A Practical Adaptive & Novel Designs and Analysis (PANDA) Toolkit

This project aims to create an online platform to educate researchers across disciplines on the practical application of adaptive designs in randomised trials. This “practical adaptive designs toolkit” will provide self-paced remote learning covering a wide range of practical aspects. The toolkit will be easily accessible to a broad audience in the conduct of clinical trials including statisticians and non-statisticians (e.g. clinical investigators, proposal developers and trial managers). We hope to facilitate the bridging of the practical knowledge gap and to improve the appropriate uptake of adaptive clinical trial designs. The platform will also offer opportunities for methodologists to showcase practical application of their newly developed adaptive methods and related issues. This may shorten the time between the development of new adaptive methods and their uptake in practice.

Researchers across disciplines, research funders and regulators acknowledge the need to streamline the conduct of randomised trials to address research questions as efficiently as possible avoiding unnecessary use of resources. The appropriate use of adaptive designs can help achieve this objective. The use of adaptive designs is gradually increasing1–3 and they are frequently considered at the design stage4. Nevertheless, there are known obstacles to the use of adaptive designs. Practical education on adaptive designs across disciplines encompassing awareness of potential benefits and pitfalls is a leading recommendation to address some of the obstacles3,5,6.We will therefore develop the Practical Adaptive & Novel Design and Analysis (PANDA) resource which aims to aid researchers (both statisticians and non-statisticians) get to grips with:

  • What adaptive trials are and adaptations that can be considered in randomised trials;
  • The scenarios in which they may be beneficial;
  • Practical considerations and advice for the design, conduct and analysis and reporting of adaptive design trials. This will encompass case studies to illustrate practical application;
  • Available statistical implementation resources.

This project is a collaborative initiative involving the University of Sheffield, Cardiff University, Lancaster University, University College London, and the University of Western Australia.

The project is funded by the NIHR CTU Support Funding to support efficient/innovative delivery of NIHR research focusing on “developing skills for trials staff”.

For more details or queries, please contact the lead investigator Dr Munya Dimairo.

Project Team

Dr Munya Dimairo Sheffield Clinical Trials Research Unit, University of Sheffield
Dr Philip Pallmann Centre for Trials Research, Cardiff University
Prof Thomas Jaki Lancaster University
Mike Bradburn Sheffield Clinical Trials Research Unit, University of Sheffield
Dr Graham Wheeler Cancer Trials Centre, University College London
Laura Flight Health Economics and Decision Sciences, University of Sheffield
Prof Cindy Cooper Sheffield Clinical Trials Research Unit, University of Sheffield
Dr Julie Marsh Telethon Kids Institute, University of Western Australia

References

  1. Hatfield I, Allison A, Flight L, Julious SA, Dimairo M. Adaptive designs undertaken in clinical research: a review of registered clinical trials. Trials. 2016;17(1):150. doi:10.1186/s13063-016-1273-9
  2. Bothwell LE, Avorn J, Khan NF, Kesselheim AS. Adaptive design clinical trials: a review of the literature and ClinicalTrials.gov. BMJ Open. 2018;8(2):e018320. doi:10.1136/bmjopen-2017-018320
  3. Hartford A, Thomann M, Chen X, et al. Adaptive Designs: Results of 2016 Survey on Perception and Use. Ther Innov Regul Sci. December 2018:216847901880771. doi:10.1177/2168479018807715
  4. Dimairo M, Julious SA, Todd S, Nicholl JP, Boote J. Cross-sector surveys assessing perceptions of key stakeholders towards barriers, concerns and facilitators to the appropriate use of adaptive designs in confirmatory trials. Trials. 2015;16(1):585. doi:10.1186/s13063-015-1119-x
  5. Dimairo M, Boote J, Julious SA, Nicholl JP, Todd S. Missing steps in a staircase: a qualitative study of the perspectives of key stakeholders on the use of adaptive designs in confirmatory trials. Trials. 2015;16(1):430. doi:10.1186/s13063-015-0958-9
  6. Love SB, Brown S, Weir CJ, et al. Embracing model-based designs for dose-finding trials. Br J Cancer. 2017;117(3):332-339. doi:10.1038/bjc.2017.186