One year older…one year
wiser?
Dr Stuart Linton,
AWMSG Chairman,
AWMSG Training Day
January 2016
A year as chair
• 2015: The year in retrospect
• The view from the top
• 2016 and beyond...
(SOME OF) MY PRIORITIES
As of November 2014
• Implementation of the new process for appraising orphan
and ultra-orphan medicines (CAPIG) 
• Improved access and availability of timely advice ahead of
NICE (‘One Wales’) 
• Re-accreditation of the AWMSG appraisal process 
2015: Appraisal Priorities
– Continuing alignment with NICE
• Updated MoU with NICE signed in October 2015...
• AWMSG working arrangements updated to reflect developments in NICE
( HSTs, EAMS, for example)...
• Regular AWTTC communication with NICE and SMC...
– Ensuring the AWMSG appraisal process remains...
•
•
•
•
Robust
Timely (currently 22 weeks...)
An efficient use of finite AWTTC resources (limited submission process)
Fit for purpose eg engaged with...
Medicines appraised by AWMSG
2002–2015 (November)
300
83% ‘yes’
Number of appraisals
250
200
150
100
Orphan appraisals ~ 16%
Ultra-orphan...~ 7%
68% ‘yes’
50
80% ‘yes’
0
All HTAs
Orphan medicines*†
Recommended or optimised recommendation
†Orphan
Ultra orphan medicines
Not recommended
medicines also includes all ultra orphan medicines
Wales Patient Access Scheme
(WPAS)
30
The availability of
WPAS has
increased the
number of
medicines
appraised by
AWMSG (10
medicines with a
WPAS were
appraised in 2014–
2015, double the
number in the
previous year).
Percentage of appraisals
with a WPAS
25
20
15
10
5
0
2009-2010
2010-2011
2011-2012
2012-2013
2013-2014
2014-2015
Percentage of appraisals with a WPAS by
financial year
AWMSG: The view from the top...
Steering Committee
• Meeting schedule aligned to AWMSG
• Standing agenda items aim to horizon scan
‘bumps in the road’
–
–
–
–
–
–
–
–
Welsh Government
WHSSC
NMG
Chief Pharmacists
Cancer NSAG
Contracting...
AWPAG/WAPSU/NMG
ABPI/ TDA Partnership Group
Some challenges:
Lack of engagement with AWMSG
• Insufficient cost-effectiveness data at licensing stage
• ICER expected to be too high
• Previous “no” from AWMSG for another licensed indication
for the medicine
• Small numbers of patients in Wales
• Limited impact of AWMSG modifiers
• Decision not to promote medicine in Wales
• Lack of resources to engage in Wales
Modifying/removing barriers to
engagement with AWMSG
Problems...
Modifiers...
• Insufficient cost effectiveness
data?
• Small numbers?
• Orphan /ultraorphan /CAPIG
process
• Limited submission process
• ICER expected to be too high?
Option of WPAS
• ?Eligibility for end-of-life
criteria
•
NICE decision delay
‘unacceptable, to NHS Wales
• Unlicensed medication
‘One-Wales’ cohort process
AWMSG Appraisal / Scrutiny:
Special circumstances (1)
• End of life situations
• Where the following conditions apply
– Life expectancy of patient group < 24 months (vs median survival in
study control group)
– Sufficient evidence that medicine offers an extension to life of at
least 3 months compared to NHS treatment (via robust data on
either PFS or OS)
– Cumulative population in Wales will be small (up to 400 patients)
• Limited submission
– Significant new formulation with a pro-rata or lower cost per treatment.
– Anticipated usage in NHS Wales is considered to be of minimal
budgetary impact.
AWMSG appraisal / scrutiny:
Special circumstances 2: NICE HST
• Will
Positive NICE HST decisions will continue
to be considered by AWMSG on
a case-by case basis.
This process allows Welsh Health
Specialised Services Committee
(WHSSC) to highlight any issues
or barriers to implementation...
