Interactive Infographic

Clinical Trial Phases Explained

Understanding the rigorous process drugs go through before reaching patients - and how NAMs are improving success rates at every stage

5
Trial Phases (0-4)
6-10
Years in Trials
$1.5B
Average Cost
~10%
Overall Success
50%+
NAMs Improvement
Preclinical
10,000+ compounds tested
Phase 1
~70% proceed
Phase 2
~29% proceed
Phase 3
~58% proceed
FDA Review
~85% approved
Approved Drug
~10% overall success
0 Phase

Exploratory/Microdosing

First-in-Human Studies

Very small doses (less than 1% of therapeutic dose) given to a small number of healthy volunteers. The goal is to learn how the drug behaves in the human body without causing any pharmacological effects.

10-15
Participants
<1%
Therapeutic Dose
Few weeks
Duration

NAMs Enhancement

  • Digital twins predict microdose pharmacokinetics before human testing
  • In silico models reduce need for extensive Phase 0 studies
1 Phase

Safety & Dosage

Establishing Safe Dose Range

Tests safety, tolerability, and pharmacokinetics in healthy volunteers (or patients for cancer drugs). Researchers determine safe dosage ranges and identify side effects. This is the first real test of how the drug affects humans.

20-100
Participants
70%
Success Rate
Several months
Duration

NAMs Enhancement

  • Organ-on-chip pre-screens for cardiac and liver toxicity
  • Patient-derived organoids predict individual drug responses
  • AI models optimize starting dose selection
2 Phase

Efficacy Testing

Does the Drug Actually Work?

The critical "proof of concept" phase where drugs are tested on patients with the target condition. This is where most drugs fail - only about 29% proceed to Phase 3. Researchers assess whether the drug has therapeutic benefit and refine dosing.

100-500
Participants
29%
Success Rate
1-2 years
Duration

NAMs Enhancement

  • Disease-specific organoids screen for efficacy before trials
  • Digital twins enable virtual patient population testing
  • Biomarker-guided patient selection improves response rates
  • Multi-organ chips detect drug-drug interactions
3 Phase

Large-Scale Confirmation

Pivotal Trials for Approval

Large, randomized, controlled trials that confirm efficacy, monitor side effects, compare to existing treatments, and collect data for labeling. These expensive trials are often the final hurdle before regulatory submission.

1,000-5,000+
Participants
58%
Success Rate
2-4 years
Duration

NAMs Enhancement

  • Virtual clinical trials reduce participant burden
  • Synthetic control arms from digital twin data
  • Real-world evidence integration for faster enrollment
  • Continuous monitoring via wearables and AI
4 Phase

Post-Market Surveillance

Long-term Safety Monitoring

Ongoing studies after drug approval to monitor long-term effects, discover new uses, and identify rare side effects that weren't apparent in smaller trials. This phase continues throughout the drug's market life.

Thousands+
Participants
Ongoing
Duration
Real-world
Setting

NAMs Enhancement

  • AI-powered adverse event detection from real-world data
  • Digital twins predict rare interactions in large populations
  • Continuous safety modeling with updated patient data

Historical Success Rates by Phase

70%
Phase 1
29%
Phase 2
58%
Phase 3
85%
NDA Review

Phase 2 has the lowest success rate - this is where NAMs can make the biggest impact by better predicting human efficacy before trials begin

Why Clinical Trials Fail

52%
Lack of Efficacy
Drug doesn't work as expected in humans, often due to animal model limitations
NAMs solution: Human organoids and organ-chips test efficacy in relevant human tissue
24%
Safety/Toxicity
Unexpected toxic effects in humans that weren't predicted by animal studies
NAMs solution: Human liver and cardiac organ-chips detect toxicity earlier
12%
Poor Patient Selection
Wrong patients enrolled; drug works but not in the tested population
NAMs solution: Biomarkers and digital twins identify responding subgroups
12%
Commercial/Strategic
Market changes, competitor drugs, or business decisions halt development
NAMs solution: Faster, cheaper development reduces financial risk

Experience Drug Development

Try our interactive simulations to see how clinical trials work in practice

Clinical Trial Simulator Full Drug Journey Digital Twins