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Outcomes & Effectiveness

Novel conceptual framework for cancer comparative effectiveness research

Novel therapies have improved the survival of patients with cancer but have also increased treatment costs substantially. These therapies create an enormous financial burden for patients, their families, and society in general. Furthermore, there are several critical barriers to generating reliable evidence to guide treatment decision-making. First, there is a lack of head-to-head comparative evidence. Also, as the cancers progress, patients’ regimens can involve multiple lines of treatment. Thus, the relevant research question for clinical evidence often becomes not simply how one drug compares to another, but which treatment sequence may produce the best patient outcomes. This project will combine literature reviews, secondary data analyses, and simulation modeling to identify the optimal sequential treatments for advanced non-small cell lung cancer. By integrating practice patterns from real-world data with evidence from randomized controlled trials, our proposed framework could provide a new foundation for comparative effectiveness research.

Funding source: American Cancer Society
Principal Investigator: Shi-Yi Wang
The IN4M Study: Quantifying physical function in cancer patients undergoing chemotherapy using clinician- and patient- reports along with wearable device data

This study aims to understand the measurement characteristics and relationships among various physical function assessments and activity data collected from a wearable device in cancer patients undergoing chemotherapy. The goals of the research are to: (1) collect data on physical function using clinician- and patient-reported methods, performance measurement, and wearable devices; (2) compare the challenges associated with collecting information using each physical function assessment tool; (3) compare longitudinal changes in physical function with the likelihood and magnitude of side effects from chemotherapy; and (4) conduct a structured exit interview to evaluate burden and usability across the different physical function evaluation modalities and a wearable device. The ultimate goal of this project is to provide evidence and knowledge to guide the selection of PF assessment modality/tool(s) to be incorporated in cancer clinical trials as an endpoint for regulatory and treatment decisions.

Funding source: U.S. Food and Drug Administration
Principal Investigator: Cary Gross

Inpatient Therapy and Outcomes in Acute Myeloid Leukemia

Unlike many solid tumors, treatment for acute myeloid leukemia (AML) is often urgent and provided in the inpatient setting. Not all patients receive aggressive therapy – due to age or underlying fitness for cytotoxic chemotherapy regimens. Little is known about patterns of inpatient AML treatment, complications, and short term mortality. This study will use detailed data from the Premier Perspectives database to characterize patterns of induction therapy, complications, and in-hospital mortality for AML. By triangulating with data from SEER-Medicare, we seek to develop population-level estimates of inpatient and outpatient treatment and outcomes.

Funding Source: Celgene Corporation
Principal Investigator: Xiaomei Ma