On Thursday, November 5, Fighting Blindness brought together the pharmaceutical and biotechnology industries, scientists, medical professionals, policy makers and people affected by vision loss. Embedding the patient voice at the heart of conversation, we discussed some of the current challenges facing the future of clinical trial development.
Irish and international patient experts clearly explained the gap that exists between the aims of doctors and scientists and what patients are willing to accept as a good outcome from a potential therapy. While doctors and scientists are rightly focused on how to efficiently measure and standardise visual function testing by methods such as the eye chart and visual field testing, individuals living with a condition are more concerned with increasing their functional vision. This means using their vision in everyday tasks in real life such as getting around independently or increasing their reading speed or ability.
There are however a number of approaches underway that are beginning to break down some of the barriers between the aims of the patient and the professional. Earlier this year saw the publication of clinical trial data for a gene therapy designed for a rare form of childhood blindness called LCA (Leber congenital amaurosis). For the first time, central to the design of a clinical trial for blindness was the inclusion of a mobility course as part of the trial testing. This mobility course has been validated by the drug regulators in the US – the Food and Drug Administration (FDA) – as an acceptable measurement. This demonstrates how the needs of the people affected can be included and clinical trials can evolve by meaningful patient engagement.
Another key point raised during the meeting was the need for more data about how diseases of the retina progress over time. Both the patients and ophthalmologists present explained how vital this information would be for them, while the pharmaceutical industry discussed the central importance of this information for the design of future therapies. These types of studies are known as Natural History Studies (NHS) and we will work with the international community to make these types of studies a priority.