"Unmet need" generally indicates that a particular disease cannot be adequately treated, or perhaps treated at all. In this presentation, Koonal Shah notes the definitions and the approaches to measuring "need" that have appeared in the literature. A recent exploratory empirical study also is reviewed. This research focused on the extent to which member of the general public believe that "unmet need" should be ranked high in priority in decisions that allocate health care resources.

1. "Unmet Need" as a Potential
Source of Economic Value in
Value-based Pricing Schemes
Koonal Shah
PharmAccess Leaders Forum
Berlin • 16-18 October 2013

2. Background
•
Stated objective of the UK government’s
proposed "value-based pricing" scheme:
“…to include a wide assessment, alongside
clinical effectiveness, of the range of factors
through which medicines deliver benefits for
patients and society...”
• The consultation document suggests that higher
prices will be granted to medicines that tackle
diseases that are severe or are associated with
unmet need
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3. Background
•
Lack of clarity about:
•
•
•
How these components should be defined
What evidence is needed to generate measures that
can be applied in a "value-based pricing" scheme
This presentation focuses on the definition of
unmet need and the evidence supporting the
use of unmet need as a source of value
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4. Defining "unmet need"
“Unmet need could reflect the degree to which there are
existing treatments. A condition for which there is no
effective treatment, and where there is, therefore,
significant unmet need, could be characterised by a high
QALY loss, and deemed to exhibit a high ‘Burden of Illness’.
Conversely, conditions that were already well served with
effective treatments would be scored at a lower level of this
measure – even if the untreated condition was itself severe
and life-threatening.”
VBP consultation document para 4.18
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5. Defining unmet need
•
“…‘unmet medical needs’ means a condition
for which there exists no satisfactory method
of diagnosis, prevention or treatment
authorised in the Community or, even if such a
method exists, in relation to which the
medicinal product concerned will be of major
therapeutic advantage to those affected.”
Commission Regulation (EC) No. 507/2006
(EC Regulation on the conditional marketing authorisation for medicinal
products for human use)
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6. Defining unmet need
•
Some definitions of unmet need are concerned
with whether the services are being received
rather than whether they exist – e.g. Carr and
Wolfe (1976)
•
Unmet need due to individual budget constraint
may arise for underserved individuals with low
socioeconomic status – e.g. Kataoka et al.
(2002)
•
Some researchers note that unmet need has
two dimensions: the per-patient level and the
number of patients with this level of need
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7. Unmet need in VBP schemes
Kanavos, P et al. (2009) The role of
.
funding and policies on innovation in
cancer drug development.
London: London School of Economics
and Political Science
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8. Understanding society’s
preferences
• UK VBP proposals focus on "what society
values"
• Whilst there is a growing body of evidence on
the extent to which society supports the use of
severity in health care priority setting, few
published empirical studies examine people’s
preferences regarding unmet need
• Tappenden et al. (2007) – preferences of NICE
appraisal committee members
• Green and Gerard (2009) – public preferences
• Linley and Hughes (2013) – public preferences
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9. Preference study
• Objective: to develop a greater understanding of
the extent of societal support for prioritising of
health care resources according to disease
severity and unmet need, using preference data
elicited from members of the UK general public
• By no means a definitive assessment of society’s
preferences
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10. Methods
• Respondents from the general public
• Face-to-face interviews conducted in respondents’ homes
• Survey on health care priority setting
• Seven choice tasks
•
Involved choosing between treatments for conditions which
differed in terms of unmet need and/or severity and/or
health gain from treatment
• General attitudinal questions about NHS priorities
• One budget allocation question
•
Repeat of earlier choice task but with more response options
• Open-ended comment regarding unmet need task
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11. Excerpt from questionnaire
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12. Questionnaire
Patient group A
Patient group B
Testing
what?
Life
expectancy
Life
extension
Other
treatments?
Life
expectancy
Life
extension
Other
treatments?
Q1
5
2
Yes
5
1
Yes
HG
Q2
5
1
Yes
1
1
Yes
SV
Q3
5
2
Yes
1
1
Yes
HG x SV
Q4
5
1
No
5
1
Yes
UN
Q5
5
1
No
5
1
Yes
UN+
Q6
5
1
No
1
1
Yes
UN x SV
Q7
5
1
No
5
2
Yes
UN x HG
-
-
-
-
-
-
-
-
Q10
5
1
No
5
1
Yes
UN
(but difficult
to take)
HG = health gain; SV = severity; UN = unmet need; UN+ = ‘partial’ unmet need
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13. Methods of analysis
• Distribution of responses to each question
• Comparing respondents’ responses from one
question to another
• Variety of face validity tests
• Independent selection of open-ended comments
that are of relevance to unmet need
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14. Results – sample
60 respondents (30 in London; 30 in Kent)
Characteristic
40
48
60
52
18-34
26
29
51
50
65+
Social grade
Male
35-64
Age
General
pop.
Female
Gender
%
21
21
A
0
3
B
8
20
C1
48
28
C2
22
21
DE
22
28
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15. Results – aggregate response data
Option A
Q1
Option B
Strongly
A
Slightly A
No pref
Slightly B
Strongly
B
Larger gain
Smaller gain
58%
30%
7%
2%
3%
88%
Q2
Moderately ill
Severely ill
38%
7%
15%
53%
Q3
Q4
Q5
Q6
Q7
Larger gain +
moderately ill
Smaller gain
+ severely ill
33%
Unmet need
No unmet
need
20%
Partial unmet
need
10%
Unmet need +
moderately ill
No unmet
need +
severely ill
20%
Unmet need +
smaller gain
No unmet
need + larger
gain
10%
Unmet need
20%
10%
23%
57%
18%
40%
20%
20%
37%
15%
23%
42%
15%
43%
22%
22%
5%
48%
33%
15%
15%
17%
40%
15%
22%
5%
25%
20%
27%
17%
37%
18%
20%
25%
10%
5%
10%
43%
23%
30%
53%
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16. Results – aggregate response data
Q8
I think that the NHS should give priority to
treating patients for whom there are no
other treatments available
43%
I think that the NHS should give priority to
treating patients who will get the largest
health gain from treatment
57%
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17. Results – Q10
0% to A; 100% to B
10% to A; 90% to B
20% to A; 80% to B
30% to A; 70% to B
40% to A; 60% to B
50% to A; 50% to B
60% to A; 40% to B
70% to A; 30% to B
80% to A; 20% to B
90% to A; 10% to B
100% to A; 0% to B
0%
10%
20%
30%
40%
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50%

