Condition

Pulmonary Arterial Hypertension

Clinical trials and treatment information for Pulmonary Arterial Hypertension

192K
People Affected
50
Active Trials
2,877
New Cases/Year
22K
Deaths/Year

Treatment Rankings

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

1
Treprostinil (Parenteral/Inhaled)
90% Effectiveness• 95% Confidence• 30% Safety• 1 trials• 8K participants
HIGH EvidencePoor ValueDose: Highly variable, titrated individually (e.g., IV/SC 1.25 ng/kg/min; inhaled 3-9 breaths 4 times daily)
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
30
DangerousModerateSafe

Time to Effect

1-2 months (titration)

Duration

Lifetime

Response Rate

50%

Remission Rate

7%

Number Needed to Treat (NNT)

3

Number Needed to Harm (NNH)

5

Common Side Effects:

Headache: 70%
Jaw Pain: 50%
Flushing: 40%
Nausea/Diarrhea: 30%
Injection site pain/infection (parenteral): 85%
Cough (inhaled): 35%

Annual Cost of Care

Drug Cost

$180,000

Monitoring

$10,000

Side Effects

$12,000

Total Annual

$202,000

Cost-Effectiveness

POOR

QALYs Gained

1

ICER

$400,000/QALY

Cost per Remission

$2,885,714

Cost per Responder

$404,000

Treatment Outcomes
Primary Outcomes
6-Minute Walk Distance320 meters
+8.75% (+28 meters)
Pulmonary Vascular Resistance10 Wood units
-40% (-4.0 Wood units)
N-terminal pro-brain natriuretic peptide (NT-proBNP)1500 pg/mL
-33.33% (-500 pg/mL)
Mean Pulmonary Artery Pressure (mPAP)55 mmHg
-9.09% (-5 mmHg)
Secondary Benefits
Borg Dyspnea Index at Peak Exercise6/10
-25% (-1.5 points)
CAMPHOR Activity Score20/30
-20% (-4 points)
Cardiac Index2.0 L/min/m^2
+15% (+0.3 L/min/m^2)
Common Side Effects
Headache
+70%
Jaw Pain
+50%
Flushing
+40%

Clinical Trial Phases:

Phase 3Phase 4
2
Macitentan
85% Effectiveness• 90% Confidence• 58% Safety• 20 trials• 6K participants
HIGH EvidenceModerate ValueDose: 10mg once daily
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
58
DangerousModerateSafe

Time to Effect

3-6 months

Duration

Lifetime

Response Rate

38%

Remission Rate

6%

Number Needed to Treat (NNT)

8

Common Side Effects:

Anemia: 12%
Nasopharyngitis: 10%
Headache: 10%
Peripheral Edema: 8%

Annual Cost of Care

Drug Cost

$120,000

Monitoring

$5,000

Side Effects

$1,000

Total Annual

$126,000

Cost-Effectiveness

MODERATE

QALYs Gained

0.4

ICER

$180,000/QALY

Cost per Remission

$2,100,000

Cost per Responder

$331,579

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+85%
Response Rate
+38%
Remission Rate
+6%
Common Side Effects
Anemia
+12%
Nasopharyngitis
+10%
Headache
+10%

Clinical Trial Phases:

Phase 3Phase 4
3
Riociguat
82% Effectiveness• 90% Confidence• 35% Safety• 47 trials• 2.5K participants
HIGH EvidenceModerate ValueDose: 0.5mg three times daily, titrated up to 2.5mg three times daily
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
35
DangerousModerateSafe

Time to Effect

1-3 months

Duration

Lifetime

Response Rate

35%

Remission Rate

5%

Number Needed to Treat (NNT)

4

Number Needed to Harm (NNH)

15

Common Side Effects:

Headache: 25%
Dyspepsia: 12%
Dizziness: 10%
Nausea: 10%
Hypotension: 7%

Annual Cost of Care

Drug Cost

$120,000

Monitoring

$5,000

Side Effects

$1,000

Total Annual

$126,000

Cost-Effectiveness

MODERATE

QALYs Gained

0.3

ICER

$160,000/QALY

Cost per Remission

$2,520,000

Cost per Responder

$360,000

Treatment Outcomes
Primary Outcomes
6-minute walk distance370 meters
+9.5% (+35 meters)
Pulmonary Vascular Resistance900 dyn·s·cm⁻⁵
-25% (-225 dyn·s·cm⁻⁵)
N-terminal pro-B-type natriuretic peptide1500 pg/mL
-35% (-525 pg/mL)
Secondary Benefits
Cardiac Index2.2 L/min/m²
+13.6% (+0.3 L/min/m²)
Mean Pulmonary Artery Pressure52 mmHg
-13.5% (-7 mmHg)
Borg Dyspnea Scale (peak 6MWD)3.5 points (0-10 scale)
-23% (-0.8 points)
Common Side Effects
Headache
+25%
Dyspepsia
+12%
Dizziness
+10%

