Bec and her granddaughter Ellayna attended the Global Genes Rare Patient Advocacy Summit in Huntington Beach, California on September 24 – 25, 2016.
With over 600 people in attendance and approximately 200 rare disease organizations represented, the summit was under way. Multimedia was in full force with photographers, videographers, social media screens throughout the ballroom, live streaming, and PowerPoint presentations.
The keynote speaker was Robert Ring, Ph.D., Former Chief Science Officer, Autism Speaks, shared information related to Rare Diseases in the 21st Century – The Look Ahead – Times are Changing and you need a plan. This presentation included topics such as; organizing principles, looking at the endgame of science, clinical objectives, thinking broadly about therapeutic modalities, and alternative therapies for rare diseases.
The morning tracks for day one included: Caring For Yourself As Caregiver – Natural History Clinical Trials – Building an Engaged Patient Community – Using Sequencing to Understand Rare Idiopathic Disorders – Effectively Engaging Teens In Rare Disease Advocacy – Developing and Validating Biomarkers – Partnering With Pharmaceutical Companies – Gene Sequencing: Not Just For Diagnosis
We chose Building an Engaged Patient Community presented by Sue Kahn, Executive Director, National Tay-Sachs and Allied Diseases Association and Blake Shewey, Vice President Client Services, Snow Companies. A community should have purpose, relationships, and personalization. It’s important to know your “story” to be able to tell your story, which is important in the process of building a community. A community becomes stronger when information is provided, the members feel supported, and there is a strong sense of unity. All of these factors are critical in building a community or “Family.”
The afternoon tracks on day one were mostly focused toward drug development. However, one track was directed toward the importance of Building a Clinical Care Team and Why it Matters, presented by Jodie Vento, Genetic Counselor and Manager, Center for Rare Disease Therapy, Children’s Hospital Pittsburgh of UPMC. This presentation stressed that we, in the rare disease community, have to involve more than a primary care provider to ensure the best quality of care. But, these multiple care providers must coordinate, communicate, and provide individualized care, which is key to receiving the best therapy possible. The coordinated team approach is also valuable in developing innovative approaches to rare disease treatment when standard therapies are limited or missing.
Day two was filled with informative tracks as well. This day was focused on How to Find and Read a Scientific Paper, Developing Volunteer Leadership, Talking and Engaging With Researchers, Genetic Counselors, Working With a Scientific Advisory Board, Growth and Transition, Understanding Clinical Trials, Genomics Revolution, Interpreting Clinical Research Findings, Effective Social Media, The Patient’s Role in Designing Clinical Trials, Advocating at the State and Federal Level, Big Data and Intelligence-based Medicine.
Ethan Peristein, Ph.D., Founder and CEO, Peristein Lab presented on how to find and read scientific papers. The first, and sometimes difficult, steps in engaging with researchers about a rare disease is finding them. Finding researchers who have published scientific articles about your rare disease or similar disease is one way to begin your journey. Ethan, who is a leading biomedical researcher, defined the pathways to finding research papers, how to identify the contact information for the researchers, and what the parts of the paper mean.
How to Recruit and Work With a Scientific Advisory Board was another hot topic. Navigating to receive the needed advice from scientists on what kinds of research to collaborate on, who to reach out to, and how to move forward research and drug development for a rare disease was presented by Steve Grossman, Parent Advocate and Co-Founder, ADCY5.org. Steve shared that it is best to determine goals, so you can identify what types of people to engage. He stressed the importance of supporting families affected by your disease, supporting doctors and researchers who are interested in finding treatment and a cure for your disease. When you reach out to patients, doctors and researchers, and any other interested partners the next step is to collaborate.
Melissa Hogan, J.D., Founder & President, Saving Case & Friends, Inc. and Stephanie Fischer, Patient Advocate, Senior Director of Patient Engagement and Communications, Everylife Foundation for Rare Disease presented an informative session on Effective Social Media Engagement. Sometimes more isn’t better and in certain social channels (Facebook, Instagram, Twitter), that is absolutely the case. Melissa and Stephanie explained how to become a reliable source, and that “Quality” over “Quantity” is the best practice. Flooding your social media channels with information that promotes your organization and sends the message of how “Great” you think you are, is the fastest way to turn people off. While people want to know that you’re credible, they also do not want that pushed in their face every time they login. These ladies explained how they have learned that taking the humble yet confident approach, reaches more people and engages them over a longer period of time. If the focus is on always promoting your organization, it shifts attention and does not raise awareness for your disease or support your patients.
The day came to a close with the Tribute to Champions of Hope Awards Ceremony. The Honorees included patient advocates, patient organizations, doctors, researchers, and many more. Following the awards ceremony the Reggae Funk Fest began in the courtyard. This conference was uplifting, inspiring, and motivational. Although it was a long two days for Ellayna, we hope to attend again next year!