Rare diseases aren’t really rare at all. One in ten Americans is living with a rare disease. 30% of children with rare diseases will not live to see their 5th birthday. Most rare diseases are genetic and are therefore present throughout a person’s life, even though life-threatening symptoms may not appear until adulthood. There are 7,000 different types of rare disorders, with more being diagnosed each day.
This is beyond a medical challenge-it is a human health crisis. The lack of adequate financial incentives to research, treat, and cure rare diseases is heartbreaking. The numbers speak for themselves. The Orphan Drug Act was passed in 1983; since then, only about 470 drugs have been approved by the FDA and brought to market. For some members of the rare disease community, these treatments have literally transformed-and even saved-their lives. But there is so much more to do –according to the Kakkis Everylife Foundation, 95% of rare diseases have not one single approved drug treatment. Not one.
Imagine the parents who learn that their once active toddlers have Batten Disease. This devastating condition causes progressive mental impairment, seizures, loss of sight, speech, and motor skills. Children with Batten inexorably and inevitably become blind, bedridden, and die, usually before they reach the age of 12. Researchers are trying to help these children–and an effective treatment may be just around the corner. But where is the money to come from? This is just one of thousands of examples-which leads to one basic fact. There is little financial incentive to find the answers that could save or transform the lives of the millions of people with rare diseases. Increasing involvement in and awareness of the huge impact of rare diseases is critical.
But let’s be real. Just like me, it’s human to believe that a rare disease is ever going to hit very close to home. Until it does.
My daughter, Gg, had just turned 21. Beautiful, strong, she was at the mall to buy boots for her upcoming ski trip. Buying them in time for the trip was our crisis. Four hours later, after complaining of back pain, she was completely paralyzed. She could not feel me touch her. Her particular rare disease is called Transverse Myelitis. It affects 1,400 Americans a year-anyone, any age, any time. There is no cure, no clear cause, no treatment other than steroids and plasmapheresis right after the acute attack. My daughter was lucky that the ER doctor recognized her symptoms and began treatment immediately. Six years after becoming paralyzed, Gg is now getting her PhD in Public Health, with her power wheelchair and sheer determination driving her. Our new “normal.” The truth; my daughter has resources and support as she deals with her condition. Others do not.
Isn’t it time to mobilize the forces? Our communications industry can radically raise awareness of the signs and symptoms of rare diseases. Let’s educate and get the word out so that theses diseases are diagnosed earlier. Let’s work hand in hand with government to increase funding–and advocate for the researchers and our pharmaceutical colleagues to find the resources that can ultimately result in “cure” for so many.
EVP Creative Strategist
At REALITYRx we will continue to support awareness and join the fight to help make conquering rare diseases a reality.