Center for Rare Diseases continues to engage with rare disease patient communities during COVID-19 crisis

Advocates and patients who attended the Center’s first interactive webinars held on April 1, 2020, requested further updates from the Center as we navigate the evolving COVID-19 landscape. In response, the Center hosted two follow-up webinars on Wednesday, April 29. Advocates from more than 43 different rare disease areas, as well as from key global organizations, once again registered for the webinars.

The agenda included discussion of the FDA Guidance, trends in how rare disease trials are being impacted, and how leveraging virtual and remote care practices can keep trials vital to rare communities moving forward. The webinars were organized and hosted by Scott Schliebner, Senior Vice President, Center for Rare Diseases, Amy Raymond, Director of Therapeutic Expertise – Center for Rare Diseases, and the Center’s Managers of Patient Advocacy & Engagement, Kendall Davis and Laura Iliescu.

The majority of participants reported that clinical research in their rare disease has been paused. Looking beyond the COVID-19 crisis, a majority of participants reported that the patient communities they serve are, “concerned about returning to sites.”

Many pharma and biotech sponsors have been rapidly assessing options for keeping important, rare disease clinical trials moving forward through virtual and remote care practices in response to the continued COVID-19 crisis. However, the majority of rare disease advocates who participated in the webinar reported that sponsors have not involved their organizations in assessing and evaluating these virtual options.

As we continue to navigate the uncertainties of the COVID-19 crisis, close collaboration with rare disease patient communities will play an important role in de-risking rare disease clinical programs. This collaboration is crucial to ensuring the feasibility of challenging rare disease studies. It is more critical now than ever as vital rare disease clinical trials face unprecedented challenges.

The series of webinars demonstrate PRA’s leadership in human centricity in clinical research. It speaks to our deep connections to rare disease patient organizations and advocacy networks, as well as demonstrates our unique expertise in developing collaborations between sponsors and rare disease patients. We do this to support the clinical trials in which they are both vitally invested.

The date of the next interactive webinar will be announced shortly.