How we approach early phase clinical trial and off-label therapy consults in pediatric oncology: The New Agents and Innovative Therapy (NAIT) team experience

• Published in: EJC paediatric oncology 2024-06, Vol.3, p.100154, Article 100154
• Main Authors: Revon-Rivière, Gabriel, Tibout, Pauline, Cabral, Jennifer, Siddiqi, Aiman, Doka, Ashley, Mills, Denise, Fung, Karen, Judd, Sandra, Morgenstern, Daniel A., Cohen-Gogo, Sarah
• Format: Article
• Language: English
• Subjects: Compassionate access; Early Phase clinical trial; Ethics; Off-label prescription; Virtual care
• ISSN: 2772-610X; 2772-610X
• DOI: 10.1016/j.ejcped.2024.100154

In the context of hard-to-cure disease, pediatric oncologists may have to explore novel therapy options and explain their rationale, risks and constraints to patients and caregivers. The New Agents and Innovative Therapy (NAIT) program at Hospital for Sick Children in Toronto facilitates patient enrollment in clinical trials as well as access to innovative therapies outside of clinical trials. Here, we summarize our experience with helping patients, caregivers, and their primary oncology team navigate information and access to new therapeutic options through enrollment in clinical trials but also off-label and compassionate use. We expose our approach to exploring clinical trial and other therapy options. We share lessons learned from clinical practice regarding the specific role of NAIT consultant, as opposed to the primary oncologist or the disease expert. We expand on ways to communicate regarding the objectives of early phase clinical trials, their methods and the important commitment asked from participants. We describe our views on equipoise, uncertainty and hope in this very specific practice. We support a model of shared decision making and empowerment of patients and caregivers. We also detail the use, benefits and challenges of virtual care applied to NAIT consults. Overall, we hope to contribute and facilitate the NAIT practice not only for trained trialists but also less-specialized teams. •Innovative therapy options should be explored in the context of hard-to-cure cancer.•Innovative therapies can be accessed through clinical trials and “out-of-trial”.•Communication with patients and caregivers is paramount is this context.•Virtual care applied to these consults has benefits and challenges.