In our space, we don't have many FDA-approved drugs yet, so access isn't our main challenge. Luxturna is approved and widely reimbursed and it's actually easily accessible for appropriate patients.

Our biggest challenge is aligning around appropriate, meaningful endpoints in clinical trial design. These are rare diseases with sometimes only hundreds or thousands of appropriate patients, so we have really small clinical trials. Standard FDA endpoints for ophthalmic indications don't always apply.

For example, with optogenetics, turning the lights back on for someone who lost light perception is life-changing, but light perception isn't currently a meaningful endpoint for FDA. The agency prefers seeing lines of vision – reading letters on an eye chart – which may not be possible for some treatments. That doesn't mean meaningful, life-changing vision restoration isn't happening.

The encouraging news is we're seeing real momentum with FDA around willingness to accept alternate endpoints and work creatively with industry for rare diseases. Many CEOs in our companies report very favorable recent interactions with the agency.

Getting comfort between the agency and industry to design appropriate clinical trials for very small populations, while bringing the patient voice into discussions so meaningful outcomes for patients are reflected by the agency. That's probably the most important development for the next couple years.

In Conversation With Foundation Fighting Blindness

On Navigating NIH Uncertainty, Breakthrough Optogenetics Trials And Translating $1bn In Research Investment Into Patient Outcomes

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