Optimal design of clinical trials with biologics using dose-time-response models.

Biologics, in particular monoclonal antibodies, are important therapies in serious diseases such as cancer, psoriasis, multiple sclerosis, or rheumatoid arthritis. While most conventional drugs are given daily, the effect of monoclonal antibodies often lasts for months, and hence, these biologics re...

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Publicado en:Statistics in Medicine Vol. 33; no. 30; pp. 5249 - 5265
Autores principales: Lange, Markus R, Schmidli, Heinz
Formato: research Journal Article
Publicado: Wiley-Blackwell Dec2014
Acceso en línea:Ver este registro en EBSCOhost
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        atl: Optimal design of clinical trials with biologics using dose-time-response models.
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        au:
          Lange, Markus R
          Schmidli, Heinz
      sug:
        subj:
          Antibodies, Monoclonal Administration and Dosage
          Biological Products Administration and Dosage
          Clinical Trials Methods
          Dose-Response Relationship, Drug
          Study Design
          Algorithms
          Gout Drug Therapy
          Probability
          Computer Simulation
          Human
          Immunologic Factors
          Regression
      ab: Biologics, in particular monoclonal antibodies, are important therapies in serious diseases such as cancer, psoriasis, multiple sclerosis, or rheumatoid arthritis. While most conventional drugs are given daily, the effect of monoclonal antibodies often lasts for months, and hence, these biologics require less frequent dosing. A good understanding of the time-changing effect of the biologic for different doses is needed to determine both an adequate dose and an appropriate time-interval between doses. Clinical trials provide data to estimate the dose-time-response relationship with semi-mechanistic nonlinear regression models. We investigate how to best choose the doses and corresponding sample size allocations in such clinical trials, so that the nonlinear dose-time-response model can be precisely estimated. We consider both local and conservative Bayesian D-optimality criteria for the design of clinical trials with biologics. For determining the optimal designs, computer-intensive numerical methods are needed, and we focus here on the particle swarm optimization algorithm. This metaheuristic optimizer has been successfully used in various areas but has only recently been applied in the optimal design context. The equivalence theorem is used to verify the optimality of the designs. The methodology is illustrated based on results from a clinical study in patients with gout, treated by a monoclonal antibody.
      pubtype: Academic Journal
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        research
        Journal Article
      ougenre: Article
    language: English
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