In this episode of “In the Interim…”, Dr. Scott Berry recounts a presentation he gave to the Speak Foundation, a patient organization focused on limb-girdle muscular dystrophy and makes the case for master protocols directly to the stakeholder trials exist to serve. Opening with the deliberately absurd premise of a sports league that constructs a four-billion-dollar stadium for every single game and demolishes it at the final whistle, Scott reframes the conventional one-off trial as a two-year construction project dismantled the moment the contest ends. The episode distinguishes platform trials and basket trials as standing “arenas,” explains the modular role of the master protocol described in Woodcock and LaVange’s 2017 New England Journal of Medicine paper, and draws on worked examples including I-SPY 2, the HEALEY ALS Platform Trial, GBM AGILE, Precision Promise, and the ROAR basket trial. Scott takes each stakeholder in turn from the treatment arm sponsor, the site, the regulator, and the patient. Scott lays out how three-to-one randomization lets an arm fund 133 patients while reading out inferences with more than 200, how 667 patients in a single platform can do the work of 1,000 across five separate trials, and why far fewer patients are assigned to placebo. He closes on the genuine bottleneck: the first arena is the heavy lift, and in rare disease it is frequently patients themselves who make it happen.

Key Highlights

• The one-game stadium analogy of two years to build a trial, one contest, then demolition.
• Platform trials and basket trials as standing arenas for multiple arms and multiple subtypes.
• Master protocols as modular documents, with arms plugging in and out as appendices.
• Worked examples from I-SPY 2, HEALEY ALS, GBM AGILE, Precision Promise, and the ROAR basket trial.
• A stakeholder-by-stakeholder view: arm sponsor, site, regulator, and patient.
• 667 patients in one platform versus 1,000 across five separate trials, with far fewer on placebo.
• Bayesian borrowing across 39 limb-girdle subtypes, and the rare-disease shots never otherwise taken.

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