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The proportion of North American cancer trials that evaluate novel targets

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Summary

Major advances in cancer care often emerge from the development of novel targets. We randomly sampled 10% of cancer trials on clinicaltrials.gov with start dates 2013–2016 to determine the proportion of trials and research subjects directed at evaluating novel targets. We found that 87 of 378 trials (23.0%) enrolling 9225 of 44,525 patients (20.7%) tested interventions that are directed towards novel targets. 146 of 378 trials (38.6%) enrolling 19,132 of 44,525 patients (43.0%) investigated treatments that were not FDA approved but utilized a previously studied target for treating cancer. Combined, 233 of 378 trials (61.6%) enrolling 28,357 of 44,525 patients (63.9%) investigated treatments that were not FDA approved. Furthermore, 36 of 378 trials (9.5%) enrolling 6592 of 44,525 patients (14.8%) investigated FDA approved anticancer drugs in their approved indication and combination while 109 of 378 trials (28.8%) enrolling 9576 of 44,525 patients (21.5%) investigated FDA approved anticancer drugs outside of their approved indication or combination. Logistic regression found that phase 1 trials were significantly more likely to test novel target interventions than phase 2 and 3 trials (p value = 0.00197 and 0.00130 respectively). Industry sponsored trials were also significantly more likely to involve novel target interventions than non-industry trials (p value <0.001). In conclusion, most cancer trials involve unapproved treatments, but a majority of these treatments are well-characterized or involve a previously studied target to treat cancer.

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Data availability

The data that support the findings of this study are available from the corresponding author upon reasonable request. Tables 1 and 2 have associated raw data.

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Acknowledgements

We thank Mark Ratain, Benjamin Carlisle, Deborah Zarin and Patrick Bodilly Kane for advice on this manuscript.

Code availability

There is no custom code or mathematical algorithm that is deemed central to the conclusions of this manuscript.

Funding

This work was supported by the Canadian Institutes of Health Research in Ottawa, Canada. CIHR (EOG 111391).

Manuscript Writing: EG, JK.

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Authors and Affiliations

  1. Biomedical Ethics Unit/Social Studies of Medicine, McGill University, 3647 Peel Street, Montreal, QC, H3A 1X1, Canada

    Eli Gumnit, Aden C. Feustel, Sandy Wong, Rafia Bosan & Nora Hutchinson

  2. Division of Ethics and Policy/School of Population and Global Health, McGill University, 3647 Peel Street, Montreal, QC, H3A 1X1, Canada

    Jonathan Kimmelman

Authors
  1. Eli Gumnit
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  2. Aden C. Feustel
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  3. Sandy Wong
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  4. Rafia Bosan
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  5. Nora Hutchinson
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  6. Jonathan Kimmelman
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Contributions

Trial Extraction and Screening: AF,SW.

Novelty Analysis: RB, EG, NH.

Corresponding author

Correspondence to Jonathan Kimmelman.

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Conflict of interest

Eli Gumnit declares that he has no conflict of interest. Aden C. Feustel declares that he has no conflict of interest. Sandy Wong declares that she has no conflict of interest. Rafia Bosan declares that she has no conflict of interest. Nora Hutchinson declares that she has no conflict of interest. Jonathan Kimmelman serves in a remunerative capacity on a DSMB for Ultragenyx Inc.

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This article does not contain any studies with human participants or animals performed by any of the authors. All information was gathered from publicly available databases.

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Gumnit, E., Feustel, A.C., Wong, S. et al. The proportion of North American cancer trials that evaluate novel targets. Invest New Drugs 39, 256–259 (2021). https://doi.org/10.1007/s10637-020-00971-w

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  • DOI: https://doi.org/10.1007/s10637-020-00971-w

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