Patient-Centered Outcomes Research In Advanced Stage Lung Cancer: Study Design and Population

Background: In 2013, researchers at the University of Nebraska Medical Center (UNMC) received funding from the Patient-Centered Outcomes Research Institute to establish a study that outlines treatment options for patients who have stage IV lung cancer. With collaborators at 4 cancer centers, UNMC researchers will compare treatment preferences among these patients when available drugs offer the same survival but different side effects. Patient preferences identified in this study can be used to inform clinical practice. This paper describes proposed study methods and population for this patient-centered outcomes research (PCOR) and highlights their significance for PCOR in lung cancer. Lung cancer is the leading cause of cancer-related deaths in the United States. Treating physicians have traditionally measured treatment success for stage IV non-small cell lung cancer (NSCLC) in terms of survival. It is not known whether individual patients also see success in terms of survival. There is no evidence to suggest that patient preferences regarding adverse treatment effects are systematically considered when choosing a treatment regimen for stage IV NSCLC. Not much is known about patient perspectives on the trade-offs between possible additional survival and possible side effects related to a treatment regimen. Study design: We will conduct a multicenter mixed method study design, which includes focus groups, and cross-sectional, prospective, and randomized experimental designs, to answer PCOR questions. Study population: Baseline data from participating cancer centers were obtained to determine sample size. On the basis of this data, we expect a study population of lung cancer patients that is 48% female and 52% male, with average age about 70 years. We also expect the population to be about 50% rural and 50% urban. Conclusion: A complex design may be necessary for a PCOR study to reflect patients’ preferences. Analysis of existing data from participating cancer centers is essential to understand the characteristics of the lung cancer patient pool for study and to estimate an accurate study sample.