June 2020
Volume 61, Issue 7
Free
ARVO Annual Meeting Abstract  |   June 2020
Characteristics of Clinical Studies Evaluating Off-Label Indications for Novel FDA-Approved Therapeutics in Ophthalmology
Author Affiliations & Notes
  • Anand Gopal
    Department of Ophthalmology, Wills Eye Hospital, Philadelphia, Pennsylvania, United States
  • Joshua Wallach
    Department of Internal Medicine, Yale University School of Medicine, New Haven, Connecticut, United States
  • Saloni Shah
    Sidney Kimmel Medical College at Thomas Jefferson University, Philadelphia, Pennsylvania, United States
  • Carl Regillo
    Department of Ophthalmology, Wills Eye Hospital, Philadelphia, Pennsylvania, United States
  • Joseph Ross
    Department of Internal Medicine, Yale University School of Medicine, New Haven, Connecticut, United States
  • Footnotes
    Commercial Relationships   Anand Gopal, None; Joshua Wallach, None; Saloni Shah, None; Carl Regillo, Allergan (C), Allergan (F), Genentech/Roche (C), Genentech/Roche (F), Kodiak (C), Kodiak (F), Novartis (C), Novartis (F), Regeneron (F); Joseph Ross, Agency for Healthcare Research and Quality (F), Blue Cross Blue Shield Association (F), Centers of Medicare and Medicaid Services (F), Johnson and Johnson (F), Laura and John Arnold Foundation (F), Medical Device Innovation Consortium (F), Medtronic (F), National Heart, Lung and Blood Institute of the National Institutes of Health (F), U.S. Food and Drug Administration (F)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science June 2020, Vol.61, 3070. doi:
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    • Get Citation

      Anand Gopal, Joshua Wallach, Saloni Shah, Carl Regillo, Joseph Ross; Characteristics of Clinical Studies Evaluating Off-Label Indications for Novel FDA-Approved Therapeutics in Ophthalmology. Invest. Ophthalmol. Vis. Sci. 2020;61(7):3070.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : Little is understood about how novel therapeutics garner evidence for off-label (i.e., non-FDA approved) use in ophthalmology. While the US Food and Drug Administration (FDA) prohibits off-label promotion, industry may sponsor and distribute peer-reviewed studies supporting off-label indications, regardless of the studies’ rigor. We characterized and compared clinical studies of FDA-approved and off-label indications for novel therapeutics in ophthalmology and assessed differences between industry and non-industry funded studies of off-label use.

Methods : We performed a retrospective analysis of prospective clinical studies, registered on ClinicalTrials.gov or PubMed-indexed through December 2018, of novel therapeutics approved by FDA for an ophthalmologic indication between 2008 and 2012. We characterized the proportion of studies evaluating original FDA-approved indications, supplemental FDA-approved indications, and off-label indications, stratified by therapeutic and study characteristics. We used Chi-squared testing to assess differences with a corrected Type I error of 0.006.

Results : There were 7 novel therapeutics approved by FDA for an ophthamologic indication between 2008 and 2012; we identified 469 unique prospective clinical studies of these therapies. Over half (256; 54.6%) assessed the original FDA-approved indication, while 24.3% (114) assessed off-label indications. 67.0% (314) were for a single therapeutic (aflibercept) (Figure). Median number of off-label indications studied was 4 (IQR, 0-12; range 0-27). Median number of studies evaluting an off-label indication was 1 (IQR, 1-2; range 1-19). Compared to FDA-approved indication studies, off-label indication studies were smaller (p<0.0001) and less frequently used an active comparator (44.4% vs 68.1%; p=0.002). Industry-funded off-label studies did not differ statistically from non-industry funded off-label studies, but tended to be larger and more rigorously designed (Table).

Conclusions : For most therapies, few studies of any indication are conducted after FDA approval. Most off-label indications are infrequently studied. Understanding the rigor of these studies is important when considering adopting approved therapies for new uses.

This is a 2020 ARVO Annual Meeting abstract.

 

Clinical studies across time, by therapeutic and study indication.

Clinical studies across time, by therapeutic and study indication.

 

Comparison of industry vs. non-industry funded studies of off-label indications, by study characteristics.

Comparison of industry vs. non-industry funded studies of off-label indications, by study characteristics.

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