The Patient-Centered Outcomes Research Institute (PCORI) recently awarded $5 million to the University of Iowa College of Public Health to conduct a comparative effectiveness research (CER) study on the treatment of NETS. Currently, there are no clear guidelines regarding the sequencing of NET therapeutic options.
The three-year study, which will be led by Michael O’Rorke, assistant professor of epidemiology, will follow patients for up to five years to track their outcomes in the hopes of determining which care options work, for whom, and under which circumstances.
“Patients with NETs face difficult treatment decisions such as, what therapy would be best to try next? If I were to take this option now, what treatment options will be closed off to me in the future?” observes O’Rorke. “Clinicians are also unsure how best to tailor treatment selection to the characteristics of the patient and his or her tumor.”
The Neuroendocrine Tumor Research Foundation (NETRF) will be participating in this project as a member of a patient advisory group. NETRF will advise on study design, testing of the patient survey portal, and will facilitate patient recruitment to the study.
The study will enroll approximately 3,000 patients from 14 participating research centers throughout the United States with gastroenteropancreatic (GEP-NETs) and lung NETs.
The study hopes to achieve four goals:
- Describe the frequency and sequencing of common treatment regimens currently in use and their outcomes.
- Determine the impact of the following characteristics on the choice of therapies:
- Patient — age, race and gender
- Clinical — preexisting conditions, medications and treatments
- Tumor — stage, grade and nodal involvement
- Based on the findings from the first goal, formulate premise-reasoning-outcomes (PROs) and combine them with the medical record data obtained from the second goal to compare the effectiveness of peptide receptor radionuclide therapy regimens on outcomes of renal toxicity, disease progression and quality of life.
- Share the findings generated by the study in the National Patient-Centered Clinical Research Network (PCORnet) to aid future Comparative Effectiveness Research (CER) into NETs and other rare diseases.
“This large comparative effectiveness study, leveraging data from electronic medical records, chart abstractions, and patient reported outcomes, will go some way to defining the risks and benefits of the different therapeutic options currently available – findings which will be of benefit to patients and their caregivers, clinicians, and other stakeholders involved in their care,” says O’Rorke. “The infrastructure generated will also be of importance for future NET research using the PCORnet resources, and for CER in other rare diseases.”
PCORI is a nonprofit organization established by Congress that funds research to guide patients and those who care for them to make better-informed decisions about healthcare and treatment choices