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: [12–15] | Key publications: [17, 20–23] |
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, 27–30] | Key publications: [33, 34, 36–38, 40, 41] |
Open-source R software: dfpk [26], dfped [29] | Open-source R software: bayesmeta [35], nmaINLA [42] |