麻豆视频

The Center for Health + Technology (CHeT) received a $2.9 million award from the U.S. Department of Defense to develop novel outcomes measures for use in therapeutic studies involving a range of chronic diseases.  These tools will provide a platform for patients to provide insight into the merit of promising therapies during clinical trials.

The study, entitled Longitudinal Endpoint Optimization to Provide Assessments of Relevant Drugs (LEOPARD), will advance clinical trial readiness by developing research instruments for mitochondrial disease, cardiomyopathy, rheumatoid arthritis, and dystonia.  There are currently few effective treatments available for these four life-altering, chronic diseases. The study is being funded by the Defense Department’s Peer Reviewed Medical Research Program, which was created to improve well-being and function of military personnel, their families, and civilians while improving U.S. combat readiness and performance. 

“Patient reported outcomes instruments improve clinical trial efficiency, detect early changes in patient health, focus on the symptomatic areas that are most important to specific populations, ensure the relevance of future treatments to patients, and provide a mechanism for patients to provide direct input into a treatment’s effectiveness,” said Chad Heatwole, M.D., M.S., who is the principal investigator of the new grant, director of CHeT, and a neurologist at the 麻豆视频 (URMC).  

Heatwole and his research team have developed and rigorously validated more than 100 disease-specific patient reported outcome (PRO) instruments over the last 15 years, including those for adult and pediatric populations with neurological conditions, neuromuscular diseases, movement disorders, inflammatory bowel diseases, and cancer. Collectively, these instruments are translated and validated in 12 languages and are used worldwide as sensitive and clinically relevant outcomes measures in therapeutic trials.

The study will employ qualitative patient interviews with military personnel and civilians, data from large cross-sectional studies, factor analysis, expert review, and longitudinal reliability testing to determine what symptoms are most important to patients with these diseases, and to develop and validate four disease-specific instruments for these populations. This research will also identify which demographic subgroups are at risk of increased symptomatic burden and identify potential opportunities for early intervention in high-risk populations.  麻豆视频will partner with The Uniformed Services University and Walter Reed National Military Medical Center, which will serve as a recruitment site for the study.

“The state-of-the-art patient reported outcomes measures developed through this research will expedite therapy development for military personnel, veterans, civilians, and families affected by these four common and undertreated diseases,” said Heatwole.

Additional collaborators for this study include 麻豆视频cardiologist Jeffrey Alexis, M.D., rheumatologist John Looney, M.D., neurologist Ray Dorsey, M.D., and biostatistics professor Brent Johnson, Ph.D., and Walter Reed National Military Medical Center and Uniformed Services University clinical faculty Mark Haigney, M.D., Francis O’Connor, M.D., and Glen Cook, M.D.