Condition

Hemophilia

Genetic bleeding disorder affecting blood clotting

831K
People Affected
11
Active Trials
40K
New Cases/Year
10K
Deaths/Year

Treatment Rankings

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

1
Emicizumab
95% Effectiveness• 98% Confidence• 58% Safety• 48 trials• 3K participants
HIGH EvidenceModerate ValueDose: 3 mg/kg weekly for 4 weeks, then 1.5 mg/kg weekly (or 3 mg/kg every 2 weeks, or 6 mg/kg every 4 weeks) subcutaneous
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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

2-4 weeks

Duration

lifetime

Response Rate

98%

Remission Rate

%

Number Needed to Treat (NNT)

1.5

Number Needed to Harm (NNH)

500

Common Side Effects:

Injection site reactions: 15%
Headache: 12%
Arthralgia: 12%
Thrombotic microangiopathy/Thromboembolism (with aPCC): 0.5%

Annual Cost of Care

Drug Cost

$500,000

Monitoring

$3,000

Side Effects

$2,000

Total Annual

$505,000

Cost-Effectiveness

MODERATE

QALYs Gained

0.7

ICER

$150,000/QALY

Cost per Responder

$515,306.12

Treatment Outcomes
Primary Outcomes
Annualized Bleeding Rate (ABR) - All treated bleeds15.0 bleeds/year
-85% (-12.75 bleeds/year)
Annualized Joint Bleeding Rate (AJBR) - Treated joint bleeds8.0 bleeds/year
-90% (-7.2 bleeds/year)
Annualized Spontaneous Bleeding Rate (ASBR) - Treated spontaneous bleeds5.0 bleeds/year
-95% (-4.75 bleeds/year)
Secondary Benefits
Bypassing Agent Consumption for breakthrough bleeds50 doses/year
-90% (-45 doses/year)
Health-Related Quality of Life (HRQoL)Haemo-QoL-A score: 50/100
+15% (+7.5 points)
Hemophilia Joint Health Score (HJHS)HJHS score: 20/60
-10% (-2 points)
Common Side Effects
Injection site reactions
+15%
Headache
+12%
Arthralgia
+12%

Clinical Trial Phases:

Phase 4
2
Recombinant Factor IX
90% Effectiveness• 95% Confidence• 68% Safety• 61 trials• 30K participants
HIGH EvidenceGood ValueDose: Prophylaxis: 25-100 IU/kg 1-2 times per week; On-demand: 10-60 IU/kg
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Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
68
DangerousModerateSafe

Time to Effect

immediate

Duration

lifetime

Response Rate

95%

Remission Rate

%

Number Needed to Treat (NNT)

2

Number Needed to Harm (NNH)

50

Common Side Effects:

Inhibitor development: 3%
Allergic reactions: 0.5%
Injection site reactions: 3%

Annual Cost of Care

Drug Cost

$300,000

Monitoring

$5,000

Side Effects

$5,000

Total Annual

$310,000

Cost-Effectiveness

GOOD

QALYs Gained

0.3

ICER

$85,000/QALY

Cost per Responder

$326,315.79

Treatment Outcomes
Primary Outcomes
Annualized Bleeding Rate (ABR)
0%
Annualized Bleeding Rate (ABR) Overall
0%
Number of Participants Who Discontinued the Use of Nonacog Gamma (Genetical Recombination)
0%
Number of Participants Who Developed a Factor IX (FIX) Inhibitor
0%
Annual Bleed Rate (ABR): Number of Times of Bleeding During the Study
0%
Number of Doses to Treat A Bleed of Participants on An On-Demand Regimen
0%
Hemostatic Effectiveness of Rixubis for Participants on An On-Demand Regimen Based on a 4-Point Ordinal Scale (Excellent, Moderate, Good, Poor)1 Participants
-100% (-1.0 Participants)
Secondary Benefits
Concentration (Cmax)
0%
Area Under the Curve (0-inf)
0%
Mean Residence Time (MRT)
0%
Common Side Effects
Inhibitor development
+3%
Allergic reactions
+0.5%
Injection site reactions
+3%

