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Published on: September 20, 2019
TFOS DEWS II Clinical Trial Design Report
Gary D Novack1, Penny Asbell2, Stefano Barabino3
1Pharma Logic Development, San Rafael, CA, USA; Departments of Pharmacology and Ophthalmology, University of California, Davis, School of Medicine, CA, USA.
Abstract:
The development of novel therapies for Dry Eye Disease (DED) is formidable, and relatively few treatments evaluated have been approved for marketing. In this report, the Subcommittee reviewed challenges in designing and conducting quality trials, with special reference to issues in trials in patients with DED and present the regulatory perspective on DED therapies. The Subcommittee reviewed the literature and while there are some observations about the possible reasons why so many trials have failed, there is no obvious single reason other than the lack of correlation between signs and symptoms in DED. Therefore the report advocates for conducting good quality studies, as described, going forward. A key recommendation for future studies is conduct consistent with Good Clinical Practice (GCP), including use of Good Manufacturing Practice (GMP) quality clinical trial material. The report also recommends that the design, treatments, and sample size be consistent with the investigational treatment, the objectives of the study, and the phase of development. Other recommendations for pivotal studies are a priori selection of the outcome measure, and an appropriate sample size.
Insights
Developing new Dry Eye Disease (DED) therapies is challenging due to trial complexities. The report emphasizes rigorous Good Clinical Practice (GCP) and Good Manufacturing Practice (GMP) standards for future DED treatment trials.
Area of Science:
- Ophthalmology
- Clinical Trial Design
- Regulatory Science
Background:
- Developing novel Dry Eye Disease (DED) therapies faces significant hurdles, with few treatments achieving marketing approval.
- Numerous clinical trials for DED have failed, with no single clear reason identified, though a lack of sign-symptom correlation is noted.
Purpose of the Study:
- To review challenges in designing and conducting quality clinical trials for DED.
- To present the regulatory perspective on DED therapies.
- To advocate for improved trial methodologies and standards.
Main Methods:
- Literature review and analysis of challenges in DED clinical trials.
- Examination of regulatory considerations for DED therapeutics.
- Subcommittee review of trial design and execution issues.
Main Results:
- Identified lack of correlation between clinical signs and patient symptoms as a key issue in DED trials.
- Highlighted the need for high-quality studies adhering to established guidelines.
- Emphasized the importance of consistent trial design, treatment, and sample size.
Conclusions:
- Future DED trials must adhere strictly to Good Clinical Practice (GCP) and Good Manufacturing Practice (GMP).
- Consistent and appropriate study design, including outcome measure selection and sample size, is crucial for pivotal studies.
- Improved trial conduct is essential for advancing DED therapeutic development.
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