Thomas Jaki1. 1. Medical and Pharmaceutical Statistics Research Unit, Department of Mathematics and Statistics, Lancaster University, Lancaster, UK. jaki.thomas@gmail.com
Abstract
BACKGROUND: In recent years, the success rate of confirmatory studies has been poor resulting in more emphasis on the conduct of exploratory studies. As one possibility to improve decision-making during the early stages of development, adaptive and Bayesian methods have been recommended. PURPOSE: To investigate the current practice in designing early-phase studies in UK public sector research institutions and the use of adaptive and Bayesian methods in particular and to determine factors that hinder the penetration of methodological advances into practice. METHODS: A questionnaire was sent to all UK clinical trials units (CTUs) to gauge their involvement in early-phase studies and to learn about the designs used in these studies. Follow-up visits to units conducting early-phase studies with round-table discussions around the methods used and the obstacles faced when using adaptive methods were undertaken. RESULTS: More than half of the CTUs are involved in early-phase studies, but conservatism in the methods used in these studies is present. Reasons for novel methodology not being used include a lack of expertise, incompatible funding and unit structure, and a lack of software. LIMITATIONS: Information is collected from UK CTUs, which undertake a large portion (but not all) publicly funded trials. CONCLUSIONS: The use of adaptive and Bayesian methods for early-phase clinical studies in the UK public sector is at present limited. Various different initiatives aim to support and facilitate the use of these methods, however, so that an increased use of these methods can be anticipated in the future.
BACKGROUND: In recent years, the success rate of confirmatory studies has been poor resulting in more emphasis on the conduct of exploratory studies. As one possibility to improve decision-making during the early stages of development, adaptive and Bayesian methods have been recommended. PURPOSE: To investigate the current practice in designing early-phase studies in UK public sector research institutions and the use of adaptive and Bayesian methods in particular and to determine factors that hinder the penetration of methodological advances into practice. METHODS: A questionnaire was sent to all UK clinical trials units (CTUs) to gauge their involvement in early-phase studies and to learn about the designs used in these studies. Follow-up visits to units conducting early-phase studies with round-table discussions around the methods used and the obstacles faced when using adaptive methods were undertaken. RESULTS: More than half of the CTUs are involved in early-phase studies, but conservatism in the methods used in these studies is present. Reasons for novel methodology not being used include a lack of expertise, incompatible funding and unit structure, and a lack of software. LIMITATIONS: Information is collected from UK CTUs, which undertake a large portion (but not all) publicly funded trials. CONCLUSIONS: The use of adaptive and Bayesian methods for early-phase clinical studies in the UK public sector is at present limited. Various different initiatives aim to support and facilitate the use of these methods, however, so that an increased use of these methods can be anticipated in the future.
Authors: Timothy C Guetterman; Michael D Fetters; Laurie J Legocki; Samkeliso Mawocha; William G Barsan; Roger J Lewis; Donald A Berry; William J Meurer Journal: Clin Res Regul Aff Date: 2015-09-18
Authors: Cody Chiuzan; Jonathan Shtaynberger; Gulam A Manji; Jimmy K Duong; Gary K Schwartz; Anastasia Ivanova; Shing M Lee Journal: J Biopharm Stat Date: 2017-02-06 Impact factor: 1.051