Demonstrating clinical efficacy is only one of the challenges companies must overcome to bring new retinal therapies to patients. Regulatory uncertainty, clinical trial design, manufacturing capacity, reimbursement, and the logistics of treatment delivery can all influence whether a promising technology reaches clinical practice, according to industry leaders speaking at the Eyecelerator meeting in New Orleans. Aleksandra V. Rachitskaya, MD, and Rachel Huckfeldt, MD, PhD, moderated the panel discussion “Emerging Innovations in Retinal Therapies,” which brought together representatives of companies developing pharmaceuticals, gene and cell therapies, retinal implants, and artificial intelligence (AI) technologies.

Anthony P. Adamis, MD, cofounder and chief scientific officer of EyeBio, a Merck company, described the differences between developing treatments at startups and large pharmaceutical companies. Smaller companies can make decisions quickly, he said, but may devote considerable resources to raising capital. Larger organizations have greater financial and technical capabilities, although decision-making involves more processes. “Each has its tradeoffs. There’s no perfect solution, but we’re seeing innovation coming out of both,” said Dr. Adamis.

Erin Kimbrel, PhD, head of cell and gene therapy research at Astellas Pharma, said companies must demonstrate a compelling value proposition early in development, particularly when competing for resources across multiple therapeutic programs. “An incremental improvement may just not be enough,” Dr. Kimbrel said.

Rethinking Clinical Trial Design

A recurring theme was the potential for AI, imaging analysis, and real-world evidence to improve clinical trial efficiency and reduce development risk. Daniela Ferrara, MD, PhD, chief medical officer of Topcon Healthcare, discussed how digital technologies could address problems such as inadequate statistical power, endpoint selection, patient recruitment, and disease heterogeneity. She said AI-based imaging analysis could help identify eligible patients, develop more sensitive endpoints, and support clinical trial design. However, regulatory considerations differ depending on how a technology is incorporated into a study, particularly when an AI-derived measurement serves as an efficacy endpoint.

Dr. Ferrara also emphasized the need for greater standardization of ophthalmic imaging data, citing the need for broad adoption of DICOM standards across medical specialties. “We have a lot to learn from radiology and cardiology and others,” she said. The discussion also addressed the limitations of conventional visual acuity endpoints for therapies intended to restore vision in patients with severe retinal degeneration.

Frank Brodie, MD, MBA, chief medical officer for ophthalmology at Science Corporation, said assessments of emerging treatments must capture functional improvements meaningful to patients. “We have to look at how our therapies will impact patients day to day, what functions we give them, what we bring back for them,” Dr. Brodie said.

Samuel B. Barone, MD, chief medical officer of Nanoscope Therapeutics, similarly encouraged developers to consider endpoints that reflect their treatments’ mechanisms of action and potential patient benefits. Dr. Adamis emphasized the importance of engaging regulators early, particularly as new technologies challenge established approaches to evaluating efficacy.

Partnerships and Manufacturing

For companies developing therapies for rare retinal diseases, panelists identified patient recruitment, natural history data, and partnerships with clinical investigators as additional challenges. Dr. Barone said smaller companies must rely on external partners whose expertise complements their internal capabilities, including contract research organizations and investigators experienced with the targeted diseases. Dr. Kimbrel pointed to Astellas’s collaborations with academic institutions and its investments in translational research to improve patient identification and disease characterization.

Manufacturing was another concern, particularly for cell and gene therapies. Dr. Kimbrel said improvements in manufacturing processes and automation have helped reduce costs and variability. “We’re chipping away at all of the arguments against it: 'Oh, it’s too expensive. It takes too long. It’s not robust enough,'” she said.

Dr. Barone noted that accelerated clinical development can create difficulties when manufacturing capacity has not advanced at the same pace, requiring companies to make substantial investments before regulatory approval. Dr. Kimbrel identified product storage, preparation, surgical training, and patient awareness as additional considerations for companies developing cell and gene therapies.

Panelists also discussed the challenges that emerge after a therapy demonstrates efficacy. Dr. Brodie described Science Corporation’s approach to commercializing its PRIMA retinal implant in Europe, where the company has received the CE mark for geographic atrophy. Beyond surgical implantation, the company has developed a rehabilitation network to help patients use the technology, including in-home services. “The patient’s journey doesn't stop at surgery,” he said.

Reimbursement planning must begin well before regulatory approval, advised Dr. Adamis, and developers must assess how new therapies could affect health care costs as well as clinical outcomes. “If it’s not reimbursed, the patients are not going to be able to access the drug,” he said.

What the Future Holds

Looking ahead, panelists cited sustained drug delivery, vision restoration, personalized treatment, and the use of retinal imaging to identify systemic diseases as areas of particular interest. Dr. Ferrara said the ability to identify systemic disease through retinal imaging could extend ophthalmology's role in patient care.

“The opportunity to make that diagnosis at scale with more confidence and earlier in disease will change health care,” she said. The discussion highlighted the challenges shared by companies developing retinal therapies. Demonstrating clinical efficacy is essential, but bringing new treatments to patients also requires addressing manufacturing, regulatory, reimbursement, and treatment delivery obstacles. RP