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"Quality of Life Promotional Claims: Methodological Issues and Potential Solutions"
Fabricated Promotional Claim I
TICKERPEP® HELPS YOUR CHF PATIENTS GAIN MORE ACTIVE LIVES
Study Summary
Tickerpep (beateramidol) is indicated for the treatment of mild to moderate (New York Heart Association [NYHA] class II or III) heart failure to reduce the progression of disease as evidenced by cardiovascular death, cardiovascular hospitalization, or the need to adjust other heart failure medications. Most frequent side effects of Tickerpep are fatigue and dizziness. Progression of heart failure and exercise tolerance were measured in four US multicenters, double-blind, placebo-controlled studies of patients with NYHA class II-III heart failure. Secondary endpoints included NYHA classification, patient and physician global assessments, cardiovascular hospitalization, death, and health related quality of life as measured by the Living with Heart Failure (LHF) Questionnaire and the Health Utilities Index (HUI). Patients were followed for 24 months. Heart failure progression was reduced by 38% when the results were pooled among all four studies. In none of the studies did Tickerpep show a statistically significant effect on exercise tolerance. There was at least a trend toward improvement in NYHA class in all studies. Pooled changes in the Living with Heart Failure Questionnaire global score showed significant differences between placebo and Tickerpep. No differences were observed on the HUI, but the ambulation domain was statistically improved in two studies. However, the assignment of significance values to these secondary endpoints is complex. No statistical multiplicity adjustment was made in any study.
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TICKERPEP® HELPS YOUR CHF PATIENTS GAIN MORE ACTIVE LIVES
SUPPORTING DATA SUMMARY
(Note: All data are fabricated to stimulate discussion)
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Study |
N |
Duration |
Primary Endpoints |
Death at:3 |
Discontinuations (not due to death) at:4 |
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Progression of Disease1 (Risk reduction) |
Exercise Tolerance2 (Increase) |
0-12 m |
12-24 m |
0-6 m |
6-12 m |
12-24 m |
| 1 |
200 |
Upto 2 yr. |
20% |
12 |
A=6 |
A=5 |
A=5 |
A=12 |
A=19 |
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P=7 |
P=5 |
P=7 |
P=11 |
P=20 |
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| 2 |
300 |
Upto 2 yr. |
35%* |
7 |
A=8 |
A=9 |
A=6 |
A=9 |
A=14 |
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P=11 |
P=12 |
P=6 |
P=11 |
P=18 |
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| 3 |
400 |
Upto 2 yr. |
45% * |
10 |
A=10 |
A=15 |
A=11 |
A=15 |
A=32 |
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P=13 |
P=19 |
P=13 |
P=20 |
P=47 |
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| 4 |
400 |
Upto 2 yr. |
36% * |
9 |
A=10 |
A=11* |
A=17 |
A=21 |
A=43 |
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P=17 |
P=20 |
P=12 |
P=25 |
P=55 |
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| 1. | Progression of Disease is defined as cardiovascular death, cardiovascular hospitalization, or the need to adjust other heart failure medications. Risk reduction is measured as compared to placebo. |
| 2. | Exercise Tolerance is defined as increase in exercise time (seconds), calculated as difference between active (Tickerpep) and placebo. |
| 3. | Number of patients with A = active (Tickerpep); P = placebo |
| 4. | Number of patients with A = active (Tickerpep); P = placebo; Reasons for discontinuations include lost to follow-up, side effect, disease progression, and patient decision. |
| * | p<0.05 compared to placebo |
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Study |
N |
NYHA class1 |
Patient Global Assessment2 |
Physician Global Assessment2 |
LHF Questionnaire3 |
HUI4 |
HUI Ambulation Domain5 |
| 1 |
200 |
A=51% |
A=39% |
A=38% |
9 |
0.08 |
7% |
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P=39% |
P=33% |
P=29% |
p=0.11 |
p=0.33 |
p=0.11 |
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| 2 |
300 |
A=49% |
A=31% |
A=27% |
11 |
0.09 |
10% |
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P=40% |
P=25% |
P=22% |
p=0.06 |
p=0.23 |
p=0.04 |
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| 3 |
400 |
A=59% |
A=39% * |
A=37% |
11 |
0.04 |
6% |
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P=50% |
P=29% |
P=28% |
p=0.04 |
p=0.27 |
p=0.11 |
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| 4 |
400 |
A=54% |
A=34% |
A=35% |
13 |
0.10 |
10% |
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P=45% |
P=26% |
P=29% |
p=0.001 |
p=0.09 |
p=0.02 |
| 1. | Percent of patients remained at the same class (most patients) or improved one class (few); A = active (Tickerpep); P = placebo |
| 2. | Patient and physician global assessment are summarized as percent of patient with improvement from baseline; A = active (Tickerpep); P = placebo |
| 3. | Living with Heart Failure (LHF) Questionnaire was measured at baseline, month 6, 12, and 24, or upon discontinuation. Data are summarized as difference from placebo. Primary time point of assessment for health-related quality of life was based on month 24 measurement; last observation carried forward (LOCF) used. Baseline was 35 |
| 4. | Health Utility Index (HUI) was measured at baseline, month 6, 12, and 24, or upon discontinuation. Data are summarized as difference from placebo. Primary time point of assessment for health-related quality of life was based on month 24 measurement; last observation carried forward (LOCF) used. Baseline was 0.6 |
| 5. | Health Utility Index (HUI) Ambulation Domain difference is the proportion of patients reporting level 1or 2 problem on the domain. At baseline approximately 30% of patients reported level 1 or 2 problems for the HUI ambulation domain. |
| * | p<0.05 compared to placebo |
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