Highly Specialised Technologies Evaluations

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• Highly Specialised Technologies Evaluations
• Josie Godfrey, Associate Director Highly
  Specialised Technologies and Topic Selection

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Topic Selection Scoping
Process starts approximately 2 years before a
drug is licensed
Suggestions received from topic sources
Reject or refer elsewhere e.g. National
Screening Committee
Suggestions are assessed according to NICE/DH
criteria to prioritise topics Decision point 1
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
Ideally 1215 months before a drug is licensed
Referral by Minister Decision point 5
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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 NHS
• 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 cost
• The technology has the potential for life long
  use
• The need for national commissioning of the
  technology is significant

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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
  benefits
• Impact of the technology on the delivery of the
  specialised service

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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.

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Highly specialised technologies programme process

• NICE produces provisional list of topics
• Consultees identified
• Scope prepared and consulted on all comments
  considered during scoping workshop
• 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

27 weeks from submission
Excludes appeal period and reconsideration
points
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How patients and carer organisations can
contribute to a NICE Highly Specialised
Technology Evaluation
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Overview of patient involvement

• Scoping usually occurs
• before a licence has been granted for a
  technology
• Before the topic has been referred
• Guidance development is usually
• After referral
• As close to issue of licence as possible (the
  licence has to be issued before the evaluation
  consultation can begin)

Stage 1 Scoping Pre-referral
Stage 2 Guidance Development Post-referral
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Scoping (pre-referral)

• Patient organisations can
• Comment in writing on the draft
• remit
• scope
• matrix (stakeholder list)
• Participate in scoping workshop

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Key information in a scope

• Population informed by clinical trial
  populations and anticipated marketing
  authorisation
• 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.

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Guidance development after referral

• Provide written evidence submission to Committee
• Nominate patient experts to attend Committee
  meeting
• Comment on Evaluation Consultation Document
• After the Final Evaluation Determination is
  issued provide factual inaccuracies or appeal
  against decision

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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, relationships)
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

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How patients and carers can contribute to a NICE
Highly Specialised Technology evaluation
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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

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How can NICE support patient experts?

• 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

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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 1400
  (refreshments from 1330)
• If you would like to discuss your organisations
  representatives, please contact Heidi
  Livingstone, Public Involvement Adviser, Public
  Involvement Programme heidi.livingstone_at_nice.org.u
  k
• If you have any queries please contact Michelle
  Adhemar, Scoping Project Manager,
  michelle.adhemar_at_nice.org.uk,