New Push Needed for Peds Oncology Drug Development: Tufts
Senior leaders from drug companies, government agencies, and academia convened by the Tufts Center for the Study of Drug Development for a roundtable discussion say that companies need to prioritize where to place resources to help pediatric oncology drug development. The center’s latest R&D Management Report (subscription required) says that pumping billions of additional dollars into pediatric academic research might create more findings and publications, and might perhaps improve bedside care, but it is unlikely to result in new pediatric drugs. “Putting such resources into a program that would set up a framework for new pediatric drug research and development, however, would likely yield more new medicines,” the report says.
The panel noted that while the list of unmet medical needs in pediatric oncology remains long, so does the list of R&D challenges, including recruitment of patients, availability of experienced investigators, high development costs, and increasingly complex regulatory requirements.
The rationale for conducting clinical trials, the report says, is to generate data, which cannot be obtained by other means, that can lead to generalizable knowledge as part of a larger information plan. “A plausible, but not exclusive, sequence for an intervention development plan is to first target population selection,” the panelists said, “followed by outcome measure selection, then calibration of the intervention. The intervention development plan will generate its own set of milestones, with each one typically followed by a decision to continue or adjust the plan, or stop and redirect resources.”
A hierarchy of data known as the Clinical Trial Universe is described with steps covering enrollment, retention, exposure, outcome measurements and assessments, and informative events. The report says there are steady losses of information throughout the process, and the process can be improved by emphasizing enabling information, efficiency of data hierarchy, and selection of outcome measures.
Three case studies briefly describe companies that prioritize the portfolio while matching children with cancer to the right drug, offer a perspective on challenges from global launches to children’s taste preferences, or look at a multi-stakeholder model for collaboration.