Parents', Health Care Professionals', and Scientists' Experiences of a Precision Medicine Pilot Trial for Patients With High-Risk Childhood Cancer: A Qualitative Study.

PURPOSE

Children with high-risk cancers have low survival rates because current treatment options are limited. Precision medicine trials are designed to offer patients individualized treatment recommendations, potentially improving their clinical outcomes. However, parents' understanding is often limited, and expectations of benefit to their own child can be high. Health care professionals (HCPs) are often not familiar with precision medicine and might find managing families' expectations challenging. Scientists find themselves working with high expectations among different stakeholders to rapidly translate their identification of actionable targets in real time. Therefore, we wanted to gain an in-depth understanding of the experiences of all stakeholders involved in a new precision medicine pilot trial called TARGET, including parents, their child's HCPs, and the scientists who conducted the laboratory research and generated the data used to make treatment recommendations.

METHODS

We conducted semistructured interviews with all participants and analyzed the interviews thematically.

RESULTS

We interviewed 15 parents (9 mothers; 66.7% bereaved), 17 HCPs, and 16 scientists. We identified the following themes in parents' interviews: minimal understanding and need for more information, hope as a driver of participation, challenges around biopsies, timing, and drug access, and few regrets. HCP and scientist interviews revealed themes such as embracing new technologies and collaborations and challenges managing families' expectations, timing of testing and test results, and drug access.

CONCLUSION

Educating families, HCPs, and scientists to better understand the benefits and limitations of precision medicine trials may improve the transparency of the translation of discovery genomics to novel therapies, increase satisfaction with the child's care, and ameliorate the additional long-term psychosocial burden for families already affected by high-risk childhood cancer.