N-of-1 Clinical Trials: A Scientific Approach to Personalized Medicine for Patients with Rare Retinal Diseases Such as Retinitis Pigmentosa

Marco A. Zarbin, Gary Novack

Research output: Contribution to journalReview articlepeer-review

Abstract

N-of-1 trials are randomized, prospective, controlled, multiple crossover trials in a single patient. Effects of one or more treatments are studied by following individual patients who receive alternative treatments (eg, therapeutic intervention). Such trials may provide a path to assess treatments for rare diseases with rigor equal to or greater than that afforded by parallel group randomized clinical trials provided that the condition is reasonably stable during the trial and has a sign/symptom that responds reversibly to the therapy and that can be measured repeatedly. In this article, the authors propose that N-of-1 trials may improve the feasibility and affordability of clinical trials for patients with rare inherited retinal diseases.

Original languageEnglish (US)
Pages (from-to)495-501
Number of pages7
JournalJournal of Ocular Pharmacology and Therapeutics
Volume37
Issue number9
DOIs
StatePublished - Nov 2021

All Science Journal Classification (ASJC) codes

  • Ophthalmology
  • Pharmacology
  • Pharmacology (medical)

Keywords

  • clinical trial design
  • retinitis pigmentosa
  • statistics

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