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How to pay for individualized genetic medicines

Nature Medicine volume 30, pages 1816–1818 (2024)Cite this article

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For precision genetic medicines to fulfill their potential as treatments for ultra-rare diseases, fresh approaches to academic–industry partnerships and data sharing are needed, together with regulatory change and adaptation of reimbursement models.

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Fig. 1: The comparable costs of transplantation and gene therapy.

References

  1. Kim, J. et al. N. Engl. J. Med. 381, 1644–1652 (2019).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  2. Korobeynikov, V. A. et al. Nat. Med. 28, 104–116 (2022).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  3. Tran, H. et al. Nat. Med. 28, 117–124 (2022).

    Article  CAS  PubMed  Google Scholar 

  4. Kim, J. et al. Nature 619, 828–836 (2022).

    Article  Google Scholar 

  5. US Food & Drug Administration. Draft Guidance for Industry https://go.nature.com/3RimwVu (December 2021).

  6. Nguengang Wakap, S. et al. Eur. J. Hum. Genet. 28, 165–173 (2020).

    Article  PubMed  Google Scholar 

  7. Richter, T. et al. Value Health 18, 906–914 (2015).

    Article  PubMed  Google Scholar 

  8. The Transplantation Society. Four Decades of International Cooperation, Innovation, Growth and Progress https://go.nature.com/4aYWU76 (2006).

  9. Bently, T. S., Ortner, N. Milliman (2020).

  10. Manalac, T. BioSpace (20 March 2024).

  11. US Health Resources & Services Administration. Organ Donation and Transplantation Legislation History https://go.nature.com/3RhbYWm (April 2021).

  12. US Congress. H.R. 2617: Consolidated Appropriations Act, 2023 (2022).

  13. S.5002 - FDA Modernization Act 2.0. 17th Congress (2021–2022).

  14. Wong, C. H., Siah, K. W. & Lo, A. W. Biostatistics 20, 273–286 (2019).

    Article  PubMed  Google Scholar 

  15. Auty, S. G., Griffith, K. N., Shafer, P. R., Gee, R. E. & Conti, R. M. J. Health Polit. Policy Law 47, 691–708 (2022).

    Article  PubMed  PubMed Central  Google Scholar 

Download references

Acknowledgements

The authors thank partners at the N=1 Collaborative, the Yu laboratory and EveryONE Medicines for contributions and collaboration. We thank J. Cummings for editorial assistance.

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Author notes

  1. These authors contributed equally: Julia M. Y. Pian, Nana Owusu.

Authors and Affiliations

  1. Boston Children’s Hospital, Boston, MA, USA

    Julia M. Y. Pian & Timothy W. Yu

  2. N=1 Collaborative, Boston, MA, USA

    Julia M. Y. Pian, Julia Vitarello, Winston X. Yan & Timothy W. Yu

  3. Columbia University Vagelos College of Physicians & Surgeons, New York, NY, USA

    Nana Owusu

  4. Mila’s Miracle Foundation, Boulder, CO, USA

    Julia Vitarello

  5. MIT Sloan School of Management, MIT Laboratory for Financial Engineering, MIT Computer Science and Artificial Intelligence Laboratory, Cambridge, MA, USA

    Andrew W. Lo

  6. Santa Fe Institute, Santa Fe, NM, USA

    Andrew W. Lo

  7. Harvard Medical School, Boston, MA, USA

    Timothy W. Yu

Authors
  1. Julia M. Y. Pian
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  2. Nana Owusu
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  3. Julia Vitarello
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  4. Winston X. Yan
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  5. Andrew W. Lo
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  6. Timothy W. Yu
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Corresponding authors

Correspondence to Julia M. Y. Pian or Timothy W. Yu.

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Competing interests

J.M.Y.P. is an associate at Atlas Venture. J.V. is a co-founder of EveryONE Medicines and the N=1 Collaborative. W.X.Y. is a founder, employee and shareholder of Arbor Biotechnologies. A.W.L. reports personal investments in private biotechnology companies, biotechnology venture capital funds and mutual funds; is a co-founder and principal of QLS Advisors LLC, a healthcare investments advisor, and QLS Technologies LLC, a healthcare analytics and consulting company; a director of AbCellera, Annual Reviews, Atomwise, BridgeBio Pharma, Uncommon Cures and Vesalius Therapeutics; an advisor to Apricity Health, Aracari Bio, BrightEdge Impact Fund, Enable Medicine, FINRA, Health at Scale, MIT Proto Ventures, Quantile Health, Roivant Social Ventures, Swiss Finance Institute, Thalēs, Think Therapeutics and xCures; and during the most recent 6-year period has received speaking/consulting fees, honoraria, or other forms of compensation from AbCellera, AlphaSimplex, Annual Reviews, Apricity Health, Aracari Bio, Atomwise, Bernstein Fabozzi Jacobs Levy Award, BridgeBio, Cambridge Associates, CME, Enable Medicine, Journal of Investment Management, Lazard, MIT, New Frontier Advisors, Oppenheimer, Princeton University Press, Q Group, QLS Advisors, Quantile Health, Research Affiliates, Roivant, SalioGen Therapeutics, Swiss Finance Institute, Think Therapeutics, Vesalius Therapeutics and WW Norton. T.W.Y. has received research funding from EveryOne Medicines, has served as a scientific consultant to Biomarin and Servier Pharmaceuticals, is a board member of the Oligonucleotide Therapeutics Society and serves as a volunteer scientific advisor to several nonprofit rare disease foundations. J.M.Y.P., J.V., T.W.Y. and W.X.Y. are volunteers with the N=1 Collaborative, a non-profit organization.

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Pian, J.M.Y., Owusu, N., Vitarello, J. et al. How to pay for individualized genetic medicines. Nat Med 30, 1816–1818 (2024). https://doi.org/10.1038/s41591-024-03071-x

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