White Paper: What You Don't Know Can Hurt You

"The pivotal trial failed," said a senior pharma executive in private conversation. "I got the call on Friday. I was dumbstruck—given its success in earlier work I could hardly believe it."

This type of shock is all too typical. Late-stage disappointments in clinical development programs are common, and many times come as a complete surprise to everyone involved.

That's exactly what happened in 2007 when a biotech CEO received an unexpected FDA rejection letter in response to his company's application to market a new biologic treatment of brain metastases. The failed Phase III study had an estimated enrollment of 550 patients with a 25-patient lead-in phase that established methods for evaluating efficacy based on novel radiologic (MRI), neurocognitive, and neurologic progression endpoints.