The journey to
sustainable and
widespread
improvement –
medicines matter
Prof Dyfrig Hughes PhD MRPharmS
Centre for Health Economics and Medicines Evaluation
Bangor University
Overview
1.
Economics of medicines

New medicines
 Wasted medicines
2.
Patients’ perspective

Preferences
 Adherence
Demand
Lancet DOI:10.1016/S0140-6736(12)60240-2
Supply
(Scotland corrected to -1.3%)
(Wales corrected to -8.3%)
BMJ 2011;342 doi: 0.1136/bmj.d2982
Cost-effectiveness threshold
A = <£20,000 per QALY gained
B = >£30,000 per QALY gained
Probability of
rejection on
grounds of cost
infectiveness
Increasing cost/QALY (log scale)
Rawlins and Culyer, BMJ 2004;329:224-227
QALY is a QALY is a QALY

“An assumption that underlies most of NICE's technology
appraisals has been that “a QALY is a QALY is a QALY.”
By this NICE means that a QALY gained or lost in
respect of one disease is equivalent to a QALY gained or
lost in respect of another. It also means that the weight
given to the gain of a QALY is the same, regardless of
how many QALYs have already been enjoyed, how
many are in prospect, the age or sex of the beneficiaries,
their deservedness, and the extent to which the
recipients are deprived in other respects than health.”
Rawlins & Culyer. BMJ 2004;329:224
not
not
QALY is a QALY is a QALY
^

^
“I am uneasy about the mantra of ‘a QALY
is a QALY is a QALY.’ It means that an
increase in utility from 0.3 to 0.5 is valued
the same as an increase from 0.7 to 0.9. I
am not sure this is fair.”
Rawlins. Value in Health 2012;15:568-9
Departing from the threshold

For each £1m spent on a medicine whose
ICER is twice the threshold (e.g.
£50k/QALY):
 Gain
20 QALYs
 Lose 40 QALYs

Net population loss of 20 QALYs
Departing from the threshold

End-of-life criteria
 Weightings
applied to quality of life
experienced at the end of life for lifeextending medicines
Cancer Drugs Fund
 Ultra-orphan drugs

 Additional
allowances in recognition of ICERs
exceeding the threshold

Value-Based Pricing criteria
Value-Based Pricing: Aims to





Improve outcomes for patients through better access to
effective medicines;
Stimulate innovation and the development of high value
treatments;
Improve the process for assessing new medicines,
ensuring transparent predictable and timely decisionmaking;
Include a wide assessment, alongside clinical
effectiveness, of the range of factors through which
medicines deliver benefits for patients and society;
Ensure value for money and best use of NHS resources.
Value-Based Pricing: Criteria

Society may place a greater weight on treating particularly
severe or life threatening conditions - SEVERITY

The current system [of appraisal] may not fully reflect
society’s preferences if there are no existing alternative
treatments and so a significant unmet need – UNMET NEED

A treatment representing a significant breakthrough … could
also be represented by a qualitative assessment of the
innovation reported by a new medicine reflecting, for example,
new modes of action - INNOVATION

Impacts of a product beyond direct health effects e.g. benefits
related to reduced reliance on carers and other wider societal
factors – NON-HEALTH-RELATED BENEFITS
Aligned with public preferences?
Cross sectional survey (n=4,118)
 Asked respondents to choose between
competing hypothetical patient groups

Criterion
Comparison
Rationale
Children
Children vs. adults
NICE
Disease rarity
Common disease vs. rare disease
ACNSS;
AWMSG; SMC
Disease severity
Severe disease vs. moderate disease
NICE; VBP
Unmet need
Several other treatment options available
vs. none
VBP
Cancer
Cancer vs. non-cancer disease
CDF
End of Life
treatment
Short life expectancy (18mths) vs. Longer
life expectancy (60mths)
NICE;
AWMSG
Disadvantaged
populations
Disadvantaged patient populations vs. Nondisadvantaged populations
NICE
Innovative
Medicine works in similar way to others vs.
Medicine works in a new way
NICE; VBP
Wider societal
benefits
Patients reliant on carers vs. patients not
reliant on carers
VBP
Results

Funding preferences exists for:
 Severe
disease
 Medicines that address unmet needs
 Medicines having wider societal benefits
 Medicines that work in new way, but only when
coupled with considerable improvement in health

No funding preference for other criteria
Policy implications

Value-based pricing
 All
4 proposed criteria for rewarding new
medicines with higher prices are supported

Cancer Drugs Fund
 Not

supported
Medicines for rare diseases
 Policies
that prioritise funding for rare diseases
are not supported

End-of-life treatments
 No
support for preferential funding allocation
Patients’ perspectives
Patients have views too!
 Most patients are non-adherent most of
the time
 Intentional non-adherence

 can
be thought as a revealed preference for a
medicine

Unintentional non-adherence
 E.g.
forgetting, cost barrier
% Adherence to
cardiovascular
medicines
Am J Med 2012; 125: 882-887
Tamoxifen: Data from
The Netherlands
Breast Cancer Res Treat. 2010 Aug;122(3):843-51
RCT of 2 vs. 5 yrs tamoxifen
J Clin Oncol. 2011 May 1;29(13):1657-63
ABC project
EU-funded “Ascertaining Barriers for
Compliance” n=2,595 patients
 Prevalence

 Antihypertensives

Determinants
 Multiple
patient-, therapy-, condition-, socialand healthcare-system-related factors
determine adherence
Netherlands (237) [24%]
10
Germany (274) [33%] 9
Austria (323) [34%]
8
Wales (323) [38%]
7
Belgium (180) [39%]
6
England (323) [41%]
5
Greece (289) [50%]
4
Poland (323) [58%]
3
■ Overall non-adherence
□ Intentional non-adherence
Hungary (323) [70%] 2
Total (2595) [44%]
1
0
0%
0%
20%
40%
60%
Non adherence
80%
100%
Main survey results
Age
Employment
Number of medicines
Dosage frequency
Self-efficacy
Barriers (TPB)
Satisfaction with practitioner
Barriers
Personal control
Concern about illness
Borrowing money
Constant
Odds Ratio
0.98***
0.74*
0.90***
1.28**
0.73***
1.10*
1.01*
1.25**
0.93**
0.96*
0.82***
34.25***
[95% CI]
0.97
0.99
0.58
0.95
0.86
0.94
1.09
1.50
0.69
0.77
1.01
1.19
1.00
1.02
1.06
1.47
0.89
0.97
0.93
0.99
0.75
0.90
12.29
95.46
Stated preference
Pay patients to adhere?
Am J Med 2012;125(9):888-96
Conclusions

Cost of new medicines set to increase
 serious
questions need to be asked about the
value of new medicines
 Many current criteria for prioritising treatments
do not reflect societal preferences

Reassuring that VBP criteria seem to be
supported
Conclusions

Patients don’t take their medicines
 Clinical
consequences
 Economic consequences

Methods for improving adherence needed
 Must
first understand the underlying reasons
He was prone to memory lapses when not taking
medication for mental health problems