Patients Need More Education Regarding the Purpose of Precision Medicine Trials
Precision medicine may offer new hope to children with high-risk cancer, but only if families and healthcare professionals (HCPs) are fully educated on the benefits and limitations of precision medicine trials, according to a study in Journal of Clinical Oncology.
While precision medicine trials offer patients access to complex treatment platforms, they can be difficult to understand. For instance, patients and parents often have a limited understanding of genomics and may not understand the purpose of an experimental trial.
“Parents often have high expectations regarding the potential therapeutic benefit for their child,” the authors explained. “These high expectations may be misconstrued as misunderstanding the trial’s primary objective but often are associated with parents’ hope of tumor response. Even when parents know that their child is unlikely to benefit, they may still hope that an effective treatment will be identified.”
It is up to the HCPs working with families to understand the purpose of precision medicine trials; however, the HCPs may have trouble explaining the purpose of an experimental trial or managing expectations.
This study followed 3 participant groups—parents, HCPs, and scientists—who had been involved with the Australian Targeted Agents for High-Risk Groups of Paediatric Tumours (TARGET) pilot study. Families who were enrolled in TARGET were told that the trial was assessing the feasibility of a new precision medicine protocol. They were also told that if any personalized therapies were identified, their child might not benefit. The researchers then analyzed paired tumor and blood samples for all patients. Those with germline mutations were referred to the genetics team for testing and counseling.
A total of 17 parents, from 10 families, opted into the trial, but the researchers were only able to reach and interview 15 of these parents. More than half (66.7%) of the parents were bereaved and were, on average, 1.9 years from their child’s death when the interview took place. A total of 33 HCPs and scientists participated in the study.
Interviews with the parents made it clear that they had a minimal understanding of the trial and needed more information. Some parents could not remember the aim of the study, and the researchers found that some parents had not understood that “TARGET was an analysis platform and not one specific drug.” However, there were parents who had a clear understanding.
In addition, the decision to enroll children in the trial was often motivated by hope their child would benefit but also by necessity since they had no other treatment options. However, the researchers found that parents had few regrets and, not only would they make the same decision again, but they would recommend participating in studies to other parents.
Among the HCPs, the researchers found the HCPs appreciated that TARGET gave them potential access to new therapies, and they valued the fact that they could offer state-of-the-art care. However, they expressed challenges managing expectations, unexpected findings, and drug access. They found it difficult to tell families there were no actionable findings.
In the interviews with scientists, the researchers found similar results. The scientists valued being able to collaborate with clinicians to learn and generate new knowledge. They also identified challenges around the time pressure to generate findings for the patient and the emotional impact of the study. For instance, they described often being upset when they learned a child had died before they completed analysis.
“Supporting families to better understand the benefits and limitations of precision medicine will be critical to enhance satisfaction with their child’s care and reduce potential negative psychological outcomes,” the researchers concluded. “Easy-to-understand educational resources in multiple languages explaining the main concepts of precision medicine trials are needed.”
Vetsch J, Wakefield CE, Duve E, et al. Parents’, health care professionals’, and scientists’ experiences of a precision medicine pilot trial for patients with high-risk childhood cancer: a qualitative study [published online November 17, 2019]. J Clin Oncol. doi: 10.1200/PO.19.00235.