Research and Development at NICE
Sarah Garner PhD BPharm
Associate Director R&D NICE
The background: why NICE was
set up
• Established in 1999
• Aim: to reduce variation in the
availability and quality of
treatments and care (the so
called ‘postcode lottery’)
• To resolve uncertainty about
which medicines and
treatments work best and
which represent best value for
money for the NHS
NICE Guidance by Year
300
Evidence
updates
Number of publications
250
Medical
devices
Diagnostics
Quality
Standards
CCGO
QIPP
200
QOF
150
Clinical
Guidelines
100
Interventional
Procedures
Public
Health
Accreditation
Appraisals
50
0
00/01
01/02
02/03
03/04
04/05
05/06
06/07
Year
07/08
08/09
09/10
10/11
11/12
12/13
Fund within
3 months
NICE
guidance
and
information
programmes
4
NICE’s procedural principles for
guidance development
Scientific
Rigour
Timeliness
Support for
implementation
Inclusiveness
Accountability
for
reasonableness
Transparency
Independence
Review
Challenge
NHS constitution 2012
You have the right to
drugs and treatments
that have been
recommended by
NICE for use in the
NHS, if your doctor
says they are clinically
appropriate for you.
• We will introduce a NICE
Compliance Regime to reduce
variation and drive up compliance
with NICE Technology Appraisals.
• We will require that all NICE
Technology Appraisal
recommendations are incorporated
automatically into relevant local
NHS formularies in a planned way
that supports safe and clinically
appropriate practice.
• We will establish a NICE
Implementation Collaborative to
support prompt implementation of
NICE guidance.
• We will develop and publish an
innovation scorecard to track
compliance with NICE Technology
Appraisals.
Our role: how does NICE
make decisions?
A spotlight on NICE’s
technology appraisals
programme
Cost
effectiveness
Clinical
effectiveness
Drug
development
Regulatory
approval
HTA
Use in
healthcare system
Risk/benefit balance
Risk/benefit ‘ratio’ (?)
Value
• Added clinical benefit
• Pricing negotiations
• Cost effectiveness
What is a NICE Technology
Appraisal?
• A review of clinical and economic
evidence leading to recommendations
on the appropriate use of new and
existing technologies for the NHS
• Funding regulations (to ensure
compliance with recommendation)
• Opportunity to challenge decision
(formal appeal process)
Therapeutic areas in technology appraisal topics
Respiratory
Skin Urogenital
Blood & immune
Musculoskeletal
Mouth & dental
Mental health &
behavioural
conditions
Cancer
Injuries, accidents &
wounds
Infectious diseases
Gynaecology,
pregnancy & birth
Eye
Endocrine,
nutritional &
Ear & nose
metabolic
Digestive system Central nervous
system
Cardiovascular
The Process
Scoping
Review
Submission
Consultation
Guidance
Assessment
Appraisal
NICE and the world of
research
The misconception
• NICE is not really a key player in the world
of research
The reality
• NICE is a research organisation: we
conduct and commission research, and
we play an important role in driving and
prioritising topics for research in the UK
Why does research
matter to NICE?
Committee decision making
Clinical
effectiveness
End of life medicines
(Supplementary advice)
Other health
benefits
Costeffectiveness
Recommendations
Uncertainty
Social Value Judgements
Innovation
Equality
legislation
Why uncertainty arises
No evidence
• Doesn’t exist
• Not identified
• Not reported
Existing evidence
• Not robust
• Cannot be
generalised
(setting, patients,
country etc)
• Does not answer
the question
• Out of date
• Conflicting
What type of uncertainties?
