21st Century Cures Act Discussion Draft Released

House Energy and Commerce Committee Chairman Fred Upton (R-MI) released a discussion draft of the 21st Century Cures Act on January 27. The draft bill is the culmination of a year of hearings and roundtable discussions held by the Committee. Its release was accompanied by a section-by-section discussion of the document and a one-pager highlighting the legislative ideas. The Committee has repeatedly stated that the draft is a “starting point in the legislative process to spur discussion.” Accordingly, they are seeking public feedback on the proposals. The Committee also cautioned that the “inclusion of a policy in the draft should not be seen as an endorsement.”

While the 393-page discussion draft was officially released as a part of a supposedly bipartisan measure, Democrats, including Subcommittee member Diana DeGette (D-CO) who worked with Upton on a series of hearings and roundtables in 2014, did not endorse the measure. In addition, Ranking Member Frank Pallone Jr. (D-NJ) released a statement citing his disappointment that the draft “does not reflect true bipartisan collaboration.” Pallone highlighted the fact that the draft did not include authorization of funding for “additional basic research” at the National Institutes of Health (NIH), adding, “increased funding was a common theme during last year’s public engagement, from both sides of the aisle, and is fundamental to truly advancing 21st Century Cures.”

The draft includes a number of provisions that, depending on the implementation, could have unintended consequences on social and behavioral science research specifically, and the operations of the NIH in general.

Longitudinal Study on Outcomes of Patients with a Chronic Disease

Included in the bill is a provision, Subtitle N—21st Century Chronic Disease Initiative Act, Sec. 2241. Plan for Longitudinal Study on Outcomes of Patients with a Chronic Disease (page 215),which would require the Secretary of Health and Human Services, in consultation with the NIH director to

“…develop and submit to the appropriate committees of the Congress a plan to carry out a longitudinal study designed to improve the outcomes of patients with 12 chronic diseases through better understanding of risk, transition from wellness to disease, disease progression, diagnosis, and other factors related to chronic disease, including by identifying potential targets for preventive or therapeutic intervention.”

Specifically, the draft bill would require NIH to:

(1)    Ensure that the longitudinal study’s design and execution can support the goal of improving the outcomes of patients with a chronic disease;

(2)   Address the roles of the following types of people in developing the plan and implementing the longitudinal study: scientific and medical researchers, patient representatives, experts in the design and implementation of longitudinal studies related to chronic disease, health care providers with expertise in chronic disease, ethicists, academic researchers, government researchers, representatives of clinical research organizations, and scientific or medical 6 staff from biopharmaceutical manufacturers and developers;

(3)   Identify existing and ongoing studies that are relevant to informing and developing the longitudinal study;

(4)  Include in the plan a description of how patient cohorts will be utilized, coordinated, and expanded in support of the longitudinal study to ensure sufficient enrollment; and

(5)   Include a description of how the efforts of researchers and investigators participating in the longitudinal study will interact and be coordinated with other chronic disease research efforts, including research under the National Alzheimer’s Project Act.

Funding Research by Emerging Scientists through the Common Fund

Another of the bill’s provision, Section 2261, Funding Research By Emerging Scientists Through Common Fund (page 216), would use Common Fund monies to support “emerging scientists,” defined as “an investigator who will be the principal investigator or the program director of the proposed research” and who has “never been awarded, or has been awarded only once, a substantial competing grant” by NIH for independent research. The investigator is further required to have completed his or her degree within the past 15 years. The provision also states that the funds “shall be used to supplement, not supplant, the funds otherwise allocated by NIH for young investigators.”

The NIH would be required, no later than six months after enactment, to submit a report to Congress explaining why, over the past 30 years, “there has been a substantial increase in the age of investigators receiving their first major research grant” from NIH; there has been a substantial increase in the average age of all NIH grantees and there has been a dramatic drop in the number of investigators under 40 who have been awarded an NIH grant. NIH would also be required to include in the report steps recently taken to address these trends.

