Evidence guide

How Clinical Trials Work

Clinical development moves from early safety and dosing to larger tests of benefit, harms and comparative value.

Phases are not grades

Phase 1 usually emphasizes safety and dose, phase 2 looks for activity and refines design, and phase 3 tests benefit and harms in larger populations. Phase 4 continues after approval.

Endpoints matter

A biomarker can change without improving how people feel, function or survive. Surrogate outcomes need independent validation.

Registration protects the question

Public trial registration and a prespecified protocol reduce the temptation to promote whichever result looked best after the data were seen.

A practical reading checklist

  1. Which phase is it?
  2. What was the primary endpoint?
  3. Was it registered before enrollment?
  4. Who funded and controlled the data?