Safeguarding the Rights of a Vulnerable Population

NORD celebrates 35 years of advocating for people with rare diseases.

NORD staffers cheer on the Running for Rare team at the Hartford Marathon
NORD staffers and patients cheer on the Running for Rare team at the Hartford Marathon, one of many programs sponsored by the Hartford Marathon Foundation whose mission is to encourage individuals and families to adopt and maintain a healthy and active lifestyle.

Vita interviews National Organization for Rare Disorders (NORD) Executive Director Peter L. Saltonstall about the organization’s 35th anniversary advocating for rare disease patients and the road ahead.

 

Vita: Congratulations on NORD’s 35th Anniversary!  Can you talk about some of the ways in which the advocacy landscape has changed since NORD was established 35 years ago?

PS: The patient advocacy landscape has changed considerably over the past 35 years, particularly in the rare disease community. More patients are engaging in activism, and existing advocates are maturing in their knowledge of complex systems including the FDA drug approval and regulatory process. Social and peer-to-peer networking has provided patients with effective ways to amplify their voices and messages digitally. Not only has this resulted in the creation of new communication platforms and tactics, but it has accelerated the ability to raise awareness around specific causes. 

I believe we are entering into a new generation of patient advocacy. While sharing patient experiences and stories will always be an important advocacy tool, advocacy can only drive real change when coupled with empirical data. NORD is leading the charge in this data-driven approach to advocacy.

Vita: Looking back on the organization’s many accomplishments advocating on behalf of individuals with rare diseases, what stands out as some of NORD’S most important achievements?

PS: Aside from NORD's advocacy for the Orphan Drug Act of 1983, which stands front and center among our most important achievements, we are proud to have undertaken a number of major projects over the years. It’s difficult to highlight just a few, but there are several that stand out.  Establishing the first Patient Assistance Program in 1987 and awarding $7 million in research funding are two that come to mind.  At least two FDA-approved products began with small research projects funded by NORD.

In 2009 we launched Rare Disease Day in the US in partnership with EURORDIS and provided advocacy for the establishment of the ClinicalTrials.gov website. Hosting a Natural History/Patient Registry Platform to collect longitudinal data, which is data gathered for the same subjects repeatedly over a period of time, is an important accomplishment to support research and better understand diseases. NORD also led the advocacy initiative to maintain the Orphan Drug Tax Credit when it appeared this important incentive might be eliminated altogether.

Vita: What are the biggest challenges facing people with rare diseases today?

PS: The greatest challenges for rare disease patients are still the frequent delays in diagnosis and lack of available information and data on their disease. Without this information it is difficult for researchers to study their disease, which hinders the development of new treatments or cures, and for physicians to accurately diagnose patients. Registries and natural history studies are critical to addressing this challenge.

Vita: Are people more open and willing to share and talk about their rare disorders today than 35 years ago and if so, has it affected awareness, connectedness, etc.?

PS: It may be that people are more open to sharing today but more importantly, the rise of platforms through which they can now share their stories and experiences has amplified their voices significantly over the years. The speed at which communication now moves, and the methods there are to choose from as opposed to the channels that were available in 1983, have made raising awareness and forming connections infinitely easier and more immediate.

Vita: How has social media influenced/impacted patient advocacy?

PS: Whereas in the past rare disease patients often felt isolated and disconnected, many rare disease communities today – even those with less than 100 patients – are able to find each other on social media, especially Facebook. There are now hundreds of private disease-specific Facebook groups that have become hubs of activity, welcoming and supporting newly diagnosed patients, while galvanizing their communities for national and international advocacy.

NORD saw firsthand the impact that social media can have in patient advocacy when we engaged individuals to make a difference in our effort to protect the Orphan Drug Tax Credit. Patient advocates who couldn't fly to D.C. or be at their state capitol got the chance to make their voices heard using social media. During this effort, we saw more than 4,000 messages using our hashtag, #SaveOrphanDrugs.

Vita: How do you envision the role of patient advocacy 35 years from now when NORD celebrates its 70th anniversary?

PS: Patient roles in research have taken on more importance than ever and will continue in that direction. This year NORD entered into a first-of-its-kind memorandum of understanding through which the FDA and NORD will work together to identify patient community representatives and coordinate listening sessions where patients can share their experiences with FDA review division staff. Our goal is to facilitate the patient’s voice being heard more directly in the regulatory decision-making process, an invaluable opportunity for the rare disease community to advance medical research and new product approvals.

Moving forward, I envision seeing an even more active and involved role for patients on all fronts - research, policy, education, and advocacy. For example, NORD’s Rare Action Network (RAN) has grown by leaps and bounds over the last few years. RAN now has more than 4,000 advocates in all 50 states. We are quickly moving in the direction of what I think of as next-generation advocacy, which couples patient stories with empirical data to drive real change, not just awareness.