Humanity + technology: Talking to patients about the science and benefits of clinical trials

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I have been passionate about research since my time in academia when I was investigating retinopathy of prematurity. This interest has carried over into my private practice, where I have created a state-of-the-art clinical trial organization and have now been the primary investigator (PI) on close to 40 studies, most of which centered around diabetic eye disease and age-related macular degeneration (AMD). I value being connected to research and find it rewarding, and it has greatly benefited my practice, staff, and patients.

Running successful clinical trials hinges on establishing a stellar team formed by nurturing staff from within who have potential, are conscientious, and are interested in research and ophthalmology. Thanks to their professionalism and dedication, we are able to meet enrollment criteria and run trials in our practice.

We have found it exciting and gratifying to be part of the development of so many therapies that now help a countless number of retina patients. When a novel therapy that my team has studied in a clinical trial becomes FDA approved and available, our experiences and familiarity with the agent are greatly beneficial. We can talk to patients with the utmost confidence and share that we are comfortable administering that agent.

Clinical trial as a care option

When it comes to talking to patients about clinical trials as a care option, I have found it is often easier to engage those with diabetes than it is patients with macular degeneration. For the latter condition, enrollment criteria usually require patients to have untreated disease. This is typically not the case with diabetic eye disease (DED) studies. Many times, patients with DED have been down the road of standard of care, are struggling, and are eager to try something different—especially if it might improve their vision.

Patients with AMD who are eligible for trials may just be finding out they have the disease. To be told they need multiple intravitreal injections for an unspecified amount of time to stave off possible vision loss is understandably overwhelming. Then having them agree to come back for screening and undergo the qualifying process associated with a clinical trial can be a challenge. Many prefer to begin treatment as soon as possible.

Yet, many patients with AMD participate because they are driven to try to make the best of a bad situation. They often ask, “Why did I get this?” When I tell them, “It is 70% genetic,” they think about their children and are motivated by a desire to protect them. Not only are we as physicians and researchers thinking about how we can provide better care for the next group of patients, but they are too.

Spotting a good study participant

Patients who are more hands-off and want as little treatment as possible do not make good study patients, and they are easily identified. Some are more in the middle, and I might let them know there is standard of care but also a research option. I tell them briefly about the opportunity and gauge their interest. If patients say, “So I’ll be your guinea pig,” I know not to pursue them further.

Good candidates will indicate interest and say they want to investigate it further and talk to their family. From there, I provide more information and pass them on to my clinical study coordinator, who sits down with them to really dive in. Patients in the third group jump at the opportunity as soon as I mention it is an option.

We have a steady group of patients who want to enroll in another study once they have finished the previous one, underscoring how positive the experience can be. When patients start a trial, they think 2 or 3 years is so long, but almost before they know it, it’s over. Often, they want to know if they can keep coming to our practice. Because they have such a positive experience, almost all of my patients continue into available extension studies. They appreciate getting to know the other patients and develop a camaraderie, feeling as though they are part of a team. Most want to know how they were randomly assigned and are keenly interested in the study results.

Discussing participation

When we discuss trial participation with patients, we stress that the goal of these studies is to obtain better visual outcomes and ease the treatment burden. Can we do better than laser and anti–VEGF-A injections? Can we treat less often? One example is a topical eye drop for the treatment of diabetic macular edema. Obviously, that is an extremely desirable option and therefore easy to enroll patients.

We emphasize that for most retina studies, the control arm is standard of care—everyone gets treated. That makes enrolling easier, as participants know they will receive some therapy. Patients often say, “I want to be in the arm that gets the new drug,” and I explain that is not how it works. In most studies, approximately two-thirds of participants do get the new drug. We explain that our results are constantly analyzed and patients receiving standard of care may be crossed over to the study drug. Plus, if efficacy is seen, they often have the option of getting the new drug once the trial is completed.

I tell trial patients that people are watching over my shoulder to make sure I do a really good job. They know that our equipment is checked and monitored, that we watch for every complication, and that there is frequent testing. They understand they are getting up-to-date care of the highest quality and that we are on top of all the research protocols. They receive transportation and a small stipend that offsets any costs they may have. Of course, it does require a commitment on their part, as they must come on set study days and the visit is longer.

Current trials in my practice

I am enrolling patients in a number of phase 2 clinical trials, including the SIGLEC study (NCT05839041) looking at a new molecule to control geographic atrophy associated with dry AMD (AVD-104; Aviceda Therapeutics) and 2 investigating oral therapies: One for Stargardt disease (ALK-001/gildeuretinol; Alkeus Pharmaceutical) and one for diabetic macular edema (RZ402; Rezolute Bio).

Another study I am currently enrolling is the COAST phase 3 study ((NCT04757636 ;Opthea) looking at adding a new molecule that inhibits VEGF-C and VEGF-D called sozinibercept (OPT-302) in combination with standard of care anti–VEGF-A therapy of aflibercept (another phase 3 study, ShORe [NCT04757610], of sozinibercept with ranibizumab is being run concurrently at other retina research clinics) for neovascular AMD. It is hoped that patients will have better visual results compared with standard of care. These phase 3 trials are building off of an encouraging phase 2 program, and we are looking for patients who are newly diagnosed with wet AMD and have yet to start treatment.

Conclusion

Running clinical trials makes a practice more attractive to patients, who do their homework when they are looking for a physician. It is exciting to be part of important research, constantly learning and finding out about advancements through direct participation. I find it meaningful to be working with my own hands vs only reading about a study in a journal.

With hundreds of studies of retinal disease currently recruiting—more than 250 in AMD alone, according to ClinicalTrials.org—there are countless opportunities to get involved. One does not need to have a large practice to set up a research section nor be in a university setting. Even with a small group and myself as the only retina specialist and PI, I created an efficient process by enlisting the involvement of a local eye surgeon as the treating ophthalmologist. If a physician is interested in research, the size of one’s practice setting does not matter.

Image courtesy of Pamela Ann Weber, MD

Pamela Ann Weber, MD

Pamela Ann Weber, MD, is in practice at Island Retina, part of Vitreoretinal Consultants, with locations in Shirley and Commack, New York.