"Patients, physicians and clinical resources are precious assets to a clinical trial, and it is vital to the sponsoring company that these assets are not wasted. By continually reassessing and adapting the trial design, one can zero in on the best dose for Phase III, identify patient subsets who will most benefit from
the drug or quickly identify potential safety issues. Adaptive approaches can optimize the trial on the fly, conserving resources and potentially shaving months and millions of dollars off the cost of developing a new drug. In this panel, we discuss how adaptive clinical trials integrate the use of biomarkers, statistical analysis, modeling and simulation, and describe the technology used to capture data in real time to support decision making."

Objective1: "Assess the status of orphan drugs in terms of applications and approvals from a historical and current standpoint . . . where are we trending?"

Objective2: Review the challenges of obtaining an orphan drug approval from a regulatory standpoint.

Objective3: Assess the overall risk/benefit profile of bringing an orphan drug to market and review post-approval obligations.