Managing evaluation research on behalf of the National Institute for Health Research

1. Managing evaluation research on behalf of the National Institute for Health Research RDS and CTU Training and Information Event 8 November 2011

2. Managing evaluation research on behalf of the National Institute for Health Research NIHR Evaluation, Trials and Studies Coordinating Centre (NETSCC) & NETS Programmes – An overview Dr Martin Ashton-KeyRDS & CTU Information Event8 November 2011

3. Outline • To provide RDS and CTU staff with an overview of the NIHR Evaluation, Trials and Studies Coordinating Centre (NETSCC) & the NETS programmes • To highlight the key functions and interactions • To then hand over to the individual NETS programmes

4. Background – Research pathway

5. The NIHR Health Research System Faculty Associates Investigators &Senior Investigators Trainees Universities Infrastructure Research Clinical Research Networks Research Projects & Programmes NHS Trusts Patients&Public Clinical Research Facilities, Centres & Units Research Schools Research InformationSystems Research GovernanceSystems Systems

6. Background: NETSCC • Health related research now funded through 2 main routes: NIHR and MRC • NIHR Evaluation, Trials and Studies Coordinating Centre - (NETSCC) • Funded by the National Institute for Health Research (NIHR)

7. Managing evaluation research programmes on behalf of NIHR • NIHR Evaluation, Trials and Studies (NETS) programmes • Efficacy and Mechanism Evaluation (EME) programme • NIHR Health Services Research (HSR) programme • NIHR Health Technology Assessment (HTA) programme • NIHR Public Health Research (PHR) programme • NIHR Service Delivery and Organisation (SDO) programme

8. Managing evaluation research programmes on behalf of NIHR • NIHR Evaluation, Trials and Studies (NETS) programmes • Efficacy and Mechanism Evaluation (EME) programme • NIHR Health Services and Delivery Research (HS&DR) programme • NIHR Health Technology Assessment (HTA) programme • NIHR Public Health Research (PHR) programme

9. NIHR Evaluation, Trials and Studies (NETS) programmes- from 2012 NETSCC Health Services and Delivery Research Will be established from 2012 HS&DR Public Health Research Established: 2008 Health Technology Assessment Established: 1993 PHR HTA Efficacy and Mechanism Evaluation Funded by the MRC and NIHR Managed by NIHR Established: 2008 EME

10. Research themes

11. Research for public health at NETSCC • The NETS programmes play a vital role in producing high quality, relevant work that aims to add significant value to the implementation of public health practice • The NETS funded research provides valuable information to public health professionals, researchers and government to ensure that public health is at the centre of improving the nation’s health • NETSCC provides a coordinated, joined-up approach to public health research across a wide research remit • NETSCC is alert to emerging national research priorities in public health and responds by giving them appropriate emphasis within the NETS programmes, e.g. obesity and dementia

12. Needs-led and science-added approach for NETS programmes • We are: Needs-led because we assess what the important questions to the NHS and other information consumers are, and in what priority they should be answered • We are: Science-added because we ask does the proposal meet the scientific quality we insist on, and how can we support the delivery of quality • We provide: Value for money because assessment of need and scientific support maximises the relevance and benefits achievable within a funding stream

13. Funding workstreams • Commissioned workstream • Starts with the information needs of decision makers typically, but not exclusively, within the NHS but also Public Health outside the NHS • Topics are refined and prioritised by people with expertise in the field, and commissioning briefs are advertised • Applications are then assessed for compliance with the commissioning brief, scientific quality, feasibility and value for money • Researcher-led workstream • Open calls for researchers to apply for funding for their own topics and questions • Applications are prioritised in terms of NHS or other information need in a process similar to that in the commissioned workstreams • Applications are then assessed for scientific quality, feasibility and value for money

14. Feasibility studies • Other definitions are used elsewhere but we apply the following in order to help us communicate with applicants • Feasibility study • Pieces of research done before a main study • Used to estimate important parameters that are needed to design the main study • Feasibility studies for randomised controlled trials may not themselves be randomised • They do not evaluate the outcome of interest; that is left to the main study

15. Pilot studies • Pilot study • Pilot studies are a small scale versions of the main study that run to test whether the components of the main study can all work together • Focused on the processes of the main study, for example recruitment, randomisation, treatment, and follow-up assessments • An internal pilotis a pilot that may be the first phase of the substantive study and data from the pilot phase may contribute to the final analysis • An external pilot is one where the data may be analysed but set aside in respect of the substantive study

16. Full and appropriate funding • We provide: • Full and appropriate funding because we place no upper limit on the amount of funding granted for a project • If the question is important enough and the science requires it, we will fund it • University based projects: we fund up to 80% of the Full Economic Cost (FEC) • NHS Trust based projects: we fund 100% of the direct costs • Other organisations are also welcome to apply

17. Eligibility • EME researcher-led workstream uses research council eligibility criteria and is open to applicants from the whole of the UK. The EME commissioned workstream requires all applications to be in the form of a significant collaboration between at least two of the following partners; industry, academia, and the NHS. • HTA, PHR and HS&DR [HSR, SDO] have broader eligibility criteria and will consider applications from any applicants who consider that they are able to conduct the study.

