Experimental medicine 2
This call is now closed for applications and is only available for reference purposes.
Building on an earlier initiative in this area the MRC launched a second wave of funding for ‘proof of concept’ studies to continue to strengthen experimental medicine research in the UK.
The British Heart Foundation (BHF) partnered the MRC with this call. This call is one of a suite of initiatives and funding opportunities available for experimental medicine over the next few years.
- Funding available
- Objectives and remit
- Partnerships
- Advice to applicants
- Assessment criteria
- Panel membership
Funding available
Funding of at least £15 million was available for this call. The scale of the proposal should reflect the resources needed for thorough and high quality investigation. The size of awards will vary widely, but overall MRC expects that the funds will be sufficient for 20-30 awards.
The next available funding for Experimental Medicine is likely to be announced during 2008.
Objectives and remit
This call was limited to research on the early testing and exploration of new ways of treating and preventing ill health in human participants. This was a “first human use” studies aimed at proof of concept or proof of mechanism; exploring therapeutic or preventative potential and where possible, providing insight into underlying mechanisms of disease/treatment. Interventions may include: drug or cell based treatments; vaccines; devices and instruments; surgical, dietary, psychological interventions, and novel applications of existing therapeutics.
Partnerships with industry and charities were strongly encouraged and priority was given to areas of high health need.
From the experience of earlier initiatives, the statistical design of studies is a key issue. We do not anticipate proposals will be powered to provide definitive evidence; they are likely to be based on smaller numbers of participants and will inform the design of more definitive studies, through providing greater precision on issues such as effect size estimates etc.
In summary, all studies must:
- Have a clear and testable hypothesis, and a quantifiable measure relating to it;
- Be based on a sound scientific rationale with supporting preliminary data;
- Involve measures of effect in either healthy volunteers or patients;
- Have a clear endpoint but also collect information that can be used to support or refute the proposed mechanism of effect;
- Use sufficient numbers of participants to give confidence that the main observed effects were a consequence of the intervention; a series of observations that show a trend in the same direction may be sufficient; formal power calculations are not mandatory, but applicants are expected to demonstrate rigorously the appropriateness of their experimental design;
- Contain a clear plan for data analysis, relating explicitly to the primary research question;
- Consider the applicability and next steps for knowledge transfer and translation into patient benefit in the short to medium term;
- Include all required expertise, especially in relation to statistics and study design;
- Demonstrate strong understanding of the appropriate regulatory frameworks and clinical research governance;
- Make any data originating from the study publicly available.
The call specifically excludes:
- Phase III studies, irrespective of scale;
- Animal studies;
- Support for infrastructure or facilities;
- Large five year programmatic proposals.
Details of the successful awarded applications in response to the first Experimental Medicine call are available under ‘Awards made in Experimental Medicine’.
Partnerships
Applications including partnerships with charities or industry were strongly encouraged where these add value to the research e.g. in terms of access to expertise, technologies, reagents, patient groups or funding. MRC does not have the capacity to broker these arrangements.
Applications involving tangible charity contributions may be announced as joint MRC/Charity funded studies. BHF co-funding will also be recognised in award announcements.
The terms of collaboration, particularly in relation to industry, must be determined early in the study development and relevant agreements must be put in place. Partnership arrangements should ensure transparency in the development of the study design and in the analysis and publication of results (including if these are negative). Consideration should also be given to issues such as: relative responsibilities, governance arrangements, regulatory approvals, indemnity, intellectual property rights, reporting and access to data and samples.
Advice to applicants
This call is a fast track funding stream with a single, full application stage. Applicants should note that because of anticipated demand there will be limited scope for feedback during the assessment process.
It is anticipated that proposals generally will be up to three years in duration with a timescale and resources appropriate to the research proposed. Costings should be on a full economic costs basis. MRC will pay 80 per cent FEC.
As there will be future opportunities to apply for experimental medicine research funding, applicants were advised to ensure their proposal is fully developed before submission to this call. Unsuccessful proposals in this call are unlikely to be eligible for re-submission to future initiatives (at least not within the MRC’s usual 12 month re-submission term).
The following will form part of the assessment process:
- Applications were invited across the whole range of disease areas. However, proposals that address an unmet clinical need were strongly encouraged.
- Applicants were encouraged to submit proposals developed by strong, multidisciplinary research teams which reflect the range of expertise required including basic and clinical researchers as well as statisticians. The ability of the team to design and deliver a methodologically robust study will be a key assessment criterion.
