1. The Efficacy and Mechanism Evaluation Programme (EME)
Robert Gray
Research Fellow, EME

2. The EME Programme EME’s position on the translational pathway
What will EME fund?
What won’t EME fund?
Examples of EME funded projects
The EME Progamme vision

3. MRC/NIHR clinical research

4. MRC Can it work? EME Does it work? HTA Is it worth it?
MRC/NIHR clinical research
In simple terms:
MRC
Can it work?
EME
Does it work?
HTA
Is it worth it?

5. MRC Can it work? EME Does it work? HTA Is it worth it?
MRC/NIHR clinical research
In simple terms:
MRC
Can it work?
Discovery science and
“first in man”
EME
Does it work?
Efficacy
HTA
Is it worth it?
Effectiveness

6. The EME Programme What will EME support?
Research to determine proof of clinical efficacy, size of effect, and safety in a well-defined population.
The evaluation of a broad range of interventions which have the potential to maintain health, treat disease or improve recovery.
Hypothesis-driven research based on an efficacy study, to explore the mechanisms of action of interventions, causes of differing responses or disease mechanisms. These studies use data or samples obtained and stored from both treatment and control groups of a clinical study, to arrive at conclusions that would not arise from a simple cohort study.
Proposals that include a series of linked stages with progression to the main clinical evaluation dependent on the outcome of the previous stage(s).

7. The EME Programme What will EME support?
Research to determine proof of clinical efficacy, size of effect, and safety in a well-defined population.
The evaluation of a broad range of interventions which have the potential to maintain health, treat disease or improve recovery.
Hypothesis-driven research based on an efficacy study, to explore the mechanisms of action of interventions, causes of differing responses or disease mechanisms. These studies use data or samples obtained and stored from both treatment and control groups of a clinical study, to arrive at conclusions that would not arise from a simple cohort study.
Proposals that include a series of linked stages with progression to the main clinical evaluation dependent on the outcome of the previous stage(s).

8. The EME Programme

9. The EME Programme What will EME support?
Research to determine proof of clinical efficacy, size of effect, and safety in a well-defined population.
The evaluation of a broad range of interventions which have the potential to maintain health, treat disease or improve recovery.
Hypothesis-driven research based on an efficacy study, to explore the mechanisms of action of interventions, causes of differing responses or disease mechanisms.
include a series of linked stages with progression to the main clinical evaluation dependent on the outcome of the previous stage(s).

10. The EME Programme What will EME support?
Research to determine proof of clinical efficacy, size of effect, and safety in a well-defined population.
The evaluation of a broad range of interventions which have the potential to maintain health, treat disease or improve recovery.
Hypothesis-driven research based on an efficacy study, to explore the mechanisms of action of interventions, causes of differing responses or disease mechanisms.
Studies using novel or infrequently-used study designs which increase the value of a study, by maximising the chances of demonstrating the benefit of an intervention, increasing the knowledge that can be gained.

11. The EME Programme Ways to increase “pull through” of studies MRC
Can it work?
EME
Does it work?
HTA
Is it worth it?

12. The EME Programme Ways to increase “pull through” of studies
Embedded pilot and feasibility studies where the main study would be within the remit of the EME programme.

13. The EME Programme Ways to increase “pull through” of studies
Embedded pilot and feasibility studies where the main study would be within the remit of the EME programme.
The final development of an intervention prior to proceeding to the main clinical evaluation within the same application.

14. The EME Programme Ways to increase “pull through” of studies
Embedded pilot and feasibility studies where the main study would be within the remit of the EME programme.
The final development of an intervention prior to proceeding to the main clinical evaluation within the same application.
Proposals that include a series of linked stages with progression to the main clinical evaluation dependent on the outcome of the previous stage(s).

15. The EME Programme Ways to increase “pull through” of studies
Embedded pilot and feasibility studies where the main study would be within the remit of the EME programme.
The final development of an intervention prior to proceeding to the main clinical evaluation within the same application.
Proposals that include a series of linked stages with progression to the main clinical evaluation dependent on the outcome of the previous stage(s).
Development Funding Scheme

16. The EME Programme What will EME not support?
Large effectiveness studies that test the impact of the introduction of an intervention in the wider NHS.
Hypothesis-generating studies based on sample or data collections from patient cohorts.
Confirmatory studies, equivalence studies, 'confidence in effect' studies or studies of incremental modifications to existing interventions.
Research into areas where the health need is identified primarily outside the UK.
Any research involving animals or animal tissues.

17. The EME Programme What will EME not support?
Large effectiveness studies that test the impact of the introduction of an intervention in the wider NHS.
Hypothesis-generating studies, e.g. biomarker discovery.
Confirmatory studies, equivalence studies, 'confidence in effect' studies or studies of incremental modifications to existing interventions.
Research into areas where the health need is identified primarily outside the UK.
Any research involving animals or animal tissues.

18. The EME Programme What will EME not support?
Large effectiveness studies that test the impact of the introduction of an intervention in the wider NHS.
Hypothesis-generating studies, e.g. biomarker discovery.
Confirmatory studies or minor modifications. Research into areas where the health need is identified primarily outside the UK.
Any research involving animals or animal tissues.

19. The EME Programme What will EME not support?
Large effectiveness studies that test the impact of the introduction of an intervention in the wider NHS.
Hypothesis-generating studies, e.g. biomarker discovery.
Confirmatory studies or minor modifications.
Research into areas where the health need is identified primarily outside the UK.
Any research involving animals or animal tissues.

