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Use of Composite End Points in Early and Intermediate Age-Related Macular Degeneration Clinical Trials: State-of-the-Art and Future Directions.
Terheyden, Jan Henrik; Schmitz-Valckenberg, Steffen; Crabb, David P; Dunbar, Hannah; Luhmann, Ulrich F O; Behning, Charlotte; Schmid, Matthias; Silva, Rufino; Cunha-Vaz, José; Tufail, Adnan; Weissgerber, Georges; Leal, Sergio; Holz, Frank G; Finger, Robert P.
Affiliation
  • Terheyden JH; Department of Ophthalmology, University Hospital Bonn, Bonn, Germany.
  • Schmitz-Valckenberg S; Department of Ophthalmology, University Hospital Bonn, Bonn, Germany.
  • Crabb DP; John A. Moran Eye Center, University of Utah, Salt Lake City, Utah, USA.
  • Dunbar H; Division of Optometry and Visual Sciences, School of Health Sciences, City University of London, London, United Kingdom.
  • Luhmann UFO; UCL Institute of Ophthalmology, London, United Kingdom.
  • Behning C; Roche Pharmaceutical Research and Early Development, Translational Medicine Ophthalmology, Roche Pharma Research and Early Development, Roche Innovation Center, Basel, Switzerland.
  • Schmid M; Institute for Medical Biometry, Informatics and Epidemiology, University Hospital Bonn, Bonn, Germany.
  • Silva R; Institute for Medical Biometry, Informatics and Epidemiology, University Hospital Bonn, Bonn, Germany.
  • Cunha-Vaz J; Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal.
  • Tufail A; University of Coimbra, Coimbra Institute for Clinical and Biomedical Research (iCBR), Faculty of Medicine, Coimbra, Portugal.
  • Weissgerber G; Department of Ophthalmology, Centro Hospitalar e Universitário de Coimbra (CHUC), Coimbra, Portugal.
  • Leal S; Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal.
  • Holz FG; Moorfields Eye Hospital, London, United Kingdom.
  • Finger RP; Novartis Pharma AG, Basel, Switzerland.
Ophthalmologica ; 244(5): 387-395, 2021.
Article in En | MEDLINE | ID: mdl-33285549
ABSTRACT
The slow progression of early age-related macular degeneration (AMD) stages to advanced AMD requires the use of surrogate end points in clinical trials. The use of combined end points may allow for shorter and smaller trials due to increased precision. We performed a literature search for the use of composite end points as primary outcome measures in clinical studies of early AMD stages. PubMed was searched for composite end points used in early/intermediate AMD studies published during the last 10 years. A total of 673 articles of interest were identified. After reviewing abstracts and applicable full-text articles, 33 articles were eligible and thus included in the qualitative synthesis. The main composite end point categories were combined structural and functional end points, combined structural end points, combined functional end points and combined multicategorical end points. The majority of the studies included binary composite end points. There was a lack of sensitivity analyses of different end points against accepted outcomes (i.e., progression) in the literature. Various composite outcome measures have been used but there is a lack of standardization. To date no agreement on the optimal approach to implement combined end points in clinical studies of early stages of AMD exists, and no surrogate end points have been accepted for AMD progression.
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Full text: 1 Database: MEDLINE Main subject: Macular Degeneration Type of study: Diagnostic_studies / Prognostic_studies / Qualitative_research / Systematic_reviews Limits: Humans Language: En Journal: Ophthalmologica Year: 2021 Type: Article Affiliation country: Germany

Full text: 1 Database: MEDLINE Main subject: Macular Degeneration Type of study: Diagnostic_studies / Prognostic_studies / Qualitative_research / Systematic_reviews Limits: Humans Language: En Journal: Ophthalmologica Year: 2021 Type: Article Affiliation country: Germany