Investigational agents for pancreatic neuroendocrine tumors: what clinical progress have we seen in the last 5 years?

Introduction: Pancreatic neuroendocrine tumors (pNETs), a group of endocrine tumors that arise in the pancreas from the hormonal cells of the islets of Langerhans, are among the most common gastroenteropancreatic neuroendocrine tumors. The incidence of pNETs has increased in recent years to about 1.5 per 100,000 population and prognosis correlates with histologic grade. This review aims to provide an overview of the newly approved or currently developing drugs over the past five years that have shown a significant improvement in prognosis for patients affected by this rare disease. Areas covered: The literature search was conducted using PubMed, focusing on English-language publications from 2020 to 2025. Keywords included ‘pancreatic neuroendocrine tumors,’ ‘new treatments,’ ‘targeted therapy,’ and ‘clinical trials.’ Studies selected for inclusion comprised clinical trials, systematic reviews, and meta-analyses reporting data on treatment efficacy, safety, and patient quality of life. Expert opinion: Future research holds great potential, especially in improving personalized treatment predictions. Therefore, it is not easy to define a ‘definitive endpoint’ for research, as discoveries and technological innovations continue to evolve.