Clinical Trial Phases:

Phase 4
3
Recombinant Factor VIII
90% Effectiveness• 95% Confidence• 65% Safety• 351 trials• 50K participants
HIGH EvidenceGood ValueDose: Prophylaxis: 20-50 IU/kg 2-3 times per week; On-demand: 10-50 IU/kg
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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

immediate

Duration

lifetime

Response Rate

95%

Remission Rate

%

Number Needed to Treat (NNT)

2

Number Needed to Harm (NNH)

4

Common Side Effects:

Inhibitor development: 25%
Allergic reactions: 0.5%
Injection site reactions: 3%

Annual Cost of Care

Drug Cost

$350,000

Monitoring

$5,000

Side Effects

$10,000

Total Annual

$365,000

Cost-Effectiveness

GOOD

QALYs Gained

0.3

ICER

$80,000/QALY

Cost per Responder

$384,210.53

Treatment Outcomes
Primary Outcomes
Annualized Bleeding Rate (ABR)25 episodes/year (without prophylaxis)
-85% (-21.25 episodes/year)
Annualized Joint Bleeding Rate (AJBR)18 episodes/year (without prophylaxis)
-90% (-16.2 episodes/year)
Factor VIII Trough Activity Level0.5% of normal activity (for severe hemophilia)
+900% (+4.5% of normal activity (e.g., achieving 5% trough))
Hemophilia Joint Health Score (HJHS)35 (on a 0-124 scale, higher score indicates worse joint health)
-15% (-5.25 points)
Secondary Benefits
Quality of Life (SF-36 Physical Component Summary)40 points (on a 0-100 scale, higher score indicates better quality of life)
+10% (+4 points)
Annual Days of Work/School Missed due to Bleeding15 days/year
-70% (-10.5 days/year)
Average Pain Score (Visual Analog Scale)4/10 (on a 0-10 scale, higher score indicates more pain)
-30% (-1.2 points)
Common Side Effects
Inhibitor development
+25%
Allergic reactions
+0.5%
Injection site reactions
+3%

Clinical Trial Phases:

Phase 4
4
Extended Half-Life (EHL) Recombinant Factor IX
90% Effectiveness• 92% Confidence• 68% Safety• 40 trials• 6K participants
HIGH EvidenceGood ValueDose: Prophylaxis: 50-100 IU/kg once weekly or every 10-14 days; On-demand: 10-60 IU/kg
Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
68
DangerousModerateSafe

Time to Effect

immediate

Duration

lifetime

Response Rate

95%

Remission Rate

%

Number Needed to Treat (NNT)

2

Number Needed to Harm (NNH)

70

Common Side Effects:

Inhibitor development: 2%
Allergic reactions: 0.5%
Injection site reactions: 3%

Annual Cost of Care

Drug Cost

$380,000

Monitoring

$5,000

Side Effects

$5,000

Total Annual

$390,000

Cost-Effectiveness

GOOD

QALYs Gained

0.35

ICER

$100,000/QALY

Cost per Responder

$410,526.32

Treatment Outcomes
Efficacy Outcomes
Overall Effectiveness
+90%
Response Rate
+95%
Common Side Effects
Inhibitor development
+2%
Allergic reactions
+0.5%
Injection site reactions
+3%

Clinical Trial Phases:

Phase 4
5
Extended Half-Life (EHL) Recombinant Factor VIII
90% Effectiveness• 92% Confidence• 65% Safety• 2 trials• 8K participants
HIGH EvidenceModerate ValueDose: Prophylaxis: 25-65 IU/kg 2 times per week or every 3-5 days; On-demand: 10-50 IU/kg
Find Trials

Comparative Safety Scale(Higher is safer)

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

Time to Effect

immediate

Duration

lifetime

Response Rate

95%

Remission Rate

%

Number Needed to Treat (NNT)

