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UNC Lineberger’s Jennifer Lund, PhD, assistant professor of epidemiology at UNC Gillings School of Global Public Health, recently was awarded a $732,000 grant from the Patient-Centered Outcomes Research Institute (PCORI). Lund serves as principal investigator for the project, “Enhancing hybrid study designs for comparative effectiveness research.”

UNC Lineberger’s Jennifer Lund, PhD.

The hybrid study design is an emerging approach for evaluating population-level benefits and harms of treatments. While randomized controlled trials (RCTs) have been considered the gold standard, those trials often underrepresent certain subgroups of patients (e.g., adults age 65+ years, women, racial and ethnic minorities) and are conducted in highly controlled environments with close monitoring of patients.

Hybrid studies combine information from patients enrolled in RCTs used to establish drug approval with observational data from patients treated in clinical practice following the drug approval.

“As a pharmacoepidemiologist,” Lund said, “much of my work is focused on generating evidence about the population-level benefits and harms of treatments when they are used in more diverse patient populations treated in everyday clinical practice.”

To that end, Lund and her team will use the PCORI Methods Award to develop research tools to visualize differences between colon cancer patients enrolled in RCTs and those treated in clinical practice, with respect to their demographics (e.g., age, sex, race/ethnicity) and clinical characteristics, as well as how their treatment is administered (e.g., dose reductions, delays and omissions).

“We then will generate guidance documents and tutorials for researchers demonstrating the use of these tools, drawing upon an example of two treatments for colon cancer,” said Lund. “This information then can be used in the analysis phase of studies to generate information about population-level treatment benefits and harms that are relevant to patients and their health care providers, as well as insurance payers and regulators.”

Colon cancer was selected as an example because the kinds of colon cancer patients receiving one of the treatments has changed over time and the way treatments are administered in these patients differs from the way they were administered in the RCT.

“Older adults are still underrepresented in randomized trials,” said Til Stürmer, MD, PhD, Nancy A. Dreyer Distinguished Professor and chair of the Gillings School’s epidemiology department and one of the co-investigators of the study. “If the treatment effect varies by age, the treatment effect estimate from the trial therefore does not translate into the treatment effect we expect in the population actually treated. [The project team] will develop methods that will result in more realistic population-level treatment effect estimates by combining data from trials with real-world data.”

Other investigators on the study are Gillings School epidemiology faculty members Daniel Westreich, PhD, associate professor, M. Alan Brookhart, PhD, associate professor, and Alexander Keil, PhD, assistant professor, as well as Hanna Sanoff, MD, MPH, clinical medical director of the North Carolina Cancer Hospital and associate professor of medicine in the UNC School of Medicine.

Lund and her team will engage a range of health care stakeholders, including doctors, insurers, health care systems and drug regulators.

“By continually incorporating their feedback into our work, we will be able to generate tools and guidance that will be useful to a broad spectrum of researchers,” said Lund. “Ultimately, improving the use of the hybrid study designs will lead to more accurate and contemporary information about the population-level benefits and harms of new therapies that can inform both clinical and policy decisions and ultimately lead to improvements in outcomes that matter to patients.”