Journal Article (Review Article)

DZNE-2026-00879

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies.

Nolen, N. ; Aartsma-Rus, A. ; Caneva, C. ; Coughlin Ii, C. R. ; Cousin, M. A. ; Douthwright, C. ; Graessner, H. ; Kim-McManus, O. ; Kuniholm, A. ; Leonard, S. ; Martinsen, A. ; Meserve, M. ; Synofzik, M.DZNE* ; Yandava, B. ; Yu, T. W. ; Demarest, S. ; Paxton, R. J.

2026
BioMed Central London

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Please use a persistent id in citations: doi:10.1186/s13023-026-04388-1

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.

Keyword(s): Humans (MeSH) ; Precision Medicine: economics (MeSH) ; Rare Diseases: genetics (MeSH) ; Genetic Therapy: economics (MeSH) ; Finance ; Funding ; Individualized medicine ; Personalized healthcare ; Precision medicine ; Rare disease ; Ultra-rare disease

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Contributing Institute(s):

1. Parkinson Genetics (AG Gasser)

Research Program(s):

1. 353 - Clinical and Health Care Research (POF4-353) (POF4-353)

Appears in the scientific report 2026

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Database coverage:
; ; ; ; Article Processing Charges ; BIOSIS Previews ; BIOSIS Reviews Reports And Meetings ; Clarivate Analytics Master Journal List ; DOAJ Seal ; Ebsco Academic Search ; Essential Science Indicators ; Fees ; IF < 5 ; JCR ; PubMed Central ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

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