Bringing the patient voice to the States

NORD’s President and CEO urges drug and medical device companies to help bring the patient voice to educate state legislators and health officials about the challenges faced by patients with rare diseases.

In his State of the Union address, President Obama struck the hearts of the Congress and the Nation when he described the medical challenges faced by Cory Remsburg, an Army Ranger who was seriously injured in Afghanistan but who is overcoming his injuries. The reception by Members of Congress was overwhelming.

Ranger Remsburg went through medical challenges due to injuries. He did not have a rare disease. The reason that his story moved me so deeply is because he reminded me of the many millions of patients who face similar medical challenges on a daily basis, and whose stories are worth recounting.

President Obama told Remsburg’s story to make a point about American dedication and heroism. It was memorable because the story was about one individual who fought to survive.

“There are many millions of patients with a rare disease – all have a story worth hearing”

Within the rare disease community, there are an unlimited number of similar stories that need to be told. Last May, at NORD’s gala dinner, we invited patients with rare diseases to tell their stories, sharing the stage with representatives of the drug companies with drugs approved for their disease. The impact on the audience was dramatic.

I write this as Rare Disease Day 2014 approaches. Rare Disease Day is the last day of February, and is one of the signature events sponsored by NORD to call attention to rare diseases and their impact on patients and on the community at large.

This year, one of the core activities being planned involves events to educate state legislators about rare diseases.

Rare Disease Day 2014 presents the ideal opportunity for those concerned about rare diseases, particularly representatives of the drug and medical devices industries (because of their existing contacts in the states), to tell the stories of the rare disease patient directly to state legislators and health officials.

For Rare Disease Day 2013, there were State House events in California, Connecticut, Massachusetts, New Jersey and Texas. In each case, they represented collaborations of patient organizations, companies, and medical professionals.

Each of these events had memorable moments. In New Jersey, 12-year-old Brooke Foster demonstrated the commitment of the next generation of rare disease patient advocates with a moving presentation about the rare disease of particular interest to her family – mastocytosis.

In Connecticut, several members of the State Legislature, including the head of the Public Health Committee, participated in the program and affirmed the value of greater understanding of rare diseases. Families coping with specific rare diseases such as Friedreich’s ataxia and adrenoleukodystrophy shared their stories with the legislators and were interviewed by TV reporters.

The Texas event began with a rousing rally on the State House steps, and the California event drew a strong representation from both patient organizations and industry partners.

In Massachusetts, where it all began, representatives of MassBio and the Shwachman Diamond Syndrome Foundation – who organized the first Rare Disease Day State House event several years ago — hosted a program in which numerous rare disease stakeholders shared their vision for a better future with the state’s decision-makers.

The fact that NORD and our members and corporate partners consider this a priority reflects a fundamental change in thinking among rare disease advocates.

For years, we focused almost entirely on national legislation and the U.S. Congress. But now, with the implementation of the Affordable Care Act and other economic and social forces, it’s become increasingly clear that there is a need, and exciting new opportunities, to have an impact at the state level while also continuing our national advocacy.

“Rare disease patients need to be heard at state level”

From newborn screening to prescription drug health coverage, very significant healthcare decisions are made at the state level. We are seeking to maintain an ongoing dialogue with state officials. We urge our friends in the drug and device industries to help us mobilize this effort. We have a common interest in assuring that patients have access to health care and to the therapies they need.

One specific way is to collaborate with patient organizations to bring the voice of the patient to the state house, if not on Rare Disease Day 2014, then on a later date. Drug and device companies increasingly have strong presences at the state level, and harnessing the voice of the patient is one way to demonstrate that we are all in this fight together.

To help, NORD has created a toolkit that is available to all on the official national website for Rare Disease Day –

Anyone representing a rare disease patient organization, academic institution, company or medical society can seek NORD’s assistance in organizing a State House event. We will connect that individual or organization with others in the state, and we’ll provide advice, guidance and encouragement as the event is being organized.

“Companies are invited to work with patients to educate state officials”

State outreach is an important new initiative for NORD, in recognition of the new health care environment. We are actively soliciting the support of companies with a common interest. There are many ways that companies can have an impact at the state level, and pairing with patients to educate state officials is just one of them.

All of our Presidents since Ronald Reagan have invited individuals to the State of Union address because they know that individual stories are memorable. Patients with rare diseases have moving stories. I urge you to learn from the Presidents and help bring the patient voice to our states.


About the author:

Peter L. Saltonstall is President and CEO of the National Organization for Rare Disorders (NORD). Visit the NORD website at

Closing thought: How can rare disease patients make their voice heard most effectively?