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The three stages a new drug passes through: phase 1 finds a safe dose in a few dozen patients, phase 2 looks for signs of activity in a hundred or so, and phase 3 compares it with the standard treatment in hundreds or thousands.
Phase 1 escalates the dose in small groups until side effects limit it, defining the maximum tolerated dose and the dose for later study; in oncology, phase 1 patients have usually exhausted standard options and modern phase 1 trials often expand into hundreds of patients with early efficacy readouts. Phase 2 tests the chosen dose for response in a specific cancer, sometimes randomised and sometimes single-arm, and phase 3 is the large randomised, often blinded, comparison against the standard of care that regulators normally require for full approval. Phase 4 studies follow a marketed drug. Only around 5% of oncology drugs entering phase 1 reach approval, and around a third of phase 3 trials fail, which is a major driver of drug cost.
Shares Bayesian trial design, Absolute versus relative benefit (number needed to treat), Statistical power, sample size and re-estimation, Endpoint.
Shares Decentralised trial, Stratified randomisation, allocation concealment and minimisation, Ethics review (IRB, research ethics committee), Trial protocol and statistical analysis plan.
Shares N-of-1 trial, Estimands and intercurrent events (ICH E9(R1)), Clinical equipoise, Kaplan-Meier curve, censoring and proportional hazards.
Shares Trial registration and results reporting (ClinicalTrials.gov, EU CTR), Estimands and intercurrent events (ICH E9(R1)), Group sequential design, stopping rules and alpha spending, Trial protocol and statistical analysis plan.
Shares Statistical power, sample size and re-estimation, Data monitoring committee (DSMB, IDMC), Group sequential design, stopping rules and alpha spending, Trial lifecycle: from protocol to label.
Shares Stratified randomisation, allocation concealment and minimisation, Clinical equipoise, Endpoint, Randomised trial.
Shares Bayesian trial design, Statistical power, sample size and re-estimation, Group sequential design, stopping rules and alpha spending, Non-inferiority trial.
Shares Response-adaptive randomisation, Statistical power, sample size and re-estimation, Data monitoring committee (DSMB, IDMC), Group sequential design, stopping rules and alpha spending.