Precision oncology promises a new standard of care where therapies are tailored to the molecular profile of a specific tumor. For the full potential of precision medicine to be realized, regulatory, technical, clinical, and economic frameworks need to evolve to the nuances of these novel treatments.
Along with demonstrated efficacy in hematologic malignancies, chimeric antigen receptor (CAR) T-cell therapies have the capacity to elicit serious toxicities. Safety considerations related to CAR T-cells may impact both trial design and trial management, as the adverse events associated with immuno-oncology agents differ from those associated with cytotoxic therapies.
By closing the orphan drug exemption loophole and enabling earlier discussions with the FDA, the Research to Accelerate Cures and Equity (RACE) for Children Act has the potential to accelerate the development of novel treatment options for children with cancer. Read more about how RACE and the FDA guidance will affect the future of pediatric oncology.
There are many challenges inherent in pediatric clinical studies, where the traditional paradigms for evaluating pharmaceutical agents in adults often do not apply. These occur on ethical, physiological, pharmacometrics, and economic grounds, among many others. In this webinar, the featured speakers discuss challenges in implementing clinical studies in children and creative solutions to effectively implement them.
This report, based on a survey of ACRO members, reveals that risk-based monitoring (RBM) makes clinical trial quality review more efficient and effective. It found that when a company reviews data through a centralized system using the RBM model, CROs and technology companies are better able to detect quality issues earlier and make rapid corrections at the site level.