CHRISTIAN K SCHNEIDER
Head of Biopharma Excellence and Chief Medical Officer for Strategic Product Development Consulting, PharmaLex, Denmark

Abstract

Targeted medicines continue to make headway in the battle against chronic disease, changing the prognosis for many cancer patients, with the promise of breakthroughs in other fields. But the path to development and regulatory approval for these transformational products is complex, requiring a high degree of collaboration from the outset. This is particularly critical considering the urgent need of patients awaiting innovative treatments.
There are clear benefits in therapeutic and diagnostic teams working closely together during the development of targeted therapies. Similarly, sponsors that make the most of opportunities to engage with regulators as early as possible are better positioned to navigate the path to approval.

REVOLUTIONIZING HEALTHCARE

Targeted medicine typically refers to a product that binds to a defined target to treat a specific subset of patients (1).
A targeted therapy can selectively target abnormal signaling pathways or biological processes underlying a given disease, offering treatment tailored to a patient’s genetic mutations or protein expression profiles (2).
They have proved particularly effective in the field of oncology, where targeted therapies such as Imatinib (Glivec) and trastuzumab (Herceptin) are improving the survival rate of patients with certain types of cancer (3, 4).
Precision medicine can span myriad drug classes, including small molecules, monoclonal antibodies, antibody-drug conjugates, radioligand therapeutic agents and immunotherapies, as well as cell and gene therapies (5).
Connecting the right treatment with the right patient is enabled by biomarkers and companion diagnostics (CDx), while artificial intelligence has proved invaluable in target identification.

MAKING THE RIGHT CONNECTIONS

Given how interlinked therapeutics and diagnostics are, a coll ...