A randomized controlled trial analyzes all patients in the groups to which they were originally assigned, regardless of whether they actually took their allocated drug or crossed over to the other arm. The main purpose of this approach is to:
- A Preserve the benefits of randomization and reflect real-world effectiveness ✓
- B Estimate efficacy under ideal adherence conditions
- C Maximize the statistical power of the trial
- D Eliminate the need for a placebo group
Correct answer: A. Preserve the benefits of randomization and reflect real-world effectiveness
Explanation
Intention-to-treat analysis keeps every participant in the original randomized group, preserving the balance in known and unknown confounders that randomization created. It answers the pragmatic effectiveness question of what happens once treatment is assigned in routine practice, including imperfect adherence. Per-protocol analysis estimates efficacy under ideal adherence but sacrifices the protection offered by randomization through self-selection of adherent patients.
Reference: Gordis Epidemiology, 6th ed.
High-yield for: NEET PGINI-CETNExTFMGEUSMLEPLABMRCP
Written and medically reviewed by the StethoPrep medical team.