Eg Eculizumab
AWMSG advised Ministerial approval
subject to developing robust
commissioning advice around
Gatekeeping
Use of a specialist centre
Starting/stopping criteria
Registry development
AWMSG appraisal / scrutiny:
Special circumstances 3: Orphan drugs
• Some definitions (EMA)...
• Orphan EMA status:
– A medicine...which includes
conditions affecting fewer
than 2,500 people in a
population of 5,000,000 (ie
1,500 patients in Wales)
• Ultra-orphan EMA medicine
– A medicine...which is used
to treat a condition with a
prevalence of 1 in 50,000 or
less in the UK ( ie around 60
patients in Wales)
• Recognition that in such
settings problems with a
conventional approach to
the data might include
– Challenges in clinical trial
design & recruitment
– QALY a ‘blunt tool’
– Unmet need
– Requirement for innovation
Form B submitted for an orphan / ultraorphan medicine or medicine developed
specifically for rare diseases
AWTTC prepares an assessment of the evidence (the
ASAR) and provides comment on the applicability of
the orphan /ultra-orphan criteria
Draft ASAR sent to applicant company for comment
ASAR may be subsequently updated in light of comments
received
Preliminary appraisal by the New Medicines Group (NMG)
Preliminary recommendation and final ASAR sent to applicant
company for comment within 5 working days from NMG meeting
Applicant company requests a meeting of
CAPIG following a negative NMG
recommendation
Appraisal process is suspended and a
meeting of CAPIG is convened (an
additional 8-12 weeks may be added to the
normal appraisal timeline)
CAPIG meeting held
Applicant company accepts the NMG
preliminary appraisal recommendation
The appraisal process continues and
appraisal by AWMSG is undertaken within
normal timelines
The information submitted by CAPIG is
considered by AWMSG along with the usual
meeting documentation
Orphan / ultra-orphan process
• Review of AWMSG orphan/ultraorphan policy during May 2013.
• OUTCOME:
– Commitment to strengthen input from
patients’ groups and clinicians -via
Clinical and Patient Involvement
Group (CAPIG).
• January 2015 to August 2015
new process piloted
• August 2015 endorsed by
AWMSG
• From Jan 2015 to date five
ultra orphan and four orphan
medicines have been
appraised
• Eight medicines were
recommended or
recommended for optimised
use by NMG and by AWMSG
• CAPIG convened for first
appraisal in November 2015
A slide on process
Clinical effectiveness
Cost effectiveness
Clinical expert input
? End-of-life criteria
NMG
preliminary negative
Recommendation for
O/U-O medication
CAPIG convened
AWMSG
Clinical effectiveness
Cost effectiveness
Clinical expert input
Budget impact
Societal impact
Welsh Government
and it is the Minister who has the final decision in
relation to the availability of the medicine within NHS
Wales
Independent review (IR) process
• Current IR process available in writing via Chair
– Within 10 days of the meeting
– On grounds that...
• there has been a misinterpretation
of scientific information OR
• a complaint in relation to process
• NB: A request for reconsideration by AWMSG does not
assume an inappropriate decision or outcome...
Members’ responsibilities!?
• Give good notice of...
– Any conflicts of interest
– The need to arrange a deputy
• Come prepared to contribute...
• Respect
– The confidentiality of discussions and materials...
– The corporate nature of AWMSG decisions
What’s new for 2016....
• NICE developments
– Review of CDF (AWTTC to input
to process as interested stakeholder)
– EAMS (AWTTC representation)
– ?Projects in early evaluation
• Commissioning through evaluation
• Adaptive regulatory pathways
•
AWMSG initiatives
– Orphan/ultra-orphan/CAPIG
– One Wales cohort process
– Membership appraisal...
offer i
A huge thank you to...
• The supporting cast..
– Continuing and retiring members of AWMSG,
AWPAG, CAPIG and NMG
– Workforce of AWTTC and WAPSU
– Partners in pharmacy, WHSSC, Welsh Government,
clinical networks and industry