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Foundation urges expedited drug approval for rare disorders

A local foundation named for a young boy with a lethal disease is urging Congress to pass legislation that would speed up the Food and Drug Administration's lengthy drug approval process. 

Suneel's Light Foundation Chief Science Officer George Hajduczok says the FAST (Faster Access to Specialized Treatments) Act removes regulatory uncertainty and formalizes the process for getting drugs approved faster.  

"Sometimes for normal clinical trials you need thousands of patients to get FDA approval for a drug before it goes on a market. For many of these disorders, there aren't even a thousand people around to qualify for the normal process," Hajduczok said.

Hajduczok says the act can affect the entire pharmaceutical industry.

"It should also incentivize smaller companies for the rare disorders, one where there isn't that much market for some of these drugs, but yet it will help. If you know that you can get a drug through a little quicker, if the protocols are a little different, and yet not sacrifice safety, they'll be all for it," said Hajduczok.

Hajduczok says for people with a limited lifespan, urgency is of the utmost importance, and the FDA's current drug approval process takes years. 

Suneel's Light Foundation, based in Williamsville, funds research for treating and curing Dushenne Muscular Dystrophy, a lethal genetic childhood disease that only targets boys.