Wishing an Act Happy Birthday may seem strange to you, but it makes perfect sense to me. The Orphan Drug Act, signed into law by President Ronald Reagan in 1983, turned 25 this year and unlike some other Acts, this one is truly hard to follow!
Why? Because the Act encourages drug companies and medical researchers to study the most rare medical conditions, also known as Orphan Diseases, defined as those which affect less than 200,000 people in the United States. And, the O.D.A. has had some pretty remarkable results. In fact, it's effect on rare diseases has been nothing less than miraculous. Tax incentives, grants for clinical trials, extended patent exclusivity for drug companies who develop drugs and treatments for Orphan Diseases, is what fueled drug companies to invest millions of dollars in research leading to the development of more than 1,100 new therapies since the Act's inception. What was life like for patients with Orphan Diseases before the Act? In the 10 years prior to the O.D.A., only 10 new drugs for rare diseases were developed, leaving patients with little hope for treatment or cure.
So I for one, am donning a party hat and singing merrily, because I am one of the 6,500 U.S. patients diagnosed with an Orphan Disease known as Hypoparathyroidism. It's quite a mouthful to say, and not an easy disease to live with. This rare endocrine disorder affecting my body's ability to regulate its calcium levels, was brought on by a botched thyroid cancer surgery I underwent in 2001. I nearly died from complications of the disease three times, as there is currently no good treatment and many physicians know very little about it. My work days as an attorney have dwindled too, from a 60 hour week, to an average of 20 hours. My eight year old daughter is often frustrated with her perpetually tired Mama, and the vibrant wife my husband married has vanished. In all honesty, I really feel lousy, but at least I have hope. For the first time since this disease was discovered, it is seriously being studied with clinical trials and drug development taking place in Massachusetts, New York and elsewhere. Hypoparathyroidism is the last area of endocrinology to be explored, the last great frontier of the endocrine research world. So as I blow out its 25 candles, I say, bless you and thank you Orphan Drug Act, and many happy returns!
--Christine Kapsos is a Milwaukee attorney and member of the Hypoparathyroidism Association. For more information about hypoparathyroidism, go to www.hpth.org.