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Senate committee recommends bill to facilitate genetically targeted drug development
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The U.S. Senate Committee on Health, Education, Labor and Pensions recommended legislation today designed to speed up development of genetically targeted drugs for patients with rare and serious or life-threating conditions.
The “Advancing Targeted Therapies for Rare Diseases Act of 2015” (S. 2030), sponsored by Sens. Michael Bennet, D-Colo., Richard Burr, R-N.C., Elizabeth Warren, D-Mass., and Orrin Hatch, R-Utah, will allow drug companies seeking approval of a candidate therapy to leverage data from previously approved drug applications and to maximize the use of certain scientific methods, such as surrogate endpoints, to facilitate drug development.
Michael Bennet
“Currently, patients with rare diseases, many of them children, have a specific gene mutation at the root of their condition,” Bennet said during an executive session. “Companies usually target one specific mutation of disease when they are creating their drug. One mutation may only capture 20% of the children with the disease, however. Without changing the overall chemistry of the drugs, some of these therapies can be easily altered to target the other 80% of patients with various mutations of the same disease. This bill would not change FDA approval standards, but rather affirm that the FDA needs to use all the tools necessary to fight for these patients’ lives.”
Patty Murray
Sen. Patty Murray, D-Wash., the ranking Democrat on the committee, added that any bipartisan effort to approve biomedical innovation legislation must include mandatory funding of the nation’s top health agencies.
“We all share the goal of getting safe, affordable, effective new treatments to patients across the country who are waiting for them,” Murray said. “I believe we cannot realize that goal without sustained investments in innovative medical research and efforts to ensure patient and consumer safety. So I’ve made clear the path to a bipartisan deal on medical innovation must include mandatory investments in the NIH and the FDA and must uphold the gold standard of FDA approval.”