supported with expense reimbursement debit cards as well as transportation and concierge services.
Principle 3 – Appreciation
Showing appreciation through patient newsletters, comfort items, and milestone awards for study accomplishments perpetuate a positive trial experience and help patients and families become strong advocates in the community for research participation.
Customized communication is a vital part of mitigating risks in every stage of a pediatric study and helps build trust. Trust and close relationships between site staff, parents, and children are major factors for why parents give consent for their child to participate in a clinical trial and stay compliant throughout the trial. Educating and engaging parents and young study participants can change their negative attitudes about research and decrease parents’ worries about disruption to their daily lives. Showing appreciation to study participants for their contributions and rewarding them and their parents and caregivers is important in perpetuating a positive trial experience.
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