Evidence map›Paper›PMID 37935855›Full record

ArticleGene therapy2023

The estimated annual financial impact of gene therapy in the United States.

Chi Heem Wong, Dexin Li, Nina Wang, Jonathan Gruber, Andrew W Lo, Rena M Conti

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Gene therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 45 papers.

0numbers the graph read from it
0cells of the map it votes in
45citing papers in PubMed
36.0field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

45 citing papers in PubMed, 63 citations in OpenAlex.

  1. Review
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  6. Review
  7. Receptor-guided AAV Tropism Engineering via MATCH.bioRxiv : the preprint server for biology · 2026
    Article
  8. Effects of Cannabidiol on TAFAZZIN-Deficient B-Lymphoblastoid Cells.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2026
    Article
  9. Article
  10. Article
  11. Review
  12. Review
  13. Review
  14. Review
  15. Review
  16. Genetic applications in arthroplasty: A review.Bulletin of the Hospital for Joint Disease (2013) · 2025
    Review
  17. Review
  18. Treating Hearing Loss: From Cochlear Implantation to Gene Therapy.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Review
  19. Review
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 5 institutions in 1 country.

Chi Heem WongResearch Affiliate, MIT Computer Science and Artificial Intelligence Laboratory & Department of Electrical Engineering and Computer Science, Cambridge, MA, 02139, USA.
Dexin LiGraduate Student, Department of Economics, Princeton University, Princeton, NJ, 08544, USA.
Nina WangUndergraduate Student, Laboratory for Financial Engineering, MIT, Cambridge, MA, 02142, USA.
Jonathan GruberFord Professor of Economics, Chair, Department of Economics, MIT, Cambridge, MA, 02142, USA.
Andrew W LoCharles E. and Susan T. Harris Professor, Department of Finance, MIT Sloan School of Management, Director, Laboratory for Financial Engineering & CSAIL, MIT, Cambridge, MA, 02142, USA.
Rena M ContiAssociate Professor and Dean's Scholar, Department of Markets, Public Policy and Law, Questrom School of Business, and Co-Director Technology Policy and Research Institute, Boston University, Boston, MA, 02115, USA. rconti@bu.edu.ORCID 0000-0002-4190-1839
Boston University · USFord Motor Company (United States) · USIIT@MIT · USMassachusetts Institute of Technology · USPrinceton University · US

Funding

NCI NIH HHS
6 · The paper itself

Abstract

Gene therapy is a new class of medical treatment that alters part of a patient's genome through the replacement, deletion, or insertion of genetic material. While still in its infancy, gene therapy has demonstrated immense potential to treat and even cure previously intractable diseases. Nevertheless, existing gene therapy prices are high, raising concerns about its affordability for U.S. payers and its availability to patients. We assess the potential financial impact of novel gene therapies by developing and implementing an original simulation model which entails the following steps: identifying the 109 late-stage gene therapy clinical trials underway before January 2020, estimating the prevalence and incidence of their corresponding diseases, applying a model of the increase in quality-adjusted life years for each therapy, and simulating the launch prices and expected spending of all available gene therapies annually. The results of our simulation suggest that annual spending on gene therapies will be approximately $20.4 billion, under conservative assumptions. We decompose the estimated spending by treated age group as a proxy for insurance type, finding that approximately one-half of annual spending will on the use of gene therapies to treat non-Medicare-insured adults and children. We conduct multiple sensitivity analyses regarding our assumptions and model parameters. We conclude by considering the tradeoffs of different payment methods and policies that intend to ensure patient access to the expected benefits of gene therapy.

Indexed as

Costs and Cost AnalysisGenetic TherapyHumansUnited States

Identifiers

PMID37935855
PMCPMC10678302
OpenAlexW4388479699

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.