1. The future of market access – the
national picture
PM Society / Wellards Forum
London 29 November 2012
Meindert Boysen
Programme Director Technology Appraisals & PASLU
National Institute for Health and Clinical Excellence
No part of this talk can be reproduced without the expressed permission of NICE

2. Health and Social Care Act - 2012:
paying for value

3. Outcomes Framework and Standards

4. http://review2011-2012.nice.org.uk/

5. Current NICE products & services
BRAND MAP HERE

6. New system relationships

7. 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.

8. • 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.

9. Use of NICE appraised medicines in
the NHS in England – 2010 and 2011
Experimental publication of the Health and Social Care
Information Centre 2012

10. Value Based Pricing
The Government view:
“We need a system that encourages the development of
breakthrough drugs addressing areas of significant unmet need.
And we need a much closer link between the price the NHS pays
and the value a new medicine delivers, sending a powerful signal
about the areas that the pharmaceutical industry should target for
development.”
“Most importantly, using our cancer drugs fund in the interim, and
value-based pricing for the longer-term, we will move to an NHS
where patients will be confident that where their clinicians believe
a particular drug is the right and most effective one for them, then
the NHS will be able to provide it for them.“
Quote from A. Lansley in Guardian 29 Oct 2010

11. Overview of rationale for VBP
Current Process Issues VBP solutions
Does drug give enough We may care more about Apply QALY
benefit* to justify moving some patients… weightings
funds and depriving some • eg with severe • Burden of Illness
other patients of their
condition, large • Therapeutic Innovation
treatment? and Improvement
unmet need
Right decision if:
Treatments affect people Include “Wider Societal
• Care equally about
beyond patients Benefits”
all patients • Effect on contribution
• Family, carers
• Only care about to society…
patients • Beneficiaries of • …and use of
goverment spending society’s resources
*measured in Quality-Adjusted Life Years (QALYs), the universal unit of health gain

12. VBP key features
• Applies to new medicines on the market from 1 Jan 2014
• Government to set out a range of thresholds or maximum prices
reflecting different values that medicines offer
• Use of a (basic) cost effectiveness threshold
• Use of QALYs
• Application of weighting to benefits implying of maximum price
thresholds
• Higher price thresholds for medicines that tackle disease of high
unmet need or severity
• Higher price thresholds for medicines demonstrating greater
therapeutic improvement and innovation
• Higher price thresholds for medicines that can demonstrate wider
societal benefits
• Categories and weights will be determined by the Secretary of
State, on the basis of expert advice, within a framework
determined in advance.
VBP consultation 2010/11

14. So, how about NICE?
“As enshrined within the NHS Constitution, the NHS in England
will continue to fund existing drugs that have been recommended
by NICE. And that right will continue and will apply to new
medicines to which VBP applies.
“NICE will examine the evidence on the potential clinical and
cost effectiveness of new drugs as they become available;
drawing on its world-leading expertise in the field.
“And, importantly, under the new system of VBP, NICE will no
longer be obliged to make yes/no decisions on access, based on
its own cost per QALY thresholds.
“Instead, you'll be free to focus on the rigorous appraisal of
evidence to show the relative benefits of a new medicine.
Andrew Lansley, Secretary of State for Health
NICE Conference, 17 May 2012

15. Appraising value

16. Procedural Principles
s
le ne s
so n ab
r r ea
o
i ty f
bil
ou nta
c
Ac

17. Most technologies are worth using ...
80%
o
recom f NICE
mend
s are
Single Multiple posit ation
Recommendation ive
categories Technology Technology Total
Appraisal Appraisal
Recommended 63 (58%) 234 (64%) 297 (62%)
Optimised 15 (14%) 68 (19%) 83 (18%)
Only in Research 3 (3%) 22 (6%) 25 (5%)
Not Recommended 27 (25%) 43 (11%) 70 (15%)
Total 108 (100%) 367 (100%) 475 (100%)
Based on 493 recommendations published in 265 technology appraisal guidance
between March 2000 and October 2012

18. Timeliness targets

19. Clarification Evidence
Review
CHMP +
8 weeks
Evidence
Submission Committee
Decision Preliminary
meeting
Problem 8 weeks recommendations
Experts
(incl PCT)
Invitation
Clarification
MA
Including
Consultation
compan
Review y 4 weeks
Scoping
MA
Final guidance Committee
Publication meeting
Meet
compan [ 26-34 weeks]
y Experts?
Including
STA Appeal (or not) company?
Process

20. The Quality Adjusted Life Year
1 Initial QALY loss
due to side effects
Health-related quality of life
New treatment
Current
treatment QALYs
gained
0
Length of life (years)

21. Fixed Budget & Opportunity Cost

22. Consideration of cost effectiveness:
threshold range
less
between
than
£20,000 and more than £30,000
£20,000
£30,000 per
per
per QALY gained
QALY
QALY gained
gained
Make explicit
reference to these
factors:
Probably • Certainty Need to identify an
cost • Health related increasingly strong case
effective Quality of life with regard to same factors.
adequately captured?
• Innovative nature
technology

23. Application of the ‘end-of-life’ advice
in 2009 - 2012
End-of life criteria
37
considered
Criteria met &
10
recommended
Criteria met & not
6
recommended
Criteria not met & not
21
recommended

