ideasIdea

Seamless phase 2/3 with pre-registered go rules as the default for new agents

Instead of stopping after the mid-sized trial, waiting a year, then starting the big one, run them as one study with a clear pre-agreed rule for continuing. This saves a year or more per drug.

Adaptive seamless designs where the phase 2 portion (dose or population selection) rolls into the phase 3 portion without a pause, with a pre-registered decision rule, an independent committee applying it, and appropriate statistical control of type I error. The idea is to make this the expected design for agents with a plausible registrational path, with regulators offering fast-track review of the adaptation plan.

Hypothesis

Programmes using seamless 2/3 designs will reach primary phase 3 read-out at least 12 months sooner than matched programmes with separate phases, without a higher rate of failed phase 3s.

Rationale

Operational pauses between phases account for a sizeable share of development time. Seamless designs have supported approvals in several cancers; the remaining barrier is habit and the need for early commitment to the confirmatory endpoint.

What would test it

Compare development timelines (first-in-human to pivotal read-out) for seamless versus sequential programmes in the same drug class, using registry start and completion dates.

Maturity

being tested at scale

Who has to act

industry

Cost to try

Large (over $50M)

Years to first evidence

Bottlenecks it attacks

Trial design, endpoints and cost · A phase 3 trial takes years and hundreds of millions of dollars, and often answers a question that has already moved on.

Connected

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terms

Overall survival (OS)Progression-free survival (PFS)

bottlenecks

Trial design, endpoints and cost