Longitudinal End Point Optimization to Provide Assessments of Relevant Drugs
Abstract
Mitochondrial disease, cardiomyopathy, rheumatoid arthritis, and dystonia are chronic conditions that collectively affect ~1/60 people.These four conditions markedly reduce the well-being and function of military personnel, their families, and civilians while simultaneously limiting U.S. combat readiness and performance. In order to facilitate the development, evaluation, and approval of promising treatments for these four chronic diseases, researchers require valid and sensitive clinical outcome measures that reliably detect relevant changes in all of the symptoms that are most important to patients. The U.S. Food and Drug Administration (FDA) has identified properly validated disease-specific patient reported outcome measures (PROMs) as an acceptable and, at times, preferred mechanism to measure therapeutic change during clinical trials and support drug labeling claims. Clinical trial infrastructure in mitochondrial disease, cardiomyopathy,rheumatoid arthritis, and dystonia is currently limited. The development, validation, and optimization of multifactorial disease-specific PROMs for these populations will significantly bolster this infrastructure. This research will utilize qualitative patient interviews with military personnel and civilians, four large cross-sectional studies, factor analysis, expert review, and longitudinal reliability testing to: 1)determine what symptoms are most important to patients with these diseases; and, 2) 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. The multifaceted state-of-the-art PROMs developed through this proposal are transformative technologies that will expedite relevant therapy development for military personnel, veterans, civilians, and families affected by these four common and undertreated diseases.
Document Details
- Document Type
- Technical Report
- Publication Date
- Aug 01, 2023
- Accession Number
- AD1226989
Entities
People
- Chad R Heatwole
Organizations
- University of Rochester