In the past 5 years, Rho has conducted more than 50 studies in pediatric populations involving more than 18,000 patients and 800 sites.
While patient recruitment can be challenging in any study, there are additional challenges to recruiting pediatric patients. Parents may be more risk averse to giving an unproven therapy to their child than they would towards receiving it themselves. To improve the chances of successfully enrolling a study, it is important to consider potential motivators for participation:
- Therapeutic benefit: If you are working on a therapy for a rare disease or for an indication where there is no approved or effective product, parents may be motivated by the opportunity to receive treatment that could improve their child’s condition even if it isn’t proven and if there is a chance they will receive placebo. When there is an approved effective treatment available parents are likely to be reluctant to sign their child up when they may receive placebo, receive a treatment whose effectiveness is unknown, or receive a treatment with unknown side effects and safety issues.
- Financial incentives: Many studies offer financial incentives to participants, and this can be a motivating factor for some parents. Additionally, patients may receive study related medications, assessments, or more routine care that could be cost prohibitive otherwise.
- Research benefit: Particularly for studies in rare disease or orphan indications, parents may see the benefit in research that provides a better understanding of the disease or the prospect of better treatment options in the future even if their child does not receive a direct benefit in participation.
Getting pediatric patients enrolled in a study is great, but it is just as important to make sure most patients are completing the study. There are a number of factors that make this more difficult in a pediatric study:
- Multiple schedules to coordinate: Each study visit requires both the parent and child to be available. Studies with numerous visits can become a significant hassle for parents, which can lead to discontinuations. Making sure that every visit is necessary and being as accommodating as possible with scheduling, such as including flexible visit windows can mitigate this risk. (Remember: Most of the parents still have to work and kids attend school).
- Parents don’t see the therapeutic benefit: If parents come to believe that their child is receiving placebo or that the treatment is ineffective, they may withdraw their child from the study. Providing clear information about what the trial is evaluating and encouraging frequent communication will help facilitate the parent voicing any concerns.
- Discomfort of participation: No one likes long doctor visits or being stuck repeatedly with a needle, but these discomforts are even harder on pediatric patients and their parents. Evaluate each assessment carefully during protocol development (even ones like blood pressure and temperature monitoring) to reduce the overall burden to the patient.
What can be done to improve retention? Encourage investigators to talk with parents about the importance of completing the study. Consider what incentives may be appropriate to improve retention and work within the limitations of what the IRB will allow based on your study. Finally, keep visits as short as possible, limit blood draws and invasive procedures, that every procedure and assessment is truly necessary to determine the safety or efficacy of the investigational product.
Close collaboration between the Rho team, the Sponsor and sites results in study completion a month ahead of schedule
Situation: A phase III ADHD laboratory classroom study
The Success: For a phase III ADHD laboratory classroom study, we:
- Completed enrollment early
- Reduced time from last patient last visit to database lock
- Produced top line results two days after database lock
- Completed the study more than a month ahead of schedule
We achieved these results by:
- Selecting experienced central sites
- Setting site start-up expectations during qualification visits
- Using online ADHD assessment tools that reduced effort for sites and parents and reduced errors in scoring
- Using sites experienced in EDC
- Understanding the sponsor’s goals to deliver top lines as soon as possible after database lock
Enrollment completed early on two pediatric studies
Rho successfully completed enrollment early on two pediatric otic studies. These studies involved more than 50 sites and over 500 subjects. The project leader identified some key factors in successfully completing enrollment early, including:
- Building and maintaining strong relationships between site personnel and CRO project team members.
- Staying in close contact with site staff to keep them motivated and identify challenges early.
- Keeping the Sponsor well informed, so decisions could be made quickly with all the risks and implications of the decisions understood.
- Streamlining site identification by re-using high performing sites previously used by the Sponsor or us.
The project team also met their timelines for database lock and topline results.
Pediatric Study Indications
- Otitis media
- Rare and ultra-rare diseases
- Cystic fibrosis
- Peanut allergy
- Transplant (liver, renal, kidney, heart, lung)
- Dental caries
- Periodontal disease
- Type 1 diabetes
Rho's CRO Services
- Integrated product development
- Clinical operations and monitoring
- Project management
- Medical monitoring
- Product safety and pharmacovigilance
- Biostatistics and statistical programming
- Clinical data management
- Regulatory operations
- Data standards and CDISC
- Regulatory and medical writing
- Quality assurance