1. HTA Efficient Study Designs Peter Davidson Head of HTA at NETSCC

2. Aim Reduce the costs, and/or duration of HTA Provide robust evidence to the NHS Value for money Fund more research from a limited budget Programme efficiency –Adding value and avoiding waste

3. Adding value in research

4. Purpose and remit –To deliver information about the effectiveness, costs and broader impact of healthcare treatments and tests for those who plan, provide or receive care in the NHS Health technologies include –All interventions used to promote health, prevent or treat disease, improve rehabilitation or long-term care including drugs, devices, procedures, settings of care and screening Health Technology Assessment Programme Evaluation, Trials and Studies Coordinating Centre

5. HTA Programme workstreams Types of Research Specific technology assessment Primary research Feasibility & pilot Evidence synthesis NICE Researcher Primary research Evidence synthesis Theme from HTA Programme Question from researcher Continuous with 3 closing dates Primary research Feasibility & pilot Evidence synthesis HTA Programme3 calls 1-2 calls Direct referral to On-contract teams Who defines the question? Frequency Per year Commissioned Evaluation, Trials and Studies Coordinating Centre NICE Guidance Researcher-led Clinical Evaluation and Trials Clinical Evaluation and Trials Themed Calls Efficient study designs call

6. –Randomised controlled –Multidisciplinary and multi-centre –Clinical and cost-effectiveness –Pragmatic and externally valid –Median number of patients = 700 (Range of 15 to 75,000) –Average duration ~4 years –Costs ~£1.5million Typical HTA trial: Evaluation, Trials and Studies Coordinating Centre

7. –Randomised controlled ? –Multidisciplinary and multi-centre –Clinical and cost-effectiveness ? –Pragmatic and externally valid –Median number of patients = 700 (Range of 15 to 75,000) –Average duration ~4 years ? –Costs ~£1.5million ? Typical HTA trial: Evaluation, Trials and Studies Coordinating Centre Works well – change not needed

8. Statistical or operational efficiency: study design and conduct –Encouraging use of different study designs –Simpler studies that can be rapidly started and conducted –Efficiencies such as routinely collected data when possible –Anticipating difficulties with operational delivery –Streamlining trial support What’s different about efficient studies? Evaluation, Trials and Studies Coordinating Centre

9. –Full or partial use of routinely collected data, registries, or electronic health records; –Exploitation of existing cohorts; –Adaptive designs for trials; –Factorial or other efficient designs; –Non-randomised evaluations (retrospective or prospective) where bias can be reduced to acceptable levels; –Reanalysis of existing datasets (eg. from other trials or IPD). Examples of different study designs Evaluation, Trials and Studies Coordinating Centre

10. Examples of operational efficiency –Simple randomised trials allowing rapid recruitment and follow-up; –Other innovations to promote rapid or more efficient recruitment; –Simplified design and/or follow up by focusing on a limited number of the most important outcomes; –Health economics not always needed –Where feasibility has already been demonstrated or where pilot studies have already been conducted or are underway; –Involving teams experienced in a topic

11. Carefully consider and minimise issues such as : Are there any barriers to timely implementation, such as complex pharmacy issues. Whether you are testing a novel therapy still needing development; Where high excess treatment costs are likely, we will need expect support from commissioners. Proposals for international studies may be complex and outside of remit for this particular call – contact the programme if you are considering this type of application. Operational issues in efficient studies Evaluation, Trials and Studies Coordinating Centre

12. Methodology The MRC methodology panel will offer projects short- listed from expressions of interest the chance to submit proposals for bolt-on methodology research. Applicants could indicate in the EOI if they might apply for methodology alongside the HTA research

13. Focussing on study design to justify the application Not justifying the research question Not giving attention to potential innovative design features Not demonstrating the benefits of efficiency Common pitfalls Evaluation, Trials and Studies Coordinating Centre

14. Making your research question stand out as important to the NHS to a multi-disciplinary committee Clearly define the intervention. Describe the current evidence and the gap. The potential to change practice. Ensure your primary outcomes matter to patients. Beware topic fatigue Think about its place in the portfolio Top tips Evaluation, Trials and Studies Coordinating Centre

15. Getting the methods right Clearly describe the methods inc PICO. Justify your study design carefully Pay attention to detail even in the expression of interest Anticipate queries and criticisms from committee members –Design-related concerns esp about bias Top tips Evaluation, Trials and Studies Coordinating Centre

16. Visit our calls for research at www.nets.nihr.ac.uk/funding Any questions? Evaluation, Trials and Studies Coordinating Centre