Highly Specialised Technologies Evaluations
Josie Godfrey, Associate Director – Highly Specialised
Technologies and Topic Selection
Topic Selection & Scoping
or refer
Suggestions received from topic sources
Suggestions are assessed according to NICE/DH criteria
to prioritise topics
Decision point 1
Process starts
approximately 2 years
before a drug is licensed
DH/NICE jointly agree on topics that should proceed to
draft scope creation
Decision point 2
DH/NICE/NHS England jointly agree on draft scopes to be
issued for consultation
Decision point 3
Consultation on the draft scope and scoping workshop
DH/NICE/NHS England post-scoping meeting
Decision point 4
Referral by Minister
Decision point 5
Ideally 12–15 months
before a drug is licensed
Highly specialised technologies
programme: prioritisation criteria
• The target patient group for the technology in its
licensed indication is so small that treatment will
usually be concentrated in very few centres in the
• The target patient group is distinct for clinical reasons
• The condition is chronic and severely disabling
• The technology is expected to be used exclusively in
the context of a highly specialised service
• The technology is likely to have a very high acquisition
• The technology has the potential for life long use
• The need for national commissioning of the technology
is significant
HST criteria
Nature of the condition
Impact of the new technology
Cost to the NHS and Personal Social Services
Value for money
Impact of the technology beyond direct health
• Impact of the technology on the delivery of the
specialised service
HST: other considerations
• Para 41…
When evaluating cost to the NHS and PSS, the Committee will take
into account the total budget for specialised services, and how it is
allocated, as well as the scale of investment in comparable areas of
medicine. The committee will also take into account what could be
considered a reasonable cost for the medicine in the context of
recouping manufacturing, research and development costs from
sales to a limited number of patients.
Highly specialised technologies
programme: process
* Excludes appeal period and reconsideration points
~27 weeks from submission*
NICE produces provisional list of topics
Consultees identified
Scope prepared and consulted on – all comments considered during scoping
Topics referred by Minister to NICE
Evidence submitted by manufacturer and other consultees, comments invited on
potential clinical effectiveness and value
Evidence review group (ERG) report independently commissioned and prepared
Committee papers prepared: Evidence submissions from manufacturer, patients,
clinical specialists and NHS England, ERG report, pre-meeting briefing
Evaluation committee considers all evidence
Evaluation committee document (ECD) produced only if recommendations are
more restrictive than license; public consultation for 4 weeks
Evaluation committee considers responses to public consultation
Final evaluation determination (FED) produced; any appeals considered
Guidance issued
How patients and carer organisations
can contribute to a NICE Highly
Specialised Technology Evaluation
Overview of patient involvement
Stage 1
Stage 2
Scoping usually occurs:
1. before a licence has been
granted for a technology
2. Before the topic has been
Guidance development is usually:
1. After referral
2. As close to issue of licence as
(the licence has to be issued
before the evaluation
consultation can begin)
Scoping (pre-referral)
Patient organisations
1. Comment in writing
on the draft
matrix (stakeholder
2. Participate in
scoping workshop
Key information in a scope
Population –informed by clinical trial populations and anticipated marketing
Intervention – name of technology and whether it is given alone or in
combination with other treatments
Comparators – alternative treatment options currently used to treat condition
in established clinical practice
Outcomes – important outcomes to show the effects of treatment
Subgroups – any subgroups of the population in which the intervention is likely
to be more clinically effective or provide more value for money
Equality issues – consideration of whether the evaluation could lead to
recommendations that have a different impact on people protected by the
equality legislation than on the wider population
The scope also contains the remit/evaluation objective, a brief background
to the condition and current treatment options, a short description of the
technology and any relevant clinical trials.
Guidance development
after referral
1. Provide written evidence
submission to Committee
2. Nominate patient experts to
attend Committee meeting
3. Comment on Evaluation
Consultation Document
4. After the Final Evaluation
Determination is issued –
provide factual inaccuracies or
appeal against decision
What information do patients and
carers provide to NICE?
1. What it is like to live with the condition
2. Outcomes that patients and carers most value
3. Perceived risks and benefits of treatment
4. Difference the treatment will make to physical wellbeing
5. Impact of condition on daily activities (work, social life,
6. Psychological health of patients/carers (mood, anxiety)
7. Emotional health of patients/carers (wellbeing, relationships)
8. Balance between health related quality of life and length of life
9. Treatment choices that matter to patients
10. Impact of condition on the lives of family members and carers
How patients and carers can
contribute to a NICE Highly
Specialised Technology evaluation
The role of patient experts
Patient Experts
– provide statements which will help the
Committee consider key criteria such as the
nature of the condition
– attend Committee meetings as individuals
They will have
– experience of the broader patient population
relevant to the evaluation and/or
– relevant personal experience
How can NICE support patient
– Provide help to patient organisations with the
process from scoping to guidance publication
– Support patient experts before, during and
after the committee meeting
– Providing patient support materials – hints and
tips and factsheets.
– Liaise with patient groups to include their
details in the patient version of the guidance
Ataluren for Duchene Muscular
Dystrophy – next steps
• Scoping:
– Deadline for written responses to consultation:
Wednesday 11 February 2015
– Scoping workshop: NICE offices, central Manchester on
Monday 16 March 2015 at 14:00 (refreshments from
– If you would like to discuss your organisation’s
representatives, please contact Heidi Livingstone, Public
Involvement Adviser, Public Involvement Programme
[email protected]
– If you have any queries please contact Michelle Adhemar,
Scoping Project Manager, [email protected],