Cost Analysis Results

1. Initial Search

Check for any company-disclosed trial costs via web search

2. Core Model

Apply Moore 2020 methodology using two key cost drivers:

• Number of patients (from clinicaltrials.gov)
• Visits per patient (estimated via protocol review)
• Values are input into the statistical model developed in the paper for estimating trial costs (R sq = 0.7, F=257.9, p<0.01)

3. Adjustments

• Protocol complexity multiplier (high/medium/low assessment)
  • Multiplier calculated by IQR of cost/patient / median cost/patient; low multiplier uses 25% quartile, high uses 75%
  • Applies 50% shrinkage factor because some of trial complexity can be explained by visits / patient
• Phase and therapeutic area multipliers (Moore 2020, Sertkaya 2016 data)
  • Moore only estimates pivotal trial costs
  • To get Phase 1 or 2 trial costs, we apply a phase multipler using Phase X/Phase 3 cost ratio from Sertkaya
  • Therapeutic area multiplier based on average cost/patient for target therapeutic area / average cost/patient for all therapeutic areas from Sertkaya
• Incremental screening visits based on screen failure rates and screening visits per patient.
• Inflation adjustment to May 2025 dollars
• Applies 2.5x CRO cost multiplier for rare diseases per Jayasundara 2019

4. Additional Costs

Add 25% overhead plus estimated study drug and comparator costs