This is an archived article that was published on sltrib.com in 2016, and information in the article may be outdated. It is provided only for personal research purposes and may not be reprinted.
I am writing on behalf of the Utah Rare Disease community about Sen. Orrin Hatch's leadership in helping so many of Utah's most vulnerable.
As the Utah Ambassador for the National Organization on Rare Disorders and the chair of Utah Rare, a coalition of groups dedicated to raising awareness on the impact of rare diseases in our state, I am encouraged by legislation from Hatch called the Advancing Targeted Therapies for Rare Diseases Act (S. 2030) that was just passed by the Senate Committee on Health, Education, Labor, and Pensions.
We have seen numerous advances in precision medicine. However, the generic subtypes of a rare disease may have only a few dozen patients in the U.S., making it very difficult for innovators to find populations large enough to run the necessary clinical trials for the drug to be approved by the Food and Drug Administration (FDA).
This bill clarifies that the FDA has the authority to allow innovators to rely on the work they've done getting a previous drug approved when seeking approval for a drug with a similar chemical backbone targeting a different genetic subtype of the same disease. Many therapies can be easily altered to target a different genetic mutation without altering the overall chemistry of the drug.
This will lead to treatments being approved for families that otherwise would have no hope.
Gina Szajnuk
Park City