Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies
Year: 2026
Type: article
Abstract: Individualized medicine has the potential to be a transformative approach to healthcare that tailors medical treatments to the unique makeup of each patient. This report explores the potential of individualized genetic medicines to meet the pressing need for effective treatments for individuals with rare genetic diseases, and funding models to support these treatments. Although recent successes have been achieved, significant administrative and financial challenges remain in implementing individualized treatment trials. This report investigates funding associated with such therapeutics. Funding challenges, key strategic operational factors, potential payor support, historic funding models, and ethical elements ultimately leading to financial support of patient treatment are considered. By addressing these questions, this report aims to provide an overview of the complex issues surrounding individualized medicine, offering insights into balancing its promises with practical and ethical considerations as the field and associated funding models continue to evolve.
Authors: Nicole Nolen, Annemieke Aartsma-Rus, Christine Caneva, Curtis R. Coughlin II, Margot A. Cousin, Catherine Douthwright, Holm Graessner, Olivia Kim-McManus, Ashley Kuniholm, Stefanie Leonard, Andrea Martinsen, Margaret Meserve, Matthis Synofzik, Booma Yandava, Timothy W. Yu, Scott Demarest, Roger J. Paxton
Institutions: Leiden University Medical Center, Children's Hospital Colorado, University of Colorado Anschutz, Mayo Clinic, University of Massachusetts Chan Medical School, University of Tübingen, University of California San Diego, Rady Children's Institute for Genomic Medicine, Boston Children's Hospital, German Center for Neurodegenerative Diseases, Hertie Institute for Clinical Brain Research, Bentley University, Harvard University, Manton Center for Orphan Disease Research, University of Colorado Denver
Language: English
Subfield: Economics and Econometrics
Domain: Social Sciences
Open Access status: gold
Estimated APC paid: $2,990
OpenAlex ID: W7162013317