- Applications with strong industry or charity collaboration were particularly welcome. However, MRC does not have the capacity to broker partnerships.
- Applications must demonstrate access to appropriate clinical infrastructure for the conduct of the study. Proposals building on existing infrastructure and facilities will be particularly welcome.
- Applications must demonstrate a strong understanding of the regulatory environment and requirements for good clinical practice and research governance. Although approvals are not required prior to submission, applicants were advised to have early discussions with the regulatory bodies, particularly if MHRA approval is required. This is to help prevent awarded research proposals being unduly delayed.
- Applications must discuss the potential audience for and impact of the outcomes of the proposed research, particularly in terms of knowledge transfer and translation for patient benefit.
Applicants are referred to the following literature on experimental medicine methodology and governance in developing their research plans:
- Royal Statistical Society’s Working Party on Statistical Issues in First-in-Man Studies
- Duff Report (Final Report of the Expert Scientific group on Phase I clinical trials)
- Clinical Trials Tool Kit
- EMEA guidance on first-in-man clinical trials
- ABPI guidelines for phase 1 clinical studies 2007 edition
Assessment criteria
Proposals are assessed by a specially convened panel including representatives of MRC Research Boards, BHF nominees and additional independent experts. The composition of this panel will be published on the MRC website in due course and will reflect the range of applications received.
Key assessment criteria will be:
- Potential impact of the research in terms of knowledge transfer or translation for patient benefit;
- Significance of the topic (either health burden, unmet clinical need or biological importance);
- Novelty/ priority/ importance /timeliness of the research;
- Robustness of the design, methodology and analysis plans to address the research questions;
- Ability of the investigators to deliver the research proposed;
- Strength and clarity of collaborations, especially with industrial partners;
- Quality and suitability of the research environment and of the facilities;
- Value for money;
- Ethical considerations and governance arrangements.
Panel membership
- Professor Stephen Holgate, Chairman, Southampton General Hospital, University of Southampton
- Professor Graham Dunn, Ordinary Member, School of Epidemiology & Health Sciences, University of Manchester
- Professor John Ellis, Ordinary Member, Biopharmaceutical CEDD, GSK
- Professor Charles ffrench-Constant, Ordinary Member, Department of Pathology, University of Bath
- Professor Michael Frenneaux, Ordinary Member, Department of Cardiovascular Medicine, University of Birmingham
- Professor Stephen Gillespie, Ordinary Member, Department of Medical Microbiology, UCL
- Professor John Gribben, Ordinary Member, Institute of Cancer, Barts & the London School of Medicine & Dentistry
- Professor Andrew Grieve, Ordinary Member, Department of Public Health Sciences, KCL
- Professor George Griffin, Ordinary Member, St George’s Hospital, University of London
- Professor Ian Hall, Ordinary Member, Therapeutics and Molecular Medicine, University of Nottingham
- Professor Doug Higgs, Ordinary Member, John Radcliffe Hospital, Oxford
- Professor Adrian Hill, Ordinary Member, Department of Physiology, University of Oxford
- Professor Patrick Johnston, Ordinary Member, Department of Oncology, Queens University Belfast
- Professor Paul Matthews, Ordinary Member, Clinical Imaging Centre, GSK
- Professor David Nutt, Ordinary Member, Department of Community Based Medicine, University of Bristol
- Professor Steven O’Rahilly, Ordinary Member, Department of Clinical Biochemistry, University of Cambridge
- Mr Richard Peck, Ordinary Member Lily Industries UK
- Professor Rodney Phillips, Ordinary Member, Nutfield Department of Clinical Medicine, University of Oxford
- Professor Charles Pusey, Ordinary Member, Division of Medicine, Imperial
- Professor William Rosenberg, Ordinary Member, Liver Research Group, University of Southampton
- Professor Caroline Sabin, Ordinary Member, Department of Public Health and Primary Care, UCL
- Professor Stephen Senn, Ordinary Member, Department of Statistics, University of Glasgow
- Professor Lee Shepstone, Ordinary Member, School of Medicine, University of East Anglia
- Professor Edith Sim, Ordinary Member, Department of Pharmacology, University of Oxford
- Professor Nigel Stallard, Ordinary Member, Warwick Medical School, University of Warwick
- Professor Charles Streuli, Ordinary Member, Faculty of Life Sciences, University of Manchester
- Professor Martin Wilkins, Ordinary Member, Department of Clinical Pharmacology, Imperial College
If you have a query about this call please email
Email: grants@headoffice.mrc.ac.uk