The questions your board asks, with the numbers behind them
Charts marked with a model code come from a free example model you can open in Excel. The others are marked illustrative.
Where each product fits:
1Phase IModelChoice
Run Phase II, find a partner, or shelve?
Weigh the efficacy trial against partnering before the money is committed. Running Phase II has the best expected value, $135.1M, against $41.8M for partnering, even with a 20% chance of a safety concern ending the programme.
Simulate site start-up and enrolment instead of assuming every site recruits at the planned rate. With the plan's 40 sites the last patient is in by month 18 in 15% of futures; 60 sites make it 83%.
Cost per patient, monthly running costs and the risks of a clinical hold or slow enrolment all feed the budget. The base estimate is enough in 13.5% of futures; a P80 budget needs $14.54M.
At the interim, 14 of the first 40 patients have responded. With the Phase Ib result as the prior, the chance of reaching 29 responders out of 100 is 83%, so the trial carries on.
Develop it ourselves, or license it after Phase II?
Most of the value sits in the 18.1% of futures where the drug launches. Developing alone has the higher mean, $61.5M against $45.5M for licensing, and loses money in 82% of futures against 65%.
Build peak sales from the patient funnel, the uptake curve and the competition. The base case says $924M; there is a 46% chance of passing $1 billion, and the year-five market share drives most of the range.
Launch at risk, or wait for the patent settlement?
A biosimilar of an $8B biologic, with patent litigation pending. Launching at risk has the best expected value, $130.0M, against $117.1M for waiting for a settlement and $80.9M for licensing in.
Three of the last 40 batches failed, and that record cannot pin the failure rate down. Allowing for it, 29 batches give a 95% chance of meeting launch demand; taking the rate as known would say 27.
Compare portfolios on value, pipeline and budget at once. Funding all eight projects has the highest mean NPV, $540M, but overruns the budget in every future; the four that fit the budget give $482M with a 2% chance of overrunning.
What is the difference between rNPV and a simulated NPV?
A risk-adjusted NPV weights each stage by the chance of reaching it and gives one number. A simulation keeps the outcomes apart, so you also see how often the compound loses money. In the free Phase II example the rNPV is $76.7M, and 82% of futures lose money if the company develops it alone.
Can we update the chance of success with interim data?
Yes. The free interim example combines a prior from Phase Ib with 14 responders in the first 40 patients and gives an 82.8% predictive probability of success from its 10,000 futures; the exact Beta-Binomial answer, 83.0%, is on the sheet as a check.
How many sites does a trial need?
Enough to meet the enrolment deadline at the confidence you choose. In the free recruitment example 40 sites meet the 18-month deadline in 15% of futures, and 60 sites are the fewest that meet it 80% of the time.
Can we try it on one of our own models?
Yes. ModelRisk and ModelChoice each have a free 15-day trial, and our team can go through one of your own models with you.
Bring one trial plan. We'll show you the odds.
Free 15-day trials of ModelRisk, ModelChoice and Tamara. For Pelican, we'll show it to you on risks like yours.