The FDA Changed Everything

Abstract

On May 23, 2017, the FDA approved the first cancer treatment (pembrolizumab) for any solid tumor with a specific genetic biomarker: microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR). For the first time in history, a solid cancer treatment was approved based on the genetic makeup of tumor not on the location in the body where the cancer originated, for example lung or breast cancer (TNM staging). This indication covers patients with solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options and patients with colorectal cancer that has progressed following treatment with certain chemotherapeutics. All cancer drug approvals in the last 30 years were grounded on TNM staging independent of the therapy type (chemotherapy, monoclonal antibodies, TKI inhibitors, immune therapies or targeted therapies) and despite the huge and fast advances in understanding tumor biology. In fact, the FDA previously approved pembrolizumab taking into consideration the TNM staging, for the treatment of certain patients with metastatic melanoma, metastatic non-small cell lung cancer, recurrent or metastatic head and neck cancer, refractory classical Hodgkin lymphoma, and urothelial carcinoma. The archaic TNM staging will probably be changed under the disruptive wave of molecular biology. The recent FDA approval could be considered the certificate of birth for a truly new dimension of personalized medicine in cancer. We recommend European Union to follow the FDA approach of tissue-agnostic cancer drugs in order to speed up the development of next-generation oncologic therapies and to increase the access of patients to truly personalized medicine.

Keywords: Cancer drug approval; Genetic biomarker; Oncologic therapy; Pembrolizumab; TNM staging; Tissue-agnostic cancer drug.