A Multistakeholder Perspective on Advancing Individualized Therapeutics

Abstract

Precision medicine has evolved from the application of pharmacogenetic biomarkers to the prospective development of targeted therapies in patients with specific molecular/genetic subtypes of disease to truly “N‐of‐1” medicines targeted to very small numbers of patients – in some cases, a single identified patient. This latter iteration of precision medicine presents unprecedented opportunities for patients with severe, life‐threatening, or life‐limiting diseases. At the same time, these modalities present complex scientific, clinical, and regulatory challenges. To realize the promise of individualized medicines, a multistakeholder approach to streamlining medical diagnoses, advancing the technologies that enable development of these therapeutic modalities, and re‐envisioning collaborative environments for access and evidence generation is of critical importance. Herein, we highlight some of these challenges and opportunities.