Clinical Trial Phases:

Phase 3Phase 4
4
Sildenafil
80% Effectiveness• 95% Confidence• 60% Safety• 125 trials• 12K participants
HIGH EvidenceExcellent ValueDose: 20mg three times 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

Weeks to months

Duration

Lifetime

Response Rate

40%

Remission Rate

8%

Number Needed to Treat (NNT)

5

Common Side Effects:

Headache: 50%
Flushing: 25%
Dyspepsia: 18%
Nasal Congestion: 12%
Visual Disturbances: 10%

Annual Cost of Care

Drug Cost

$2,000

Monitoring

$4,500

Side Effects

$300

Total Annual

$6,800

Cost-Effectiveness

EXCELLENT

QALYs Gained

0.2

Cost per Remission

$85,000

Cost per Responder

$17,000

Treatment Outcomes
Primary Outcomes
6-Minute Walk Distance330 meters
+12.12% (+40 meters)
Pulmonary Vascular Resistance1000 dyn·s·cm⁻⁵
-25% (-250 dyn·s·cm⁻⁵)
Mean Pulmonary Artery Pressure55 mmHg
-14.5% (-8 mmHg)
Secondary Benefits
N-terminal pro-BNP (NT-proBNP)1500 pg/mL
-25% (-375 pg/mL)
Cardiac Index2.0 L/min/m²
+15% (+0.3 L/min/m²)
Borg Dyspnea Scale Score5/10
-30% (-1.5 points)
Common Side Effects
Headache
+50%
Flushing
+25%
Dyspepsia
+18%

Clinical Trial Phases:

Phase 3Phase 4
5
Tadalafil
78% Effectiveness• 95% Confidence• 60% Safety• 44 trials• 9K participants
HIGH EvidenceExcellent ValueDose: 40mg once 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

Weeks to months

Duration

Lifetime

Response Rate

35%

Remission Rate

7%

Number Needed to Treat (NNT)

6

Common Side Effects:

Headache: 30%
Flushing: 15%
Dyspepsia: 12%
Nasal Congestion: 8%
Back Pain: 8%

Annual Cost of Care

Drug Cost

$2,500

Monitoring

$4,500

Side Effects

$300

Total Annual

$7,300

Cost-Effectiveness

EXCELLENT

QALYs Gained

0.2

Cost per Remission

$104,286

Cost per Responder

$20,857

Treatment Outcomes
Primary Outcomes
6-Minute Walk Distance320 meters
+10% (+33 meters)
Pulmonary Vascular Resistance10 Wood units
-25% (-2.5 Wood units)
Mean Pulmonary Artery Pressure55 mmHg
-7% (-4 mmHg)
WHO Functional Class Score2.8 points (on 1-4 scale, 1=best)
-18% (-0.5 points)
Secondary Benefits
NT-proBNP1500 pg/mL
-25% (-375 pg/mL)
SF-36 Physical Component Summary Score35 points (on 0-100 scale)
+11% (+4 points)
Tricuspid Annular Plane Systolic Excursion (TAPSE)15 mm
+17% (+2.5 mm)
Common Side Effects
Headache
+30%
Flushing
+15%
Dyspepsia
+12%

Clinical Trial Phases:

Phase 3Phase 4
6
Bosentan
75% Effectiveness• 90% Confidence• 55% Safety• 73 trials• 7K participants
HIGH EvidenceGood ValueDose: 62.5mg twice daily for 4 weeks, then 125mg twice daily
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
55
DangerousModerateSafe

Time to Effect

1-3 months

Duration

Lifetime

Response Rate

32%

Remission Rate

5%

Number Needed to Treat (NNT)

7

Number Needed to Harm (NNH)

15

Common Side Effects:

Headache: 18%
Flushing: 8%
Peripheral Edema: 12%
Anemia: 12%
Liver enzyme elevation: 10%

Annual Cost of Care

Drug Cost

$10,000

Monitoring

$6,000

Side Effects

$800

Total Annual

$16,800

Cost-Effectiveness

GOOD

QALYs Gained

0.25

ICER

$85,000/QALY

Cost per Remission

$336,000

Cost per Responder

$52,500

Treatment Outcomes
Primary Outcomes
6-Minute Walk Distance340 meters
+10% (+34 meters)
Pulmonary Vascular Resistance850 dyn·s·cm⁻⁵
-20% (-170 dyn·s·cm⁻⁵)
Mean Pulmonary Arterial Pressure55 mmHg
-10% (-5.5 mmHg)
Cardiac Index2.1 L/min/m²
+12% (+0.25 L/min/m²)
Secondary Benefits
NT-proBNP1500 pg/mL
-30% (-450 pg/mL)
SF-36 Physical Component Summary (PCS)SF-36 PCS score: 38 points
+10% (+3.8 points)
Common Side Effects
Headache
+18%
Flushing
+8%
Peripheral Edema
+12%

Clinical Trial Phases:

Phase 3Phase 4