Title: Improving treatment for patients by design
Funder: Department of Health and Social Care

Abstract: The development of new medicinal products and health technologies is a time consuming and very expensive process. Among the largest contributors to both time and cost are confirmatory (Phase III) clinical trials that often involve thousands of patients with follow-up period frequently lasting years. In recent years, however, around 50% of confirmatory clinical trials have been unsuccessful resulting in a large number of participants in these trials being exposed to an ineffective or even harmful treatment while at the same time costing substantial amounts of money. Even among successfully registered treatments, however, dose changes after registration are common. CHAR(13) + CHAR(10)The challenge is therefore to seek improvements throughout the drug development process to eliminate ineffective or harmful treatments quickly and provide better estimates of the optimal dose for treatments which in turn will reduce the number of unsuccessful confirmatory clinical trials and allow for more efficient development of medicinal products and health technologies overall.CHAR(13) + CHAR(10)While there exist an obvious need for method development, the success of any such undertaking will depend heavily on training end users in these methods. As a consequence, this proposal is for the development of novel methods for clinical trials that integrates application of and training in the methods within its programme. More specific aims are to: develop relevant and efficient methods for designing and analysing clinical trials; better understand the drivers and hurdles for patients to take part in studies utilizing novel designs; implement the developed methods in publicly funded clinical trials; familiarise applied researchers with novel methods for designing and analysing clinical trials; publish the results in leading scientific journals and to present them at national and international conferences. The project will investigate adaptive and Bayesian methods to allow accurate decision making that utilizes previously available information in a structured manner. The project will focus on four different work-streams: Bayesian adaptive dose finding trials with multiple objectives Adaptive designs in small populations Adaptive equivalence studies Estimation after adaptive trials These areas build upon work previously being undertaken and are motivated by work with clinical trials units and the MRC North-West Hub for Trials Methodology Research and have not been adequately addressed to date.

Lay abstract: Developing new medicines and health technologies is lengthy and expensive. Many development programmes do, however, fail late in the process exposing large numbers of participants to ineffective/harmful treatments while costing substantial amounts of money. The success of any new methods developed depend heavily on training users in these methods. Consequently, this proposal is for the development of novel methods for clinical trials and integrates training in these methods within its programme. Specific aims are to: develop efficient methods for designing and analysing clinical trials; better understand the drivers and hurdles for patients to take part in studies utilizing novel designs; implement methods in clinical studies; familiarise applied researchers with these methods; publish results in leading journals.

Research activity codes: 8.4 RESEARCH DESIGN AND METHODOLOGIES (HEALTH SERVICES)
Health categories: GENERIC HEALTH RELEVANCE