Risk Analysis for Pharma and Life Sciences | Vose Software

Industries / Pharma and life sciences

When will the trial actually read out?

Value R&D pipelines with rNPV, and make go/no-go calls at every phase gate.

  • ModelRisk
  • ModelChoice

Duration of a Phase II trial

10,000 futures

P80 33.6 months
80%on time20%late
Months to top-line results
80% on time20% late
From the free example model PH-01 Clinical Trial Cost and Duration. 10,000 futures.

The 28-month plan holds in 12.5% of futures. With 80% confidence, top-line results come in October 2029, not May.

Clients in pharma and life sciences

  • Roche
  • Merck KGaA
  • Abbott
  • Boehringer Ingelheim
  • Fresenius
  • ALK

Questions we answer

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.

Proceed to Phase IIEV $135.1MBestPartner / Out-LicenseEV $41.8MShelve CompoundEV $0.0M

From the free example Clinical Trial Go/No-Go Decision, rolled back in ModelChoice.
2Trial designModelRisk

How many sites to enrol on time?

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%.

Deadline month 18
15%by the deadline85%late
Month the last patient is enrolled
15% by the deadline85% late

Median month 21.4 · P80 month 25.0

From the free example model PH-02 Patient Recruitment and Trial Timelines. 10,000 futures.
2Trial designModelRisk

What budget does the trial need?

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.

Base estimate $12.77M
13.5%enough86.5%over it
Total cost of the trial, US$ million
13.5% enough86.5% over it
From the free example model PH-01 Clinical Trial Cost and Duration. 10,000 futures.
3InterimModelRisk

Stop at the interim, or carry on?

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.

Success: 29 or more
17.0%falls short83.0%success
Responders out of 100 at the final analysis
17.0% falls short83.0% success
From the free example model PH-08 Bayesian Estimate of a Trial's Success Probability: the exact predictive distribution, which the model uses to check its 10,000 futures.
4ValuationModelRisk

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%.

NPV $0
82%lose money18%make money
NPV, US$ million
82% lose money18% make money

Mean $61.5M · rNPV $76.7M

From the free example model PH-03 Drug Development Risk-Adjusted NPV. 10,000 futures.
5LaunchModelRisk

Will it be a blockbuster?

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.

$1 billion
54%below $1B46%blockbuster
Peak sales, US$ million a year
54% below $1B46% blockbuster
From the free example model PH-05 Launch Sales Forecast: Peak Sales and Uptake. 10,000 futures.
5LaunchModelChoice

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.

At-Risk LaunchEV $130.0MBestWait for Patent SettlementEV $117.1MLicense-In from ManufacturerEV $80.8MDon't Enter MarketEV $0.0M

From the free example Biosimilar Launch Strategy Decision, rolled back in ModelChoice.
6SupplyModelRisk

How many batches for the launch year?

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.

24 batches, in house56.6%
26 batches, 2 bought in83.4%
27 batches, 3 bought in90.5%
29 batches, 5 bought in97.0%
27 batches, rate taken as known97.7%
From the free example model PH-06 Batch Yield and Manufacturing Capacity: the chance of meeting launch demand. 10,000 futures.
+Every projectModelRisk

Which projects should we fund?

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.

NPV $0
32%lose money68%make money
Portfolio NPV, US$ million
32% lose money68% make money

Mean NPV $540M · no launch 15% · over budget 100%

From the free example model PH-04 R&D Portfolio: Which Projects to Fund. 10,000 futures.

How it fits together

One development model, from the first patient to peak sales

Built in ModelRisk, in Excel. Each step has a free example model you can open.

  1. Step 1 · ModelRiskRecruitmentSite start-up and enrolment rates.PH-02
  2. Step 2 · ModelRiskTrial cost and timelinePhases, risks and contingency.PH-01
  3. Step 3 · ModelRiskChance of successInterim data updating the prior.PH-08
  4. Step 4 · ModelRiskPeak salesPatient funnel, uptake and competition.PH-05
  5. Step 5 · ModelRiskRisk-adjusted valueStage by stage, develop or license.PH-03

Training

Risk analysis training

Risk analysis modelling courses can focus on your industry and on the problems you model, online or face-to-face.

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Consulting

Bring one model

Our consultants review one of your own models before a critical decision or a public review.

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FAQ

Common questions

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.