Hepatoblastoma is an exceptionally rare type of malignant tumor that originates from abnormal growth of liver cells. Despite its low incidence, it’s also the most common type of liver cancer and typically occurs in children younger than 3 years of age. First-line therapy for this form of cancer involves resecting the tumor out of the liver so that it can regenerate new healthy hepatocytes. However, this is usually preceded with chemotherapy to reduce tumor size before removal and minimize the chances of recurrence.
Thanks to the availability of these interventions, the cure rate for pediatric hepatoblastoma is approximately 70%, which is a staggering 40% jump since the 1970s. This highlights why hepatoblastoma clinical trials are so vital for advancing cancer research and ensuring biopharma/biotech sponsors can bring these much-needed treatment options into the market. However, running these hepatoblastoma clinical trials smoothly is no small feat. In this next installment of Vial CRO’s Oncology Trial Management series, we present the top 5 challenges sponsors face when running hepatoblastoma clinical trials and how to overcome them.
1 | Recruiting Pediatric Patients for Hepatoblastoma Clinical Trials
Recruitment goals are challenging enough for most clinical trials to meet when the target population consists of adults. Only about 50-70 new cases of hepatoblastoma are diagnosed in the United States annually, which significantly narrows the potential candidate pool for these studies. More importantly, parental concern and hesitation about clinical trials is another issue to account for when recruiting pediatric patients. In addition to ensuring the selected sites truly have access to the right participant population, biopharma companies can improve pediatric enrollment by developing safe, child-friendly advertising materials which could be shared with parents through local pediatricians, oncologists, childcare centers, and schools.
2 | Study Design Considerations for Rare Diseases
Aside from the patient availability aspect, rare cancers inherently require more unique study designs than other conditions. For example, the small pool of potential participants statistically limits how accurately traditional designs can determine the safety and efficacy of a new therapy. To get around this, sponsors will often have to enlist sites globally, as well as build effective partnerships with patient advocacy groups, regulatory authorities, and patients’ families. Alternative trial approaches intended for rare indications like hepatoblastoma with smaller sample sizes include enrichment, crossover, adaptive, and N-of-1 designs.
3 | Measuring Patient-Reported Outcomes
Electronic patient-reported outcome (ePRO) platforms are a central piece of technology that provides biopharma and biotech sponsors with essential information on how a clinical trial therapy is impacting a patient’s quality of life. Given that hepatoblastoma primarily affects children under the age of 5, it can be difficult to collect ePRO data. One method is to adapt the questionnaires into an engaging, even gamified, image series so that young children can communicate their emotions and experiences more clearly. Of course, if the patient is an infant or toddler, their guardians will have to complete ePROs on their behalf. Regardless, sponsors should take care to standardize data collection methods to account for discrepancies between pediatric and parental reporters.
4 | Obtaining Consent from Vulnerable Populations
Children are classified as a vulnerable population in the context of clinical research, which necessitates a more complex informed consent process before they can be included in a clinical trial. This will differ if the patient is an infant or a young child able to communicate, but sponsors may benefit from creating standardized competency criteria for determining which subjects are eligible to provide their assent. The content of assent forms should also contain engaging, playful animations of trial information to maximize the level of understanding achieved by the child. These creative approaches can help sponsors maximize safe, informed enrollment into their pediatric hepatoblastoma trials.
5 | Decision-Making with all Influential Stakeholders
Cancer diagnoses are difficult as is for families, but treatment decisions become that much more emotionally charged when the individual affected is a young child. Depending on their age, interactive decision tools can be used to obtain input from pediatric patients, as they’ve shown some success in previous research. Additionally, sponsors can potentially provide or help sites locate cancer support resources for parents emotionally struggling with difficult decisions involving their child’s care. Hepatoblastoma trials already have so few subjects meeting the eligibility criteria that the sponsor’s retention plan should find ways to include families in the decision-making process. Strategies like this are essential for helping both parents and their children feel understood and comfortable during the trial.
Vial, The CRO for Oncology Clinical Trials
Pediatric oncology research can be a complicated maze to navigate, but Vial’s Oncology CRO has you covered. Our clients benefit from our next-generation approaches to clinical research, led by a team of clinical professionals with specialized expertise in oncology. Connect with a team member today to learn how working with us can get you ahead with your next clinical trial!
