Top 10 Takeaways from the 2023 RARE Advocacy Summit

September 21, 2023

Did you LEVEL UP while attending or live streaming the 2023 RARE Advocacy Summit? We certainly did!

Here are the top 10 things that the Global Genes team learned during this year’s RARE Advocacy Summit:

10. The internet and technology allow for more than just access to data and information. It’s really about access to each other. We are learning from each other about how to collect data, how to conduct personal science and how to build medical and assistive devices. – Susannah Fox, Author, Digital Health Expert, and Advisor for the Patient-Led Research Collaborative (Rebel Health: How Rare Disease Communities Lead the Revolution)

9. Because we tend to grieve in a silo, when one member of the family is enveloped by their grief, that leaves a hole. If a parent is unable to function in the way that they typically functioned, sometimes the children will step in and become the parent/caretaker. The entire family dynamic can be impacted. – Ronda Thorington, LPC, Clinician, Parent Coach (Grieving Diagnosis, Lack of Diagnosis and Loss)

8. Patient advocates are the only ones that have the connections to the community to drive and create that success [for research and clinical trials], but also, it doesn’t make sense to pursue research in the disease if you don’t understand the patient voice – their needs, their issues. – Eric Sid, MD, Program Officer, National Center for Advancing Translational Sciences Division of Rare Diseases Research Innovation, National Institutes of Health (Finding Your Flock: Emerging Opportunities to Accelerate Research Through Disease Community Collaborations)

7. We have to do our homework as moms and dads. We don’t have to be scientists. We’re asking for a treatment. FDA standards are safe, and efficacious. Our standard is urgency. Do everything you can to have your words respected. – Mark Dant, Executive Director, The Ryan Foundation for Rare Disease Research (7 Ways to Engage the FDA)

6. Without a diagnosis, I felt very alone. By sharing his [her son’s] story, I could connect with others and learn things that would help with getting a diagnosis and therapies. We have documented through video about his diagnosis process, and we found this to be therapeutic. It also connects me with others so I’m not isolated. It’s like the door opens, and you want to step out and share that story. – Caroline Cheung-Yiu, Founder, CURE (Coping with the Challenge of Rare Disease – With or Without a Diagnosis)

5. Data is so important, so no matter how you are collecting it, make sure that you are doing it in a way that’s meaningful. Figure out what your community really needs so that they can be contributing to these data sets. – Kendall Davis, MPH, Director of Advocacy and Engagement, ICON, plc (Becoming Clinical Trial Ready)

4. To realize there was “pre-grieving,” “ambiguous grief,” “anticipatory grief” and they are worth knowing about so you can seek out the support you need. Giving the grief a name empowers you. – Daniel DeFabio, Director, Community Engagement, Global Genes (Grieving Diagnosis, Lack of Diagnosis and Loss)

3. We’re dealing with rare diseases, but they all have common issues and common needs. We can try to fight barriers together and figure out how to move forward. – Joni L Rutter, PhD, Director, National Institutes of Health (NIH) National Center for Advancing Translational Sciences (Strengthened by Our Past, Working Together to Create Our Future) 

2. Your flock is in this room. You find your flock at these meetings by engaging and talking with other groups and realizing that there are common opportunities to work together. This isn’t isolated to foundation groups. At the academic level, I collaborate with lots of institutions and other providers. In rare disease, everything you accomplish that’s meaningful happens because of collaboration. – Scott Demarest, MD, Associate Professor, Children’s Hospital of Colorado (Finding Your Flock: Emerging Opportunities to Accelerate Research Through Disease Community Collaborations)

1. “Let’s continue to work tirelessly toward our ultimate goal to ensure that no patient with rare disease is ever left helpless, hopeless and abandoned.” – Ravi Bhargava, MD, Roche Global Informatics, Global Medical Director, AI Digital Health (Open Science Data Challenge Presentation)

Now what did you learn?

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