“CHI encourages research and development efforts dedicated to advancing new treatments for patients suffering from rare diseases." - David L. Gollaher, Ph.D., President and CEO, CHI
Sacramento (PRWEB) February 28, 2013
Today at the State Capitol, Assemblyman Marc Levine (D-San Rafael), in partnership with the California Healthcare Institute (CHI), presented a House resolution recognizing Feb. 28, 2013 as Rare Disease Day.
“On this 30th anniversary of the Orphan Drug Act, we recognize the need for continued incentives for biomedical innovators to bring much needed treatments to patients in need,” Levine said.
It is estimated that there are 7,000 diseases considered rare in the U.S. affecting almost 30 million Americans or about 1 in 10 people. Most rare and ultra-rare diseases have no treatment.
In 1983, Congress enacted the Orphan Drug Act to provide incentives for medical pharmaceutical and product developers to focus on treatments for victims of rare diseases. Since that time, there have been more than 2,700 products studied as possible treatments for rare diseases, and more than 400 of those have been approved by the U.S. Food and Drug Administration. Most of these were for diseases that had no approved treatment.
A disease is considered rare in the U.S. if it affects fewer than 200,000 people. Approximately 50 percent of the people affected by rare diseases are children. Even more prominent are ultra-rare diseases that affect only a few thousand individuals.
Patients with rare diseases and ultra-rare diseases are frequently misdiagnosed or are undiagnosed. Few drug companies conduct research into rare diseases since it is difficult to recover the costs of developing treatments for small, geographically dispersed populations.
BioMarin Pharmaceuticals Inc., based in Novato, Calif., is a pioneer in the area of rare disease research. Dedicated to having a meaningful impact in the lives of underserved patients suffering from diseases that are often ignored, BioMarin has successfully brought four therapies for rare genetic diseases to market. The company has five additional therapies in various phases of clinical trials and multiple pre-clinical programs devoted to treating additional rare diseases. Due to their global success of providing effective therapies, BioMarin has nearly doubled their number of employees in the last five years.
“CHI encourages research and development efforts dedicated to advancing new treatments for patients suffering from rare diseases,” said CHI President and CEO David L. Gollaher, Ph.D. “Rare Disease Day serves as a reminder that while millions of patients have benefited from new treatments, many more remain in need of timely and accurate diagnosis, along with adequate treatment options.”
CHI represents more than 275 leading biotechnology, medical device, diagnostics, and pharmaceutical companies, and public and private academic biomedical research organizations. CHI’s mission is to advance responsible public policies that foster medical innovation and promote scientific discovery. CHI’s website is http://www.chi.org. Follow us on Twitter @calhealthcare, Facebook, LinkedIn and YouTube.