Related Experiment Video
Updated: Jul 5, 2025
Author Spotlight: Finding New Therapeutic Targets for Malignant Peripheral Nerve Sheath Tumor Through Genome-Scale shRNA Screens
Published on: August 25, 2023
Making progress against rare cancers: A case study on neuroendocrine tumors
Michael O'Rorke1, Elizabeth Chrischilles1,
1Department of Epidemiology, College of Public Health, University of Iowa, Iowa City, Iowa, USA.
Abstract:
In April 2023, the National Cancer Institute offered a roadmap for cancer research to achieve Cancer Moonshot goals. To reach these goals requires making progress for all cancers, not just those that are most common. Achieving progress against rare cancers, as well as common cancers, requires involvement of large clinical research networks. In 2020, the Patient-Centered Outcomes Research Institute (PCORI) launched an initiative on Conducting Rare Disease Research using PCORnet, the National Patient-Centered Clinical Research Network. The purpose of this commentary is to introduce the broader community of cancer researchers to the PCORnet NET-PRO study (comparing the effects of different treatment approaches for neuroendocrine tumors on patient-reported outcomes) thereby demonstrating how researchers can use the PCORnet infrastructure to conduct large-scale patient-centered studies of rare cancers.
Insights
Large clinical research networks are essential for advancing rare cancer research. The PCORnet NET-PRO study demonstrates how to leverage this infrastructure for patient-centered rare cancer investigations.
Area of Science:
- Oncology
- Clinical Research Networks
- Patient-Centered Outcomes Research
Background:
- National Cancer Institute's Cancer Moonshot initiative emphasizes progress for all cancers, including rare types.
- Large clinical research networks are crucial for advancing research in both common and rare cancers.
- The Patient-Centered Outcomes Research Institute (PCORI) launched an initiative in 2020 to support rare disease research using PCORnet.
Purpose of the Study:
- To introduce cancer researchers to the PCORnet infrastructure.
- To highlight the PCORnet NET-PRO study as a model for rare cancer research.
- To demonstrate the utility of PCORnet for large-scale, patient-centered studies of rare cancers.
Main Methods:
- Utilizing the PCORnet (National Patient-Centered Clinical Research Network) infrastructure.
- Focusing on the NET-PRO study, which compares treatment approaches for neuroendocrine tumors.
- Measuring patient-reported outcomes to assess treatment effectiveness.
Main Results:
- The PCORnet infrastructure facilitates large-scale clinical research for rare cancers.
- The NET-PRO study serves as an example of patient-centered research within PCORnet.
- The study demonstrates the feasibility of conducting comparative effectiveness research for rare tumors.
Conclusions:
- PCORnet provides a valuable platform for rare cancer research.
- Patient-centered outcomes are key in evaluating treatments for rare cancers.
- Leveraging research networks like PCORnet is vital for achieving equitable progress in cancer research.
