Condition

Emphysema

Lung condition causing shortness of breath

200M
People Affected
70
Active Trials
4M
New Cases/Year
3.5M
Deaths/Year

Treatment Rankings

Ranked by evidence volume first. Medicine has spent centuries ranking things by vibes. This is an improvement.

1
Pulmonary Rehabilitation
88% Effectiveness• 95% Confidence• 95% Safety• 34 trials• 25K participants
HIGH EvidenceExcellent ValueDose: 2-3 sessions per week, 1-2 hours per session
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
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95
DangerousModerateSafe

Time to Effect

6-12 weeks

Duration

6-12 weeks intensive, then maintenance

Response Rate

78%

Remission Rate

0%

Number Needed to Treat (NNT)

4

Common Side Effects:

Musculoskeletal discomfort/injury (minor): 8%
Fatigue (during sessions): 15%

Annual Cost of Care

Drug Cost

$0

Monitoring

$3,000

Side Effects

$50

Total Annual

$3,050

Cost-Effectiveness

EXCELLENT

QALYs Gained

0.4

ICER

$20,000/QALY

Cost per Responder

$3,935

Treatment Outcomes
Primary Outcomes
6-Minute Walk Distance (6MWD)300 meters
+15% (+45 meters)
St. George's Respiratory Questionnaire (SGRQ) Total Score55/100 points
-10% (-5.5 points)
Modified Medical Research Council (mMRC) Dyspnea Scale2.5/4 points
-20% (-0.5 points)
Quadriceps Strength (Peak Torque)50 Nm
+15% (+7.5 Nm)
Secondary Benefits
Hospitalizations for COPD Exacerbations1.5 exacerbations/year
-33.3% (-0.5 exacerbations/year)
Hospital Anxiety and Depression Scale (HADS) - Anxiety Subscale10/21 points
-20% (-2 points)
Arm Ergometry Peak Workload20 watts
+25% (+5 watts)
Common Side Effects
Musculoskeletal discomfort/injury (minor)
+8%
Fatigue (during sessions)
+15%

Clinical Trial Phases:

Phase 4
2
Oxygen Therapy (long-term)
90% Effectiveness• 95% Confidence• 95% Safety• 2 trials• 10K participants
HIGH EvidenceExcellent ValueDose: 1-5 liters/minute via nasal cannula
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
95
DangerousModerateSafe

Time to Effect

immediate for oxygenation, months-years for survival

Duration

lifetime

Response Rate

95%

Remission Rate

0%

Number Needed to Treat (NNT)

3

Common Side Effects:

Nasal irritation/dryness: 25%
Skin breakdown (from cannulas): 5%
Fire hazard (rare): 0.01%
CO2 retention (if flow too high): 3%

Annual Cost of Care

Drug Cost

$0

Monitoring

$4,800

Side Effects

$100

Total Annual

$4,900

Cost-Effectiveness

EXCELLENT

QALYs Gained

0.8

ICER

$25,000/QALY

Cost per Responder

$5,158

Treatment Outcomes
Primary Outcomes
Arterial Oxygen Partial Pressure (PaO2)50 mmHg
+30% (+15 mmHg)
2-year Mortality Rate40% (without LTOT)
-25% (-10 percentage points)
Mean Pulmonary Arterial Pressure (PAP)28 mmHg
-14.3% (-4 mmHg)
6-Minute Walk Distance (6MWD)300 meters
+13.3% (+40 meters)
Secondary Benefits
St. George's Respiratory Questionnaire (SGRQ) Total Score55 points (range 0-100)
-9.1% (-5 points)
Respiratory Hospitalization Rate1.5 hospitalizations/year
-20% (-0.3 hospitalizations/year)
Hematocrit52%
-9.6% (-5 percentage points)
Common Side Effects
Nasal irritation/dryness
+25%
Skin breakdown (from cannulas)
+5%
Fire hazard (rare)
+0.01%

Clinical Trial Phases:

Phase 4
3
Umeclidinium/Vilanterol (LABA/LAMA combination)
75% Effectiveness• 90% Confidence• 65% Safety• 50 trials• 20K participants
HIGH EvidenceGood ValueDose: 62.5 mcg umeclidinium / 25 mcg vilanterol once daily
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
65
DangerousModerateSafe

Time to Effect

within 15 minutes

Duration

lifetime

Response Rate

75%

Remission Rate

0%

Number Needed to Treat (NNT)

12

Number Needed to Harm (NNH)

40

Common Side Effects:

Upper respiratory tract infection: 9.5%
Nasopharyngitis: 7.5%
Headache: 5.5%
Cough: 3.5%
Dry mouth: 2.5%
Urinary tract infection: 2.5%

Annual Cost of Care

Drug Cost

$6,000

Monitoring

$800

Side Effects

$100

Total Annual

$6,900

Cost-Effectiveness

GOOD

QALYs Gained

0.25

ICER

$65,000/QALY

Cost per Responder

$9,200

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+75%
Response Rate
+75%
Common Side Effects
Upper respiratory tract infection
+9.5%
Nasopharyngitis
+7.5%
Headache
+5.5%

Clinical Trial Phases:

