A drug development team saw three paths to get to a successful launch:
1. Best Case: Test the response rate in a small cohort of 20 patients. If there are exceptional response rates, then run a larger pivotal trial using CRR and get approval based on that trial.
2. Upside: If at first this is not exceptional, add another cohort of patients. If results are now exceptional from the combined trials, run the CRR pivotal as in (1).
3. Base Case: After the second CRR cohort, run a regular Phase 3 trial using PFS/OS and register based on those results.
After modeling these three potential outcomes, it’s possible to quickly analyze the trials' timing and determine the likelihood of being first, second, or third to market based on early study outcomes. There are also NPV, POL, risks, and development costs for a particular path or the entire product.
Including all potential outcomes in the forecasting model will help the team prepare for when clinical study reality diverges from the plan.
How do you model multiple paths? Do you differentiate between decisions and outcomes?