LETTER: Counsel needed for rare diseases
According to the National Institutes of Health, one in 10 Americans live with a rare disease. Rare diseases and disorders are those which affect small patient populations, typically populations smaller than 200,000 individuals in the United States. Such diseases and conditions include Huntington's disease, Lou Gehrig's disease, Tourette syndrome, Crohn's disease and cystic fibrosis, and even certain types of cancers. Of the nearly 7,000 rare diseases identified by the NIH, only about 200 have Food and Drug Administration-approved treatments. Although categorized as "rare," most of us have family or friends whose lives are impacted by a rare disease. I have a friend who lost her husband to cystic fibrosis at the age of 38 and know of many people who suffer from Crohn's disease.
On Jan. 26, state Representative Marcy Toepel introduced House Bill 239 that would create a rare disease advisory council. The establishment of the council will create a unique collaborative partnership between patients, providers, researchers, agencies and insurers in an effort to improve the lives of those living with one of the 7,000 devastating rare diseases. The council's focus will be on the needs of the rare disease community and the actions necessary to address those needs.
The fact that only 200 approved treatments exist for over 7,000 rare diseases epitomizes the need for a focused plan to address rare diseases. The implementation of a rare disease advisory council in Pennsylvania would be a significant step in the effort to identify and improve treatments for rare diseases. I would therefore encourage you to contact your state Representative and state Senator and ask them to cosponsor and support House Bill 239.
Harleysville, Pa. (formerly of York)