Rare But Equal

PhRMA Staff
PhRMA Staff March 1, 2011
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The theme to Rare Disease Day 2011 was "Rare But Equal."

I glossed over that at first when I saw it, but the more I thought about it, the more chilling it is.

It suggests that patients with rare diseases - which, by definition, affect fewer than 200,000 (though roughly 80 percent are much smaller, affecting fewer than 6,000 patients in the U.S) - feel that they may be seen or treated less-than-equal to patients suffering from more-common illnesses.

It's tragic that they should feel this way; they already face enough challenges in their lives without that added turmoil. But it's wholly understandable.

Many patients with rare diseases suffer for years before receiving accurate diagnoses, because their illnesses are not easily recognized. They may struggle to find specialists to treat them, and likely receive many blank stares from people who've never heard of, for example, myelodysplastic syndromes, Castleman's disease or maple syrup urine disease.

They don't usually get to see national annual walkathons to raise money, or telethons featuring celebrities. Perhaps they don't benefit from aid and living assistant programs that exist for other diseases.

And unfortunately, many do not have a wealth of treatment options available to them.

Still, each life is as important as any other, and each patient is equal to the next. That's why biopharmaceutical research companies continue to invest in rare disease research, even though it can be costlier, riskier, more time-consuming and, ultimately, more frustrating.

Rare disease research presents significant challenges. For example, with small patient populations, it can be nearly impossible for biopharmaceutical research companies to enroll enough clinical trial participants to meet the Food and Drug Administration's demands for new drug approval.

However, the need is clear. Learn more about just what we mean when we talk about rare diseases. Visit the Rare Disease Day Web site to hear directly from patients suffering from these little-known diseases and give them the attention they deserve.

Topics: Rare Diseases, Patients