24. Application of ‘special circumstances’
Rawlins, Barnett, Stevens Br J Clin Pharmacol 2010

25. “different ways of doing things
which bring improved outcomes”
(Cooksey)
“new, constitutes an improve-
ment on existing products ,
step-change” (Kennedy)
“…a plausible gain of at least
1 QALY would be a reasonable
threshold for judging the
usefulness of a supposedly
innovative technology…”
(Ferner et al, 2010)
“…..whether a new medicine
represented a significant
improvement relative to
Ferner R., Hughes D. and Aronson J. ‘NICE and new:
existing treatments...” (DH
appraising innovation’ BMJ 2010; 340: 245-247.
Consultation Paper on VBP)

26. How often were the HR-benefits not
captured in the QALY?
30 published appraisals or consultation documents*
7 some HR-benefits not captured in the QALY
2 5
Impact on decision No impact on decision
HR-benefits of reduced hospital (changing utility values had
visits in last year of life little effect on ICERs)
HR- benefits of delaying toxic
chemotherapy
*July 2010 to January 2011

27. Schemes in operation in England and Wales
Pre-PASLU
NICE Product & clinical area Details
TAG*
129 Bortezomib (Velcade) – multiple myeloma Rebate for non-responders
155 Ranibizumab (Lucentis) – age related macular oedema Dose cap
162 Erlotinib (Tarceva) – non-small cell lung cancer Simple discount
169 Sunitinib (Sutent) – renal cell carcinoma Free stock
171 Lenalidomide (Revlimid) – multiple myeloma Dose cap
176 Cetuximab (Erbitux) – colorectal cancer Rebate
179 Sunitinib (Sutent) – gastro intestinal stromal tumour Free stock
180 Ustekinumab (Stelera) – psoriatic arthritis Free stock
185 Trabectedin (Yondelis) – soft tissue sarcoma Dose cap
186 Certolizumab pegol (Cimzia) – rheumatoid arthritis Free stock
192 Gefitinib (Iressa) – non-small cell lung cancer Single fixed price
*See http://www.nice.org.uk/guidance/ta/published/index.jsp

28. Schemes in operation in England and Wales
PASLU
NICE Product & clinical area Details
TAG*
202 Ofatumumab (Arzerra) – chronic lymphocytic leukaemia Simple discount
205 Eltrombopag (Revolade) – thrombocytopenic purpura Simple discount
215 Pazopanib (Votrient) – renal cell carcinoma Simple discount
218 Azacitidine (Vidaza) – myodysplastic syndromes Simple discount
220 & 225 Golimumab (Simponi) – psoriatic & rheumatoid arthritis Free stock
221 Romiplostim (Nplate) – thrombocytopenic purpura Simple discount
227 Erlotinib (Tarceva) – maintenance advanced or metastatic nsclc Simple discount
233 Golimumab (Simponi) – ankylosing spondylitis Free stock
235 Mifamurtide (Mepact) – non-metastatic osteosarcoma Simple discount
238 Tocilizumab (RoActemra) – juvenile idiopathic arthritis Simple discount
241 & 251 Nilotinib (Tasigna) – Chronic myeloid leukaemia Simple discount
247 Tocilizumab (RoActemra) – moderate to severe rheumatoid arthritis Simple discount
250 Eribulin (Halaven) – advanced breast cancer Simple discount
254 Fingolimod (Gilenya) – relapsing remitting multiple sclerosis Simple discount
*See http://www.nice.org.uk/guidance/ta/published/index.jsp

30. Quality evidence submissions; a real
challenge ...

31. Highly Specialised Technologies
a ie a n o d
S
bc e y n
re tim
lin ti lit y a
C nd nlef ts t f
se u
im po cti lini ris iafe o
pa abi rit
ca k ty
in a la
ve
n r
pa Ne ov ch ting
t
Se
s
g fo &
ti ed at an
ov n t ne l
in ial ss
so ent s o ion d
he r
th
ci s a f
al
pr te ve
C
et n
y d
fe
ef
Patients’
B
e
de a c Need
pr st
. oV or c l c en st
Eciv ct lin
l
in af p ral at co
co m polu da t an os t
Ac Co effseon ri ice ica
al m onrtue fbili d t
e
im ver p ge
o n
i
ce f nt icirviomg in l
pe e r a
te pa e nitor ty
ps prin e c i t
iv to st
o
ro o u n e c h
Av
rn re y y c
ge bal sivi viis cy e
f a
b
di o an isit tyoo
O
l i
gr c oyn nf
es
at d
st
pa
rib ap ed an
ut hi d
io c cl
n

32. The ‘new’ challenge for HTA; ‘adaptive
licensing’!
Eichler et al. 2012 www.nature.com/cpt

33. ?

34. For discussion …?
• How will the new commissioning structure in the NHS
deal with, and shape, NICE’s approach to technology
appraisals?
• What is to (further) gain in market access from VBP
knowing that the majority already receives positive
guidance.
• Will VBP reflect all value, and if not, how much of a role
should there be for a deliberative process?
• Do patient access schemes have a future in the context
of VBP and sPPRS?
• How different is (should) the situation (be) for highly
specialised technologies?

35. MEINDERT.BOYSEN@NICE.ORG.UK