The Biotherapy Development Association convened a two-day workshop in January 2014 to assess access to innovative cancer medicines in Europe. This presentation by OHE's Adrian Towse covers the situation in England, examining challenges that are peculiar to England as well as the English experience with issues common across countries.

1. The Rationale and Procedure for
Oncology Pricing and Reimbursement
Decisions in England
Adrian Towse
BDA1 Workshop:
Access to Innovative Oncology Medicines
Bonn • 16-17 January 2014
1Biotherapy
Development Association

2. Agenda
•
Pricing and reimbursement (P&R) context
•
Where are we now?
•
Issues for HTA in assessing oncology medicines
•
Value-based assessment and the end-of-life
(EoL) criteria
•
Emerging issues
– HTA for companion diagnostics
– differential pricing
P&R for oncology drugs in the UK
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3. UK P&R context
• In the UK, companies set prices, NHS decides
use
• England uses NICE to perform an HTA and
recommend on use; Scotland uses the Scottish
Medicines Consortium
• Hospitals use tenders when relevant
• Companies can offer discounts via patient
access schemes in England and the Scottish
equivalent
P&R for oncology drugs in the UK
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4. UK P&R context
Concerns 10 years ago: (1) poor uptake of new cancer
medicines in UK and (2) poor 5-year survival rates in the UK
Cancer a priority for the Government
NICE assesses all new cancer medicines
Introduction of EoL criteria
Introduction of Cancer Drugs Fund (CDF)
P&R for oncology drugs in the UK
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5. Improving UK cancer outcomes
•
•
•
•
•
Higher rates of curative
surgery
Increased drug use
Public health measures
Tackling late presentation
Increasing speed of
primary care referrals
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6. Richards’ report on drug uptake
Brings together IMS data with
manufacturers’ own information.
Strong industry engagement in data
validation. Support and engagement
from industry trade bodies.
Data findings tested and potential
causes of variation explored with a
range of UK experts, including
clinicians, pharmacists, public health
doctors, academics, patients’
representatives and industry
Result: the most comprehensive
insight to date on international
variations in drug usage
P&R for oncology drugs in the UK
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7. International Variations in Drug Usage
Comparison of UK drugs use (by ranking and percentage of
14-country average)
Source: Presentation by Sir Mike Richards 2010
7

