California Healthcare Institute Partners with Asm. Kevin Mullin to Recognize Rare Disease Day

Resolution Promotes Awareness of Rare Diseases 

(Sacramento, Feb. 27, 2014) – Today at the state Capitol, Assemblymember Kevin Mullin (D-South San Francisco), in partnership with the California Healthcare Institute (CHI), presented House Resolution 32 recognizing Feb. 28 as Rare Disease Day in California. CHI is a nonprofit, public policy research organization, representing leading California academic institutions, biotechnology, medical device, diagnostics and pharmaceutical firms.

“31 years after the Orphan Drug Act was signed into law, we recognize the need for continued incentives for biomedical innovators to bring much needed treatments to patients with rare diseases,” said Assemblymember Mullin. “While many innovators continue to bring novel treatments into the pipeline, it is essential that sound public policy is developed to support small subsets of patients with rare diseases.”

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. 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. Before Congress acted, the majority of these diseases had no approved treatment.

“CHI members are intimately involved in research and development efforts dedicated to advancing new treatments for rare diseases,” said Eve Bukowski, CHI’s vice president of state government affairs. “Rare Disease Day serves as a critical 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.”

To increase Rare Disease Day awareness and education, CHI released this report on the many therapies and treatment options CHI members are developing. The report also highlights a patient’s perspective and delves into the challenges these patients and their families face daily.

CHI’s Washington team also participated in Rare Disease Day activities, including presenting at the Rare Disease Day Legislative Advocates (RDLA) Legislative Conference earlier this week.

About CHI-California Healthcare Institute
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 Follow us on Twitter @calhealthcare, Facebook, LinkedIn and YouTube.

Will Zasadny