HORSHAM, Pa., Oct. 18, 2012 /PRNewswire/ --A panel of parents who became patient advocates after their children were diagnosed with rare diseases will discuss their struggles and successes in navigating the drug development process on Monday at a Washington, D.C., conference sponsored by DIA and the National Organization for Rare Disorders (NORD).
The work carried out by these parents through rare disease associations can be one of the most important factors in getting lifesaving help for their children. For many parents, the news that their child has a rare disease has changed the course of their lives, including their leaving other careers to work full time to advance the development of therapies to treat severe illnesses that may affect as few as 500 people in the United States. Five of these parents and one patient will speak at the 2012 U.S. Conference on Rare Diseases and Orphan Products.
"People are going along living their lives and then learn their child has a rare disease. These parents are diving into a world unknown to them to navigate the complicated path to find a treatment," said Peter L. Saltonstall, NORD president and CEO. "We see it over and over again. The result can be critical funding and advocacy for groundbreaking research."
Patient advocate Ron Bartek lost his son, Keith, to Friedreich's ataxia, a genetic disease that affects the heart and muscles. There is no treatment for the disease. Bartek and his wife, Raychel, founded the Friedreich's Ataxia Research Alliance, working tirelessly to raise money for research and clinical trials to advance the likelihood of developing an effective treatment.
Bartek said pharmaceutical companies showed no interest in developing a drug for the disease because it is so rare. Fourteen years later, he is confident that clinical trials under way will result in the first treatment for the diseasethanks to the money the alliance raised for research and clinical trials.
"They got interested because we did all of that," said Bartek. "It's almost purely a financial decision. Whether a treatment gets developed often depends on the ability of patients or their advocates to get the process started."
Pharmaceutical companies traditionally have turned their attention to developing therapies for diseases that affect larger patient populations. Because the drug development process is very expensive, time-consuming and challenging, companies have had few incentives to commit the resources to develop therapies that affect very small numbers of patients. However, data show a recent renewed interest by pharmaceutical and biotech companies in the development of rare disease therapies.
Other panelists include:
WHAT:
Panel discussion for members of the media featuring advocates for rare disease patients at the U.S. Conference on Rare Diseases and Orphan Products
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Patient Advocates Tell of Struggles in Getting Drugs Developed for Rare Diseases