What I Hope to Get From Attending the Global Genes Rare Patient Advocacy Summit

By Janet Mills

As I count down the days before I leave Wyoming to attend the Global Genes Patient Advocacy Summit and Champions of Hope Gala in Newport Beach, California, I feel a mixture of excitement and anxiety. This will be the first time I go to a rare disease event without another member of the CADASIL community or a family member along for the experience. While I am grateful that I have met several people in person who will be participating in the event, I will be traveling alone. Thankfully, I will have a roommate when I get there, to split the cost of the room and to chat about the activities we attend. We have met on Facebook and share the same passion of bringing awareness to rare diseases. I doubt we’ll be strangers for long!

The evening before the summit, I get to meet a fellow CADASIL-ian for dinner somewhere between her southern California home and Newport Beach. We have known one another for three years online, but seeing her in person will be a rare treat. There’s something about getting together with another person who shares your disease (and in this case, a birthday!) that resembles a family reunion. You “get” one another and the bond formed is instantaneous.

The objectives for the summit are excellent. I have copied them here and added some personal comments.

Attendees will:

• Learn how to get started: obtain 501c3 status, write grants, leverage PR effectively and utilize social media to spread your message.

The CADASIL Association has made it successfully through the 501c3 approval process and is ready to embark on the tasks of grant writing and finding additional ways to spread our message. Grant writing is a particularly challenging topic, and I hope to learn about resources to help.

• Collaboration: Understand how to successfully work with other rare disease stakeholders, patient advocates, the FDA and other government entities.

This is a key part of the summit, in my opinion. Joining with others toward the same goal of getting our voices heard is so important. Divided, we replicate the work others have done, sometimes starting at Square One. Together, we learn from one another in order to progress at a faster and more successful rate.

• Learn the importance of patient registries, the different types of registries and how advocates can support them.

Members of CADASIL Association have been looking into rare patient registries. There are a number of them, which makes it difficult to know which direction to go. Which one is right for us?

• Explore the role of foundations and advocates related to scientific discovery and drug development.

I joined the Global Genes Corporate Alliance Patient Advocacy Committee to help better understand these roles and to offer a rare patient voice in the process. The science of our rare diseases can be mind-boggling. Deciding which treatments to try (if there are treatments available) is a huge challenge and a source for concern for many of us.

I am excited to participate in these sessions and discussions. I can’t wait to make more friends in the rare disease community. I look forward to attending the Corporate Alliance Patient Advocacy Committee meeting in person on Saturday morning following Friday’s summit. Then there’s the Champions of Hope Gala on Saturday night (AKA: par-tay!) and a reason to put on a new blue dress.

Top it all off with the fact that these events take place near the beach, and this former California resident is rarin’ to go!

About the Author

Janet Mills is a CADASIL patient and a trustee of CADASIL Association. She is active in several patient advocate and support groups online. Janet worked as an elementary school teacher for twenty years and is now on medical retirement. Her previous published writing credits include romantic contemporary and historical fiction. (www.janetmills.net)