Governor Declares February 28th Rare Disease Day in Massachusetts

February 26, 2009

The last day of February has been designated as Rare Disease Day in Massachusetts to call attention to the public health issues associated with rare diseases, which affect nearly 30 million Americans and countless others around the world.

At the request of the Massachusetts Biotechnology Council (MBC), Gov. Deval Patrick recently signed a proclamation declaring February 28th as Rare Disease Day. (To see the proclamation, click here .) The Commonwealth joins a coalition of organizations recognizing the day, being coordinated by the National Organization for Rare Disorders (NORD), that includes patient organizations, professional societies, government agencies, medical researchers, and pharmaceutical and biotechnology companies.

Massachusetts-based biotechnology companies, including Shire, Genzyme and FoldRX Pharmaceuticals have taken an active role in the Rare Disease Day coalition by supporting efforts to designate February 28th as Rare Disease Day in Massachusetts. Additionally, some several companies are hosting special events in recognition of Rare Disease Day.

"The Massachusetts biotechnology community has long been at work discovering treatments and cures for rare diseases. We want people to learn about these diseases and to better understand the importance of maintaining a regulatory climate that encourages this vital innovation," said Robert K. Coughlin, President and CEO of the Massachusetts Biotechnology Council. "We are proud to support this initiative, and draw attention to the special challenges facing patients with rare diseases and their families every day."

A rare disease is one that affects fewer than 200,000 Americans. According to the National Institutes of Health (NIH), there are nearly 7,000 such diseases affecting nearly 30 million Americans.

Rare Disease Day activities in the U.S. will include a nationwide network of online videos, patient stories and blogs; newspaper, radio, and television reports; state and municipal proclamations; a Rare Disease Hall of Fame for researchers; and other activities designed to raise awareness of what it means to have a rare disease.

People with rare diseases often face challenges that occur less frequently with more common diseases, including delay in getting an accurate diagnosis, few treatment options, and difficulty finding medical experts. Many rare diseases have no approved treatment, and insurance may not cover treatments that aren't approved. Medical and social services may be denied because those making the decisions are not familiar with the diseases. Also, treatments for rare diseases tend to be more expensive than treatments for more common diseases.

In 1983, the Orphan Drug Act was passed by Congress to create financial incentives for companies to develop treatments for rare diseases. Since then, nearly 330 "orphan" (for rare diseases) drugs and biologics have been approved by the U.S. Food and Drug Administration (FDA). FDA estimates that from 11 to 14 million Americans benefit from these products, but that still leaves more than 15 million Americans with diseases for which there is no approved treatment.

Rare Disease Day also will highlight the unique partnership that exists among the patient community, government entities such as the NIH Office of Rare Diseases and FDA Office of Orphan Products Development, medical professionals, researchers, and companies developing orphan products.

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The Massachusetts Biotechnology Council
www.massbio.org
The Massachusetts Biotechnology Council (MBC), a not-for-profit organization that represents and provides services and support for the Massachusetts biotechnology industry, is the nation's oldest biotechnology trade association. Founded in 1985, the MBC is committed to advancing the development of critical new science, technology and medicines that benefit people worldwide. Representing over 600 biotechnology companies, academic institutions, research hospitals, and service organizations involved in life sciences and health care, the MBC works to advance policy and promote education, while providing member programs and events, industry information, and services.

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