18. Results – Q4 vs. Q10
Q10 Q4
100% A
Strongly A
Slightly A
No pref.
Slightly B
1
Strongly B
Total
1
90% A
1
1
80% A
2
70% A
4
3
1
60% A
1
3
3
50 : 50
2
6
7
60% B
1
2
1
4
8
4
23
2
2
2
80% B
2
12
12
12
1
2
5
1
2
1
90% B
Total
9
1
1
70% B
100% B
2
1
7
14
10
60
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19. Results – focus on unmet need
• Five of the choice tasks examine unmet need preferences
•
Q4 – unmet need vs. no unmet need
•
Q5 – unmet need vs. partial unmet need
–
Existing treatments are difficult to take and cause disruption to patients’ lives
•
Q6 – unmet need + less severity vs. no unmet need vs. more severity
•
Q7 – unmet need + smaller gain vs. no unmet need + larger gain
•
Q10 – unmet need vs. no unmet need (budget allocation framing)
• Whilst distribution of responses differs from task to task, none
suggests that, on average, society supports giving higher priority
to treatments that address unmet need
• But Q8 (attitudinal/opinion question) tells a different story
•
(although there remains strong evidence of a relationship between Q7 and Q8; p=0.99)
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20. Face validity of the data
•
We conducted checks of consistency across questions
•
Interviewers also reported their assessments of
respondent understand and effort
•
Interviewer assessments predicted fairly well how likely
respondents were to give inconsistent answers (as judged by
us)
•
Excluding respondents who we suspect were not
answering "properly" makes little/no difference to results
•
Both respondents who understood/concentrated and those who did
not tended to go for a 50:50 split in Q10
•
Survey administered by trained, experienced interviewers
•
Questions no more complex than those used elsewhere in
the empirical ethics literature
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21. Comments left by respondents
• 18 of the 60 respondents left comments that we
considered to be relevant in terms of unmet need
• Examples:
•
“Strong preference that if no treatments are available for
condition A – then this should be prioritised.”
•
“Being a new breakthrough treatment I thought it worth giving
slightly more of the budget.”
•
“Condition B has an alternative to choose from there is still hope
for alternative B”
•
“There should be a higher focus on finding new treatments for
patients and then on looking at treatments to replace old ones.”
•
Both conditions patients die after 5 yrs. A no treatment 5 yrs. B
Treatment 5yrs. As far as I can see the overall scenario for both
A+B is the same.”
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22. Concluding remarks
• Unmet need typically described in terms of the
availability of alternative treatment options,
though other definitions exist
• (Sparse) literature suggests that at least some
members of the society consider unmet need to
be a valid health care priority setting criterion
• Estimating the strength of society’s preferences
is challenging
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23. About OHE
To enquire about additional information and analyses, please contact Koonal Shah at
kshah@ohe.org
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