Considering tomorrow’s patients in today’s drug approvals

Responding to pressure from patients seeking access to promising new medicines,1 drug regulatory agencies have worked over several decades to adopt more patient centred approaches.234567 The decision whether to approve a drug reflects both scientific evidence and value judgments about the sufficiency of that evidence.8 Patient centred drug regulation recognises patients as experts in their conditions and aims to incorporate their experiences, needs, and preferences when making decisions that demand balancing benefits and risks, speed and certainty.9 Because patients are directly affected by drug approvals, especially when they face serious diseases and conditions that lack good treatment options, it is an ethical imperative that they have a seat at the regulatory table, with the opportunity to have their voices heard in decisions about which drugs should be allowed to come to market and how they should be studied.1011

A relatively unrecognised but persistent challenge, however, is that tomorrow’s patients—those who are presently healthy but will develop serious diseases in the future—are missing from that table. Although these individuals will eventually be affected by today’s drug approvals, they cannot be straightforwardly included in today’s decision making.12 All patients share an interest in having effective, safe drugs available when they need them, but current and future patients’ value judgments may differ in important ways (fig 1).

For example, when the US Food and Drug Administration controversially approved aducanumab for Alzheimer’s disease,13 it pointed to the willingness of some patients “to accept the trade-off of some uncertainty about …