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Table 1 Main project topics and outputs

From: Recent advances in methodology for clinical trials in small populations: the InSPiRe project

Efficient study design

 

Optimal designs for confirmatory studies using decision-theoretic and value-of-information (VOI) approaches

Design of confirmatory studies with stratified populations for personalized medicines

Key publications: [1215]

Key publications: [17, 2023]

Improved analysis and evidence synthesis

Incorporation of pharmacokinetics (PK) and pharmacodynamics (PD) data in early-phase dose-finding studies

Meta-analysis methods for small trials or small numbers of trials

Key publications: [25, 2730]

Key publications: [33, 34, 3638, 40, 41]

Open-source R software: dfpk [26], dfped [29]

Open-source R software: bayesmeta [35], nmaINLA [42]