18. NIHR Clinical Trials Unit Support Funding • Launched 2008 • Recognises the importance and crucial role played by CTUs in designing and delivering quality NETS research applications and projects • Provides ‘priming’ funding to support CTUs on a 3 year rolling contract and provide stability to units (allowing expansion and retention of skilled staff)

19. NIHR Clinical Trials Unit Support Funding • Intended to result in an increased number of high quality research applications submitted to NETS programmes • 22 CTUs in England supported to date on rolling contract basis • Three funding bands:£150, 200, 250k per annum • All payments are made directly to CTUs • Funding adjusted according to successful grant applications

20. NIHR Reviews Infrastructure • NETSCC manages the contracts for Reviews Infrastructure on behalf of the NIHR • Systematic Reviews provide decision-makers with the best possible information about the effects of tests, treatments and other interventions used in health and social care • There are four entities which comprise the NIHR Reviews Infrastructure: • UK Cochrane Centre and Cochrane Review Groups • Centres for Reviews and Dissemination • Diagnostics Evaluation Unit • The Unit for Improved Monitoring in Long Term Disease • For information on NETSCC’s role within the Reviews Infrastructure, please visit: www.netscc.ac.uk/reviews_infrastructure/index.asp

21. Public involvement in NETS programmes • Patients, service users and members of the public are involved in: • Identifying research topics • Prioritising topics to be developed further • Reviewing research briefs/vignettes as they are compiled • Prioritising research briefs/vignettes to be advertised as calls • Reviewing proposals submitted by researchers

22. Strategic development of PPI in NETS programmes • Closer working with patient/service user organisations on topic identification • Open recruitment of wider group of public contributors to prioritisation and commissioning • Panels & boards require clear evidence of commitment to PPI in design and conduct of research • Wider dissemination of ‘plain English’ findings to interested communities • Reference group of public contributors to advise on strategic development of PPI

23. Summary • NETSCC has responsibility for coordinating all the NETS programmes • NETSCC has additional functions including Reviews Infrastructure / CTU funding • NETSCC is keen to work with the RDS and CTUs to ensure applicants have best opportunity for success • NETSCC is here to help

24. Further information Visit: www.netscc.ac.uk Email: info@netscc.ac.uk

25. Thank you

26. Managing evaluation research on behalf of the National Institute for Health Research Efficacy and Mechanism Evaluation (EME) programme Lisa Douet8 November 2011

27. Outline • Background • Remit • Assessment process • Board members • Examples of funded research • Summary

28. Managed Translational Pathway

29. Translating science into better health • MRC: can it work? • EME: does it work? • HTA: is it worth it? • The EME programme is funded by the MRC and NIHR, with contributions from the CSO in Scotland, NISCHR in Wales and the HSC R&D, Public Health Agency in Northern Ireland • It is applicable to all parts of the UK

30. Background • The EME programme funds research through two main workstreams: • Researcher led workstream - funds research questions proposed directly by researchers • Commissioned workstream - advertises calls for research proposals that address specific research areas • Additionally the EME programme may commission research through: • Themed calls – advertises for research proposals that address themed areas. These are one-off calls for research in areas where a particular need has been identified and are advertised on ad-hoc basis

31. Who defines the question? Frequency Per year EME programme As required Continuous with 3 submission deadlines Researcher Types of Research Primary Research; Clinical efficacy of interventions Translational pull through Primary Research; Clinical efficacy of interventions EME Researcher-led Primary Research; Clinical efficacy of interventions Commissioned EME programme 3 calls per year

32. The EME programme remit • The EME programme is broadly aimed at supporting 'science driven' studies with an expectation of substantial health gain and aims to support excellent clinical science with an ultimate view to improving health or patient care • The remit includes evaluations of new treatments, including therapeutics (small molecule and biologic), psychological interventions, public health, diagnostics and medical devices. Treatments or interventions intended to prevent disease are also included

33. EME will support clinical trials and evaluative studies in patients that: • Provide definitive evidence of efficacy of healthcare interventions, including size of effect, safety and effectiveness • Add significantly to understanding of disease mechanisms • Break new ground – new scientific or clinical principles • Include development or testing of new methodologies • Include validated surrogate markers as indicators of outcome • Laboratory studies as part of the main study, if relevant to the EME remit • The EME programme will not support animal studies

