Preparing for the Road Ahead: Rare Disease Advocates Unite
PRA Health Sciences
PRA Health Sciences

This year's RARE Patient Advocacy Summit takes place October 3-4 at the Hotel Irvine in Irvine, California. This annual event, hosted by Global Genes, is the largest worldwide gathering of rare disease patients, advocates, and thought leaders.

The founder of Global Genes, Nicole Boice, shares her thoughts on the summit and her hopes for the future for rare disease patients:

This year’s summit theme is “Preparing for the Road Ahead”—why was this theme chosen and what does it mean?

At Global Genes, we’re all very aware of the need for education in support of patients/advocates as their role as drivers in rare disease continues to grow. Global Genes works to help equip patient advocates to be prepared for success as they drive efforts in support, community development, and community coordination, all to help drive treatments, therapies, and cures. This year’s summit works to help ensure that wherever you are on your path forward in rare disease, that you have access to the right resources, insights, and connections, preparing patients/advocates for success.

One of the tracks at the conference is “Science and Technology Innovation”—how do you think science and technology is helping the rare disease community?

The time is now! Innovations in Science and Technology are helping us diagnose patients more quickly, providing a greater understanding of rare diseases, and are driving new therapies and potential cures, especially surrounding Gene Medicine. There is incredible energy around rare disease right now, and as patients become more savvy and prepared to participate, we anticipate that all of this forward movement will only accelerate! It is the decade for rare disease!

There is another track “Patients and Partners and Drivers”—how can patients help drive change for rare disease research/advances? How can their voice be better represented in the clinical research process?

We don’t use the phrase Patient Voice anymore. Patients role in drug development is far greater than that. Patients have become as vital to the industry as is a chief commercial officer. Patients are seed-funding early stage research; they are de-risking early investment for our partners; they are coordinating their disease communities, collecting data, managing data registries/natural history studies; and they are making all of this available in a coordinated way to help advance research efforts for their disease more quickly. Patients are critical as partners…and Global Genes takes its role seriously, in helping ensure that patients are readied to help usher forward the next cure.

Global Genes is celebrating its 10th anniversary—how has the organization evolved/grown in the past decade?

What began as a small effort with seven parent advocates who understood the needs that existed to better educate, coordinate, and mobilize the rare disease community, now includes a global footprint, an expanded family of more than 500 patient advocacy organizations, thousands of patients, more than 80 biopharma partners, and countless other rare disease stakeholders. Global Genes has a clear mission and goals and is laser-focused on ensuring that wherever patients reside, they have an opportunity to be supported for success as they embark on this often complex journey. They have a family to help them and the most important element of all of this is that patient advocates move forward believing that they don’t have to go it alone.

What positive changes in the past 10 years have you seen for rare diseases?

Increased energy and awareness for rare disease. Although unfortunate, there is a growing community of patient advocates, but patients now have a place to go for help and support. We have seen advances in Science/Tech/Investment/Legislation, which have all contributed to these positive changes and steps forward for the rare disease community.

What are the ongoing challenges?

Although tremendous progress has been made, there is still much work to be done. We still are challenged and continue to need more efforts in awareness building, increased Investment in science and tech, access to testing, and access to treatments.

What is on the horizon for Global Genes?

Global Genes is readying itself to support more patients globally, creating more impact. Global Genes is launching an important “Impact 2020 Initiative” that is raising significant funding to build and expand programming, invest in tech infrastructure, and provide more access to resources for patients globally. Global Genes has focused its efforts on helping ensure that we can help ease the rare disease burden for families and ultimately help eliminate the challenges of rare disease for patients around the world.

Tim kelly artisgood
Timothy Kelly

At this year’s RARE Patient Advocacy Summit, we’re excited to host a collaborative art project for attendees using silhouette art. The artist behind this project is Timothy Kelly, who was with us last year for an art “puzzle” project:

Tell us about the Global Gene art project that is planned this year.

For this year’s Art is Good project we will be sharing our “Rare Story” with silhouette art. First we trace our silhouette onto an art making poster, then we think about our life and make art that shares our story. People often don’t think about what they are thinkin’ or what’s in their head. This project helps turn our ideas and thoughts into art. Like last year, all of the art supplies will be provided, you just have to bring your good ideas. The PRA Health Art is Good team will be there to help. I’m looking forward to seeing everyone I connected with last year.

How will it be different from last year?

The substrate we make art on and the size will be different. Last year we used a 24” x 24” foam core puzzle piece, this year a 12” x 18” poster. The artful goal is the same… Global Genes participants can use art to tell their story and/or provide information about their rare disease, condition or organization. Treat your art like your own “Public Service Announcement.”

What did you enjoy about being part of the GG summit in 2017?

Last year was a truly beautiful experience for me. I was humbled and honored that people utilized the puzzle project to tell their story and educate others about their rare disease or condition. The community, empathy and love all around was inspiring. Every time someone found someone else in the same situation because of a puzzle piece, was amazing. People connected and shared empathy and their own experiences with each other. I learned a lot about how the body works, too. Few people truly understand how their body works like the people in the Rare Disease community.

What do you hope people will take away from the silhouette art experience?

This year, I hope people again will use art to share their story, educate, find others and promote their organizations. I hope the experience is fun and a source of healing. Like last year, people can connect and collaborate while they create. Afterward, we will exhibit and celebrate what we made. We will share our rare stories together.

You started “Art is Good”—how is art is good—particularly for patients or those who might be going through a difficult time?

ART HEALS! That is a fact. Being creative in a safe place with people that truly understand the challenges of your Rare life is comforting. Making something meaningful is cathartic. Being able to tell your story with art when you may not have the words or want to say them = Healing

The Art is Good program is about “ART” and “YOU.” The goal of the program is to learn a little bit about ourselves and other with art. Art is Good for YOU!

Silo art examples 1 rs

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