February 27th, 2012
Why Rare Disease Day is So Important
When EURORDIS first created Rare Disease Day in 2008, my first reaction was a bit skeptical. There are so many awareness days. I wasn’t sure the world needed one more…or that a day to focus on rare diseases would do anything concrete to help patients and families.
My thinking has changed radically on this. On this 5th anniversary of Rare Disease Day, I am overwhelmed at how far we all have come and how much this simple gesture has done to draw the global rare disease community together.
Just think, for a moment, about the following, bearing in mind that the global theme for this year is “Solidarity”:
• While we all have different challenges and concerns, at this one time of the year, patients, government officials, medical professionals and the pharma/bio industry are all speaking together with one voice and one set of goals.
• In countries as diverse in other respects as Iran, the USA, China, and Pakistan, Rare Disease Day Partners are sharing the same theme, logo, poster, video, and key messages.
• In this era when the social media rule, it’s astonishing to see the volume of Rare Disease Day photos, stories, videos and blogs … some quite professional in nature and others obviously “home grown” … and the consistency in the messaging, regardless of the country of origin.
• The first year, Rare Disease Day was observed primarily in the EU and Canada. Last year, it was observed in 66 nations and this year the number will be even higher.
In the USA, where my organization sponsors Rare Disease Day, nearly 700 patient organizations, government agencies, medical societies, and companies have signed on as Rare Disease Day Partners, displaying the logo and supporting the goals of the day.
The videos this year have been particularly moving to me. Yann’s video message does a wonderful job of stating the themes and purposes of Rare Disease Day. Others, especially the ones featuring children and young people, tug at our heartstrings and leave us with an even greater sense of urgency to accomplish our goals. My personal favorites … and there are many … include a young girl named Gracie singing “Brave”, a song she has written about living with Schwachman-Diamond syndrome, and a six-year-old boy named Ian who concludes his video with the ringing declaration: “I’m a histio warrier!”
But the most wonderful thing about Rare Disease Day isn’t just that it draws people together and encourages solidarity. Rare Disease Day actually helps drive progress toward our stated goals: more research; more safe, effective treatments; greater access to treatments; and a greater emphasis on rare diseases globally as a public health issue.
In Europe, Rare Disease Day is a time to focus on the need for national plans and to remind everyone that the clock is ticking toward the deadline for accomplishing that. In the USA, the National Institutes of Health and several highly regarded medical research institutions are hosting scientific conferences focusing on rare diseases. The Food and Drug Administration will host its first-ever Rare Disease Patient Advocacy Day.
FDA had worried initially that it might “throw a party and no one would come.” To the contrary, the organizers actually had to close down the registration a week ago because they were at full capacity. Patients are thrilled. This is the first time most have ever ventured onto the FDA campus. And, from the Commissioner on down, senior officials will be on hand to answer questions, explain the terminology and the process, and talk about how patients can participate.
Similar things are happening around the world. So, I have long ago put away my initial skepticism. We all owe Yann and EURORDIS a debt of gratitude for creating Rare Disease Day. It has accomplished more in these five short years than anyone could possibly have anticipated when it all first began.
My thinking has changed radically on this. On this 5th anniversary of Rare Disease Day, I am overwhelmed at how far we all have come and how much this simple gesture has done to draw the global rare disease community together.
Just think, for a moment, about the following, bearing in mind that the global theme for this year is “Solidarity”:
• While we all have different challenges and concerns, at this one time of the year, patients, government officials, medical professionals and the pharma/bio industry are all speaking together with one voice and one set of goals.
• In countries as diverse in other respects as Iran, the USA, China, and Pakistan, Rare Disease Day Partners are sharing the same theme, logo, poster, video, and key messages.
• In this era when the social media rule, it’s astonishing to see the volume of Rare Disease Day photos, stories, videos and blogs … some quite professional in nature and others obviously “home grown” … and the consistency in the messaging, regardless of the country of origin.
• The first year, Rare Disease Day was observed primarily in the EU and Canada. Last year, it was observed in 66 nations and this year the number will be even higher.
In the USA, where my organization sponsors Rare Disease Day, nearly 700 patient organizations, government agencies, medical societies, and companies have signed on as Rare Disease Day Partners, displaying the logo and supporting the goals of the day.
The videos this year have been particularly moving to me. Yann’s video message does a wonderful job of stating the themes and purposes of Rare Disease Day. Others, especially the ones featuring children and young people, tug at our heartstrings and leave us with an even greater sense of urgency to accomplish our goals. My personal favorites … and there are many … include a young girl named Gracie singing “Brave”, a song she has written about living with Schwachman-Diamond syndrome, and a six-year-old boy named Ian who concludes his video with the ringing declaration: “I’m a histio warrier!”
But the most wonderful thing about Rare Disease Day isn’t just that it draws people together and encourages solidarity. Rare Disease Day actually helps drive progress toward our stated goals: more research; more safe, effective treatments; greater access to treatments; and a greater emphasis on rare diseases globally as a public health issue.
In Europe, Rare Disease Day is a time to focus on the need for national plans and to remind everyone that the clock is ticking toward the deadline for accomplishing that. In the USA, the National Institutes of Health and several highly regarded medical research institutions are hosting scientific conferences focusing on rare diseases. The Food and Drug Administration will host its first-ever Rare Disease Patient Advocacy Day.
FDA had worried initially that it might “throw a party and no one would come.” To the contrary, the organizers actually had to close down the registration a week ago because they were at full capacity. Patients are thrilled. This is the first time most have ever ventured onto the FDA campus. And, from the Commissioner on down, senior officials will be on hand to answer questions, explain the terminology and the process, and talk about how patients can participate.
Similar things are happening around the world. So, I have long ago put away my initial skepticism. We all owe Yann and EURORDIS a debt of gratitude for creating Rare Disease Day. It has accomplished more in these five short years than anyone could possibly have anticipated when it all first began.
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