CHI lauds California state Assembly's proclamation of Feb. 28, as Rare Disease Day

Feb 27 2010

CHI-California Healthcare Institute, a non-profit public policy research organization, applauds the state Assembly’s proclamation of Feb. 28, as Rare Disease Day. This day helps bring awareness of the biomedical companies that are dedicated to researching and developing treatments for patients suffering from rare diseases. A rare disease is defined as a condition that affects fewer than 200,000 patients. Because populations for rare diseases are small and the cost to develop a drug and bring it through clinical trials can be hundreds of millions of dollars, few companies can invest in creating these needed therapies. Additionally, the biggest obstacle to finding cures for rare diseases is their scarcity—so few people are affected that it is difficult to gather enough patients for clinical trials.

“As formidable as the challenges to creating treatments for rare diseases are, CHI would like to recognize those California innovators stepping up to make therapies available and support patients in need of a cure.”

“Families dealing with a rare disease often feel helpless, isolated and frustrated due to the limited options available,” said David Gollaher, Ph.D., CHI’s president and CEO. “As formidable as the challenges to creating treatments for rare diseases are, CHI would like to recognize those California innovators stepping up to make therapies available and support patients in need of a cure.”

California Healthcare Institute member companies and partnering organizations that have taken on the challenges of creating new therapies for rare diseases and supporting the patients who suffer include: Amgen and BioMarin Pharmaceuticals, which has focused its mission solely to discovering and developing therapies for rare diseases, Genzyme, CureDuchenne, The Nicholas Conor Institute for Pediatric Cancer Research, and the Children’s Rare Disease Network.

Rare Disease Day, Sunday, Feb. 28, was proclaimed by the California state Assembly as a day to recognize and support the patients and their families affected by these debilitating diseases. CHI approached Asm. Jerry Hill (D-San Mateo) with the legislative language to recognize Rare Disease Day in the state of California (ACR 117). The bill was passed on a unanimous voice vote, after 44 legislators signed on to be co-authors of the measure. The resolution now goes to the state Senate for final passage next week. The public can show its support by printing out posters or displaying the Rare Disease Day logo on their Web sites. Click here to upload your poster or logo. To learn more about Rare Disease Day and other ways to get involved visit www.rarediseaseday.org.

Statistics on rare diseases:

• One in 10 Americans, nearly 30 million people, suffer from a rare disease.
• There are nearly 8,000 rare diseases.
• Children are disproportionally affected by rare diseases—75 percent of rare diseases affect children.
• More than a third (35 percent) of patients with a rare disease die before the age of five.

SOURCE California Healthcare Institute