Requirements for a Breakthrough Therapy Designation

Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, emphasizes the importance of adequate data supporting proposals to the FDA for Breakthrough Therapy designations.

Clinical Pearls

Pazdur notes that several Breakthrough Therapy proposals were denied by the FDA because of inadequate preliminary evidence. This enhances the importance of communicating with the review division prior to submission, to ensure the requirements are met.

• Adequate preliminary evidence is crucial for a drug to receive a Breakthrough Therapy designation
• Communication with the review division prior to submission can help to ensure requirements are met