NORD’s Breakthrough Summit: Listening to the Rare Disease Patient Voice

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The National Organization for Rare Disorders (NORD) is set to host its annual Rare Diseases and Orphan Products Breakthrough Summit on October 18-19th. Ahead of the Summit, the Chair of NORD’s Board of Directors Kay Holcombe explains its significance for cross-stakeholder interaction and learning and why we are entering “a new era for rare disease patients and families.”

 

It has been my privilege over a number of years to work with the National Organization for Rare Disorders (NORD) and to observe first-hand why and how they are the leading independent advocacy organization representing all patients affected by rare diseases in the United States. On October 18 and 19, 2021, NORD will host (virtually, thanks to COVID-19) its annual premier event the Rare Diseases and Orphan Products Breakthrough Summit.® This annual gathering provides an unparalleled opportunity to learn from medical and academic leaders, regulators and policymakers, innovators and – most significantly – from patients, their families and their caregivers. The NORD Summit is a can’t miss event that helps chart the future of rare disease diagnosis, treatment and cures, highlighting the importance of sound public policy, collaboration, excellence in data collection and analysis and listening to the patient.

 

I have filled several roles at the Summit over many years but still remember my first experience of seeing from the podium the very people whose lives had been unalterably changed by a rare disease — patients, caregivers and patient advocates — sitting alongside medical experts and researchers, corporate thought leaders and government health policy officials. This was not your ordinary Summit. And it never will be. Indeed, its prescient inclusion of “the patient voice” has continued to make this the most important rare disease conference hosted every year.

 

We are on the precipice of a new era for the rare disease world, and there is more promise than ever before to make truly life-changing advances for patients

 

We are on the precipice of a new era for the rare disease world, and there is more promise than ever before to make truly life-changing advances for patients. Critical sessions at this year’s Summit will address rare diseases as a public health challenge; discuss how rare disease product development, including fundamental research and clinical trials, could be changed permanently as a result of things we’ve learned during COVID-19; applaud the increasing international collaboration in the rare community; and address healthcare inequities and making clinical trials more inclusive and diverse. These are leading, prominent discussions in which all attendees will be able to engage.

 

I am particularly excited about the innovative use of health technology and patient-generated data. NORD has made a longstanding commitment to the elevation and standardization of rare disease registries and data sharing, particularly through the IAMRARE® Database and now the Rare Disease Cures Accelerator-Data and Analytics Platform initiative (RDCA-DAP®). It is so important to see this initiative featured on the virtual main stage. The work NORD and Critical Path Institute (C-Path) are doing together, with funding and support from the US Food and Drug Administration (FDA), will make a huge contribution to the development of therapies for rare diseases. This illustrates the kinds of cutting-edge innovations that the Summit has brought to the fore each year so all stakeholders, including patients and families, can make the best use of advancing knowledge.

 

The NORD Summit is a unique event, a cross between a jam-packed agenda of terrific speakers and topics and a get-together for friends who have never met but whose lives are inextricably linked. The content is dynamic and engaging and the participants include everyone who is anyone in rare diseases – patients, families and caregivers; researchers and clinicians; private sector product developers and regulators and policymakers. And it is important for every one of these participants to be there – everyone has a role. Bringing all these stakeholders together generates an environment of growth, collaboration and progress. All Summit attendees together are helping to usher in this new era for the rare disease world.

 

NORD has been organizing rare disease conferences since the 1980s and the NORD Summit in its current form was launched as an annual event 12 years ago. Throughout all the change in the world over the years, the conference has never lost sight of its number one priority: improving the lives of rare disease patients. This conference is successful because it is accessible – it is a place that everyone can learn something new, hear inspiring and important stories and network, network network.

 

Registration for the Summit is open now. Attend the Summit with questions in your mind and on your notepad; that you will find answers is virtually guaranteed – and you may find more questions too. That’s okay, I’m right there with you – let’s continue to ask and answer those questions together. This truly is, as I have said, a new era for rare disease patients and families, and I’m excited to learn more at the Breakthrough Summit.


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