Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies

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.

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Publication Details

Journal
Orphanet Journal of Rare Diseases
Published
2026-05-21
DOI
https://doi.org/10.1186/s13023-026-04388-1
Primary Topic
Genomics and Rare Diseases
Type
article
Field-Weighted Citation Impact
0.00

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article

Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies

Margaret Meserve, Ashley Kuniholm, Margot A. Cousin, A. Aartsma-Rus et al.
Orphanet Journal of Rare Diseases
Genomics and Rare Diseases
article

Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies

Margaret Meserve, Ashley Kuniholm, Margot A. Cousin, A. Aartsma-Rus, Timothy W. Yu, Curtis R. Coughlin, Stefanie Leonard, Nicole Nolen, Scott Demarest, Booma Yandava, Andrea Martinsen, Christine Caneva, Roger J. Paxton, Holm Graessner, Catherine Douthwright, Matthis Synofzik, Olivia Kim-McManus
article en

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.

Orphanet Journal of Rare Diseases
Boston Children's Hospital (US), Children’s Institute (US), Mayo Clinic (US), Harvard University (US), Bentley University (US), University of Massachusetts Chan Medical School (US), Leiden University Medical Center (NL), Somerville Hospital (US), German Center for Neurodegenerative Diseases (DE), Children's Hospital Colorado (US), Mayo Clinic in Arizona (US), Cambridge Collaborative (United States) (US), Hertie Institute for Clinical Brain Research (DE), Mayo Clinic in Florida (US), Dana-Farber/Boston Children's Cancer and Blood Disorders Center (US), University of Colorado Anschutz Medical Campus (US), University of Tübingen (DE), University of Colorado Denver (US)
Chan Zuckerberg Initiative
Openalex Percentile: Top 6%
Genomics and Rare Diseases
DOIOpen Access PDFOpenAlex
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