10/04/2023

Research Funding: a view from the Efficacy and Mechanism Evaluation (EME) Programme

Dr Lisa Douet, Senior Research Fellow and

Ellie Monks, Programme Manager

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

Efficacy and Mechanism Evaluation Programme

Managed Translational Pathway

Efficacy and Mechanism Evaluation Programme

• 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

Translating science into better health

Efficacy and Mechanism Evaluation Programme

• 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.

Overview of EME

Efficacy and Mechanism Evaluation Programme

• Standard mode (two stage application)On going call with 3 closing dates a yearPreliminary application submission deadline to funding decision around 8 months

• Fast track (single stage application)Reduces time to funding decision by ~ 3 monthsAllowed in exceptional circumstances where reducing the timescale will:

• provide significant benefit, • allow exploitation of a narrow window of opportunity

Mechanisms of working: researcher-led work stream

Efficacy and Mechanism Evaluation Programme

• 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.

Mechanisms of working: commissioned work stream

Efficacy and Mechanism Evaluation Programme

• 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

Current EME commissioned calls

Efficacy and Mechanism Evaluation Programme

• 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

Future EME commissioned calls

Efficacy and Mechanism Evaluation Programme

• 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

Funded studies

Efficacy and Mechanism Evaluation Programme

Examples of Funded studies Researcher-led

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

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

Efficacy and Mechanism Evaluation Programme

Top 10 reasons for rejection

Reason for rejection Rank Order

Trial design issues 1Poorly written application/ case for trial needed to be stronger 2Sample size/power calculation/stats issues 3Justification of end point measurements 4Justification of costs of required 5Lack preliminary data 6Recruitment issues (including drop rates and recruitment targets) 7Issues relating to the selection of patients/population chosen 8

Lack of inclusion of CTU/statistician on study team 9

Drug dose/side effects 10

Efficacy and Mechanism Evaluation Programme

Thank you

Any questions?