Presentation held by Ben Morgan, Assistant Director and Professor Richard Watt, Panel Chair For RDS London

1. Research for Patient Benefit
Programme
Ben Morgan, Assistant Director
Professor Richard Watt, Panel Chair
RDS Workshop – 27 June 2017

2. Research for Patient Benefit (RfPB)
The RfPB Programme
• Response mode funding programme
• Grants may be:
– Costed up to £350k (Tier 1)
– Feasibility applications costed up to £250k (Tier 2)
– More upstream studies costed up to £150k (Tier 3)
• Three funding competitions per year
• Eight Regional Advisory Panels
• > 780 awards made to date totalling nearly £172m (132
projects and £33m for LDN)
• Overall success rate of ~22% (peer reviewed
applications). LDN success rate of 23%.

3. Research for Patient Benefit (RfPB)
Research for Patient Benefit
• Supports high quality investigator-led research projects
that address issues of importance to the NHS
• Supports qualitative or quantitative research that could:
– Study the way NHS services are provided and used
– Evaluate whether interventions are effective and
provide value for money
– Examine whether alternative means for providing
healthcare would be more effective
– Formally assess innovations and developments in
healthcare
– Assess feasibility of projects requiring major applications
to other funders

4. Biomedical Research Centres
Basic Research
National Institute
for Health Research
This pathway covers the full range of
interventions - pharmaceuticals,
biologicals, biotechnologies, procedures,
therapies and practices - for the full range
of health and health care delivery -
prevention, detection, diagnosis,
prognosis, treatment, care.
Patient Safety Translational
Research Centres
Research for Patient Benefit
Programme Grants for Applied Research
Health Technology Assessment
Invention for Innovation
Collaborations for Leadership in Applied Health
Research and Care
Centre for Reviews & Dissemination, Cochrane, TARs
Development Pathway Funding
Public Health Research
Health Services and Delivery Research
INVENTION EVALUATION
ADOPTION DIFFUSION
Efficacy & Mechanism
Evaluation
NHS Commissioning Board and Clinical Commissioning Groups Commissioning
National Institute for Health & Clinical Excellence Guidance on Health & Healthcare
NHS Supply Chain Support for Procurement
NHS Evidence Access to Evidence
InnovationAcademic Health Science Networks
Patient CareProviders of NHS Services
Clinical Research Facilities
Experimental Cancer Medicine Centres
Research Schools
Horizon Scanning Centre
Centre for Surgical Reconstruction & Microbiology
Biomedical Research Units
Medical
Research
Council
The central role of NIHR research in the innovation
pathway

5. Research for Patient Benefit (RfPB)
Recent Changes
• New Director
• Standard Application Form (SAF)
• Two stage process

6. Research for Patient Benefit (RfPB)
New Director
• Professor Sue Ziebland started in May this year
• Sue is Professor of Medical Sociology and Director of
the Health Experiences Research Group (HERG), in the
Nuffield Department of Primary Care health Sciences,
Oxford University

7. Research for Patient Benefit (RfPB)
RfPB funding limits guidance
Introduction of three funding tiers that aim to balance the
probability of a proposal achieving patient benefit against
its cost.
• Upper limit of £350,000 for research with a clear and close
trajectory to patient benefit
• Soft ceiling of £250,000 for feasibility studies (in exceptional
circumstances a well argued application could cost more)
• RfPB will also now consider research that is ‘higher risk’
(further from patient benefit), but should cost less than
£150,000 (exceptions for proposals that might have more
immediate patient impact can be made)

8. Research for Patient Benefit (RfPB)
Examples of £150,000 projects
• Observational studies
• Developing and refining interventions
• Developing new scales or outcome measures
• Exploratory studies that might provide insights into an
intractable problem
• Additional follow up of patients in a completed clinical trial
• Post-market surveillance for unknown side-effects of a
drug (Phase IV trials)
• Systematic reviews where the number of relevant studies
is likely to be limited

9. Research for Patient Benefit (RfPB)
Review of Tier 3
• Review of Tier 3 projects published on RfPB website
• A total of 21 projects funded over 3 competitions totalling £3m.
• Developed 4 ‘types’ of research seen within Tier 3 and
identified the expected pathways to patient benefit
Funded projects included:
• 3 systematic reviews
• 7 developing and refining interventions
• 1 meta-analysis
• 1 realist synthesis
• 1 economic evaluation
• 6 secondary data analysis (including developing predictive
models and needs assessments)
• 2 diagnostic accuracy studies