20. The EME Programme What will EME not support?
Large effectiveness studies that test the impact of the introduction of an intervention in the wider NHS.
Hypothesis-generating studies, e.g. biomarker discovery
Confirmatory studies or minor modifications.
Research into areas where the health need is identified primarily outside the UK.
Any research involving animals or animal tissues.

21. The EME Programme Examples of EME Projects
11/14/25 (Alton) A randomised double-blind placebo controlled Phase 2B clinical trial of repeated application of gene therapy in patients with Cystic Fibrosis.
11/100/50 (Maughan) Molecular selection of therapy in colorectal cancer: a molecularly stratified randomised control trial programme. (FOCUS 4)

22. 11/14/25 (Alton)

23. 11/100/50 (Maughan)
Novel and repurposed drugs for the treatment of inoperable and metastatic colon cancer.
The FOCUS4 Trials Programme
University of Oxford, £3.5million over 7 years
Joint-funded with Cancer research UK
Adaptive platform design
Multi-arm, multi-stage (Phase 2-3)
Molecular stratification
Efficacy study of several drugs against placebo or monitoring

24. 11/100/50 (Maughan)

25. 11/100/50 (Maughan)
FOCUS4 is supported by collaborations with pharmaceutical companies:
Providing study drug
Matched placebo
What next?
The trial design has been published in the Journal of Clinical Oncology
It is being adopted by other large trials, eg. The pharma-sponsored MODUL trial.
Potential for new arms to test promising new drugs
Evidence will support treatment of bowel cancer

26. The EME Programme The programme vision
To continue to fund ambitious projects which include new ways of delivering clinical studies that could:
Maximise the potential gain from the research.

27. The EME Programme The programme vision
To continue to fund ambitious projects which include new ways of delivering clinical studies that could:
Maximise the potential gain from the research.
Reduce the time or cost to evaluate promising new interventions.

28. The EME Programme The programme vision
To continue to fund ambitious projects which include new ways of delivering clinical studies that could:
Maximise the potential gain from the research.
Reduce the time or cost to evaluate promising new interventions.
Increase the breadth of the programmes portfolio in terms of the types of interventions being evaluated and the methodologies being used.

29. The EME Programme The programme vision
To continue to fund ambitious projects which include new ways of delivering clinical studies that could:
Maximise the potential gain from the research.
Reduce the time or cost to evaluate promising new interventions.
Increase the breadth of the programmes portfolio in terms of the types of interventions being evaluated and the methodologies being used.
Increase the number and extent of collaborations, acknowledging that there is a potential for very large and ambitious studies.

30. The EME Programme
To increase representation and encourage applicants to identify appropriate opportunities:
Patient stratification.
Methodological innovation.
Broader diversity of interventions.
Novel use of information enabled by digital technology.

31. The EME Programme Contact details: Useful resources
Direct
Telephone number
+44 (0)

32. *

33. The Efficacy and Mechanism Evaluation Programme (EME): The Application Process
Hints & Tips and Getting it Right

34. Funding Opportunities
Types of
Research
Primary Research;
Clinical efficacy
of interventions
Who defines
the question?
EME Programme
Researcher
Frequency
Per year
3 submission
deadlines
Continuous with
Researcher - led
EME
Commissioned

35. Funding Opportunities
Current commissioned calls
Calls closing
Epilepsy
Improving Safety and Efficacy Through Targeted Drug Delivery
Novel Interventions for Treatment-resistant depression
Ongoing rolling calls (3 dates per year)
Mechanisms of action of health interventions
Anticipated future calls
Interventions to Slow the Progression of Chronic Kidney Disease
Intraoperative Imaging for Oncological Surgery
Virtual and Augmented Reality Technologies for Treating Psychological Disorders
Chronic Obstructive Pulmonary Disease
Dates may change so please check the website

36. Key Stages Overlap checks
Initial Remit & Competitiveness Checks/Prioritisation
Outline/Expression of Interest Funding Board Review
External Review
Full Funding Board Review
Funding Board Membership
Content Experts
PPI
Methodologists

37. Key Considerations Research Question:
Is it the most important question, clearly defined in simple terms, ideally in one sentence?
Has the question already been answered?
Has a similar project already been funded by the funders?
Does it matter to patients/public?
Is it timely and will it make a difference?
Can it be delivered by the NHS/Social Care?

38. Study Design: Is the design optimised to answer the question?
Use existing support, e.g. RDS
Choose the most robust research method and describe it clearly and fully
Ensure your choice of primary outcome, and any secondary outcomes are clear
Statistical input: can your sample size/power calculation be replicated?
Explain the dosage and any side effects of the intervention

39. Multi-disciplinary team:
Do you have the expertise you need?
Ensure the roles are clearly defined and appropriate
Consider the level and range of expertise required
Ensure that PPI is demonstrated at all stages

40. Deliverability: Have you ensured your research is credible?
Recruitment: have you made a convincing case that your recruitment plan is realistic?
Is your timeline manageable?
Does your application provide value for money, and are the costs correctly allocated?

41. Research Dissemination & Impact:
Is there a clear pathway to dissemination and impact?
What are the next steps involved after the project has completed?
How will the research impact current practice?

42. Feedback
Have you followed the feedback, or made a robust defence for why you disagree?
External Reviewers
Board Members

43. Contact details: Useful resources
Direct
Telephone number
+44 (0)

44. Any Questions?