2

Number Needed to Harm (NNH)

8

Common Side Effects:

Inhibitor development: 10%
Allergic reactions: 0.5%
Injection site reactions: 3%

Annual Cost of Care

Drug Cost

$400,000

Monitoring

$5,000

Side Effects

$8,000

Total Annual

$413,000

Cost-Effectiveness

MODERATE

QALYs Gained

0.35

ICER

$110,000/QALY

Cost per Responder

$434,736.84

Treatment Outcomes
Primary Outcomes
Annualized Bleeding Rate (ABR)2.5 bleeds/year (on standard FVIII prophylaxis)
-64% (-1.6 bleeds/year)
Factor VIII Trough Activity1.0 IU/dL (just before next standard FVIII infusion)
+400% (+4.0 IU/dL)
Annualized Joint Bleeding Rate (AJBR)1.5 joint bleeds/year (on standard FVIII prophylaxis)
-66.7% (-1.0 joint bleeds/year)
FVIII Infusion Frequency104 infusions/year (2x weekly standard FVIII prophylaxis)
-35% (-36 infusions/year)
Secondary Benefits
Health-Related Quality of Life (Haem-A-QoL Total Score)50 points (Haem-A-QoL scale, higher score indicates worse QoL)
-10% (-5 points)
Physical Activity Level (Self-Reported)4 points (on a 0-10 scale, where 10 is highest activity)
+25% (+1.0 points)
Pain Severity (NRS)3.5 (on a 0-10 Numerical Rating Scale)
-28.5% (-1.0 points)
Common Side Effects
Inhibitor development
+10%
Allergic reactions
+0.5%
Injection site reactions
+3%

Clinical Trial Phases:

Phase 4
6
Desmopressin (DDAVP)
70% Effectiveness• 85% Confidence• 72% Safety• 8 trials• 5K participants
MODERATE EvidenceExcellent ValueDose: 0.3 mcg/kg IV or SC, or 150-300 mcg nasally; Max 2-3 doses in 24 hours
Find Trials

Comparative Safety Scale(Higher is safer)

Cyanide☠️
Meth💀
Cigarettes🚬
Chemo☢️
Alcohol🍺
Morphine💊
Antibiotics💉
Tylenol💊
Exercise🏃
Water💧
72
DangerousModerateSafe

Time to Effect

immediate to 1 hour

Duration

acute treatment

Response Rate

75%

Remission Rate

%

Number Needed to Treat (NNT)

3

Number Needed to Harm (NNH)

15

Common Side Effects:

Facial flushing: 35%
Headache: 20%
Hyponatremia: 8%
Dizziness: 8%

Annual Cost of Care

Drug Cost

$1,500

Monitoring

$200

Side Effects

$300

Total Annual

$2,000

Cost-Effectiveness

EXCELLENT

QALYs Gained

0.1

ICER

$10,000/QALY

Cost per Responder

$2,666.67

Treatment Outcomes
Primary Outcomes
Peak Factor VIII (FVIII) Activity15% of normal (0.15 IU/mL)
+200% (+30% of normal (+0.30 IU/mL))
Annualized Bleeding Rate (ABR)5 episodes/year
-60% (-3 episodes/year)
Procedures/Minor Bleeds Requiring Exogenous FVIII Concentrate70% of events requiring FVIII concentrate
-71.4% (-50% (reduction in proportion of events, from 70% to 20%))
Secondary Benefits
Hemophilia-specific Quality of Life (QoL) Score50/100 (lower score indicating worse QoL)
+15% (+7.5 points (from 50 to 57.5))
Hospitalizations Due to Bleeding0.5 admissions/year
-80% (-0.4 admissions/year)
Work/School Absenteeism Due to Bleeding5 days/year
-70% (-3.5 days/year)
Common Side Effects
Facial flushing
+35%
Headache
+20%
Hyponatremia
+8%

Clinical Trial Phases:

Phase 4