10. Research for Patient Benefit (RfPB)
Funding Comp 28, 29 & 30
Funding tier
Tier 1
(up to £350k)
Tier 2
(up to £250k)
Tier 3
(up to £150k)
Total
Stage 1 applications
submitted
125 220 120 465
% of applications
submitted
27 47 26 100
Number invited to stage 2 47 90 34 171
% applications at stage 2 27 53 20 100
Number of applications
funded at stage 2
18 36 19 73
% applications funded at
stage 2
25 49 26 100
Total funding committed £5,876k £8,385k £2,517k £16,779k

11. Research for Patient Benefit (RfPB)
RfPB Applications

12. Research for Patient Benefit (RfPB)
Funding Success Rates

13. Research for Patient Benefit (RfPB)
RfPB Spend 2016-2017

14. Research for Patient Benefit (RfPB)
New Investigators
• New investigators are encouraged to apply
• The team as a whole is assessed (ensure the %FTE is
appropriate)
• Relatively inexperienced lead applicant with sufficiently
experienced and resourced co-applicants is fine
• RfPB is a good option for researchers looking for their
first grant

15. Research for Patient Benefit (RfPB)
A renewed RfPB
Two changes introduced from January 2016 (Competition
28)
1. Ten Regional Advisory Committees reduced to 8
Regional Advisory Panels (‘Panels’ to ensure
consistent naming across NIHR) by merging regions
2. A new Stage 1 formative assessment to triage weak
applications and offer advice/support to all applicants

16. Research for Patient Benefit (RfPB)
Two stage application process
NIHR Standard Application Form (SAF) burdensome for
‘smaller’ RfPB grants
Stage 1:
• Complete main sections of SAF for a formative
assessment by Panel
Stage 2:
• If invited, revise and complete rest of SAF for a
summative assessment

17. Research for Patient Benefit (RfPB)
Stage 1
• Panel assesses the main sections of application (no
external reviews)
– Names of applicants
– Background/rationale for study
– Research question(s)
– Methods
– Estimate of costs
• Is Panel interested in seeing full application?
• What could be done to improve it?

18. Research for Patient Benefit (RfPB)
Stage 2
• If invited to Stage 2, Stage 1 application copied across to
SAF
• Applicant revises Stage 1 sections in light of panel
comments and completes other sections
• Applicants given 6 weeks to complete SAF for current
round or defer until next
• Stage 2 application assessed by panel

19. Research for Patient Benefit (RfPB)
How’s it going so far?
• Have run 3 competitions using two stage process
• The success rate at stage 2 typically 45%
• Outcomes sent to applicants much quicker (especially
those who were declined at Stage 1)
• Feedback on application process and assessment has
been positive from applicants and RfPB panel members
• Required 600 fewer peer reviews per year (despite more
applications)
• Peer review scores higher
• Overall, more efficient running of the programme

20. Research for Patient Benefit (RfPB)
New SAF
• New Standard Application form being launched across
all programmes – RfPB launching in July
• Gives greater flexibility for researchers to describe their
proposal
• Should enable PPI to be ‘threaded’ throughout proposal
• Should not disrupt current RfPB two stage process

21. Research for Patient Benefit (RfPB)
Know your target:
• What are the aims of the funding scheme?
• Am I eligible for support?
• How will the application be assessed?
• Who will assess the application?
• When will the application be assessed?
• What is the scale of a typical award?
• Who has received previous awards?
Applying for research funding

22. Research for Patient Benefit (RfPB)
Other things to keep in mind
• All project costs are scrutinised by CCF – particular
attention should be paid to NHS support, treatment
and excess treatment costs (at stage 2)
• Patient and public involvement must be adequately
thought through and planned as part of design
• Read the guidance and website resources
• Guidance available for feasibility studies as well as for
other specific types of research (information resources,
established interventions)

23. Research for Patient Benefit (RfPB)
RfPB: ‘me too’ applications?
Is
{pick any
intervention}
• CBT
• CBT
variant
• exercise
etc
effective
in
treating
{pick any
disease}
• depression
• anxiety
• heart dis.
• diabetes
etc
in
{pick any
population}
• men
• women
• young
• old
• Manchester
• ethnic min.
• disabled
etc
?
The Research One-arm Bandit

24. Research for Patient Benefit (RfPB)
Common areas for feedback
• Detail in the methodology lacking or appropriateness of the
design questioned
• Overall lack of clarity and focus of the application
• Inappropriate outcome measures
• Particular areas of expertise lacking in the research team
• Insufficient quality of the patient and public involvement
• Justification or detail of the intervention lacking
• Insufficient detail provided in the background information
• Inappropriate statistics or health economics analysis
• Concerns with the recruitment, sampling and overall
feasibility
• Questions regarding project impact, timescales,
generalisability or dissemination

