Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma

Abstract

Drug regulatory agencies are increasingly pressed by the challenge of finding the appropriate balance between the need for rapid access to new drugs and the need to ensure comprehensive data on their benefits and risks. This dilemma is not new, but has been made more prominent by recent high-profile drug withdrawals and conflicting demands, including the need to improve the efficiency of drug development on one hand, and the need to avoid exposing patients to unnecessary risks or possibly ineffective treatments on the other. Here, we summarize the current demands by stakeholders and the scientific and regulatory issues at stake, describe existing and emerging regulatory approaches, and speculate on future directions, such as evolution of the current regulatory model from a one-off marketing authorization to a life-cycle approach.