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.

Governance of conditional reimbursement practices in the Netherlands

“…As such, conditional reimbursement aligns well with the recent move to making the registration process of new medicines more flexible and tailormade, e.g. in terms of conditional approvals [19] and adaptive licensing [20]. These schemes require room for careful tinkering and learning in such a way that actors can carve out their responsibilities and processes.…”

Observations on Three Endpoint Properties and Their Relationship to Regulatory Outcomes of European Oncology Marketing Applications

“…Hard clinical endpoints reflect objective measures, namely, overall survival (OS), duration of response, and death. A surrogate end point (a quantitative measure that substitutes for a clinically meaningful end point and that can predict a change in the outcome based on epidemiologic, therapeutic, pathophysiologic,orotherscientific evidence) that is,progressionfree or disease-free survival (PFS/DFS) or overall response rate (ORR) might be more subjective [7][8][9].…”

Good Decision Making Requires Good Communication

“…et al, [51] European Medicines Agency, [52] Levitan et al, [7] Mussen et al [53] and Phillips et al [8] They differ from the FDA's Framework primarily in requiring more formal (and more demanding) judgements from experts and in relying more heavily on computation.…”