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Senate bill focuses on development of genetically targeted drugs
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The U.S. Senate is considering a bipartisan bill intended to speed up the development of genetically targeted drugs for patients with rare life-threatening or debilitating conditions.
The Advancing Targeted Therapies for Rare Diseases Act of 2015 (S. 2030) would “allow the sponsor of an application for the approval of a targeted drug to rely upon data and information with respect to such sponsor’s previously approved targeted drugs.”
The Senate Committee on Health, Education, Labor and Pensions (HELP) recommended the bill on Feb. 9. The bill is sponsored by Sens. Michael Bennet, D-Colo., Richard Burr, R-N.C., Elizabeth Warren, D-Mass., and Orrin Hatch, R-Utah. It was referred to the HELP committee in September 2015.
“The Academy would definitely be in favor of anything that helps bring effective treatments for rare blinding diseases to market. That, to me, is important and straightforward,” Michael X. Repka, MD, MBA, American Academy of Ophthalmology medical director for governmental affairs, told Ocular Surgery News.
Michael X. Repka
Repka said the bill would likely have a positive impact on ophthalmic research, particularly for medical retinal diseases.
“This could promote research that could impact many of the retinal dystrophies, including Stargardt’s disease and retinitis pigmentosa,” he said. “It seems to me these rare diseases with a genetic basis fit the intent of the law. I think the idea to allow the sponsor to use prior research data to expedite additional novel treatments makes great sense.”
Repka said that using information from previous research could significantly enable research for new treatments.
“It seems to me that when a condition is rare, the chance to study a new treatment ... using what you’ve learned from similarly rare disease research could make the research actually feasible where it might not be feasible before,” Repka said.
S. 2030 is designed to facilitate targeted drugs to address unmet medical needs in one or more patient subgroups or genetic variant subpopulations, and to maximize the use of scientific tools or methods such as surrogate endpoints and other biomarkers.
“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,” Bennet said during a HELP Committee executive session.
Sen. Patty Murray, D-Wash., the ranking Democrat on the committee, said 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.”
S. 2030 and six other bills contain 50 different legislative proposals that concern regulation of medical devices, next-generation medical research, improving medical rehabilitation research, advancing research for neurological diseases, preventing superbugs and improving health information technology. – by Matt Hasson and John Schoen
Disclosure: Repka reports no relevant financial disclosures.