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Functional Vision Endpoints: OCTGT Perspective

Functional Vision Endpoints: OCTGT Perspective. Samuel B. Barone, M.D. Office of Cellular, Tissue, and Gene Therapies Center for Biologics Evaluation and Research samuel.barone@fda.hhs.gov NEI/FDA Endpoints Symposium May 6, 2011. Disclosure.

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Functional Vision Endpoints: OCTGT Perspective

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  1. Functional Vision Endpoints:OCTGT Perspective Samuel B. Barone, M.D. Office of Cellular, Tissue, and Gene Therapies Center for Biologics Evaluation and Research samuel.barone@fda.hhs.gov NEI/FDA Endpoints Symposium May 6, 2011

  2. Disclosure • No financial conflicts with any FDA regulated product

  3. FDA Organization • Office of the Commissioner • Office of Combination Products • CDRH (Center for Devices and Radiological Health): devices for treatment, implants, diagnostic devices • CBER (Center for Biologics Evaluation and Research): vaccines, blood and blood products, human tissue/tissue products for transplantation, cell therapy, gene therapy, donor screening tests for blood and tissue safety, devices • CDER (Center for Drug Evaluation and Research): drugs, monoclonal antibodies, therapeutic proteins) • CVM • CFSAN • NCTR

  4. CBER Organization • Immediate Office of Director • Office of Blood Research and Review • Office of Cellular, Tissue and Gene Therapies • Office of Vaccines Research and Review • Office of Compliance and Biological Quality • Office of Biostatistics and Epidemiology • Office of Communication, Training and Manufacturers Assistance • Office of Management

  5. OCTGT Products • Cellular therapies • Gene therapies • Tumor vaccines and immunotherapy • Devices used for cells and tissues • Human cells and tissues for transplant • Combination products

  6. OCTGT Ophtho Product Submissions 5 7 5

  7. OCTGT Ophtho Product Submissions 5 4

  8. Clinical Development Considerations • Novel products/routes of administration • Size of patient population • Age of disease onset • Disease progression • Efficacy endpoints

  9. Safety Concerns • Product • Immune response • ? Need for immunosuppressive regimen • Tumor formation • Procedure • Route of administration Johnson et al., Molecular Vision, 2008; 14: 2211–2226.

  10. Patient Population • Size • Age-related macular degeneration • 1.75 millionFriedman, Arch Ophthalmol, 2004; 122(4):564. • Stargardt disease • 30,000 Riveiro-Alvarez et al., BJO, 2009; 93(10):1359. • Leber congenital amaurosis • 4,000 Stone, AJO, 2007; 144(6):791. • Age • Pediatric population

  11. 1974 1976 Stargardt Disease 1978 1984 Natural History • Time Course • Onset • Progression • Variability • Individuals • Eyes • Lack of Predictability SR Dubovy, AFIP Ophthalmic Pathology for the Ophthalmologist, 2010.

  12. Efficacy Endpoints

  13. Assessing Vision • (Best Corrected) Visual Acuity • Visual Field • Pupillary Response • Color Vision • Contrast Sensitivity • Photostress Recovery • Electrophysiologic Testing • Imaging

  14. Vision Endpoints • Visual acuity: a 3-line (15-letter) change • clinically meaningful benefit in comparison between treatment arms or before and after treatment • Visual Field • Surrogate Endpoints • Secondary Endpoints

  15. Maguire, High, et al. • Measured improvement in retinal function: • Dark adaptometry • Pupillometry • Electroretinography • Nystagmus/eye movement measurements • Ambulatory behavior • Ability to navigate a standardized obstacle course Lancet, 2009; 374(9701): 1597-605.

  16. Effectiveness? • Endpoint not obtainable? • Low vision • No well established surrogate endpoint • Objective anatomic measurements with unknown meaning to patient • Subjective improvement

  17. Summary OCTGT Perspective • OCTGT is currently evaluating ophtho products • Potential challenges: • Small and younger disease populations • Natural history variable • New therapies/treatment modalities where safety is not as well established • Functional Vision Endpoints: • Capture meaningful subjective patient experience as an objective measurable data point • May be susceptible to bias; masking is essential

  18. Cellular, Tissue, and Gene Therapies Advisory Committee • Meeting to discuss cellular and gene therapy products for the treatment of retinal disorders • Efficacy endpoints in peds/adult populations • Repeat/second eye admin safety issues • Eval of product delivery into target site • June 29, 2011 • Crowne Plaza Hotel, Silver Spring, MD http://www.fda.gov/AdvisoryCommittees/Calendar/ucm251963.htm

  19. OCTGT Regulatory Resources • OCTGT Learn Webinar Series: http://www.fda.gov/BiologicsBloodVaccines/NewsEvents/ucm232821.htm • Regulatory Questions: Contact the Regulatory Management Staff in OCTGT at CBEROCTGTRMS@fda.hhs.govor Patrick.Riggins@fda.hhs.govor by calling (301) 827-6536

  20. More Information: www.fda.gov go Search • CBER Homepage • Search • “advisory committees” for CTGAC June 29, 2011 • “OCTGT learn” for biologicals regulatory process

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