The FDA’s Indispensible Role (a guest blog from Dr. Patricia Zilliox)

To get a sight-saving treatment or therapy to the commercial market in the United States, you need three basic things: 1) scientists to discover and develop it; 2) money to support the research; and 3) Food and Drug Administration (FDA) marketing approval. While it may not seem obvious, the FDA ultimately drives everything — the researchers and the money. That’s because without FDA approval, you have no treatment, even if you have great research and adequate funding.

Of course, it would be nice if we could test a potential treatment in just four or five mice and, if it were to save vision, bypass the regulatory process and immediately get it out to everyone who needs it. But by being that hasty in the real world, we wouldn’t fight blindness at all; instead, we might make people sick, impair their vision even more, and put their lives at risk.

Therein lies the indispensable regulatory role of the FDA, its European counterpart, the European Medicines Agency(EMA), and other agencies around the world — to protect consumers by ensuring, as best as possible, that a treatment is safe and beneficial.

As the Foundation’s chief drug development officer, I have to take the FDA into consideration, directly or indirectly, in just about every decision I make. And, having worked for three decades with the FDA and the EMA, I’ve developed an understanding of what it takes to get authorization for clinical trials and marketing approvals for treatments that perform well in those studies.

So what is the FDA looking for? First and foremost, it wants to ensure that a potential treatment is safe. That means extensive testing in the lab and throughout human studies to demonstrate that the therapy is not toxic and not causing adverse events in the patients. Safety and toxicology studies must be performed and documented meticulously.

An oral drug has the potential to affect virtually every part of the body, so related safety studies must be very comprehensive. Does the drug affect the cardiac system? Is it causing an immune reaction? Does the treatment increase risk of cancer or birth defects for pregnant women? Does it lead to mood changes or mental health issues? All of these potential effects must be watched for in both lab studies and clinical trials.

Demonstrating to the FDA that a drug is saving or restoring vision is also challenging and expensive, namely because retinal diseases vary so much in their rate of progression and how they impact vision. As a result, evaluating efficacy usually requires large numbers of patients, several types of vision tests and sophisticated imaging of the retina. A recent article on the Foundation’s website describes many of the tests and imaging techniques used in clinical trials to evaluate changes in vision.

Finally, a company or institution that brings a treatment to the market must demonstrate that it is following the FDA’s Good Manufacturing Practices (GMP) to make certain that each pill, eye drop or injection is produced in a sterile environment and that there is no variation in doses, packaging and shipments. This requires specialized institutional oversight and expertise as well as rigorous production standards.

Once a treatment is FDA-approved, the company or institution distributing the drug is obligated to continue to collect and report information on side effects and adverse events. Post-marketing surveillance can reveal problems or limitations with a treatment that were not identified in clinical trials.

Ultimately, when anyone goes to a pharmacy to pick up a prescription or visit a doctor for a treatment, the FDA has overseen the development and production of those therapies at virtually every step to ensure they are safe and effective. And, as the Foundation moves treatments through clinical trials and out to the market, we and our partners are working hard to follow FDA regulations and standards, so we can get approval to deliver them to the people with retinal degenerative diseases who need them.