Application of Bayesian Borrowing Methods in Clinical Trials for Children with Type II Diabetes Mellitus.

Clinical trials for children with type II diabetes mellitus (T2DM) pose challenges often due to recruitment issues. The variability in the treatment effect for pediatrics with T2DM tends to be much larger than that for adults, therefore, a larger pediatric study is needed to independently detect a s...

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Publicado en:Therapeutic Innovation & Regulatory Science Vol. 60; no. 1; pp. 210 - 223
Autores principales: Crackel, Roberto, Ji, Yuanyuan, Kim, Yoonhee, Travis, James, Tu, Wenda, Wang, Yun, Kim, Sunghee, Bonangelino, Pablo
Formato: equations & formulas research tables/charts Journal Article
Publicado: Springer Nature Jan2026
Acceso en línea:Ver este registro en EBSCOhost
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      dt: Jan2026
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        atl: Application of Bayesian Borrowing Methods in Clinical Trials for Children with Type II Diabetes Mellitus.
      aug:
        au:
          Crackel, Roberto
          Ji, Yuanyuan
          Kim, Yoonhee
          Travis, James
          Tu, Wenda
          Wang, Yun
          Kim, Sunghee
          Bonangelino, Pablo
        affil: https://ror.org/034xvzb47 United States Food and Drug Administration, Silver Spring, MD, USA
      sug:
        subj:
          Diabetes Mellitus, Type 2 Drug Therapy
          Hypoglycemic Agents Therapeutic Use
          Drugs, Investigational Therapeutic Use
          Drug Development
          Clinical Trials
          Probability
          Research Subject Recruitment
          Sample Size
          Human
          Male
          Female
          Child
          Adolescence
          Diabetic Patients
          Methodological Research
          Drugs, Investigational Adverse Effects
          Models, Statistical
          Glycated Hemoglobin Blood
          United States Food and Drug Administration
          Drug Approval
          Funding Source
          Child: 6-12 years
          Adolescent: 13-18 years
          Male
          Female
      ab: Clinical trials for children with type II diabetes mellitus (T2DM) pose challenges often due to recruitment issues. The variability in the treatment effect for pediatrics with T2DM tends to be much larger than that for adults, therefore, a larger pediatric study is needed to independently detect a similar treatment effect. If leveraging adult information to a pediatric population can be appropriately justified, and scientific rational has been given for the relevancy of the adult information, then Bayesian borrowing methods can aid in reducing the number of patients needed for a pediatric study and therefore increase feasibility and efficiency. We introduce Bayesian borrowing methods to obtain scientifically sound and conclusive results with adequate study power in anti-diabetic products development for children with T2DM. To apply Bayesian borrowing methods, it is important to (1) identify the external data that can be leveraged, (2) pre-specify model parameters, (3) assess operating characteristics, and (4) pre-specify weights and the maximum amount of borrowing needed to achieve a study win. To protect against prior-data conflicts that may exist due to differences in T2DM between adults and pediatrics, we select a mixture prior to take an advantage that they adjust the degree of information borrowed based on the similarity between adults and pediatrics. We applied these methods to an anti-diabetic product. In conclusion, the outcomes of our step-by-step demonstration of the application of Bayesian borrowing methods provides a guide on how to pre-specify parameters and considerations that should be made when planning to implement said methods.
      pubtype: Academic Journal
      doctype:
        equations & formulas
        research
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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