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  • Washington Highlights

    Senate Panel Explores Incentives, Obstacles for Developing Cures

    Tannaz Rasouli, Sr. Director, Public Policy & Strategic Outreach

    The Senate Commerce, Science, and Transportation Subcommittee on Space, Science, and Competitiveness July 14 held a hearing titled, “Unlocking the Cures for America’s Most Deadly Diseases.”

    Subcommittee Chair Ted Cruz (R-Texas) opened the hearing by noting, “The path to achieving medical breakthroughs and cures is long and capital intensive. Yet often it seems when it comes to medical research that we are penny-wise and pound-foolish, that we pay billions or trillions on the back end, dealing with the consequences of horrific diseases, rather than investing and creating the incentives on the front end to cure these diseases once and for all.”

    Chairman Cruz added, “In addition, the regulatory burdens and bureaucratic unpredictability slow the ability of innovators to create new cures. Regulatory burdens and uncertainty are also having an effect on private investment,” citing a 2011 report from the National Venture Capital Association that found U.S. venture capitalists are shifting their focus overseas to Europe and Asia.

    Ranking Member Gary Peters (D-Mich.) noted “we must develop our science and technology workforce through education, inspiration, and opportunity…We need to make sure that our science and technology workforce is second to none…. [F]ederal investment in science and technology must at least keep pace with the growth of our economy.”

    “We need to keep our federal research portfolio balanced,” he said, adding, “Only a broad and balanced science and technology investment portfolio will preserve America's place at the forefront of innovation for generations to come.

    “And finally, we need to find creative policy avenues to incentivize breakthroughs and reduce barriers to innovation,” Peters said, citing the example of the University of Michigan Medical School’s Fast Forward Medical Innovation program.

    Witnesses included former U.S. Senator Tom Coburn, M.D., (R-Okla.), who told the committee, “I think the NIH is woefully underfunded…[B]ut I think we need to have better oversight because they make some errors in funding that are just plain stupid and cause people not to want to support them.”

    He noted the House-passed 21st Cures legislation “is a great move forward but it’s got to be better. It’s got to have some teeth in it to force the FDA to become transparent.” He also said reforms of reimbursement and intellectual property policies are just as critical as FDA reform.

    Sen. Coburn testified, “We need a drug development system that encourages innovators to pursue breakthrough cures and allows patients and physicians fighting serious illnesses to take informed risks…and allows everyone to learn from the real world evidence about drug safety and efficacy.”

    “That is not the system we have today but it is the one we need to face the health and physical challenges for our future,” he said, adding, “The double-blind placebo-controlled study in the future will have very limited value to us if we want to propel our country ahead in terms of leading on new innovation and new cures."

    Keith Yamamoto, Ph.D., vice chancellor for research, University of California, San Francisco (UCSF), told the committee, “Precision medicine is a new concept that will collect and analyze vast amounts of basic research and patient data using computational tools to build a network of knowledge that allows effective diagnosis and treatment decisions or provides for disease prevention advice tailored to individual patients.”

    He explained that “precision medicine is like Google maps where the full picture is derived from stacking together many layers of different types of information but instead of topology and roads and gas station, it's DNA sequence and glucose blood levels and exposure to second hand smoke while growing up.” Together, these layers of data produce “a knowledge network and increasingly precise picture of human health and disease.”

    He told the committee that at UCSF, “many precision medicine project pilots are underway, most of them in collaboration with industries, startups, national labs, or other universities...and our emerging knowledge network already has begun to impact our research, our health support, and our treatments of disease.”

    He identified four necessary factors for precision medicine to succeed:

    • Expanded, sustained federal support for fundamental discovery, i.e., basic research, to define biological processes and to develop powerful new research tools, such as the CRSPR/Cas9 technology;
    • Transdisciplinary research, because precision medicine requires the concepts and tools of physics, chemistry, engineering and computer science through integrative programs across federal agencies and partnering with industry;
    • Big data, interoperable data sharing, computational learning, and data security; and
    • Science-based regulatory technologies and methodologies to accelerate clinical trials, enhance post-market vigilance, and create standards for next generation sequencing, novel trial design, cell-based therapies, and risk- benefit assessments of devices and therapies.

    Christopher Frangione, vice president, Prize Development, XPRIZE, testified that prizes are powerful for many reasons, including leveraging investment, democratizing innovation, and reducing risk. He said “the public and private sectors must work together to utilize every tool available and prizes are one of those tools.”

    Frangione cautioned that prizes do not work where early stage research and discovery is needed, and emphasized that “even where we believe prizes work well, they can always compliment traditional forms of funding and should never replace them.”

    Peter Huber, senior fellow, Manhattan Institute, also testified.