Improving the efficiency of randomized trials of behavioral interventions for acute and chronic respiratory failure

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The goal of this Midcareer Investigator Award in Patient-Oriented Research is to enhance the ability of Dr. Scott Halpern to mentor students, residents, post-doctoral fellows, and junior faculty in developing and testing behavioral economic interventions to prevent and improve outcomes following, acute and chronic respiratory failure. Patients with many forms of acute and chronic respiratory failure, such as acute respiratory distress syndrome (ARDS) and chronic obstructive pulmonary disease (COPD), respectively, are at high risk of death and suffer considerable short- and long-term impairments in quality of life and functioning. Behavioral interventions, including those informed by behavioral economic principles, hold great promise for preventing these conditions and improving outcomes. Yet the knowledge produced by randomized clinical trials (RCTs) of such interventions is limited due to impediments to patient accrual, patient-centered outcomes ascertainment, and the abilities of traditional analyses to determine which interventions work best for which patients. By leveraging the conceptual models, research infrastructures, and data collected in 5 of Dr. Halpern's ongoing or recently completed RCTs including 40,000 patients, Dr. Halpern and his mentees will develop and test ways to surmount each of these three barriers to producing greater knowledge on how to prevent and treat acute and chronic respiratory failure. First, they will conduct randomized trials of “nudges” to improve enrollment and retention in real RCTs. Second, they will learn and apply state-of-the-art natural language processing and machine learning techniques to enhance the ability to extract patient-centered outcomes from electronic health records, thereby identifying more efficient methods for outcome ascertainment in large, pragmatic RCTs. Third, they will learn and apply modern methods in causal inference and the detection of heterogeneous treatment effects to better understand how effective behavioral interventions are overall, how efficacious they would be if more patients adhered to them, and which interventions work best for which patients. These scientific aims will be completed within the context of a structured training program designed to improve Dr. Halpern's skills in mentoring junior patient-oriented researchers. Dr. Halpern has already successfully mentored more than two dozen patient-oriented researchers. However, by enabling him to reduce his clinical and administrative commitments, this award will increase the time he commits to mentoring, and will improve his mentoring skills by supporting didactic training in influence and leadership and “mentor-the-mentor” meetings with more senior mentors. The award will also enable him to develop a “Junior Faculty Visiting Professor Program in Pulmonary and Critical Care Medicine,” thereby enhancing opportunities for his and other established investigators' mentees to network with other leaders in the field, receive external feedback and mentorship, and gain the confidence that comes with speaking to less familiar audiences.