Efficacy & Mechanism Evaluation Efficacy & Mechanism Evaluation

What does the funding stream focus on?
  • Trials of the efficacy of treatments in controlled settings, sometimes using proxy or intermediate measures of health outcome.

EME Key Information

Director: Professor John Simpson

Aim: To evaluate interventions with potential to make a step-change in promoting health, treating disease and improving care. EME supports clinical trials and other robustly designed studies that test the efficacy of interventions and the mechanisms of diseases and treatments. EME is a partnership between the MRC and NIHR.

Funding: Commissioned and researcher-led, no fixed limits on duration or funding.

Process: Two-stage process.

Summary of Panel Observation Tips

  • EME funds research that is earlier in the development pathway than HTA
  • The EME committee will look for detailed recruitment plans as few trials recruit > 50% of eligible patients
  • Designs involving routinely collected digital data and/or novel methodologies are strongly encouraged

Key panel observations:

  • Sample size: This includes issues such as sample size calculation justification, consideration of dropout rates, and ensuring the sample size is adequately powered.
  • Clarity and presentation: Applications should have a clear research question, benefit, and impact. They should also be well-written and easy to read.
  • PPI (Patient and Public Involvement): The level and detail of PPI involvement in the study design and execution are often scrutinized.
  • Precision medicine: When applicable, applications should clearly define how precision medicine principles are being applied.
  • Platform trials: For platform trials, common issues include a lack of clarity regarding the interventions and a lack of succession planning for the platform design.

Other issues that arise include the clarity of the primary outcome, the appropriateness of the control group, the robustness of the proof of concept, and the reliance on self-report measures.

Funding Deadlines

Call Webinar Outline submission deadline Outcome date Full submission deadline Outcome date

Swiss-UK investigator-led randomised controlled clinical trials in areas of unmet clinical need

  9 Sept 25      

Novel non-pharmacological approaches for diagnosis and treatment of Attention Deficit Hyperactivity Disorder (ADHD)

  5 Aug 25 Nov 25 Jan 26 March 26

Prevention and/or management of lymphoedema following Breast Cancer Resection

  5 Aug 25 Nov 25 Jan 26 March 26

NIHR NICE Rolling Funding Opportunity (EME Programme)

  8 April 25 July 25 Sept 25 Nov 25

NIHR James Lind Alliance Priority Setting Partnerships Rolling Funding Opportunity (EME Programme)

  8 April 25 July 25 Sept 25 Nov 25

EME Programme Researcher-led

  8 April 25 July 25 Sept 25 Nov 25

Decarbonising the health and social care system

  26 March 25 June 25 August 25 November 25

Australia-UK platform studies in areas of unmet clinical need

  23 July 25 October 25 27 November 25 Feb 26

EME Example

A randomised placebo-controlled study examining the role of anti-IgE in severe recalcitrant paediatric atopic eczema

Funding: £479k, 2014-2018

Aim: To determine if the drug anti-IgE (omalizumab) can reduce levels of IgE in children with eczema and improve their symptoms.

Other EME Examples

  • Gene therapy for choroideremia - a Phase II clinical trial
  • Paclitaxel assisted balloon Angioplasty of Venous stenosis in haEmodialysis access: The PAVE Trial. A multicentre double-blind randomised controlled trial in haemodialysis patients with a stenosis in a native arteriovenous fistula.
  • PAX-D: Randomised placebo-controlled trial evaluating the efficacy and mechanism of pramipexole as add-on treatment for people with treatment resistant depression
  • STudy Of Prevention by Aspirin anD EPA; kNowledge Of Mechanism of Action (STOP-ADENOMA)