Open AccessTargeted Therapies in Neuroendocrine Tumors (NET): Clinical Trial Challenges and Lessons Learned
Author(s) -
Yao James C.,
Lagunes Diane Reidy,
Kulke Matthew H.
Publication year - 2013
Publication title -
the oncologist
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.176
H-Index - 164
eISSN - 1549-490X
pISSN - 1083-7159
DOI - 10.1634/theoncologist.2012-0434
Subject(s) - neuroendocrine tumors , everolimus , medicine , sunitinib , clinical trial , randomized controlled trial , oncology , clinical study design , intensive care medicine , cancer
In the past 3 years, we have witnessed the completion of four randomized phase III studies in neuroendocrine tumors and the approval of two new drugs, everolimus and sunitinib, for the treatment of patients with well‐differentiated pancreatic neuroendocrine tumors. These studies demonstrate a shift from case series and single‐arm studies toward prospective, randomized controlled clinical trials and evidence‐based therapy in the neuroendocrine tumor field. However, the clinical development of these agents also highlights the potential challenges awaiting other new drugs in this area. Herein, we discuss the strengths and weaknesses of the most recent phase II and phase III neuroendocrine tumor studies and discuss how limitations inherent in current trial design can lead to potential pitfalls. We also discuss how trial design can be improved, with the hope of increasing the number of drugs successfully developed to treat patients with neuroendocrine tumors.