As 2023 began, RARE Revolution interviewed Charlene Son Rigby, incoming CEO of Global Genes, a global non-profit advocacy organisation serving the rare and genetic disease community. We learned how Global Gene’s recent merger with RARE-X will benefit the community, and how this newly strengthened organisation will be meeting the needs of the next generation of patient advocates, this year and beyond

Can you remind our readers about the respective missions of RARE-X and Global Genes?

Global Genes and RARE-X share quite a bit of history—including our founder—and the audiences that we serve: rare disease patients, families and researchers. Both organisations have a heavy focus on patients, families and advocacy groups being involved in innovation and research, and we saw that both of our missions were progressing down similar paths. 

By combining Global Genes and RARE-X, we are aligning our missions to help support and enable advocates in a deeper and more comprehensive way as they seek to drive disease understanding, spur development of therapies and change what it means to have a rare disease diagnosis.

What benefit will combining the two organisations bring for the rare disease community?

The landscape of rare disease is changing, bringing new opportunities for the rare disease community—whether recently diagnosed, living with a rare disease, building community, or involved in drug discovery and development. 

Combining Global Genes and RARE-X will provide individuals, families and advocates with a complete and integrated set of essential services to continue their work towards support and awareness, and accelerate them towards drug development through a collaborative approach of biopharma, researchers and funders, along with data as a central core.

Global Genes and RARE-X were born from the same leaders and already had an established strong partnership model. With the complementary services of Global Genes and RARE-X, the timing is ideal for the two organisations to join as one, enabling patients, caregivers and advocates to drive better, faster progress across rare diseases. 

The shared mission and vision of Global Genes and RARE-X focuses on next generation advocacy, supporting individuals who can utilise the tools and resources needed to accelerate advocacy efforts and also support opportunities to drive research and therapy development. 

Global Genes will continue its commitment to engaging and supporting advocates and other essential rare disease ecosystem partners through such programmes as the Global Advocacy Alliance, the Corporate and Research Advisory Boards, an expanded Scientific Advisory Board, and those leveraging and partnering with the RARE-X research programme. 

How will Global Genes be maximising opportunities for the rare disease community in 2023? 

The range and depth of activities that patient advocates are now engaging in has expanded significantly over the last decade. To meet the needs of next generation advocacy and support individuals, Global Genes is providing tools, connection and opportunities to help people do more, faster, for themselves and their communities. 

To simplify how our services and capabilities will be built and organised we have identified three pillars: Support – for patients and developing communities; Education – robust tools, events and educational opportunities; Research & Development – focusing on research and data enablement, with platform services to support these critical efforts.

Global Genes serves individuals and advocacy leaders as part of the Support pillar. Our Patient Services arm, RARE Concierge, serves thousands of patients and advocates each year, acting as a navigation service and hub of connection, guidance and education. It can be a first entry point into the rare disease ecosystem for undiagnosed and newly diagnosed patients, and families who are looking for answers, access to genetic counselling and testing, specialised care, clinical trials, resources and support— or simply to reach and connect with others in their disease area. 

The Global Advocacy Alliance provides support to patient advocacy groups seeking guidance on fundraising, increasing reach and growing their audience, and providing resources and support for patients and families. 

Our Education pillar is critical, as we ensure individuals and advocates can get information they need as quickly as possible. Global Genes has a rich history of providing education and information through our toolkits, in-person events, webinars, podcasts and video education. As we go forward, we are committed to continuing to make important education accessible in a timely manner so all can thrive. 

The newest addition to Global Genes, and the heart of the Research & Development pillar, is the RARE-X Data Collection Program. The RARE-X Data Collection Program enables rare patient communities to collect robust, secure, patient-reported data that is patient-owned, and makes that data widely available for research. Launched a little over a year ago, 70 patient communities and over 3,000 participants are collecting and sharing data through the platform. This expansion complements other work, like the newly published RARE Disease Research Roadmap—a comprehensive resource to support advocates to drive research and to work effectively with researchers and industry partners—that was generated from work at last year’s RARE Drug Development Symposium.

Across all our programmes, we are actively working to integrate our offerings so people can seamlessly navigate through these comprehensive services. 

How will Global Genes be rising to the challenges faced by the patient community? 

We polled our communities in 2022 to gain an understanding of where the community felt they had challenges and have identified three areas of strategic focus: mental health, health equity and research readiness. 

Mental health

Mental health is a serious and complex matter, especially impacting rare disease families. We are collaborating with our scientific advisory board, partners, advocates and mental health professionals to identify the best approach and strategy for Global Genes to support patients, caregivers and advocates, and get people to the most appropriate resources quickly. We just announced a grant programme to allow patient advocacy group to develop mental health resources, open to Global Advocacy Alliance members. 

Health equity

Health equity has far-reaching impacts in rare disease. In early 2022, RARE-X published a landmark equity, diversity and inclusion report that has led to an innovative, collaborative pilot programme, developing community-driven research networks in partnership with RareKC and University of Alabama-Birmingham. Two pilots will begin in 2023 that will help community leaders (i.e. faith-based leaders, rural community liaisons) identify rare patients, become educated about rare disease, provide family support, build trusted relationships with the programme’s community health workers and then engage where appropriate in research.

Global Genes also offers several programmes that bring health equity to the forefront. Health Equity grants are open to Global Advocacy Alliance members to develop initiatives to improve outreach strategies and address challenges that affect underserved and underrepresented communities. Our RARE Health Equity Forum, in partnership with the Rare Disease Diversity Coalition (RDDC), brings together stakeholders in the rare disease community to identify the challenges that disproportionately affect underrepresented populations and define collaborative efforts to address those inequities.

Research readiness

In the area of research enablement, we piloted a research readiness framework which we are rolling out in 2023. We are catalysing early work and research that will help advocates entice interest from research partners, including biopharma. There has never been more opportunity for patient advocates to get started with new therapies and treatments in the research readiness realm.

Finally, what are you looking forward to most in 2023?

This September, we’ll be hosting a Week in RARE, combining the RARE Health Equity Forum, RARE Advocacy Summit (formerly RARE Patient Advocacy Summit), and a RARE Partnering and Investor Forum. 

We appreciate the sacrifices advocates and organisations make to attend multiple meetings. By bookending the RARE Summit with these two additional programmes, we will create travel and time efficiencies, creating less burden for those who desire to attend the different events for their unique content. We’ve created an amazing week of support, education and networking opportunities that we hope will excite the community of patients, advocates, researchers, biopharma and other key stakeholders to want to participate. 

We also want attendees to be assured that our organisation is committed to ensuring that there will be meaningful and actionable takeaways that will positively impact the important work ahead for all of us.

About Charlene

Charlene Son Rigby has spent her career building organisations at the intersection of data, technology and life sciences, and was previously CEO of RARE-X and chief business officer at Fabric Genomics. She also co-founded the STXBP1 Foundation following her daughter’s rare genetic disease diagnosis. Charlene’s journey in her personal and professional life led to her role with Global Genes and has propelled the search for a cure for her daughter and kids like her. 

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