A Vision For Inclusion: Designing Trials That Benefit Visually Impaired Patients

By Huma Qamar, MD, MPH, CMI, chief medical officer, Ocugen

Inherited retinal diseases (IRDs) are a group of rare genetic disorders that progressively lead to vision loss and, in many cases, complete blindness. Despite their profound impact on patients, IRDs have long been under-researched, with limited treatment options available. The high unmet medical need in this area is exacerbated by the fact that large pharmaceutical companies often shy away from investing in clinical trials for such small patient populations. The challenges are substantial, but so are the opportunities for meaningful breakthroughs that could transform the lives of those affected by IRDs. Yet, while the industry has largely overlooked IRD trials, it’s crucial to recognize the broader need: clinical trials for any therapy must also accommodate visually impaired patients. Whether targeting vision-related conditions or other medical needs, trial designs must ensure these patients are included.

Challenges In Developing Clinical Trials For Small Patient Populations

One of the primary obstacles in developing clinical trials for IRDs is economic viability. Large pharmaceutical companies frequently avoid these small populations due to the significant logistical hurdles involved. The coordination required for such trials, especially with rare disease patient populations spread across various geographic regions, countries, and cultures, can be daunting.

In addition to economic and logistical challenges, there is a substantial gap in communication and outreach to patients who qualify for these trials. Many patients with IRDs are unaware of the clinical trials available to them, particularly those who may have undergone genetic testing years ago. Resources like ClinicalTrials.gov are invaluable for patients and their families, providing a centralized platform to find appropriate treatments and study locations. However, visually impaired patients often rely on caregivers to navigate these resources, which underscores the need for greater awareness and accessibility.

Unique Challenges For Visually Impaired Patients

Visually impaired patients face unique challenges in the context of clinical trials, including logistical issues such as travel and accessibility. Traveling to trial sites can be particularly challenging, as these patients often require caregivers to accompany them, adding to the complexity and effort involved. The limited availability of specialized surgeons and facilities further complicates participation in these trials.

Awareness and recruitment remain significant hurdles. Visually impaired patients, compared to their non-impaired counterparts, encounter additional barriers to learning about and accessing trial information. Assistive technologies and tailored communication methods can help bridge this gap, but more needs to be done to ensure these patients are reached effectively. For example, audio advertisements targeting visually impaired listeners can be a powerful tool in raising awareness.

Enrollment and participation in trials also pose challenges. These patients often require specialized support, such as caregivers, to assist them in navigating the trial process. Trial materials and consent processes must be accessible to those with visual impairments. Cultural and language barriers must also be addressed, particularly for non-English-speaking patients who may be eligible for these trials. Expanding outreach globally is essential to maximize patient recruitment in such rare disease populations.

While accommodating visually impaired patients in clinical trials does present real barriers — such as additional costs, regulatory uncertainty, and logistical complexities — these challenges are not insurmountable. Managing clinical trials is already a complex process, and adding accessibility measures can feel like just one more optional task in a long list of requirements. Driving meaningful change requires recognizing that inclusivity, which improves access and enrollment in clinical trials, is an investment with a return well worth the effort.

Stringent Inclusion And Exclusion Criteria

Visually impaired patients exhibit a high level of motivation due to the progressive nature of their conditions. Unlike some other patient groups, those with IRDs often have a strong sense of urgency, knowing that their vision is deteriorating over time. This urgency drives them to actively seek out treatment options and participate in clinical trials, striving to make the most of the time they have left with their vision.

For some, even when a suitable clinical trial is identified, strict inclusion and exclusion (I/E) criteria can deprive some patients of a chance for help. Balancing the critical importance of I/E criteria, essential for maintaining rigorous scientific standards and meeting patient needs is delicate and often difficult to achieve. To address this issue, alternatives such as the FDA’s Expanded Access Program (EAP) have been developed. The EAP provides alternative pathways for patients who do not meet traditional inclusion criteria, allowing them to benefit from treatment without delay. The recent FDA approval of Ocugen’s OCU400 for expanded access is a prime example of how the EAP can broaden access to potentially life-changing therapies.

Adapting Marketing And Outreach Strategies To Reach Visually Impaired Patients

To effectively reach visually impaired patients, pharmaceutical companies must adapt their outreach and marketing strategies to target hard-to-reach groups. Engaging with ophthalmologists and low-vision clinics can be done through journal articles, ads in ophthalmology-related publications, and distributing educational leaflets and brochures. Engaging with domestic and international conferences also presents an opportunity to educate doctors and patients about available trials.

