Beyond Immune Checkpoint Inhibitors to a New Era of Personalized Medicine

Immunotherapies are being touted—both in scientific literature and the general media—as the most promising advances in cancer treatment in decades. Indeed, newly approved drugs are improving survival dramatically for patients with many difficult-to-treat cancers such as melanoma, Hodgkin’s lymphoma, and cancer of the bladder, lung, kidney, and head and neck. Immunotherapies are coming to the fore in cancer treatment as we learn more about tumor genetics, pathophysiology, tumor microenvironment, and as interest grows in attacking tumors on multiple levels. Some—though still few—therapies under development are truly individualized for the patient, potentially ushering in a new, even more tailored approach to personalized medicine.

The field of research has engendered such enthusiasm that there is some concern we may be experiencing a “bubble” in immunotherapy development.1 Currently, 830 companies are developing 1,578 cancer immunotherapy drugs via 4,062 development projects across 535 different targets. (That is excluding the 624 drugs for which development has ceased and the 19 for which it has been suspended.)2 There is fear that many of these products, once in clinical practice, will not live up to the high hopes they’ve raised while in development. Furthermore, the market is unlikely to bear the cost of so many drugs at the price-point of current checkpoint inhibitors. (The Wholesale Acquisition Cost (WAC) of the four checkpoint inhibitors on the market for a 12-week dose runs from $24k to $137k, and the estimated cost of CAR-T therapy could be even higher than bone marrow transplants, which can exceed $500,000.3) Both fears are justified, but have yet to deter enthusiastic researchers.

Clinical trials of cancer immunotherapies are necessarily quite different from those in other therapy areas; they require special planning and are subject t unique medical, regulatory, and operational considerations. Here we discuss the factors that sponsors must take into account as they design and execute clinical trials in this space.

Click here to read our perspectives on the latest immunotherapies