Advancing clinical trials for inherited retinal diseases: Recommendations from the second monaciano symposium

The Monaciano Consortium

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations

Abstract

Major advances in the study of inherited retinal diseases (IRDs) have placed efforts to develop treatments for these blinding conditions at the forefront of the emerging field of precision medicine. As a result, the growth of clinical trials for IRDs has increased rapidly over the past decade and is expected to further accelerate as more therapeutic possibilities emerge and qualified participants are identified. Although guided by established principles, these specialized trials, requiring analysis of novel outcome measures and endpoints in small patient populations, present multiple challenges relative to study design and ethical considerations. This position paper reviews recent accomplishments and existing challenges in clinical trials for IRDs and presents a set of recommendations aimed at rapidly advancing future progress. The goal is to stimulate discussions among researchers, funding agencies, industry, and policy makers that will further the design, conduct, and analysis of clinical trials needed to accelerate the approval of effective treatments for IRDs, while promoting advocacy and ensuring patient safety.

Original languageEnglish (US)
Article number2
JournalTranslational Vision Science and Technology
Volume9
Issue number7
DOIs
StatePublished - Jun 2020

Keywords

  • Clinical trials
  • Counseling patients
  • Inherited retinal diseases
  • Natural history studies
  • Outcome measures

ASJC Scopus subject areas

  • Biomedical Engineering
  • Ophthalmology

Fingerprint Dive into the research topics of 'Advancing clinical trials for inherited retinal diseases: Recommendations from the second monaciano symposium'. Together they form a unique fingerprint.

Cite this