Eyeworld

WINTER 2024 | EYEWORLD | 71 C Contact Boyd: CDERTradePress@fda.hhs.gov About the physician William Boyd, MD Deputy Division Director Center for Drug Evaluation and Research U.S. FDA Silver Spring, Maryland by Liz Hillman Editorial Co-Director or a symptom, and a company may need to do an additional trial; that starts to get complex. But I think the answer to your question is we want to see two adequate, well-controlled trials demonstrating efficacy of the product. Efficacy is defined as statistical significance in sign and symptom. There are some signs of dry eye that we would accept on their own. One is complete clearing of corneal staining. We also talk about the fact that dry eye trials can be extremely difficult to demonstrate efficacy. It's the nature of the patient population and the condition being treated. We recommend the use of vehicle placebo because we would recommend a superiority trial. We would not recommend equivalence; we would not recom- mend inferiority just based on our past experi- ence and our understanding of the fact that a large number of these trials can fail even with active agents. … It doesn't mean the product doesn't work if the trial fails. There is a lot that can go wrong in trial design or selection of a patient population. It is also a more difficult in- dication than some because the patient popula- tion can be varied. You have some patients who have a lot of symptoms; you have some patients who don't have symptoms. It can be difficult to sort out, but that's why we have the recommen- dation of superiority with a placebo, and I will point out that a very effective treatment for dry eye is water. A large component of the majority of the dry eye products is water. So when you're comparing to vehicle or placebo, it's a fairly high bar. When you get success in two trials, you can be confident that the product demon- strated efficacy. EyeWorld: Is there anything else you want people to know about this topic? Dr. Boyd: If a company or industry were to come to us and have a different proposal, and after discussion with us, we found that pro- posal to be acceptable … we would consider that. You do not have to follow this guidance to get a product approved for dry eye. … I'm just pointing out these are recommendations based on our understanding of the current science and our understanding of what we've seen, but it doesn't preclude other options. I n December 2020, the FDA issued "Dry Eye: Developing Drugs for Treatment Guidance for Industry" as a draft for public com- ment. In a recent interview with EyeWorld, William Boyd, MD, Deputy Division Direc- tor with the FDA's Center for Drug Evaluation and Research, shared that the administration hopes to issue its final guidance soon. While this guidance applies mostly to industry as they design clinical trials to bring dry eye therapies to patients, Dr. Boyd said ophthalmologists should care as well. "Our hope is that the guid- ance helps industry design new trials to give the best data quality, the best current information that we have on the populations to be studied, the best information that we currently have on the endpoints. We put that in the guidance for industry, but it's our hope that an individual in ophthalmology can gain information from the package insert for the products," Dr. Boyd said. EyeWorld: Why was guidance in this area needed? Dr. Boyd: We have a policy of meeting with industry whenever they request, and frequently part of those requests are comments on how we would recommend someone design the trial. … A large number of companies were asking the same questions over and over. We give the same advice unless there is a major change in the sci- ence. If there is a major change in the science, we incorporate that in our recommendations, but the key concept of this guidance sprang from the fact that we were trying to provide recommendations for trial design for industry developing dry eye products in a single entity. EyeWorld: Can you distill some of what the guidance is in the draft regarding the need to demonstrate clinical significance vs. to a comparator placebo in signs and symptoms in two separate trials? Dr. Boyd: We recommend submission of two adequate, well-controlled trials. We want to see replication of results. For the majority of prod- ucts, they can demonstrate significance in a sign and a symptom of dry eye in a trial, then they can replicate that in a second trial. There are circumstances where a trial only shows a sign Insights on FDA guidance for developing dry eye drugs

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