Rare Disease Day 2020 kicked off in Raleigh/Durham, NC with a packed house at IQVIA, who hosted “Patient Perspectives on Clinical Research in Rare Disease” for their employees. The panel of patients and patient advocates was co-moderated by Dr. Jeffrey Keefer, Head of the Pediatric and Rare Disease Center of Excellence at IQVIA and Tara Britt, Founder and President of the Rare Disease Innovations Institute, Inc.
Dr. Keefer kicked off the event by reminding everyone of the profound significance of input from patients and patient advocates on clinical research and described some of the ways that is addressed at IQVIA. He graciously set the stage for each of the panelists.
Rare and ultra-rare diseases were represented by the following panelists: Ty Alexander Myatt, Fabry disease; Justina Williams, sickle cell disease; Jenny Wilkinson, cystic fibrosis; Meredith Huml, facioscapulohumeral muscular dystrophy; and Parvathy Ramen Krishnan, Lynch syndrome and constructional mismatch repair deficiency syndrome. Each presenter shared compelling stories of their journeys through diagnosis, the search for services, and a multitude of treatments, while maintaining hope for an improved quality of life. Regardless of their specific disease, panelists encouraged the audience to include patients and/or advocates in every part of a clinical trial.
Employees were encouraged to picture patients benefitting from the work of everyone who contributes to a clinical trial, regardless of their role. In addition, researchers and clinicians must be asking patients the right questions. Panelist Parvathy Krishnan, Director of Patient Engagement for the Rare Disease Innovations Insitute said, “It doesn’t matter what your brochure looks like if you don’t ask the right questions.”
Patient involvement also hinges on access to services. Highlighting both awareness and access, Tara Britt provided an eye-opening update regarding the state of rural health care in North Carolina and its impact on rare disease patients. Rare disease has no geographic or socio-economic boundaries.
IQVIA employees heard a resounding message of appreciation for their tireless work, and the presenters heard enthusiastic applause for their nonstop work of living with rare disease.
Panelists, moderators, and event organizers joined arm-in-arm to express appreciation to the employees attending the event and to everyone working to improve the life of rare disease patients. For additional information, please see the IQVIA LinkedIn post.