34. Researcher-led workstream • The EME researcher led workstream will not fund: • Proof of concept in humans must be established prior to applying to the EME commissioned workstream • confirmatory studies or trials of incremental modifications and refinements to existing medical interventions

35. Mechanisms of working: researcher-led workstream • Standard mode (two stage application) • On going call with 3 closing dates a year • All in remit and competitive preliminary applications are peer reviewed before Board assessment • If successful full application to the next Board. • Preliminary application submission deadline to funding decision around 8 months • Fast track (single stage application) • Normally expected to use the standard funding mode • Single stage application process reducing time to funding decision by about 3 months • Only used where reducing the timescale will provide significant benefit, or exploitation of a narrow window of opportunity • All cases for an application are discussed with the EME team to ensure that there is a need for the reduced timescale

36. Mechanisms of working: commissioned workstream • Applications to the EME commissioned work stream should be in the form of a significant collaboration. All applications should include significant contributions from at least two of the following partners; industry, academia, and the NHS • EME commissioned aims to fund large staged projects of work which have clear go/no go milestones • Early stages may include: proof of concept studies, pilot studies, feasibility studies and late stage development of the intervention. However, the majority of the study should be an efficacy study

37. Mechanisms of working: commissioned workstream • Specific topics advertised three times a year • Research areas are identified and prioritised by the Prioritisation Group • Prioritised research topics are advertised and request submission of preliminary applications • All in remit and competitive preliminary applications received will be prioritised prior to peer review before Board assessment • If successful at preliminary stage a full application is submitted to the next Board • A funding decision takes around eight months from the close of the commissioning brief

38. The EME Board • Chaired by Professor Rajesh Thakker • 26 members - Board membership comprises senior, experienced clinicians and methodologists (including clinical trial specialists and statisticians) who are all active researchers in their field • Meets three times per year • Supported by the NETSCC, EME scientific secretariat (directed by Professor Ian Cree)

39. Funded studies • Since April 2008 the EME Board has funded 31 researcher led studies • The funding projects have received ranges from £124, 000 to £1.79 million and the duration ranges from 18 to 66 months • The first funding decisions for the commissioned workstream will be in Jan 2012. It is anticipated that the majority of EME commissioned studies will cost between £500,000 to £2 million • However, for both workstreams there is no upper limit to funding as long as the funding requested is justified in the application

40. Examples of funded research A novel anti-Wilms-Tumour-1 (WT1) vaccination strategy in haematological malignancy using DNA fusion vaccines delivered with electroporation Professor Christian Ottensmeier (University of Southampton) 48 months from 1st September 2010 (£973,304) Aims to show that DNA vaccination, directed against the tumour protein, WT1, can control the progression of the disease

41. Summary • The key focus of all EME studies is to establish clinical efficacy of an intervention: • For the researcher-led workstream proof of concept in humans must be established prior to application • For the commissioned workstream, applications are expected to be in the form of large multi-stage projects for which the majority of the funding is for the efficacy study but may include earlier work • The researcher-led is an on-going call with three close dates a year • The commissioned workstream has three calls a year.

42. Thank you

43. Managing evaluation research on behalf of the National Institute for Health Research NIHR Health Technology Assessment (HTA) programme Andrew Cook8 November 2011

44. Outline • Background and Remit • Boards and Panels • The Workstreams • Portfolio • Current and Future Calls

45. NIHR Health Technology Assessment programme • Purpose and Remit To deliver information about the effectiveness, costs and broader impact of healthcare treatments and tests for those who plan, provide or receive care in the NHS • The HTA programme includes The programme includes all interventions used to promote health, prevent or treat disease, improve rehabilitation or long-term care including drugs, devices, procedures, settings of care and screening

46. Panel and Boards: Stages of Assessment

47. Prioritisation Panels • Six areas • Pharmaceuticals • Diagnostics and Screening • Interventional Procedures • External Devices and Physical Therapies • Psychological and Community Therapies • Disease Prevention • Membership • Between 12 and 20 members • Clinicians, Commissioners, Patients and Public

48. Prioritisation Group • Membership • The Programme Director • Chair of the Commissioning Board (Deputy Programme Director) • Chair of the Clinical Evaluations and Trials Board • Chairs of the Prioritisation Panels

49. Commissioning Board and CET Board • Membership • Clinical Academics • Methodologists

50. The HTA programme offers 3 funding workstreams: Types of Research Who defines the question? Frequency Per year Primary research Feasibility & pilot Evidence synthesis HTA programme 4 calls Commissioned Primary research Feasibility & pilot Evidence synthesis Continuous with 4 closing dates Clinical evaluation Researcher HTA Researcher Led Theme from HTA programme Question from researcher Themed Calls Primary research Evidence synthesis 1 call NICE Guidance Direct referral to On-contract teams Specific technology assessment NICE