Rare Disease Day organizers from around the world made “Rare Disorders Without Borders” the slogan for 2013. I see in it a potent reminder that rare disorders exist everywhere in the world, and I see in it a challenge to ensure that geographic borders don’t bar access to research initiatives, therapeutic options, and support services. The slogan is particularly poignant during the 30th anniversary year of the National Organization for Rare Disorders (NORD). This organization was born with the Orphan Drug Act of 1983—legislation that provided drug and device developers incentives to invent and market products for rare disorders. Since then, over 2,700 potential treatments have poured into the research pipeline as “orphan products”, and the U.S. Food and Drug Administration (FDA) has approved over 400. More products have become more available for more people with rare disorders as a direct result of this legislation, and more are coming.
When rare disorders crossed into commercial research and development, however, other borders remained, and new borders were revealed. Like the Haitian proverb that says, “beyond mountains, there are more mountains,” in rare disorders, beyond borders, there are more borders.* And like the Haitian proverb, we must see this statement as both realistic and hopeful. Mountains act as obstacles to overcome as well as opportunities to capture; rare disorder communities can approach borders in the same way. In forthcoming blog posts I intend to explore borders important to rare disorder constituencies and how these borders outline the challenges and opportunities facing people affected by rare disorders.
One border I intend to focus on runs within individual families. Most people imagine and plan their future lives based on family histories and hopes; they anticipate trajectories that seldom accommodate a rare disorder. Confronting any of these disorders alters expectations and can create borders—setting parents and children apart, dividing siblings, and fragmenting personal relationships for example. Another set of borders cuts across society. These can be the borders built of institutional arrangements like distribution and payment systems for rare disorder treatments as well as social policies like the current health care reforms. Other societal borders are erected by cultural views, such as common disease and illness conceptions, which in turn follow traditions and trends, public debates, and even language structures.
Borders that function as barriers to diagnosis, treatment, and support are frightening prospects to people affected by rare disorders. Borders that can be seen, however, are the borders that can be addressed. Borders that are not seen are the more worrisome. Therefore, I invite readers to comment on the borders I will be considering and to identify others that need revealing. Rare disorder groups started the conversation about geographic borders, and our conversation can continue traversing borders beyond borders.
*From, Kidder T. Mountains Beyond Mountains: The Quest of Dr. Paul Farmer, A Man Who Would Cure the World. New York; Random House: 2009.
J. Russell Teagarden recently joined NORD as the Senior Vice President, Medical & Scientific Affairs.
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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