Overcoming Challenges in Ophthalmology Trials

Although ophthalmology clinical trials require specialty equipment and technical expertise, opportunities to enable more site- and patient-centric approaches are growing. Learn how experts at the PPD clinical research business of Thermo Fisher Scientific can act as partners in creating therapies to treat ocular disease.

There is a growing need for novel therapies that more effectively treat ocular disease. This need is fueled by rising rates of diabetes and hypertension and increasing incidence of autoimmune diseases that affect eye health. Unmet patient need also continues to be an issue in age-related macular degeneration, diabetic eye disease, glaucoma, dry eyes and rare ophthalmic diseases.

Because of unique requirements for both sites and patients, ophthalmology clinical research differs greatly from other therapeutic areas. Using a site- and patient-centric lens is important to overcoming burdens and ensuring the site is fully supported throughout the study process. The PPD clinical research business of Thermo Fisher Scientific continues to investigate and develop new methods to mitigate these challenges — methods that are data-driven and rely on decentralized, virtual tools and innovative delivery methods to increase compliance and efficiency.

Creating Early Plans to Address Unique Challenges

In ophthalmology clinical research, each indication and individual trial demands a tailored approach that addresses challenges up front. Study plans must cover the usual bases — everything from optimized development to clinical supplies, vendor management, database development, site selection, patient enrollment, monitoring and quality.

However, identifying the right specialty sites early is critically important. Site needs for each trial can vary greatly, which is why developers must consider the following when choosing sites:

• Regulatory requirements by country
• Site specialty expertise in ophthalmology (including neurology, pediatric, retina, corneal, rare disease, etc.)
• Specific equipment
• Patient population availability
• Staffing and facility requirements
• IP delivery experience
• Marketing goals

Ophthalmology sites are not alone in experiencing major resourcing issues, including staffing shortages, high turnover rates, significant clinical trial workload and, often, studies competing for similar patient populations and/or the same site staff.

Ensuring Study Start-up Is as Smooth as Possible

After site selection is done, robust planning helps ensure minimal startup delays and enrollment success. First, you must budget significant time for the study coordinator and technician to fulfill ophthalmic imaging and visual acuity certification requirements, lab needs, and potential partnerships with referral or other specialty sites. Additional start-up planning should include strategies that:

• allow time for site staff to efficiently identify and contact subjects and coordinate necessary visits,
• mitigate the volume of patients who may decline study participation,
• address increased prescreen or screen fail rates,
• determine the need for treatment-naïve subjects,
• confront the fierce competition for study subjects, and
• accommodate patients who present at alternate locations and must be referred — or in cases of rare disease, who are spread over a wide geographic area.

Using the Power of Databases and Registries

PPD has identified several patient registries and ophthalmic vendors with strong potential to offer support in protocol optimization, site identification, potential referral methods and up-front patient identification. This data enables us to answer key questions such as:

• Have we planned achievable enrollment rates and timelines?
• Is there a need to adjust country mix or the total number of sites?
• Have we selected the right sites with the highest potential to enroll patients who meet all requirements?
• Are there competing studies for this indication, and are we selecting sites with minimal to no overlap?
• Is the patient population elderly, or are there long or multiday visits that necessitate patient transportation and accommodation?

Bringing the Trial to the Patient

The remote digital tools of decentralized clinical trials (DCTs) are enhancing clinical research — and ophthalmology trials are no exception. Decentralized approaches reimagine the clinical trial to bring clinical research to the patient, meeting them where they are to make participation as easy as possible.

In the past, virtual trials were tough to realize in ophthalmology due to the specialty equipment and skilled technicians required. PPD has identified partners offering unique, direct-to-patient solutions that are an extension of the local site. Mobile vision assessments make it possible for patients to participate through home visits or appointments at convenient locations. What began as a collaboration to reduce travel for rare disease patients during the pandemic has broadened into a powerful solution with a wide range of potential applications:

• Increased access for underserved populations
• Unique batch screening solutions leveraging alternate locations based on indication
• Patient visits at patient homes, including prescreening opportunities with ophthalmic assessments
• Reduction of patient burden through minimizing site visits to multiple locations

Tying it Together with PPD Expertise

PPD offers ophthalmic developers global expertise in trial execution and site management across all phases of the trial. We serve as a trusted partner for ophthalmic development, leveraging strong site relationships, powerful technologies, preferred vendors and patient insights into clinical development.

PPD ensures patients and caregivers are informed every step of the way through educational materials including consent aids, brochures specific to their condition, and patient-facing portals and newsletters.

Various PPD teams enable our site- and patient-centric approach, including our medical communications group, virtual study coordinators and patient concierge service.

• For many years, patient involvement was limited to consent and study participation. Now, our medical communicators produce robust educational materials that make patients aware of the benefits of their participation — for themselves, future patients and the advancement of science.
• Virtual study coordinators assist with many aspects of trial management and site support. Some of the services they provide to ophthalmology sites include prescreening, scheduling and inbound hotline management.
• The patient concierge team members are dedicated to enhancing the experience for patients and their families. As a single point of contact, concierges guide patients throughout the trial, organizing everything from travel to reimbursement of expenses, and coordination of and reminders about visits. This can be of critical importance in complex rare disease studies where a prior poor experience or lack of consistent engagement leads to a higher risk of patient dropout.