1. The Efficacy and Mechanism Evaluation (EME) Programme Susan Solomons Senior Programme Manager, EME Programme RDS West Midlands 9th October 2013
23/11/2018

2. Aim:
Support excellent clinical science with an ultimate view to improving health or patient care
Dual Approach:
“Science driven” – examine the efficacy of a technology intervention (i.e. pharmaceutical, diagnostic test, surgical or psychological therapies, or public health measures) and/or explore its mechanisms of action
Clear patient focussed outcomes in areas of need for health care research

3. Programme Remit We support studies in patients which seek to:
evaluate the clinical efficacy of interventions (where proof of concept in humans has already been achieved);
add significantly to our understanding of biological or behavioural mechanisms and processes;
explore new scientific or clinical principles;
Within the main clinical intervention study, if relevant to the EME remit, we will support:
the development or testing of new methodologies;
studies that use validated surrogate markers as indicators of health outcome;
laboratory based, or similar embedded studies
pilot and feasibility studies where the later main study would be within the remit of the EME programme

4. Programme Remit
The EME Programme will not support:
confirmatory studies or trials of incremental modifications and refinements to existing medical interventions;
proof-of-concept, proof-of-mechanism in humans, nor 'confidence in effect' studies;
research into 'global health', where 'global health' can be defined as 'areas where the health need is identified in developing countries (i.e. including diseases of developing countries), or where the health need does not yet exist in the UK but might in the future and the problem can be best addressed in developing countries;
research involving animals.

5. Translating science into better health
In simple terms:
MRC: can it work?
EME: does it work?
HTA: is it worth it?
There is opportunity for pull through of research, but also for push back (reverse translation) when necessary

6. Types of applications:
Researcher led, commissioned, themed calls and fast track
The researcher-led workstream is an on-going funding opportunity for questions in a broad range of disease areas which are identified by the researcher.
Applications to the researcher-led workstream are required to follow Research Council eligibility criteria.
Commercial collaborations are welcome in applications, however, the lead applicant must be from academia or the NHS.
The funding projects range from £120,000 to £3.1 million and the durations range from 18 to 66 months

7. Commissioned work stream
Applications must have a strong collaborative approach and include significant contributions from at least two of the following partners: industry, academia and the NHS.
Proposals should focus on a clinical study which is within the remit of the EME programme but may be staged and include earlier stages, such as:
the limited steps needed to progress the development of an intervention to a stage suitable for use in an accredited clinical service
prospective clinical work or retrospective research utilising existing clinical samples or data to inform the main study
pilot or feasibility studies
As a rough guide it is expected that these early stages will be complete within the first 18 months of the project and contribute approximately 25% to the total cost of the project.
Research areas suggested are from a broad range of sources
Calls pre advertised around 9-12 months in advance with commissioning brief published at the start of an individual call

8. Anticipated Commissioned call opening dates:
October 2013
Non-respiratory sleep disorders
Active implantable medical devices
Autism spectrum disorders
Mechanisms of action of bariatric surgery
Mechanisms of action of health interventions
February 2014
Self-harm and suicidal behaviours
Passive and bioactive implantable medical devices
June 2014
Bowel control and faecal incontinence in adults
Wound healing
October 2014
Endoscopic and laparoscopic optical imaging

9. The Application Journey (2 stage):
Pre-Board Processes:
Remit and Competitiveness meeting
Prioritisation Panel for Commissioned applications
Internal overlap, finance and IP checks
External peer review
Applicants will be asked to respond to questions raised.
Funding Board:
Membership: clinicians, methodologists, statisticians, PPI and industry
Assessment: scientific quality of the proposals, feasibility/deliverability and value for money.
Outcomes for full applications: fund, fund with changes, resubmission, reject.

10. Examples of areas of weakness identified by the Board:
Sample size/power calculation
Recruitment
Lack of preliminary data
Study design
Choice of patients/population: inclusion/exclusion
Endpoints
Randomisation
Standarisation of intervention across centres
Dosage
Study team
Justification of costs
Lack of embedded PPI involvement
Unclear relationship with industry
Unclear how mechanistic component fits into the study

11. Examples of Funded studies Researcher-led
A randomised double-blind placebo controlled Phase 2B clinical trial of repeated application of gene therapy in patients with Cystic Fibrosis.
Professor Eric Alton (Imperial College, London) 24 months from 1st March 2012 (£3,073,905).
Aims assess the clinical benefit of repeated doses of gene therapy (pGM169/GL67A) administered to the lungs of patients with CF over a period of 48 weeks
Remote ischaemic preconditioning in renal transplantation
Professor Raymond MacAllister (University College London) 42 months from 1st July (£818,263)
Aims to determine if remote ischaemic preconditioning improves renal function after transplantation

12. Examples of funded studies (commissioned)
Developing a novel, biopsy-based diagnostic for patient stratification: A Randomised, open labelled study in anti-TNFalpha inadequate responders to investigate the mechanisms for Response, Resistance to Rituximab versus Tocilizumab in Rheumatoid Arthritis patients
Professor Costantino Pitzalis (University of London) 33 months from 1st December 2012 (£1,002,635)
Aims to establish whether a sample of the joint lining can predict which patients will respond to treatment.
Enhanced Neoplasia Detection and Cancer Prevention in Chronic Colitis (ENDCaP-C)
Dr Glenn Matthews (University of Birmingham) 24 months from 1st February 2013 (£1,552,675).
Aims to develop a diagnostic test to be used alongside colonoscopy, and so improve the detection of tumours at an
early stage.

13. Key Points and resources:
Have good preliminary data to justify your proposal
Gather a team with the relevant scientific and project management skills
Ensure you have an appropriate study design and strong statistical support
Be realistic and accurate when costing the study
Talk your proposed study through with EME before submitting an application
Key Resources:
EME website:
EME video/podcast:
EME secretariat: contact us at or