Visualizing the target estimand in comparative effectiveness studies with multiple treatments

Autor: Gabrielle Simoneau, Marian Mitroiu, Thomas PA Debray, Wei Wei, Stan RW Wijn, Joana Caldas Magalhaes, Justin Bohn, Changyu Shen, Fabio Pellegrini, Carl de Moor

Publikováno v: Journal of Comparative Effectiveness Research, Vol 13, Iss 2 (2024)

Aim: Comparative effectiveness research using real-world data often involves pairwise propensity score matching to adjust for confounding bias. We show that corresponding treatment effect estimates may have limited external validity, and propose tw

Externí odkaz: https://doaj.org/article/ab90d666593b46258e60422b08a83b78

Estimation of treatment effects in short-term depression studies. An evaluation based on the ICH E9(R1) estimands framework

Autor: Marian Mitroiu, Steven Teerenstra, Katrien Oude Rengerink, Frank Pétavy, Kit C. B. Roes

Publikováno v: Pharmaceutical Statistics, 21, 5, pp. 1037-1057
Pharmaceutical Statistics, 21, 1037-1057

Contains fulltext : 283289.pdf (Publisher’s version ) (Open Access) Estimands aim to incorporate intercurrent events in design, data collection and estimation of treatment effects in clinical trials. Our aim was to understand what estimands may cor

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1f479f79ce29caf1224d79dceda7199a
https://doi.org/10.1002/pst.2214

Letter to the editor regarding 'Two-year results of a double-blind multicenter randomized controlled non-inferiority trial of polyetheretherketone (PEEK) versus silicon nitride spinal fusion cages in patients with symptomatic degenerative lumbar disc disorders'

Autor: Mark P. Arts, Steven M. van Gaalen, Arthur de Gast, Marian Mitroiu, F. Cumhur Oner, Kit C.B. Roes, Roel F. M. R. Kersten

Publikováno v: J Spine Surg
Journal of Spine Surgery, 7, 249-251
Journal of Spine Surgery, 7, 2, pp. 249-251

BACKGROUND: During lumbar spinal fusion, spacer cages are implanted to provide vertebral stability, restore sagittal alignment, and maintain disc and foraminal height. Polyetheretherketone (PEEK) is commonly used by most spine surgeons. Silicon nitri

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::587e66f3faa2c77700ebdbebb9bac5a7
https://pubmed.ncbi.nlm.nih.gov/33102889

A narrative review of estimands in drug development and regulatory evaluation: old wine in new barrels?

Autor: K. Oude Rengerink, Marian Mitroiu, Frank Pétavy, Kit C.B. Roes, Steven Teerenstra

Publikováno v: Trials, 21, 1
Trials, Vol 21, Iss 1, Pp 1-14 (2020)
Trials, 21
Trials

Background An estimand defines the target of estimation for a clinical trial through specification of the treatment, target population, variable, population-level summary and of the strategies for intercurrent events. A carefully defined estimand ali

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e6f6f959ee9a506263ea218eaa9019a4
https://hdl.handle.net/2066/225029

Applicability and added value of novel methods to improve drug development in rare diseases

Autor: Robin Ristl, Caridad Pontes, Stavros Nikolakopoulos, Aranzazu Sancho, Konstantinos Pateras, Roser Vives, Juan Manuel Fontanet, Ferran Torres, Martin Posch, Katrien Oude Rengerink, Stella Pesiou, Kit C.B. Roes, Marian Mitroiu, Spineli Loukia, Johanna H. van der Lee, Gerd K. Rosenkranz, Armin Koch, Susanne Urach

Publikováno v: Orphanet journal of rare diseases, 13(1). BioMed Central
Orphanet Journal of Rare Diseases
Orphanet Journal of Rare Diseases, Vol 13, Iss 1, Pp 1-16 (2018)
Orphanet Journal of Rare Diseases, 13(1), 200. BioMed Central

Background The ASTERIX project developed a number of novel methods suited to study small populations. The objective of this exercise was to evaluate the applicability and added value of novel methods to improve drug development in small populations,

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4263f98e6c0dc4e0ff5a2e0e6885d61f
https://doi.org/10.1186/s13023-018-0925-0