The EveryLife Foundation for Rare Diseases convened its 12th annual Scientific Workshop to highlight and document the COVID-19 mitigation strategies being utilized by industry partners and rare disease communities in an effort to move towards permanent solutions.
The goal of this year’s workshop was to gather key thought-leaders from the FDA, NIH, CMS, industry, patient organizations and other relevant stakeholder groups to highlight case studies of COVID-19 innovations, discuss potential longer-term models and best practices, and to identify how together, we can carve the pathway forward.
Throughout the day patient advocates, clinical experts, federal agency leaders, and industry professionals shared examples of how the COVID-19 pandemic has affected their lives, the way they deliver diagnostic testing and medical care, and how clinical trials are conducted. We are so encouraged by the commitment this community has shown to innovating and protecting rare disease patients and families during the pandemic.
Our sincere thanks to all our esteemed speakers, case study presenters, and panel moderators sharing your experiences and expertise to collectively work towards actionable takeaways throughout the day.
The EventMobi platform, which can be accessed here, will remain open only to those who attended if you would like to access any of the meeting resources. The highlight video is at the top of this page and videos from the Case Study Panel Sessions can be viewed here. Drawings of the day’s proceedings from artists at the Sketch Effect are below this text.
Topic Background
The profound effect of COVID-19 on therapeutic access paradigms and the clinical trials enterprise underscored the opportunities for innovations that have been long-championed by the rare disease community stakeholders. Telehealth and home health delivery models, patient-centered data collection through real-world evidence, remote monitoring, and decentralized trial models were introduced through statute including 21 Century Cures, PDUFA V and VI, and then further reflected in the Patient Experience Data Guidances published by the FDA. But the COVID-19 pandemic underscored the critical need for this infrastructure and again highlighted an opportunity to examine ‘real world’ dimensions of therapy delivery, clinical trial design and evaluation.
As the coronavirus paralyzed the nation, rare disease community leaders mobilized. Immediately stakeholders from across sectors began working together to identify challenges and innovations being encountered in conducting peer to peer research, clinical trials, newborn screening pilots and programs, and access to/delivery of approved therapies. In real-time, novel approaches and innovative policy solutions to address COVID-19 have been identified and applied to rare diseases.
Scientific Workshops
Thank you to our 2020 Scientific Workshop Sponsors
Regulatory Review
Clinical Trial
Pre-Clinical
Drug Discovery
