IGNITE your.... medical funding opportunities Presenter - Dr Lisa Douet, Senior Research Fellow, and Ellie Monks, EME Programme Manager will present about “Research Funding: The Efficacy and Mechanism Evaluation Programme” The EME Programme funds clinical efficacy studies. The studies it supports usually test if an intervention works as expected, in a well-defined population or group of patients. The Programmealso also provides an opportunity to use clinical studies to understand disease or treatment mechanisms, which may in turn lead to improvements in health and patient care.

1. Research Funding: a view
from the Efficacy and
Mechanism Evaluation (EME)
Programme
Dr Lisa Douet, Senior Research Fellow and
Ellie Monks, Programme Manager
11/12/2013

2. Efficacy and Mechanism Evaluation Programme
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. Efficacy and Mechanism Evaluation Programme
Managed Translational Pathway

4. Efficacy and Mechanism Evaluation Programme
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

5. Efficacy and Mechanism Evaluation Programme
Overview of EME
•
Two funding streams
MRC funded, largely researcher led, but with an eye to MRC strategy and
priorities
NIHR funded, for commissioned research into priority areas
•
Same broad remit
To support clinical trials and studies which:
• evaluate clinical efficacy of healthcare interventions (drugs,
technology, diagnostics, procedures)
• may add significantly to our understanding of biological or
behavioural mechanisms and processes;
• explore new scientific or clinical principles.

6. Efficacy and Mechanism Evaluation Programme
Mechanisms of working: researcher-led work stream
• Standard mode (two stage application)
On going call with 3 closing dates a year
Preliminary application submission deadline to funding decision around 8 months
• Fast track (single stage application)
Reduces time to funding decision by ~ 3 months
Allowed in exceptional circumstances where reducing the timescale will:
• provide significant benefit,
• allow exploitation of a narrow window of opportunity

7. Efficacy and Mechanism Evaluation Programme
Mechanisms of working: 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.

8. Efficacy and Mechanism Evaluation Programme
Current EME commissioned calls
•
•
Current calls – close 3 February 2014
• non-respiratory sleep disorders
• autism spectrum disorders
• active implantable medical devices
• mechanisms of action of bariatric surgery
• microbial therapies to treat gastrointestinal disease
• passive and bioactive implantable devices (close 3 June 2014)
On-going call opening 2013 – Mechanisms of action of health interventions
For further details about these calls, including the detailed commissioning
brief please see the EME website
www.eme.ac.uk/funding/Commissioning.asp

9. Efficacy and Mechanism Evaluation Programme
Future EME commissioned calls
•
Future calls
• Opening February 2014 – self-harm and suicidal behaviours
•
Opening June 2014 – bowel control and faecal incontinence in adults
and wound healing
•
Opening October 2014 – endoscopic and laparoscopic optical imaging
Please check the EME website for further details on each call and for up to
date information on call timings
www.eme.ac.uk/funding/Commissioning.asp

10. Efficacy and Mechanism Evaluation Programme
Funded studies
• Since April 2008 the EME Board has approved funding for 57
researcher led studies (subject to contract)
• Since January 2012 the EME Board has approved funding for 20
commissioned studies (subject to contract)
• The funding projects have received ranges from £0.12 to £3.1
million, and durations range from 18 to 66 months
• Projects cover a broad range of disease areas and interventions

11. Efficacy and Mechanism Evaluation Programme
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 2009 (£818,263)
Aims to determine if remote
ischaemic preconditioning
improves renal function after
transplantation

12. Efficacy and Mechanism Evaluation Programme
Top 10 reasons for rejection
Reason for rejection
Trial design issues
Poorly written application/ case for trial needed to be
stronger
Sample size/power calculation/stats issues
Justification of end point measurements
Justification of costs of required
Lack preliminary data
Recruitment issues (including drop rates and
recruitment targets)
Issues relating to the selection of patients/population
chosen
Lack of inclusion of CTU/statistician on study team
Drug dose/side effects
Rank Order
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5
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7
8
9
10

13. Efficacy and Mechanism Evaluation Programme
Thank you
Any questions?