A leading pharmaceutical company wanted to optimise the clinical development plan of a gene therapy indicated for a rare ophthalmic disease, and to better qualify the future clinical trial population. They also wanted to assess the clinical course of disease progression and estimate the change in visual acuity. The company aimed to capture the patient pathway and to measure the cohort when patients often exit the healthcare system due to the lack of therapy. Their goal was to publish their findings in congress and a peer reviewed journal. The company engaged Kantar because of its strong scientific and marketing expertise, as well as its robust capabilities and targets access worldwide.
We implemented a multifaceted research programme, which included qualitative, quantitative and observational studies, with the objectives of understanding the patient pathway and burden, measuring the cohort, and describing the disease history and the clinical profile of a specific type of patient. The scope of the global project covered 21 markets: the United States; Canada; the EU5; Latin America, including Brazil, Argentina, Chile, Colombia, Venezuela and Mexico; Asia Pacific, including China, Japan, South Korea, Taiwan, Malaysia, Philippines and Australia; and South Africa.
Along our three-year study programme demonstrating progression, we provided a mapping of experts’ sites and a clear description of the disease management, genotyping practices, disease progression and cohort measure. We also published results and presented posters at a recent congress of ARVO, the Association for Research in Vision and Ophthalmology.
Through our findings, we concluded that patients with an earlier onset of the disease have a more rapid progression of vision loss; however, patients with a better visual acuity tend to have a quicker loss of visual function. Additionally, variation in disease form and gravity, and factoring in local specialists’ accessibility and potentially high incurred costs, tend to create a distortion between the onset of symptoms and diagnosis, ranging from six months to five years.