Phase 3Phase 4
4
Tiotropium (LAMA)
70% Effectiveness• 90% Confidence• 65% Safety• 1 trials• 50K participants
HIGH EvidenceGood ValueDose: 18 mcg once daily
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
65
DangerousModerateSafe

Time to Effect

within 30 minutes

Duration

lifetime

Response Rate

65%

Remission Rate

0%

Number Needed to Treat (NNT)

12

Number Needed to Harm (NNH)

7

Common Side Effects:

Dry mouth: 15%
Upper respiratory tract infection: 4.5%
Pharyngitis: 2%
Sinusitis: 1.5%
Urinary tract infection: 1.5%
Dyspepsia: 1.5%

Annual Cost of Care

Drug Cost

$5,000

Monitoring

$800

Side Effects

$100

Total Annual

$5,900

Cost-Effectiveness

GOOD

QALYs Gained

0.2

ICER

$50,000/QALY

Cost per Responder

$9,077

Treatment Outcomes
Primary Outcomes
FEV1 (trough post-bronchodilator)1.2 L
+12.5% (+0.15 L)
Annualized Rate of Moderate-to-Severe COPD Exacerbations1.2 exacerbations/year
-25% (-0.3 exacerbations/year)
Dyspnea (Modified Medical Research Council (mMRC) score)2.0 points (on 0-4 scale)
-25% (-0.5 points)
Health-related Quality of Life (SGRQ total score)48 points (on 0-100 scale)
-9.4% (-4.5 points)
Secondary Benefits
Daily Rescue Medication Use3.5 puffs/day
-28.6% (-1.0 puffs/day)
Exercise Endurance (Six-Minute Walk Distance)350 meters
+7.1% (+25 meters)
Inspiratory Capacity (IC)2.0 L
+10% (+0.2 L)
Common Side Effects
Dry mouth
+15%
Upper respiratory tract infection
+4.5%
Pharyngitis
+2%

Clinical Trial Phases:

Phase 3Phase 4
5
Fluticasone/Salmeterol (ICS/LABA combination)
65% Effectiveness• 85% Confidence• 60% Safety• 80 trials• 40K participants
HIGH EvidenceModerate ValueDose: Fluticasone propionate 250 mcg / salmeterol 50 mcg, twice daily
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
60
DangerousModerateSafe

Time to Effect

within 10-20 minutes for bronchodilation, weeks for anti-inflammatory effects

Duration

lifetime

Response Rate

55%

Remission Rate

0%

Number Needed to Treat (NNT)

11

Number Needed to Harm (NNH)

40

Common Side Effects:

Oral candidiasis (thrush): 7%
Upper respiratory tract infection: 9%
Headache: 7.5%
Pneumonia: 4%
Pharyngitis: 6.5%
Bronchitis: 5.5%

Annual Cost of Care

Drug Cost

$6,000

Monitoring

$800

Side Effects

$100

Total Annual

$6,900

Cost-Effectiveness

MODERATE

QALYs Gained

0.15

ICER

$100,000/QALY

Cost per Responder

$12,545

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+65%
Response Rate
+55%
Common Side Effects
Oral candidiasis (thrush)
+7%
Upper respiratory tract infection
+9%
Headache
+7.5%

Clinical Trial Phases:

Phase 3Phase 4
6
Alpha-1 Antitrypsin Augmentation Therapy
55% Effectiveness• 80% Confidence• 70% Safety• 12 trials• 5K participants
MODERATE EvidencePoor ValueDose: 60 mg/kg body weight intravenously once weekly
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
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70
DangerousModerateSafe

Time to Effect

immediate maintenance of AAT levels, years for clinical benefit

Duration

lifetime

Response Rate

45%

Remission Rate

0%

Number Needed to Treat (NNT)

8

Number Needed to Harm (NNH)

75

Common Side Effects:

Headache: 15%
Dizziness: 7%
Nausea: 7%
Infusion reactions (skin rash, chills, fever): 3%

Annual Cost of Care

Drug Cost

$100,000

Monitoring

$2,000

Side Effects

$500

Total Annual

$102,500

Cost-Effectiveness

POOR

QALYs Gained

0.1

ICER

$500,000/QALY

Cost per Responder

$227,778

Treatment Outcomes
Primary Outcomes
Annualized Rate of FEV1 DeclineAnnual FEV1 decline: 70 mL/year
-28.6% (-20 mL/year (slowing of decline))
Annualized Rate of Lung Density Decline (15th percentile MLD)Annual rate of lung density decline: 2.2 g/L/year
-40.9% (-0.9 g/L/year (slowing of decline))
Serum Alpha-1 Antitrypsin Trough Levels30 mg/dL
+233.3% (+70 mg/dL)
Secondary Benefits
St. George's Respiratory Questionnaire (SGRQ) ScoreSGRQ score: 55 points (higher indicates worse HRQoL)
-4.5% (-2.5 points)
6-Minute Walk Distance (6MWD)350 meters
+2.9% (+10 meters)
Annualized Exacerbation Rate2.0 exacerbations/year
-15% (-0.3 exacerbations/year)
Common Side Effects
Headache
+15%
Dizziness
+7%
Nausea
+7%

Clinical Trial Phases:

Phase 3Phase 4