Putting Meat on the Bone: How to Fast-Track Innovative Medicines to Those Who Need Them and Generate Data to Justify Continued Use

Abstract

Regulatory agencies worldwide have taken significant steps to expedite approval and market authorization of medicines based on their potential to address areas of significant unmet medical need and severe disease burden. But initial approval of such medicines is often accompanied by limited evidence of benefit, posing a conundrum for payers and health systems who may desire greater certainty of their value. This paper describes a system of “accelerated access” to manage these tensions and coordinate activities across stakeholders, based on discussions held at a multi-stakeholder convening in June 2023. We focus on 6 core, near-term actions that can be taken to improve the current system: clarifying criteria for expedited regulatory approval; enhancing stakeholder coordination; creating expedited pathways in payer and health technology assessment settings; developing joint regulatory/payer/HTA guidance on study design and data needs; linking pricing policy to data uncertainty; and improving patient and public understanding of the processes involved as well as the risks and benefits of the relevant medicines. Many of these actions will require additional resources and personnel, and some will necessitate unprecedented levels of coordination. Nevertheless, each action is designed to work with minimal adjustments to the current system rather than demanding an entirely new approach.