Engaging with the MRC Methodology Hubs - NWHTMR

1. Engaging with the MRC Methodology Hubs - NWHTMR Dyfrig Hughes, Bangor University

2. MRC Hubs in Trials Methodology Research (HTMR) Why: To support increased trials activity planned for early and late phase trials What: UK-wide centres of excellence - £16m Linking innovation in trials methodology and conduct with trials activity Provide training programmes Provide advice to wider/national trials community Wide ranging methods research expertise across a range of research issues and therapeutic areas Networked together

4. Organogram

5. Development of methodological research proposals Training opportunities – post-doctoral, PhD, MSc, ACF Training and research workshops Advice on methodological issues

6. Methods Medical statistics Health economics Qualitative research methods

7. Themes Early phase trial design and analysis Later phase trial design and analysis Patients’ perspectives

8. Clinical areas Drug safety Medicines for children Epilepsy Cancer Infectious diseases

9. Example PhD studentship Clinical trial simulation and economic evaluation during early-phase drug development Purpose – to provide estimates of cost-effectiveness during the early stages of drug development

10. Methods Integration of: traditional mathematical models that predict drug response over time, based on pharmacokinetic-pharmacodynamic models with economic model to assess the cost-effectiveness of the treatment

11. Applications Value of information analysis (to assess where further research is required); Value-based pricing (setting the price of the medicine to a level which results in a cost-effectiveness ratio that is acceptable by NICE); Assessment of the impact of patient subgroups (e.g. age, genetic factors) on cost-effectiveness; Impact of adherence (how well patients take their medications) on cost-effectiveness

12. Proposed workshop topics Developing consensus around core outcome sets User engagement in clinical trials Processes in recruitment to trials Risk stratification to inform trial design Medication adherence Issues of trial conduct Economic evaluation in trials Biomarker assessment

13. Communication Website: www.nwhtmr.org.uk Sign up for email alerts

15. Launch - date for your diary Tuesday 29th September Foresight Centre, University of Liverpool Talks on methodological issues Opportunity for networking Register by e-mailing: nwhtmr@liv.ac.uk

16. Case study

17. The problem Suggestion that the effectiveness of drugs differ according to endpoint measured – time to treatment failure versus QALYs Evidence that there is little association between EQ-5D and health state utility measured by time trade off and standard gamble methods Dimensions of EQ-5D unlikely to be affected unless the person is actually having a seizure

18. How might you approach this problem? What would you expect from involving a Hub?

19. Hub involvement Project development group convened Possible approaches for a epilepsy-specific utility instrument discussed Invited Prof John Brazier, Sheffield University (not a Hub member) to lead on a proposal

20. The outcome “Health economic evaluations in epilepsy: Developing a preference-based outcome measure” Funded by Epilepsy Research UK ..to develop a new preference-based epilepsy-specific outcome measure that will enable appropriate health economic analyses of treatments and other interventions for epilepsy

21. Group work What do you want from the methods Hubs? How would you like to engage with Hubs?