Sponsors are likely to encounter compounding complexities when running pediatric rare disease trials.
Individually, rare disease and pediatric clinical studies present noteworthy challenges, but together, they can tie logistical knots that impact families and cause costly delays. With more than 150 successful rare disease clinical trials supported, including several pediatric indications, this article outlines our top lessons learned and offers advice 15 years in the making.
Rare diseases affect nearly 300 million people globallyand most of them are children1,2
In pediatric hospitals, beds are regularly filled with children battling a rare disease—approximately one third of whom will not live to see their 5th birthday.3 With an average time to diagnosis of six to eight years,4 high mortality rates, and no clear progression nor established standard of care, the margin for uncertainty in research is zero.
Participation in a clinical study can be a difficult ask, and these children are often severely ill. Managing the side effects of investigational products, transporting children to sites for visits, and running labs or assessments can create an overwhelming environment for everyone involved. For sponsors, these challenges are compounded by the increasing intensity of competition in study recruitment and gaining consent amidst heavily restricted travel.
Competition and cost make patient-centricity an essential strategy in rare disease pediatric research.
One obvious yet inescapable hurdle of rare disease pediatric research is the small pool of patients from which to recruit. With more and more choices available, families sit firmly in the driver’s seat. Variables like geography, study design, and trial goals are important factors that contribute to the willingness to enroll one’s child in a clinical trial. While some of these variables may not be within a sponsor’s control, there are ways to make studies easier and more appealing to potential patients and caregivers.
These immensely powerful tools help to ease feelings of uncertainty, giving caregivers the information necessary to feel comfortable with the prospective therapy’s mechanism of action, the study’s requirements, and what potential outcomes are expected.
Marketing is the most important way in which sponsors can raise awareness of an upcoming study. When well-executed, those information-seeking parents and caregivers will proactively inquire about enrollment criteria and share promising findings within trusted online circles.
Among the many factors affecting a patient’s experience is the transfer of information. Portals streamline data sharing, helping to mitigate the disruption of household routines due to study participation.
When in-person appointments are required, travel can be a sore subject. The special needs of very ill children make access to travel agents and seamless reimbursement a real difference-maker in this population’s decision-making.
Participating sites may also be affected by a pediatric rare disease study
Adding a complex trial on top ofthe already-taxed resources of most study sites can be a battle. In addition to the personnel requirements, there are beds and access to equipment, like infusion pumps or respirators, required to meet the needs of these very (often terminally) ill children. Experience shows us that employing strategies to minimize the burden can help encourage sites to participate. Examples include:
Providing inclusion and exclusion cards, in addition to standard recruitment and engagement materials, as well as a laminated schedule of events for easy use in clinical settings
Pocket protocols, visit checklists, and a study reference manual, together with Frequently Asked Questions, for at-a-glance information and greater time efficiencies
CRO-led trainings and streamlined communication pathways, like identifying centralized points of contact or outlining meeting cadence before study initiation, help to streamline study-related work
As rare disease pediatric patients are frequently immune-suppressed, remote care, telemedicine and in-home nursing visits have shown to further reduce the efforts of on-site staff. The adoption of decentralized clinical trials surged during COVID-19, and the outcomes have been largely favorable, demonstrating positive potential for specific populations.
Consent and assent in pediatric rare disease clinical trials.
It is essential that patients and caregivers have the time to review study consent forms in their own time at their home. This allows them to review the information, understand the risks and benefits, ask questions, and evaluate the impact participation will have on the family. Many sites and CROs are moving toward e-consent platforms which are even more accommodating to patients and their families.
Managing emotions plays a tremendous role in a rare disease pediatric study’s success.
Kids will be kids and parents…parents. Remember, enrolling a child into a clinical trial can offer hope – if not for a cure today, for one that helps future patients – but until then, it is a burden. Children experience a rollercoaster of emotions ranging from fear and isolation in unfamiliar surroundings to the inconvenience in accommodating study schedules and site visits. Caregivers too can be anxious or even wary.
Through our experience working with child participants in rare disease studies, we’ve identified several study adaptations that can make study participation easier for everyone.
Families should only be contacted by trust people
Familiar physicians or an advocacy group are ideal for reaching out and the communication should always be tailored to the audience.
Do not expect caregivers to “get it” right away
While caregivers are very much subject matter experts in their child’s disease, with innovative therapies being developed with complex mechanisms of action, avoiding medical jargon, asking questions to confirm their understanding, and providing ample time for discussions about the study and the child’s health helps build trust.
Streamline study visits when possible
Short visit times help parents who are taking time off work, managing transportation issues, and securing the extra help that may go in to transporting a child.
Put yourself in the caregiver’s shoes
Focusing on the holistic elements surrounding enrollment helps to build trust, show empathy, and reduce the emotional toll of study participation. This, in turn, creates a differentiated environment which is more attractive to families and increases the chances of enrollment and a timely study start.
To learn more about Precision’s experience overcoming enrollment challenges for rare disease clinical trials, read this case study.
Traci Fulton is Vice President of Project Management at Precision for Medicine, providing senior level oversight and support for clinical trials spanning rare disease, oncology and CNS indications. Ms. Fulton brings over 21 years’ experience with CRO teams, supporting the conduct of clinical trials across all phases, with over 18 years of her career focused on execution and delivery for complex clinical trials in oncology and rare diseases. She is passionate in her approach to support trials and her drive to support advancements in the treatment for underserved patient populations.
Precision for Medicine is part of the Precision Medicine Group, an integrated team of experts that extends Precision for Medicine’s therapeutic development capabilities beyond approval and into launch strategies, marketing communication, and payer insights. As one company, the Precision Medicine Group helps pharmaceutical and life-sciences clients conquer product development and commercialization challenges in a rapidly evolving environment.