Foundations that support the visually impaired also play a crucial role in raising awareness of clinical trials. Pharmaceutical companies should collaborate with these foundations to raise awareness around potential trials through their press offices, chapter meetings, websites, and newsletters. With such partnerships, a rising tide can lift all boats — enhancing overall awareness and engagement, which benefits both patients and the broader research community. By learning from the best practices of these foundations, pharma companies can better engage with the visually impaired community.

Social media campaigns should also be tailored to be more accessible to visually impaired patients. This includes the use of alternative text for images, which allows screen readers to describe visual content to users, ensuring they can fully engage with the material. Content should be structured in a way that is screen reader-friendly, using proper HTML tags and descriptive link text, so that visually impaired users can easily navigate and understand the information being presented. Incorporating audio descriptions into videos further enhances accessibility by providing detailed narrations of key visual elements, allowing visually impaired individuals to comprehend the full scope of the content.

Moreover, blending visual and auditory formats in social media ensures critical information is accessible in multiple forms expanding reach to both patients and their caregivers. This might involve pairing images with spoken-word content or offering video content with enhanced audio descriptions, creating a more inclusive experience. Podcasts, in particular, are an excellent resource for visually impaired patients and should be leveraged more by foundations, low-vision clinics, ocular surgeons, ophthalmologists, and biotech companies.

To build a better digital landscape for individuals with visual impairments is a broad challenge. In the realm of clinical trials, pharmaceutical companies should collaborate with internal teams like digital marketing, content creators, and compliance, while also partnering with external accessibility experts, advocacy groups, and technology providers to ensure all digital content is fully accessible. For smaller biotechs and pharmaceutical companies, it’s critical to engage with medical professionals, such as ocular surgeons and low-vision specialists, to better understand the needs of visually impaired patients and leverage their networks for outreach. For engagement to foster understanding, the industry should actively seek feedback from advocacy groups and visually impaired communities to continuously improve the accessibility and effectiveness of their clinical trial communications.

Nontraditional Approaches To Overcoming Marketing Challenges

To overcome the myriad challenges in developing clinical trials for IRDs, approaches beyond the traditional are essential. New trial designs that accommodate the unique needs of visually impaired patients, such as decentralized trials or the use of digital health technologies, can improve accessibility and participation.

Holistic gene therapy approaches are particularly promising, as they focus on resetting genetic issues rather than merely treating symptoms. Developing clinical trials that are less burdensome, with treatment regimens that do not require considerable in-person follow-up, is crucial in improving patient participation and outcomes.

At Ocugen, we are employing a variety of strategies to reach these patients, including a word-of-mouth strategy designed for the digital world. By cultivating strong relationships with specialty surgeons and low-vision clinics, we have built a foundation of trust within these communities that is crucial for effectively leveraging real-world connections to establish in the broader digital network. This approach extends into the digital realm through targeted actions such as engaging with patient advocacy groups like and contributing to discussions on associated online communities. We build upon their efforts by developing and sharing educational content, including infographics, videos, and blog posts, that seek to create a personal connection through the sharing of personal stories from individuals we’ve met, and some that we’ve treated, who are living with visual impairments.

To effectively move the conversation into these digital spaces, we seek to collaborate with patient advocates and opinion leaders who have an established and trusted online presence. We provide them with up-to-date information on clinical trials and treatment options, which they can share within their networks, to ensure the message comes from a trusted source. This approach effectively leverages the credibility of personal interactions while harnessing the reach of digital platforms and the influence of trusted voices, creating a multiplier effect that significantly amplifies a traditional word-of-mouth approach.

Expanding Clinical Trial Awareness For The Visually Impaired

Addressing the challenges of clinical trials for inherited retinal diseases is not only crucial for the patients who suffer from these debilitating conditions but also represents an opportunity for significant medical breakthroughs. By adopting innovative strategies and working closely with the visually impaired community, pharmaceutical companies can help bring much-needed treatments to these underserved populations. The call to action is clear. More attention and resources must be directed toward rare and orphan diseases, with a focus on inclusivity and accessibility to ensure that no patient is left behind.

About The Author:

Huma Qamar MD, MPH, CMI, is the chief medical officer for Ocugen, a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines. In her role, she oversees clinical development and medical operations, including vaccine trials and mid-to-late-stage clinical trials for Ocugen’s novel modifier gene therapy platform with the potential to treat multiple retinal diseases. Dr. Qamar has extensive experience in clinical research, having worked at Ivy League institutions such as Yale, Harvard, and the University of Pennsylvania. She has developed and executed Phase 1-4 clinical trials across therapeutic areas, including gene and cell therapy, oncology, and infectious diseases.