Functional Data Models for Dose Finding Studies

Abstract Number:

3420 

Submission Type:

Contributed Abstract 

Contributed Abstract Type:

Paper 

Participants:

Bahaeddine Taoufik (1), Justin Petrovich (2), Sungwook Kim (1)

Institutions:

(1) Saint Joseph's University, N/A, (2) Saint Vincent College, N/A

Co-Author(s):

Justin Petrovich  
Saint Vincent College
Sungwook Kim  
Saint Joseph's University

First Author:

Bahaeddine Taoufik  
Saint Joseph's University

Presenting Author:

Bahaeddine Taoufik  
Saint Joseph's University

Abstract Text:

The primary aim of dose-finding studies is to pinpoint the optimal dose level based on subjects' responses, focusing on 'Efficacy' and 'Toxicity.' The optimal dose is identified at the point of maximum probability, where efficacy is significant without toxicity. While some studies use Emax, quadratic, or non-linear models, they are unsuitable for non-monotonic curves. Cripper & Orsini (2016) proposed regression splines, but they may not sufficiently describe reasonable dose-response distributions. This paper introduces functional data models for dose-finding studies, presenting a novel approach by applying them to meta-analysis data. We focus on three outcome probabilities: P(Efficacy), P(Toxicity), and P(Efficacy but No Toxicity), guided by monotonic and unimodal assumptions. Our functional data models estimate these probability distributions and introduce adjusted confidence intervals. Finally, we apply these models to analyze data on alcohol consumption and colorectal cancer.

Keywords:

Functional Data|Dose Study|Meta-Analysis Data|Efficacy and Toxicity|Functional Anova|Smoothing methods

Sponsors:

Section on Nonparametric Statistics

Tracks:

Statistical Methods for Functional Data

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