Senate HELP Committee Examines “U.S. Leadership in Medical Innovation”

On March 10, the Senate Health, Education, Labor and Pensions (HELP) Committee held its first in a series of anticipated hearings on “U.S. Leadership in Medical Innovation.” Opening the hearing, HELP Committee Chairman Lamar Alexander (R-TN) announced that he and Ranking Member Patty Murray (D-WA) intend to focus on three major actions over the next two years: (1) “fixing” the No Child Left Behind Act; (2) simplifying and reauthorizing the federal government’s supervision of higher education in America; and (3) dealing with the “exciting new era of medicine.” Regarding the latter, Alexander noted that the House is moving on a parallel track via its 21st Century Cures initiative (see Update, February 24, 2015).

Alexander pointed out that he and Senator Richard Burr (R-NC) recently issued a white paper that is focused on medical innovation, which has been submitted to Senator Murray as well as to the members of the Committee for their consideration (see Update, February 24, 2015). The point of the hearing, Alexander explained, is to learn from National Institutes of Health (NIH) director Francis Collins and Food and Drug Administration (FDA) administrator Margaret Hamburg on what issues the Committee should be focusing.

Alexander also announced the formation of a HELP Committee working group comprised of committee staff for the purpose of identifying how the Committee will proceed. The Committee will  alsomonitor the House’s activities and work with Secretary of Health and Human Services Sylvia Burwell and the President, particularly on the precision medicine initiative.

Senator Murray echoed the Chairman and stated that she is pleased to be working with Alexander and the Committee on ways the Congress can continue to advance biomedical innovation for patients. She emphasized “the critical need to secure and build on the United States’ leadership in medical innovation.” To accomplish this, Murray said, Congress has to look at how to ramp-up investment in the kind of research and development that helps to drive this private sector growth. She expressed her concern regarding “the impact of sequestration and what it has done to NIH,” and indicated that her priority is the needs of women and young children in the research, development, and approval process.

Human Subjects and Revisions to the Common Rule

Responding to Alexander’s question regarding the amount of time investigators spend on administrative tasks as reported by the National Academies, Collins acknowledged that NIH is very concerned that investigators are spending 42 percent of their time dealing with administrative matters instead of directly engaged in research. To that end, the agency is undertaking a number of activities within its authority to try to limit the amount of administrative oversight, but not all of the issues that need addressing fall under the NIH’s authority. One example of action the agency has taken is the standardization of the biographical sketch that investigators have to provide when applying for grants, which has been found to substantially assist investigators.

Collins specifically noted NIH is currently examining human subjects regulations. The Common Rule, the regulation that oversees research involving human subjects, has not been revised in 20 years, Collins told the Committee. It “does not currently take account of the risk involved in a study, and applies a great deal of oversight to some studies that are truly low-risk, as if they were, in fact, invasive surgical procedures.” Alexander asked the director to work with the Committee to identify what those actions are specifically, so that if the Congress needs to make changes in the law it can.

Collins also highlighted that the approval process required for NIH staff to attend conferences has been a burden. “Scientists going to conferences is a critical part of how NIH moves things forward, how new ideas emerge, and it is very much being inhibited by this very heavy-handed oversight,” Collins maintained, adding that the agency could also benefit from the Chairman’s help there. A second area where NIH could use Congressional help, according to the director, is the ability to carry funds over into a second year similar to the authority granted to the National Science Foundation and the Department of Energy in their scientific budgeting. The NIH does not have this authority and consequently at the end of each fiscal year, the agency has money that it needs to spend or forfeit. This authority would provide more flexibility to the agency.

Hamburg responded that the FDA needs to invest more money in regulatory science that develops the know-how and the tools, approaches, and strategy that will enable the agency to assess in an effective and efficient way, the safety, efficacy, quality, and performance of a product. Regulatory science has been under-appreciated, under-developed, and under-invested in the FDA’s overall biomedical product enterprise. It has proven to be essential as the FDA is trying to take the last set of steps from research and development into a product, she stressed.

Murray asked the NIH director to explain how the sequester impacted research at NIH. Collins responded that it “was a serious blow to momentum.” The $1.56 billion removed from the agency’s budget in the middle of the fiscal year prevented the NIH from funding about 750 grants that otherwise would have been funded that year. Those very good ideas were left on the table, he added. He noted, however, that the agency was able to make up some that ground in the subsequent years, but even in 2015, it did not recover the entire amount lost in 2013. The consequence is that the U.S. is losing opportunities for science; it is losing jobs and is potentially at risk of losing young investigators, some who are beginning to give up, said Collins.

Predictable and Sustained Funding Needed                    

Expressing pride in having both the NIH and FDA in Maryland, retiring Senate Appropriations Committee Ranking Member Barbara Mikulski (D-MD) shared what she considers three criteria for reform: (1) respect for the mission of the agencies and the men and women who work there; (2) adequate resources so that the agencies can do the job and have the tools needed to do so; and (3) approach reform in a targeted way as outlined by Alexander, focusing on specific problems and specific solutions.

Responding to Mikulski’s inquiry about the need for “predictability and sustainability” for each of the agencies when it comes to funding, Hamburg stated that predictability and certainty is essential for the FDA to do its job. Collins answered that the idea of stability is crucial for biomedical researchers, especially those individuals just beginning their careers. Currently, investigators’ applications are successful only in one out of six attempts, which is discouraging to investigators, said Collins. He added that the President’s FY 2016 budget begins to provide that needed stability.

Senator Bill Cassidy (R-LA) compared the funding for HIV ($3 billion) to that of Alzheimer’s disease ($800 million), and questioned if NIH should “start shifting more aggressively resources from that which has been addressed to that which we are confronting.”

Senator Elizabeth Warren (D-MA) noted that since 2003, the NIH budget has not kept pace with inflation and its purchasing power is down nearly 25 percent. She asked Collins to share how the “collapse in congressional NIH funding has hurt the American pipeline of biomedical innovation.” The NIH director responded that the agency has had to “basically leave about half of the ideas on the table” that traditionally it would have funded, reflecting a slowing down of the innovation process from basic science to clinical trials. Following up, the Senator stated the first priority by the Committee would be to figure out how to get the NIH the resources it needs to replenish the pipeline of research.

Senator Tammy Baldwin (D-WI) expressed similar concerns regarding the agency’s budget and noted that she remained troubled that the budget cuts mandated by the Budget Control Act has put medical research at risk. She highlighted the Next Generation Research Act, a bill she introduced last year that would coordinate efforts within the NIH and streamline current programs to improve opportunities for new investigators. Baldwin asked Collins to discuss the progress made via NIH’s existing programs for investigators, such as the Early Stage Investigator program and the Director’s New Innovator Award to bolster the emerging research workforce. He responded that the agency has instituted a number of programs aimed at encouraging the next generation, including increasing the number of awards (K99/ROO) that are a bridge to independence from a postdoctoral fellowship to an academic position. In addition, Collins explained that the agency is ensuring that its graduate students and post-docs are exposed to multiple career paths because not all of them need to become research-track faculty in an intensive university.

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Posted in Issue 5 (March 24), Update, Volume 34 (2015)

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