8. Richards’ international usage report country
ranking for cancer medicines classes
Number of years on UK market since launch in 2009
0-5 years
6-10 years
Rank
Country
1
France
2
Switzerland
3
Austria
4
Spain
5
Italy
6
Germany
7
USA
8
UK
10+ years
Rank
Country
1
France
2
Italy
3
Spain
4
Germany
5
Switzerland
6
Austria
7
USA
8
Sweden
Rank
1
2
3
4
5
6
7
8
Country
France
Austria
USA
Germany
Spain
Switzerland
Sweden
Italy
9
Norway
9
Australia
9
10
11
12
Australia
UK
Canada
10
11
12
Sweden
Canada
Norway
10
11
12
13
New Zealand
13 New Zealand
Hormonal cancer
treatments
Rank
1
2
3
4
5
6
7
8
Country
Italy
Spain
Germany
France
UK
Austria
Sweden
Norway
UK
9
Switzerland
Canada
Norway
Australia
10
11
12
Canada
Australia
USA
13
New Zealand
13 New Zealand
Source: Richards, 2010. Extent and causes of international variations in drug usage
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9. Impact of patient access schemes
If all positive
decisions since 2009
where a PAS was
implemented were
assumed to be a
“not recommended”
decision in the
absence of a PAS
(bar labelled
“without PAS”) the
share of not
recommended
decisions increases
to 47%
Chart: share of decision outcome for all medicines
decisions from 2009 to Q3 2013, with and “without” PAS
Source: OHE analysis from data on NICE website
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10. Trends in decision for cancer medicines
before and after establishment of cancer
drugs fund (Q4 2010- Q3 2013)
Source: OHE analysis from data on NICE website
P&R for oncology drugs in the UK
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11. Where are we now?
Uptake has hopefully improved (new report due out soon)
Inconsistency between use of NICE and existence of CDF
HTA in cancer has issues
Introduction of VBA will throw up further issues, e.g. future
of EoL criteria
Issues for the future:
• Small patient groups for rarer cancers – orphan and “ultra
orphan” drugs
• Use of companion diagnostics
• Different prices for different indications
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12. Sources of uncertainty
•
•
•
Mapping from
progression-free survival
(PFS) to overall survival
(OS)
Correcting clinical trial
data to account for
treatment crossover
Complexity of treatment
sequencing options
Advances in Oncology Modelling: Recent
Results from POI Research
Posted on 18 September 2012 by OHE News
Editor
OHE and the Pharmaceutical Oncology
Initiative (POI) held a workshop to discuss and
debate several key issues in oncology
modelling. Participating were more than 50
experts from industry, academia and
government.
The focus of the workshop was three key
technical challenges associated with using
clinical trial data for anti-cancer medicines and
adapting it, in an unbiased fashion, to support
decisions about cost-effectiveness. These are:
extrapolating from progression-free
survival, correcting for patient crossover,
and efficient modelling of different
sequences of treatment.
http://news.ohe.org/2012/09/18/advances-inoncology-modelling-recent-results-from-poiresearch/
P&R for oncology drugs in the UK
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13. Limitations of using QALYs in cancer
Sensitivity of EQ-5D to
changes in health status of
cancer patients
Violation of constant
proportional trade-off
assumption at end of life
Using valuations from
members of the general
population
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14. Eliciting social preferences: End-oflife findings highlight the challenges
Linley and Hughes (2012)
Shah, Tsuchiya and Wailoo (2013)
P&R for oncology drugs in the UK
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15. Authors.
journal
(date)
Shah,
Tsuchiya,
Hole,
Wailoo
DSU report
(2012)
Linley and
Hughes
Health Econ
(2012)
Olsen
Value in
Health
(2013)
Elicitation
Method(s)
Responden Mode of
t sample
admin
(n)
General
population
DCE
Online
survey
EoL comparison
Life expectancy without
treatment
3, 12 ,24 36, 60m
(3969)
Choice plus
trade off
questions
General
population
18 months vs 60 m
Online
survey
Life extension 6 months
Findings relevant to EoL
EoL was not a significant driving
factor (but longest LE 60 months)
No evidence of a value premium for
EoL
(4118)
Choice plus
benefit trade
off
General
population
(503)
Online
survey
Remaining lifetime
untreated;
Total lifetime untreated;
Relative loss in remaining
life expectancy (prop
shortfall)
Source: Rachel Baker, Glasgow Caledonian University
No evidence of EoL (proximity to
death) premium
Total lifetime inequality is most
important and ‘trumps EoL’
Not proportional shortfall but
absolute loss

16. Authors.
journal
(date)
Pinto Prades
et al
Working
paper
2013
Elicitation Responden
Method(s t sample
)
(n)
WTP
PTO
3 survey
versions:
(820)
General
population
Choice
tasks
EoL comparison
Probability of gains in
LE (EoL)
QoL (EoL)
QoL (non-terminal)
Findings relevant to EoL
EoL gains valued more highly than
gains for non-EoL conditions
Respondent split into 2 groups
Gains in QoL at Eol more valued than LE
gains.
Some support for prioritising patients
with shorter life expectancy.
Face to face
1 year vs 10 year LE
(50)
Respondents in 2 groups
QoL at EoL more valued than LE at EoL.
Preparation for death may be
important (suddenness).
Rowen,
Brazier et al
HESG (2014)
General
population
Face to face
Shah,
Tsuchiya,
Wailoo
Eur J Health
Econ (2013)
Mode of
admin
General
population
DCE
(3669)
Life expectancy without
condition (5, 20, 40, 80)
Online
survey
LE with condition w/out
treatment
(3m-5 years, 3m-10
yrs,3m-30 yrs, 3m-60
yrs)
Source: Rachel Baker, Glasgow Caledonian University
Size of the health gain important
Coefficient for EoL (as per NICE
definition) positive and significant.
Not clearly evident in attitudinal
questions
1422 respondents possible
misunderstood tasks.

