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Agenda
Outcomes and variability of disease and
response to Rx and lack of correlation
between outcomes
HRQOL with particular attention to newest
Xolair analysis
Pharmacoeconomics: basics, specifics and
what current data does and doesnt tell us
Asthma is a syndrome,
syndrome not a disease
The Asthma phenotype is highly variable
(clinically, pathologically and physiologically)
Whos Wrong
NAEPP Guidelines
Severe
Moderate Mild
Intermittant
None
4.8%
10.4%
13.1%
48.6%
Mild
31.9% 47.2%
60.1%
42.3%
Moderate
41.3%
36.3%
22.1% 8.1%
Severe
21.9%
5.8%
4.5%
Symptoms
0.8%
34%
21%
35%
49%
11%
2%
% Total Sample
32%
24%
% Total Sample
42%
9%
In Previous 3 months
13%
Asthma Variability:
Moderate-Severe Asthma on -Agonist Only
12 week: mean FEV1: 64%, -agonist: 4-5/day
Albuterol: 59%
Symptoms: 45%
Weeks in Category
2 of 3
and
1 nocturnal awakening
No exacerbations
No ED visits
No therapy related adverse events
all
Different
Exacerbations
or Different People
(not all exacerbations
and not all asthmatics
are the same)
Dimensions of Control
How the Disease Affects the Organism
Physiology
Symptoms (nocturnal, exercise)
Quality of life and Activities of Daily Living
Medications (adverse events, adherence)
Health Care Utilization (function of
exacerbations)
Comorbidities
Outcomes
Functional
Symptoms/Medication Use
Exacerbation
Global: QOL, ADL
Physiologic
Lung function/BHR
Progression
Pathologic (Inflammation)
Sputum eos/ eNO
Economic
Direct and indirect
unhealthy days
147 million
unhealthy
functioning
days/year
activity limitation
mentally
unhealthy
physically
unhealthy
0
6
days/month
10
12
$325
$300
$275
$250
$225
$200
$175
$150
Excellent Mean + 10
(90%-tile)
Mean
Mean - 10
Poor
(10th %tile)
Mild Asthma
ModerateSevere Asthma
FEV1
No correlation
No correlation
Some
correlation
No correlation
Some
correlation
Some
correlation
Total: 0 to 4 barriers
No inflammation
Good lung
function
No urgent visits
Low costs
I can ...
I can ...
Play ball
Stay at my friends
who has a dog
Forget my medicine
activity limitations
6
asthma symptoms
emotional function
5
environmental exposure
Clinical relevance
Score
+ 1.5
large
+ 1.0 moderate
+ 0.5
small
4
3
2
1
Higher
scores
=
less
impairment
in AQoL
0
Juniper E et al., Am Rev Respir Dis 1993
18
% patients
*P<0.05
Kishiyama JL, et al. Allergy Clin Immunol International. 2000;Suppl 2:115. Abstract.
% patients
*P<0.05
Kishiyama JL, et al. Allergy Clin Immunol International. 2000;Suppl 2:115. Abstract.
Activities Domain
all activities done
p<0.001
Emotions Domain
fear of not having medication available
p<0.01
Environment Domain
symptoms from being exposed to dust
p<0.001
Health-Care Utilization:
Omalizumab vs. Placebo
Cost of Therapy
~0.5 exacerbations/pt/year (~1 in pts on po CS) compared to pl
$3.35-$7.50
Zafirlukast
$5.71-$12.08
Sal/FP
$3.79-$9.06
Omalizumab
$523
Xolair Cost-Effectiveness:
Issues with Current Data
RCT data not representative of real-world
Overestimates placebo arm
Underestimates active drug arm
Xolair Cost-Effectiveness:
Issues with Current Data
RCT data not representative of real-world
Overestimates placebo arm
Underestimates active drug arm
Xolair Cost-Effectiveness:
Issues with Current Data
Hospitalization rate ~16% in the literature
Placebo-3%
Xolair-<1%
Indirect costs
Dollars expended by the patient, family, employer,
and/or society because of illness (including loss of
productivity and quality of life)
Total
Indirect
Direct
Costs**
Costs
Total
Costs
Meds
Am. Care
Hospital
Use
Mild
47%
7%
4%
5%
$1681
22%
$2646
Moderate
39%
7%
5%
4%
$2473
33%
$4530
Severe
19%
7%
17%
8%
$6354
46%
$12,813
Asthma
Severity
Indirect
Costs
$5.3B (US)
Work Loss
Employed $1.5B
At Home $800M
Mortality $1.8B
Physician Services
Inpatient care $110M
Office Visits $740M
Prescriptions $3.2B
Pharmacist Services
Cost to
Patient
ARQoL
Activity avoidance
Mortality
16 Asthma deaths
per day
Missing school
Missing work
Unscheduled office
visits and visits to ER
Lifestyle disruptions
have become embedded
in patient expectations
for disease
Sullivan SD, and Weiss KB, Health economics of asthma and rhinitis, I and II. Assessing the value of interventions,
Current Reviews of Allergy and Clinical Immunology, January 2001, Volume 107, No. 1&2, p. 3-8 and 203-210.
Asthma
Control
Severity
Control
Cost of Illness
Effect of Presenteeism
Effect of Presenteeism
Condition
Prevalence Productivity
Loss
Total annual
loss
Migraine
Arthritis
LBP
Allergies/sinus
Asthma
GERD
Dermatitis
Flu (past 2 wk)
Depression
12.0 %
19.7
21.3
59.8
6.8
15.2
16.1
17.5
13.9
$434,385
865,530
858,825
1,809,945
259,740
582,660
610,740
607,005
786,600
4.9%
5.9
5.5
4.1
5.2
5.2
5.2
4.7
7.6
Cost-Sharing
In an attempt to reduce costs, payors will shift
costs to patients:
consumer-driven health plans
Utilization control and influence choice
NHISSurveys
Discussion Questions
Are the current outcomes that we consider in the
treatment algorithm for asthma adequate?
If not, what else should we be considering?
What are the benefits and challenges of looking
at these other outcomes?
What endpoints would help clarify and
communicate the value proposition for Xolair?
Discussion Questions
What indirect costs are most strongly associated with
poor control of asthma symptoms?
With increasing focus on the concept of control,
should we rethink the conventional cost-effectiveness
approach for asthma interventions?
Is an outcome measure other than the symptom free-day
warranted?
Should analyses take into account the significant burden
associated with indirect costs that may be mitigated by
therapies that reduce activity limitations?