IDEAS home Printed from https://ideas.repec.org/a/nat/natcom/v13y2022i1d10.1038_s41467-022-30908-1.html
   My bibliography  Save this article

Addressing challenges with real-world synthetic control arms to demonstrate the comparative effectiveness of Pralsetinib in non-small cell lung cancer

Author

Listed:
  • Sanjay Popat

    (Royal Marsden Hospital and Institute of Cancer Research)

  • Stephen V. Liu

    (Georgetown University)

  • Nicolas Scheuer

    (Roche Products Ltd)

  • Grace G. Hsu

    (Cytel, Inc)

  • Alexandre Lockhart

    (Cytel, Inc)

  • Sreeram V. Ramagopalan

    (Global Access, F. Hoffmann-La Roche)

  • Frank Griesinger

    (Pius-Hospital Oldenburg)

  • Vivek Subbiah

    (The University of Texas MD Anderson Cancer Center)

Abstract

As advanced non-small cell lung cancer (aNSCLC) is being increasingly divided into rare oncogene-driven subsets, conducting randomised trials becomes challenging. Using real-world data (RWD) to construct control arms for single-arm trials provides an option for comparative data. However, non-randomised treatment comparisons have the potential to be biased and cause concern for decision-makers. Using the example of pralsetinib from a RET fusion-positive aNSCLC single-arm trial (NCT03037385), we demonstrate a relative survival benefit when compared to pembrolizumab monotherapy and pembrolizumab with chemotherapy RWD cohorts. Quantitative bias analyses show that results for the RWD-trial comparisons are robust to data missingness, potential poorer outcomes in RWD and residual confounding. Overall, the study provides evidence in favour of pralsetinib as a first-line treatment for RET fusion-positive aNSCLC. The quantification of potential bias performed in this study can be used as a template for future studies of this nature.

Suggested Citation

  • Sanjay Popat & Stephen V. Liu & Nicolas Scheuer & Grace G. Hsu & Alexandre Lockhart & Sreeram V. Ramagopalan & Frank Griesinger & Vivek Subbiah, 2022. "Addressing challenges with real-world synthetic control arms to demonstrate the comparative effectiveness of Pralsetinib in non-small cell lung cancer," Nature Communications, Nature, vol. 13(1), pages 1-10, December.
    • Handle: RePEc:nat:natcom:v:13:y:2022:i:1:d:10.1038_s41467-022-30908-1
    DOI: 10.1038/s41467-022-30908-1
    as

    Download full text from publisher

    File URL: https://www.nature.com/articles/s41467-022-30908-1
    File Function: Abstract
    Download Restriction: no
    File URL: https://libkey.io/10.1038/s41467-022-30908-1?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    References listed on IDEAS

    as
    1. David M. Phillippo & Anthony E. Ades & Sofia Dias & Stephen Palmer & Keith R. Abrams & Nicky J. Welton, 2018. "Methods for Population-Adjusted Indirect Comparisons in Health Technology Appraisal," Medical Decision Making, , vol. 38(2), pages 200-211, February.
    Full references (including those not matched with items on IDEAS)

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Manuel Gomes & Nick Latimer & Marta Soares & Sofia Dias & Gianluca Baio & Nick Freemantle & Dalia Dawoud & Allan Wailoo & Richard Grieve, 2022. "Target Trial Emulation for Transparent and Robust Estimation of Treatment Effects for Health Technology Assessment Using Real-World Data: Opportunities and Challenges," PharmacoEconomics, Springer, vol. 40(6), pages 577-586, June.
    2. B. Muresan & C. Mamolo & J. C. Cappelleri & M. J. Postma & B. Heeg, 2021. "Cost-Effectiveness of Bosutinib for the Treatment of Adult Patients with Chronic Phase Chronic Myeloid Leukemia in the Second-Line Setting," Applied Health Economics and Health Policy, Springer, vol. 19(6), pages 929-940, November.
    3. Dasom Lee & Shu Yang & Lin Dong & Xiaofei Wang & Donglin Zeng & Jianwen Cai, 2023. "Improving trial generalizability using observational studies," Biometrics, The International Biometric Society, vol. 79(2), pages 1213-1225, June.
    4. Jiyeon Kang & John Cairns, 2023. "“Don’t Think Twice, It’s All Right”: Using Additional Data to Reduce Uncertainty Regarding Oncologic Drugs Provided Through Managed Access Agreements in England," PharmacoEconomics - Open, Springer, vol. 7(1), pages 77-91, January.
    5. Nan Qiao & Ralph Insinga & Thomas Burke & Gilberto Lopes, 2021. "Cost-Minimization Analysis of Pembrolizumab Monotherapy Versus Nivolumab in Combination with Ipilimumab as First-Line Treatment for Metastatic PD-L1-Positive Non-small Cell Lung Cancer: A US Payer Per," PharmacoEconomics - Open, Springer, vol. 5(4), pages 765-778, December.
    6. Doug Coyle & Isabelle Durand-Zaleski & Jasmine Farrington & Louis Garrison & Johann-Matthias Graf von der Schulenburg & Wolfgang Greiner & Louise Longworth & Aurélie Meunier & Anne-Sophie Moutié & Ste, 2020. "HTA methodology and value frameworks for evaluation and policy making for cell and gene therapies," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 21(9), pages 1421-1437, December.

    More about this item

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:nat:natcom:v:13:y:2022:i:1:d:10.1038_s41467-022-30908-1. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Sonal Shukla or Springer Nature Abstracting and Indexing (email available below). General contact details of provider: http://www.nature.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.