Giving more patients with rare diseases access to innovation

March 21, 2019

New and innovative therapeutic options hold exciting potential for those living with rare diseases. Yet long before the drugs might be approved by the FDA, operationalizing a clinical trial with an advanced therapeutic requires robust patient recruitment, regulatory planning, specialized physicians, and multiple clinical tracking tools.

The brave and dedicated patients must be identified, educated, navigated to the proper treatments, and then turned into advocates for the new treatments. The burden of clinical trial participation in a rare disease is often a limiting factor for recruitment. What’s more, keeping track of patients, especially pediatric patients, for the years of post-treatment follow up presents a challenge for engaged and active participants in the clinical trial process.

At 83bar we work with sponsors and CROs to continue to evolve and engage patients using our technology and content through apps, devices, social media, personal calls, and other communications channels.

Many experts feel an area of innovation involves improving the diagnostic journey of patients. With so many rare diseases being genetic and effecting children, it’s critical we remember the first step to finding and caring for patients is to facilitate a timely, accurate, and accessible diagnosis.

At 83bar this helps remind us of the true meaning of patient centricity. Because so many genetic diseases are not on standard screening panels, many families go through a heartbreaking process of misdiagnosis and delayed treatment options as they seek to identify the disease that’s ailing them. That’s why we’re excited to collaborate with pharmaceutical companies, diagnostic makers, academic institutions, and patient advocacy groups to further rare disease drug research and development.

The expanded role of patient activation can help boost public awareness, increase request from patients and their families for new treatment, and help drive the scientific industry to create new treatments for these orphan disorders.