18/170 Efficacy and Mechanism Evaluation Programme – Researcher-led

Applications are sought for research into interventions that are based or used by the NHS and its partners. This document provides the detailed call specification for applications to the EME researcher-led workstream.

Applications are expected to set out programmes of work which may contain distinct stages. It is expected that the early stages of the study will, if successful, lead onto a full evaluative clinical study or trial, which is in the remit of the EME Programme. This study must also be included and clearly specified within the application. Clinical trials embedded within the programme of work must be large enough to detect a meaningful effect.

Applications to this call may also include initial 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 big data or clinical samples 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 must not contribute more than 25% to the total cost or duration of the project.

Applicants will need to make a strong case for the future importance of the intervention through providing a measurable positive impact on health, innovation or future wealth creation and for the ultimate benefit of individual patients’ or the wider NHS. Applicants should read the details of the EME remit at https://www.nihr.ac.uk/eme

Programme remit

The EME Programme funds ambitious studies evaluating interventions that have the potential to make a step-change in the promotion of health, treatment of disease and improvement of rehabilitation or long-term care. Within these studies EME supports research to improve the understanding of the mechanisms of both diseases and treatments. The programme supports translational research evaluating a wide range of novel or repurposed interventions. The interventions may include diagnostic or prognostic tests and decision-making tools, drugs or biological compounds, psychological treatments, medical devices, and public health initiatives delivered within the NHS.

The EME Programme primarily supports clinical trials, and other robustly designed studies that test the efficacy of interventions. The interventions should have the potential to improve patient care or benefit the public. The programme will only support studies where there is sufficient evidence that the intervention might work in man, i.e. that there is ‘proof of concept (pdf, 55.19 KB) ’.

Innovative study designs involving stratification, the use of routinely collected digital data or novel methodologies are strongly encouraged. Where appropriate, the programme encourages hypothesis-testing mechanistic studies (pdf,  124.55 KB) integrated within the main efficacy study. These studies could explore the mechanisms of action of the intervention, the causes of differing responses, or promote an understanding of any potential adverse effects and how these could be reduced; they could also contribute to understanding of the disease. The programme will also support mechanistic studies that follow on from on-going or completed clinical studies funded by the NIHR which can use data or samples from these studies.

The programme will accept applications for studies that use clinical or well-validated surrogate outcomes. It will also consider studies that validate potential surrogate outcomes against a primary clinical outcome, within the main clinical trial.

The EME programme WILL support:

  • Research to determine proof of clinical efficacy, size of effect, and long-term safety in a well defined population.
  • The evaluation of a broad range of interventions that have the potential to maintain health, treat disease or improve recovery.
  • Hypothesis-testing 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 (usually 2 to 3) with progression to the main clinical evaluation dependent on the outcome of the previous stage(s). The criteria for progression must be clearly defined. The main clinical evaluation should require more than 75% of the total project costs and commence within 18 months of the project start date.
  • Pilot and feasibility studies where the main study would be within the remit of the EME programme. These studies may be either stand-alone or can be the initial part of a staged project that includes the main clinical evaluation as a subsequent stage.
  • The limited steps needed to progress the development of an intervention to a stage suitable for use in an accredited clinical service when included as an initial stage prior to commencing the main clinical evaluation.
  • Studies using novel or infrequently-used study designs that increase the value of a study, by maximising the chances of demonstrating the benefit of an intervention, increasing the knowledge that can be gained through the study, or by making the study more efficient.
  • The EME Programme welcomes studies adopting novel and efficient study designs or that include the development or testing of new methodologies in an embedded methodological study (EMS). Where delivery of the EMS is integral to the main efficacy trial the additional costs should be modest and the purpose should be to explore issues that may potentially increase the efficiency of trials and value for money. The proposed work should be included in the EME application. Where the EMS requires more substantial funding and can be delivered independently from the running of the main trial, then applicants might wish to consider applying to the MRCNIHR Methodology Research Programme (MRP). http://www.mrc.ac.uk/funding/browse/methodology-research-programme/ Any such application would be independent of the application to the EME Programme. For more information please contact the MRP programme: MRPGrants@headoffice.mrc.ac.uk

Of particular relavence to the Dementia Research community is that NIHR programmes currently have the following highlight notice:

Complex Health and Care Needs in Older People. This highlight notice indicates the continuing interest of NIHR in receiving research proposals in this area, and particularly those addressing Frailty; Transitions in care, service delivery and models of care; Medicines  anagement/polypharmacy; Promoting healthy ageing/preventing ill health; Patient-centred decision making.

 

Visit funding web page
(https://www.nihr.ac.uk/funding-and-support/funding-opportunities/18170-efficacy-and-mechanism-evaluation-programme-researcher-led/9511)