17. Institutional processes for the value
assessment of new diagnostics
Source: Garau, M., Towse, A., Garrison, L., Housman, L. and Ossa, D. (2013) Can and should
value-based pricing be applied to molecular diagnostics? Personalized Medicine. 10(1), 61-72.
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18. Need for flexible pricing and more
outcomes-based PAS
Garrison et al (2013)
Academy of Medical Sciences (2013)
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19. A reordering of process?
Criteria: broader definition of value
(risks, benefits)
Affordability
(BIA)
Safety
Value for
money
(CE)
Efficacy,
effectiveness
Overall D-M Framework:
Other factors of value to D-M (ethical issues, social values, feasibility of Opportunity costs
implementation, unmet needs, innovation value, legal issues …)
(value-for-money)
Source: Professor Ron Goeree, Director PATH Research Institute, McMaster University
P&R for oncology drugs in the UK
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20. Conclusions
1.
England’s access to innovative oncology medicines has
(hopefully) improved as a result of a series of policy initiatives
2.
Inconsistencies in policy in England exist, e.g. role of CDF
3.
Issues for HTA in assessing oncology medicines
4.
Value-based assessment will have a big potential impact on
oncology medicines and the EoL criteria
5.
Social preferences as well as cost-effectiveness are now at the
core of value assessment in HTA, but they are not easy to
establish using empirical research
6.
Other issues for the future include addressing HTA for
companion diagnostics and differential pricing
7.
Price flexibility by indication / subgroup and outcomes-based
CED/ PBRSA schemes are important to effective use of drugs
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21. References
Academy of Medical Sciences. (2013) Realising the potential of stratified medicines. London: Academy of Medical
Sciences.
Baker, R. (Glasgow Caledonian University). (2014) Plenary session presentation. Health Economists’ Study Group.
Sheffield. 8-10 January 2014.
Garau, M., Shah, K., Mason, A., Wang, Q., Towse, A. and Drummond, M. QALYs in cancer: A review of the methodological
limitations. PharmacoEconomics. 29(8), 673-685.
Garau, M., Towse, A., Garrison, L., Housman, L. and Ossa, D. (2013) Can and should value-based pricing be applied to
molecular diagnostics? Personalized Medicine. 10(1), 61-72.
Linley, W.G. and Hughes, D.A. (2012) Societal views of NICE, Cancer Drugs Fund, and value based pricing criteria for
prioritising medicines: A cross-sectional survey of 4118 adults in Great Britain. Health Economics. 22(8), 948-964.
OHE Editor. (2012) Advances in Oncology Modelling: Recent Results from POI Research. OHE News. [blog] 18 September.
Available at: http://news.ohe.org/2012/09/18/advances-in-oncology-modelling-recent-results-from-poi-research/
[Accessed 14 January 2014].
Richards, M. (2010) Extent and causes of international variations in drug usage. London: Department of Health. Available
at https://www.gov.uk/government/publications/extent-and-causes-of-international-variations-in-drug-usage.
Richards, M. (2011) Achieving world class outcomes in cancer treatment. London: Office of Health Economics/ Available at
http://www.ohe.org/publications/article/achieving-world-class-outcomes-in-cancer-treatment-3.cfm
Shah, K., Tsuchiya, A. and Wailoo, A. (2013) Valuing health at the end of life: An empirical study of public preferences.
European Journal of Health Economics. Epub ahead of print. doi: 10.1007/s10198-013-0482-3.
P&R for oncology drugs in the UK
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22. About OHE
To enquire about additional information and analyses, please contact Professor Adrian Towse at
atowse@ohe.org.
To keep up with the latest news and research, subscribe to our blog, OHE News.
Follow us on Twitter @OHENews, LinkedIn and SlideShare.
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