Gene therapy has the promise of being one and done, but it isn’t always that way for all patients, which leaves questions about where these therapies fit in the commercial landscape, said Charles C. Wykoff, MD, PhD, of Retina Consultants of Texas and the Blanton Eye Institute at Houston Methodist Hospital.

Gene therapy has the promise of being one and done, but it isn’t always that way for all patients, which leaves questions about where these therapies fit in the commercial landscape, explained Charles C. Wykoff, MD, PhD, director of research at Retina Consultants of Texas; chair of research, Retina Consultants of America; and deputy chair of ophthalmology for the Blanton Eye Institute, Houston Methodist Hospital.

Wykoff presented data on RGX-314, a gene therapy being developed to treat wet age-related macular degeneration, during the Angiogenesis, Exudation, and Degeneration 2023 meeting.

Transcript

How do we balance the lower treatment burden but higher cost of gene therapy with the lower cost but higher treatment burden of other therapies?

Gene therapy certainly holds the promise of being one and done. But as we've seen in this data set, while many patients do appear to be one and done through 6 months, some are not, and they need ongoing therapy. Even if we are using gene therapy, it's important to realize that these patients will continue to need retinal care and retinal follow up. They will still need to come back to the clinic. You're looking for signs of efficacy, you're monitoring them for safety, you're making sure that they get any retreatments if they need them. Of course, there's a host of other retinal issues that may come up in these patients. They're going to continue to need retina care, certainly, even in the context of gene therapy.

Then you bring up an important topic: cost is always a consideration. Many insurers now use prior authorizations and step therapies. Where this will fit into that paradigm, I think still has yet to be defined. Part of how that's going to be defined will be what the data looks like at the pivotal trial stage. We have very good long-term data now from the phase 1/2 data out many years for these patients, and many of them very, very well, needing very few retreatments, if any, and none in some cases. This phase 2 data adds to that positive data set.

But we really need a comprehensive, pivotal trial data set to really gauge what this what this potential gene therapy is going to look like in the commercial landscape. From there, I think, the various stakeholders can decide where this fits on the financial scale, and certainly where it fits in the in the issue of step therapy and prior authorizations.