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

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. P · 2026

Read Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies by 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. P (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-13474725.txt.

Description

For a handful of children with unique gene faults, scientists can now craft a treatment for one patient alone, but who pays for such N-of-1 trials is unresolved. Nicole Nolen and co-authors map the funding question in this 2026 report.

They review past funding models, the role insurers might play, and the practical and ethical hurdles that surround paying for a therapy built for a single person. Administrative friction and high unit costs stand out as the main brakes. The authors aim to give researchers, families, and payers a shared picture of workable routes, so promising one-off treatments do not stall for money alone.

Catalog record

CREATORNicole 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. P
YEAR2026
COLLECTIONPAPERS
FORMATTXT
FILE SIZE45 KB
VERSION1.0
LICENSECC BY 4.0
RIGHTSCC BY
SOURCEEurope PMC

Source: https://europepmc.org/articles/PMC13474725

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