"Bayesian Methods for Clinical and Observational Studies" was the winning project of the University of Florence Young Independent Researcher call, funded by NextGenerationEU (BayesMeCOS Grant no. B008-P00634).
It all started in 2022 when Alejandra Avalos-Pacheco, Matteo Pedone, and I were at the DiSIA @Unifi. Despite being involved in different projects with different collaborators, we were all directly and indirectly facing the issue of how the traditional development of new drugs and biomarkers following the “one drug, one target disease” strategy is costly and inefficient. Hence, we thought it would have been helpful to research together to create Bayesian Methods for Clinical and Observational Studies that study heterogeneous populations, target several diseases, and test various treatments from the pre-clinical and clinical stages. We then proposed the project intending to help speed up the drug development process while increasing the power of results and biomarker discovery using adaptive randomization. Since then, our research group has grown, first welcoming Norma Alejandra Vergara Lope Gracia to the team and then Giulio Grossi.
The project developed in three directions:
Precision Medicine and adaptive designs: change the randomization ratios using covariate-dependent models; help evaluate the potential variations in global genomic architecture associated with bacterial genomic information.
External Data Integration: integrate data coming from non-concurrent trials.
Causal Inference: provide a way to analyze the study in the presence of intercurrent events, i.e., events that take place after the randomization and may bias the study results in a causal framework.
Ballerini V., Bornkamp B., Mealli F., Wang C., Zhang Y., Mattei A. (2025) Evaluating causal effects on time-to-event outcomes in an RCT in Oncology with treatment discontinuation. Biometrical Journal, 67(7), e70092. doi.org/10.1002/bimj.70092
Ballerini V. (2025) From Association to Causation: The COVID-19 Wildfire Data. In: di Bella, E., Gioia, V., Lagazio, C., Zaccarin, S. (eds) Statistics for Innovation II. SIS 2025. Italian Statistical Society Series on Advances in Statistics. Springer, Cham. doi.org/10.1007/978-3-031-96303-2_18
Ballerini V., Mattei A., Mealli F. (2025) Principal stratum strategy for safety evaluation under principal ignorability. In: Pollice, A., Mariani, P. (eds) Methodological and Applied Statistics and Demography II. SIS 2024. Italian Statistical Society Series on Advances in Statistics. Springer, Cham. doi.org/10.1007/978-3-031-64350-7_33
Ballerini V., Grossi G., (2025) Synthetic control method for clinical trials and precision medicine. In: Pollice, A., Mariani, P. (eds) Methodological and Applied Statistics and Demography IV. SIS 2024. Italian Statistical Society Series on Advances in Statistics. Springer, Cham. doi.org/10.1007/978-3-031-64447-4_27
Ballerini V., Liseo B. (2024) Inferring a population composition from survey data with nonignorable nonresponse: Borrowing information from external sources. Journal of Survey Statistics and Methodology. Advance online publication, pp. 1-20. doi.org/10.1093/jssam/smae041
Avalos-Pacheco A., Ballerini V., Pedone M., Müller P. (2023) Contributed discussion: "Causal Inference Under Mis-Specification: Adjustment Based on the Propensity Score (with Discussion)", Bayesian Analysis, 18(2), 680-682.
Braito L., Ballerini V., Bocci C., Rocco E. (2024) “Integrating traditional and innovative data sources”. Report for the European Commission, SPES Project - Horizon. Available here
Mattei A., Ding P., Ballerini V., Mealli F. (2024) Assessing causal effects in the presence of treatment switching through principal stratification. Bayesian Analysis, Advance Publication, pp. 1-28. doi.org/10.1214/24-BA1425
Niccolai E., Pedone M., Martinelli, I. . . ., Stingo F.C., Mandrioli J., and Amedei A. (2024). Amyotrophic lateral sclerosis stratification: Unveiling patterns with virome, inflammation, and metabolism molecules. Journal of Neurology. 1-16. 10.1007/s00415-024-12348-7
Pedone M., Argiento R., Stingo F. C. (2023) Personalized treatment selection model for survival outcomes. Book of Abstract and Short Papers, 14th Scientific Meeting of the Classification and Data Analysis Group. Salerno, September 11-13, 2023 edited by Carla Rampichini, Michele La Rocca, Pietro Coretto, Giuseppe Giordano, Maria Lucia Parrella. Available here