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Ideas
Require a randomised phase 2 before any phase 3

Require a randomised phase 2 before any phase 3

Phase 3 trials are often launched on a small single-arm response-rate study with no comparison group, and most then fail. Requiring a randomised phase 2 with a concurrent control first, with exceptions only for large effects in refractory settings, would filter out weak drugs earlier.

Overview Key papers1 Connected5

Overview

Sponsors and regulators adopt an expectation that phase 3 in a new indication is preceded by a randomised phase 2 (or the phase 2 portion of a seamless design) with a concurrent control, rather than a single-arm response-rate study. Exceptions for very large single-arm effects in refractory settings.

Hypothesis

Programmes with a randomised phase 2 will have a phase 3 success rate at least 15 percentage points higher than programmes proceeding from single-arm phase 2, and overall development cost per approval will fall despite the added step.

Rationale

Response rates in single-arm studies are poor predictors of survival benefit, and phase 3 oncology failure rates are among the highest of any therapeutic area. Randomised phase 2 also yields early estimates for planning the phase 3.

Proposed test

Compare phase 3 success rates for programmes with and without randomised phase 2 across a decade of registry data, adjusting for indication and mechanism.

Maturity

early clinical

Who acts

industry

Cost to try

Medium ($1M to $50M)

Years to first evidence

3

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.
Failures are hidden · Negative trials, failed drugs and abandoned programmes are rarely published, so the same mistakes are repeated.

Key papers

observationalBMJ 2017390 citations

Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13

Two bottleneck pages and 15 idea pages on OnCo cite this paper by its DOI; this record gives the citation a page of its own so a reader can follow it without leaving OnCo. Read the abstract above alongside the citing pages listed under Related; the record was created automatically from the Europe PMC entry and its figures have not been checked by hand.

Connected

Similar pages

Idea
Seamless phase 2/3 with pre-registered go rules as the default for new agents
Shares Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13, Progression-free survival (PFS), Trial design, endpoints and cost.
Idea
Bayesian shrinkage for subgroup claims to stop false 'works in this group' stories
Shares Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13, Failures are hidden, Trial design, endpoints and cost.
Idea
Stop failing trials earlier with pre-registered aggressive futility rules
Shares Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13, Failures are hidden, Trial design, endpoints and cost.
Idea
Aggregated single-patient crossover trials for symptom and supportive treatments
Shares Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13, Trial design, endpoints and cost.
Idea
Pre-specified crossover-adjusted survival in every trial that allows crossover
Shares Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13, Trial design, endpoints and cost.
Idea
Validate real-world progression endpoints so pragmatic trials can use them
Shares Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13, Progression-free survival (PFS), Trial design, endpoints and cost.
Idea
Competing sponsors share one control arm in the same indication
Shares Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13, Trial design, endpoints and cost.
Idea
Report time toxicity, the days spent in healthcare, as a standard outcome for older patients
Shares Progression-free survival (PFS), Trial design, endpoints and cost.

terms

Objective response rate (ORR)Progression-free survival (PFS)

bottlenecks

Failures are hidden Trial design, endpoints and cost

key papers

Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13

ProvenanceLast edited 2026-09-11 · Jude Gomila · TL;DR clarity pass · diff · file

Sources & links

Bottleneck evidence (Trial design, endpoints and cost): Davis et al., Availability of evidence of benefits on survival and quality of life of cancer drugs approved by EMA 2009-13 (BMJ 2017)

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Terms

Objective response rate (ORR)
Progression-free survival (PFS)

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