IDEAS home Printed from https://ideas.repec.org/p/ehl/lserod/125440.html
   My bibliography  Save this paper

Optimum designs for clinical trials in personalized medicine when response variance depends on treatment

Author

Listed:
  • Duarte, Belmiro P.M.
  • Atkinson, Anthony C.

Abstract

We study optimal designs for clinical trials when the value of the response and its variance depend on treatment and covariates are included in the response model. Such designs are generalizations of Neyman allocation, commonly used in personalized medicine when external factors may have differing effects on the response depending on subgroups of patients. We develop theoretical results for D-, A-, E- and D (Formula presented.) -optimal designs and construct semidefinite programming (SDP) formulations that support their numerical computation. D-, A-, and E-optimal designs are appropriate for efficient estimation of distinct properties of the parameters of the response models. Our formulation allows finding optimal allocation schemes for a general number of treatments and of covariates. Finally, we study frequentist sequential clinical trial allocation within contexts where response parameters and their respective variances remain unknown. We illustrate, with a simulated example and with a redesigned clinical trial on the treatment of neuro-degenerative disease, that both theoretical and SDP results, derived under the assumption of known variances, converge asymptotically to allocations obtained through the sequential scheme. Procedures to use static and sequential allocation are proposed.

Suggested Citation

  • Duarte, Belmiro P.M. & Atkinson, Anthony C., 2024. "Optimum designs for clinical trials in personalized medicine when response variance depends on treatment," LSE Research Online Documents on Economics 125440, London School of Economics and Political Science, LSE Library.
    • Handle: RePEc:ehl:lserod:125440
    as

    Download full text from publisher

    File URL: http://eprints.lse.ac.uk/125440/
    File Function: Open access version.
    Download Restriction: no
    ---><---

    References listed on IDEAS

    as
    1. A. C. Atkinson, 2015. "Optimum designs for two treatments with unequal variances in the presence of covariates," Biometrika, Biometrika Trust, vol. 102(2), pages 494-499.
    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. Yu, Jun & Kong, Xiangshun & Ai, Mingyao & Tsui, Kwok Leung, 2018. "Optimal designs for dose–response models with linear effects of covariates," Computational Statistics & Data Analysis, Elsevier, vol. 127(C), pages 217-228.
    2. Waite, Timothy W., 2024. "Replication in random translation designs," Statistics & Probability Letters, Elsevier, vol. 215(C).
    3. Yu, Jun & Meng, Xiran & Wang, Yaping, 2023. "Optimal designs for semi-parametric dose-response models under random contamination," Computational Statistics & Data Analysis, Elsevier, vol. 178(C).
    4. Atkinson, Anthony C. & Biswas, Atanu, 2017. "Optimal response and covariate-adaptive biased-coin designs for clinical trials with continuous multivariate or longitudinal responses," LSE Research Online Documents on Economics 66761, London School of Economics and Political Science, LSE Library.
    5. Atkinson, Anthony C. & Biswas, Atanu, 2017. "Optimal response and covariate-adaptive biased-coin designs for clinical trials with continuous multivariate or longitudinal responses," Computational Statistics & Data Analysis, Elsevier, vol. 113(C), pages 297-310.
    6. Qiong Zhang & Amin Khademi & Yongjia Song, 2022. "Min-Max Optimal Design of Two-Armed Trials with Side Information," INFORMS Journal on Computing, INFORMS, vol. 34(1), pages 165-182, January.
    7. Belmiro P. M. Duarte & Anthony C. Atkinson & David Pedrosa & Marlena van Munster, 2024. "Compound Optimum Designs for Clinical Trials in Personalized Medicine," Mathematics, MDPI, vol. 12(19), pages 1-20, September.
    8. Hanan Hammouri & Muna Salman & Mohammed Ali & Ruwa Abdel Muhsen, 2025. "Advances in Clinical Trial Design: Employing Adaptive Multiple Testing and Neyman Allocation for Unequal Samples," Mathematics, MDPI, vol. 13(8), pages 1-23, April.

    More about this item

    Keywords

    ;
    ;
    ;
    ;
    ;

    JEL classification:

    • C1 - Mathematical and Quantitative Methods - - Econometric and Statistical Methods and Methodology: General

    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:ehl:lserod:125440. 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: LSERO Manager (email available below). General contact details of provider: https://edirc.repec.org/data/lsepsuk.html .

    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.