Published: 15 January 2021
Version: 1.0 Janaury 2021Print this document
This guide outlines which sorts of primary research studies will be of interest to the Developmental Pathway Funding Scheme (DPFS) (funded by the Medical Research Council (MRC), or the Efficacy and Mechanism Evaluation (EME) Programme (an NIHR and MRC partnership).
DPFS Research: Developmental Pathway Funding Scheme (DPFS) is aimed at providing proof of concept in humans. They are generally supported by pre-clinical evidence (where appropriate).
EME Research: The Efficacy and Mechanism Evaluation (EME) Programme supports explanatory trials (later in the development pathway than DPFS), undertaken when there is some initial evidence that the technology is efficacious in patients (where proof of concept in humans has already been achieved) but a large-scale study is needed to determine definitive proof of clinical efficacy and safety.
|Evaluates||Studies should focus on establishing safety and proof of concept in humans. The principal focus of the study should not be to understand disease mechanism.||Studies should be aimed at establishing clinical efficacy of a therapeutic intervention under controlled conditions which already have proof of concept in humans established.|
|Stage of research||
DPFS supports early stage clinical studies up to and including safety and proof of concept in humans. Studies would generally aim to evaluate the treatment in tightly controlled conditions. The development of the intervention may still require some refinement.
Where appropriate, proof of concept in animal models will have been undertaken. All forms of intervention will be considered, for example drug or cell-based treatments (apart from stem cells), vaccines, devices and instruments, diagnostics, surgical, dietary, psychological interventions, and novel applications of existing therapeutics.
EME research would be specifically designed to evaluate efficacy and whether the treatment works under ideal conditions, but may gather some further information on safety.
EME studies would start where proof of concept in humans has already been established and where the required intensity/level/dose of an intervention has been decided.
A clinical study. Studies may be proposed with multiple “phases”, where there are clear and measurable milestones between them, which must be met in order to progress to the next phase.
|A large-scale clinical trial or evaluative study (not necessarily an RCT). May also include laboratory based (or similar) studies embedded within the main EME study.|
|Participant eligibility criteria||Relevant patient groups or healthy volunteers.||Relevant patient groups: often narrowly defined by the entry and exclusion criteria, and not necessarily representative of the UK population as a whole.|
|Technology*||A new intervention (or a new indication for an existing intervention) which requires proof of concept (or safety) in humans.||Mostly developed and defined but some refinement / fine tuning may still be needed.|
|Outcome Measures||Studies will demonstrate proof of concept in humans and may include surrogate outcome measures or safety. The research will provide supporting data for a large scale evaluative trial.||Validated surrogate markers as indicators of health outcome are acceptable. The output may provide the justification for a pragmatic trial.|
|Animal studies||Can be included where pre-clinical toxicology/ manufacture/ scale-up work is required to enable the proposed trial to be undertaken.||Research involving animals is not supported.|
|Can a mechanistic evaluation be included as part of the main study?||The principal focus of the clinical study should not be to understand the aetiology or mechanism of a disease. However, opportunistic mechanistic work is encouraged. A significant proportion of the research should involve human participants.||Mechanistic evaluations which help to understand how an intervention works, or how behaviour affects or is affected by an intervention, may be included as part of the main EME study.|
|Diagnostic evaluations||Likely to support the development of a diagnostic test. Unlikely to support the validation of a diagnostic test.||Likely to support validation of a diagnostic test.|
*By “technology” we mean any method used to promote health, prevent and treat disease and improve rehabilitation or long-term care. “Technologies” in this context are not confined to new drugs but include procedures, devices, tests, settings of care, screening programmes and any intervention used in the treatment, prevention or diagnosis of disease.