25. Research for Patient Benefit (RfPB)
Possible reasons for failure
• Failure to demonstrate importance of topic or patient benefit
• Research question is ill-defined, unfocused or unsupported
by preliminary data
• Omission of critical literature references
• Research team lacks relevant experience
• Methodology unsuitable, flawed or unlikely to yield results
• Insufficient methodological detail to convince reviewers that
the team knows what it is doing
• Insufficient recognition of potential problems

26. Research for Patient Benefit (RfPB)
PPI
Stage 1 – looking at the wider picture
• Focus on question
• Patient burden/experience
• Patient relevant outcomes
Stage 2 – focus on the detail of PPI plans

27. Research for Patient Benefit (RfPB)
PPI at stage 1
• PPI box taken out at stage 1
• PPI should be a constant thread through the proposal
and should be explicitly referenced throughout where
appropriate
• If the research question, outcomes etc have been
revised due to PPI then say so in the relevant places.

28. Research for Patient Benefit (RfPB)
The ‘make it clear’ campaign was launched in May 2014
and a good quality plain English summary is now a
requirement for NIHR funding.
• New guidance for applicants, reviewers and panels has
been developed by INVOLVE and implemented across
NIHR to help develop and evaluate good quality plain
English summaries
• The NIHR standard application form and review forms
have been revised
• Resources have been developed,
including examples to support applicants
http://www.invo.org.uk/makeitclear/
Plain English Summaries

29. Research for Patient Benefit (RfPB)
RfPB: the research question
What is an interesting/important question?
• How important is the new knowledge for achieving
patient benefit?
• What is the trajectory from this question to patient
benefit?
• Will the methods provide a clear answer to the question?
• Is the cost of the research commensurate to the ‘risk’ it
won’t deliver?

30. Research for Patient Benefit (RfPB)
Stage 1 Assessment
• Panel will be primarily looking at the following:
1. The relevance and importance of the research
question, such as is it relevant to patients and the
NHS? Patient Benefit
2. The appropriateness of the methodology; will the
proposed methods achieve the stated aims? Does the
proposed method imply a burden for patients that is
unwarranted? Are the endpoints (such as the outcome
measure in a clinical trial) sufficiently patient oriented?
Methods and the Team
3. The amount of improvements required to make any
subsequent Stage 2 application competitive.

31. Research for Patient Benefit (RfPB)
Team composition
• Needs to reflect nature of proposed work – under/over
costing will be picked up by panel
• Each co-applicant needs a clearly described role – if
panel cannot determine what each member is doing it
undermines application
• Including senior colleagues at 1% is not well received by
panels and looks suspicious
• If the PI is ‘junior’ make it clear who is performing a
‘mentor’ role and give this person a suitable %FTE.

32. Research for Patient Benefit (RfPB)
Involvement of CTUs
• When is a CTU needed and what is required?
• Typically expected if research is a definitive trial
• Full trial management or specific services required?
– Often see tasks such as randomisation conducted by
CTUs
• Programme wrote to NIHR funded CTUs to gauge
expected level of involvement – RDS and programme
can help find suitable CTUs

33. Research for Patient Benefit (RfPB)
Defining feasibility aims
• Need to reflect nature of proposed work
• What genuinely needs to be assessed? Patient
recruitment/retention? Acceptability of intervention?
Willingness to be randomised (patient/clinician)?
• Parameters needed to determine if definitive trial is
feasible will be different for each feasibility study

34. Research for Patient Benefit (RfPB)
Appropriate Dissemination
• Dissemination plans need to be proportionate to the
nature of the work and likely outcomes
• Feasibility studies – do they need disseminating via
high-impact journals, international conferences etc?
• Dissemination needs to link with how output could be
integrated into care (especially information resources)

35. Research for Patient Benefit (RfPB)
Summary - my personal opinion
Three key things for a successful RfPB application
• Patient benefit has to be very clear (or path to patient benefit)
–Including specifics about the numbers of people affected
–Evidence that the question is important to patients/public
• Methods must be clear
–What is the research question?
–Will the proposed project answer the research question?
• The Team needs to be convincing
–Does not need to be the great and the good but needs to have the right
expertise; research as well as clinical skills are important
–Tiny amounts of time from seniors does not convince the panel project
will be delivered

36. Questions?