• clinical
effectiveness
• cost effectiveness
• implementation
• outcomes
• accuracy of a test
• clinical prediction
rules
• diagnosis
•
•
•
•
prognosis
rates of harms
patients’ experience
measurements of
outcome
• service delivery
• organisation
• methods
Our committees
have a key role to
play in identifying
gaps in the evidence
base
NICE’s Research
Recommendations Database
• We develop research recommendations outlining
what’s needed to fill evidence gaps
• NICE is a policy partner with the NIHR Evaluation,
Trials and Studies (NETS) team
• Priority research recommendations are fast-tracked by
the NIHR into their Priority Strategy Group for
consideration and possible advertisement
http://www.nice.org.uk/guidance/GuidanceResearchRecc
ommendations.jsp (or search ‘research recommendations
at www.nice.org.uk)
Case study: funded research
NICE Public Health guidance published: Alcohol Use
Disorders - preventing harmful drinking
Research recommendation identified by NICE: “Which
screening tool should be considered as the ‘Gold Standard’
for assessing the drinking behaviour of under-18s?”
Research funded by NIHR. Dr Dorothy Newbury, University
of Newcastle: A feasibility trial of screening and brief
alcohol intervention to prevent hazardous drinking in young
people aged 14-15 in a high school setting.
Publication due in November 2014
Challenges
How can we ensure that the gaps in the evidence base,
identified by NICE’s committees, are prioritised
systematically across research funders?
Other research
activities at NICE
CASMI is the Centre for the
Advancement of Sustainable
Medical Innovation, a partnership
between Oxford University and
UCL, created to develop new
models for medical innovation.
The centre aims to address the
issues that have led to current
failures in the translation of basic
bioscience into affordable and
widely adopted new treatments.
26
Re-designing the biomedical
innovation paradigm
Adaptive licensing pilot project
• NICE is a key partner in the EMA’s adaptive licensing
project
• Project aims:
 To bring together regulation, research and HTA bodies to
make sure the right trials are conducted, providing the
best and most appropriate evidence for the MHRA, EMA
and HTA bodies like NICE
 To streamline development, regulation and market
access for selected products
 To balance timely access for patients with the need to
provide adequate evolving information on benefits and
harms
Key elements of the concept
Conditional
Approval (or
approval on
conditions)
Stakeholders:
-Sponsor
-Regulator
-HTA
-Patients
Co-design of
confirmatory
trials
Adaptive
licensing
Real world
effectiveness
tracking
EMA, based
on rapporteur
evaluation of
submission
Managed
Market Entry
Outcome, safety
data collection via
reliable network
Patients
treated;
sponsor
reimbursed
Challenges: efficacy vs effectiveness
• Clinical trials: efficacy
• How a drug performs under
controlled conditions
• Most precise estimates
• Data needed for regulation
• Real world: effectiveness
• How a drug likely to perform in
healthcare systems
• A lot of variability and biases
• Data needed for HTA
• Innovative Medicines Initiative (IMI)
– Europe's largest public-private initiative
– joint undertaking between European Union and European pharmaceutical
industry association EFPIA.
• GetReal
•
Understanding how real-world data can contribute to decision-making
– October 2013 to December 2016 (39 months)
– 29 partners
– Total budget: €18 million
• 50% staff from the 15 participating pharma companies
• 50% cash contribution from the EU to fund ‘public’ sector
The research leading to these results has received support from the Innovative Medicines Initiative Joint Undertaking under grant
agreement no [115303], resources of which are composed of financial contribution from the European Union’s Seventh Framework
Programme (FP7/2007-2013) and EFPIA companies’ in kind contribution.
www.imi.europa.eu
WP3
Overcoming
practical barriers to
the design of realworld studies
WP2
Understanding the
efficacy-effectiveness
gap
Identifying best
practice and creating
new methods for
evidence synthesis and
predictive modelling
simulation of trials to
improve design
WP1
Frameworks
• Standardising terminology
• Interviews to understand and
the perspectives and policies of
different stakeholders
• Designing a framework for
decision-making during
development
WP4
Processes
Policies
• 5 Case studies using drugs that
had difficulty at regulation and
HTA
• 360 degree reviews
• Re-designing development
pathways to include realworld data
• Simulation
• Ascertaining impact on
decision makers
The research leading to these results has received support from the Innovative Medicines Initiative Joint Undertaking under grant
agreement no [115303], resources of which are composed of financial contribution from the European Union’s
Seventh Framework
Slide 32
Programme (FP7/2007-2013) and EFPIA companies’ in kind contribution.
www.imi.europa.eu
Thank you
sarah.garner@nice.org.uk