BIO Comments on Upcoming ODAC Pediatric Oncology Subcommittee Meeting

On Wednesday, April 27th, BIO submitted comments regarding an upcoming meeting of ODAC’s Pediatric Oncology Subcommittee on PREA Waivers for Same-in-Class Drugs. In particular, the subcommittee will meet to discuss the development of a conceptual framework that will inform the decision making of FDA on sponsor plans and requests for waivers of early pediatric investigations of molecularly targeted cancer drugs and biologics when multiple same-in-class products are approved and/or in development, recognizing that the rarity of pediatric cancers may preclude the feasibility of investigations of multiple products. In the comments submitted, BIO stated support for the development of a conceptual framework as the absence of an effective mechanism for making decisions about the feasibility of preliminary efficacy studies may increase the likelihood that studies cannot be fully enrolled, an outcome undesirable to all stakeholders. However, BIO emphasized that the construction of a conceptual framework to assist FDA’s regulatory decision making should avoid unduly burdening one sponsor more than its competitors, utilize scientific data (e.g., mechanistic, nonclinical, clinical, etc.), and have a patient-centric approach.