Rare Disease Diversity Coalition Publishes Roadmap to Achieving Health Equity in Rare Disease

On February 26, 2021, Travere Therapeutics was honored to have Linda Goler Blount, president of the Black Women’s Health Imperative (BWHI), present a keynote address at our Rare Disease Day program. The opportunity for Travere team members to hear Linda speak came on the heels of the first public meeting of the Rare Disease Diversity Coalition, which is led by BWHI.

At the coalition’s meeting earlier that week, more than 200 rare disease community members came together to endorse an ambitious platform of 11 initiatives that had been researched and developed in working groups since the previous spring. These initiatives set out to achieve tangible solutions to address the delays in diagnosis and treatment for diverse patients, and the historic and cultural barriers to medical care and research. The complete roadmap document is available at https://www.rarediseasediversity.org/action-plan.

During Travere’s Rare Disease Day program, Linda shared some of the specific recommendations the RDDC is advocating for on behalf of patients of color living with rare disease. In recognition of national minority health month, in gratitude for her partnership, and honor of all those living with rare disease, we are sharing a piece of Linda’s inspiring message below.

…We want to give hope to those people who are struggling to go on, or who are on the brink of giving up. That’s why we created the first of its kind Rare Disease Diversity Coalition, a broad, committed coalition of doctors, researchers, patient advocates, and industry leaders. We were born out of a collective commitment to be a catalyst for change for people of color with rare disease. 

So let me share with you a little bit about how we arrived at our roadmap to achieving health equity in the rare disease community, and then some of the specific recommendations we’re advocating for, on behalf of patients of color living with rare disease. As we developed our ideas, we’ve been encouraged by the momentum for change over the past few months. Key policymakers have expressed the will to develop policy to address racial health disparities. We’ve been further encouraged after engaging some of these policymakers about the prospect of working cooperatively with the coalition. 

Our coalition formed its plans through evidence-based research, inclusion, advocacy, and transparent acknowledgement of the challenges we faced. A great example were the coalitions that led to the Orphan Drug Act, which resulted in the development of the National Organization for Rare Disorders that demonstrated that together, we can drive progress on both rare disease and health equality. The coalition undertook a six-month study to assess the most pressing challenges faced by people of color with rare disease, and to identify potential solutions. Based on an extensive literature review, research, working group analysis, and input from patients, and the Steering Committee, we identified priority recommendations that directly address systemic and structural barriers to health. We landed on three critical areas.

One, reduce the time from the onset of symptoms to a diagnosis.

Two, educate, equip and engage patients and healthcare providers on racial bias and recruit more medical students of color, and fund more researchers of color.

And three, increase medical literacy in rare disease patients and caregivers, by communicating the importance of knowing their family medical history, and educating them on their right to treatment and inclusion in clinical trials.

We have come a long way since my first meeting with Eric and the Travere team just one year ago, and I had no idea at the time that a simple get to know you meeting would have such a profound impact on my life, and that of my family. Achieving health equity in the rare disease community won’t be easy. It requires a willingness to rethink how the nation approaches research and healthcare, investment of time and resources, and a relentless commitment. But what Travere and the other pharmaceutical companies in the coalition can do is set the example for other drug development companies for what inclusion looks like. It must be done, and the Rare Disease Diversity Coalition is proud to have taken that first step. Without Travere, this coalition would not have been possible. The coalition’s key recommendations are just the beginning, and you can find the recommendations, along with all of our future work on the coalition webpage, rarediseasediversity.org.

I want to thank all of you. I hope each of you shares in the sense of pride that I feel doing something truly remarkable and groundbreaking on behalf of rare disease patients of color, and frankly, on behalf of the work for health equity. Although we’ve got a long way to go, look at what can be accomplished when a committed group of people have the will to change. We are confident that as this nation learns more about our mission, vision and goals, others too will join us and contribute to inspire hope, hope that comes with the knowledge that patients of color living with rare disease can expect to be treated with value, with dignity, with respect, that their journeys from symptoms to diagnosis to treatment, can be short because of who they are, and not because of who they know.

Learn more about the Rare Disease Diversity Coalition.

Achieving health equity in the rare disease community won’t be easy. It requires a willingness to rethink how the nation approaches research and healthcare, investment of time and resources, and a relentless commitment to justice and compassion, and it takes political will.

^{Linda Blount, President, Black Women’s Health Imperative}