The bill also references the use of “tap” Public Health Service Evaluation Funds and states that the tap “shall not be subject to section 241” an issue addressed in the FY 2015 CRonimbus. (see COSSA’s analysis).

High-Risk, High-Reward Science

Subtitle P—Fostering High-Risk, High-Reward Science, Sec. 2281. High-Risk, High-Reward Research Program, would require that each of NIH’s 27 institute and center directors, in collaboration with other scientists “establish programs to conduct or support research projects that pursue innovative approaches to major contemporary challenges in biomedical research that involve inherent high risk, but have the potential to lead to breakthroughs.” The institutes would be required to “set aside a specific percentage of funding” determined by the NIH director for each.

Clinical Research Modernization

In TITLE III—Modernizing Clinical Trials, Subtitle A—Clinical Research Modernization, Sec. 3001. Protection Of Human Subjects In Research; Applicability Of Rules, the draft includes provisions addressing human subjects, stating “that all human subject research shall be conducted in accordance with the HHS Human Subject Regulations, and as applicable to the human subjects involved in such research, with vulnerable-population rules.” This section also applies to human subject research that is conducted or supported by the Department of Health and Human Services or otherwise subject to HHS regulations under a provision of Federal law.

Twelve months after enactment of the 21st Century Cures Act, the statute would require the HHS Secretary acting “through the relevant agencies and offices of HHS, including the Office for Human Research Protection (OHRP) and relevant agencies and the offices of the Food and Drug Administration (FDA), to issue regulations and guidance and “take such other actions as may be necessary.” The guidance should address “delineating the roles of institutional review boards [IRBs] in multistate or cooperative, multisite studies where one or more local [IRBs] are relied upon, or similar arrangements are used,” the risks and benefits to human subjects, the standardization of informed consent and other processes and legal documents, and incorporating community values through the use of local [IRBs] while continuing to use central or lead [IRBs].” The HHS Secretary is directed to avoid duplication. The measure would require the Secretary to consult with stakeholders, including researchers.

Broader Application of Bayesian Statistics and Adaptive Trial Designs

Subtitle B—Broader Application of Bayesian Statistics and Adaptive Trial Designs, Sec. 3021. Clinical Trial Modernization (page 232) would direct the HHS Secretary to implement a framework that addresses incorporating the use of Bayesian methods or “other alternative statistical methods into proposed clinical protocols and marketing applications for drugs, biological products, or devices.”

Global Pediatric Clinical Trial

Subtitle D — Pediatric Research Network Improvement, Sec. 3041. National Pediatric Research Network (page 237) expresses “the sense of Congress” that the NIH should support a global pediatric clinical trial network through the allocation of grants to supplement the salaries of young researchers who participate; NIH grants should be awarded, solely for the purposes of supplementing the salaries of young researchers, to entities that participate in the global pediatric clinical trial network; FDA should engage the European Medicines Agency and other foreign regulatory entities during the formation of the network to encourage their participation; and once the network is established, the draft directs the FDA to continue to work with the European Medicines Agency and other foreign regulatory entities.

NIH Research Strategic Investment Plan

Title IV—Accelerating The Discovery, Development, And Delivery Cycle And Continuing 21st Century Innovation at NIH, FDA, CDC, AND CMS, Subtitle A—National Institutes of Health, Sec. 4001. NIH Research Strategic Investment Plan.
Beginning in FY 2016, the bill would direct the NIH director, in consultation with the institute and center directors, researchers, patient advocacy groups, and industry leaders, to “develop and maintain a 5-year biomedical research strategic investment plan.” According to the draft, the strategic investment plan is designed “to increase the efficient and effective focus of biomedical research in a manner that leverages the best scientific opportunities through a deliberative planning process.” It would identify areas, “to be known as strategic focus areas,” in which NIH resources can “best contribute to the goal of expanding knowledge on human health” in the U.S. through biomedical research. It would also require measureable objectives for each strategic area.

The IC directors would be further directed to use the strategic investment plan to “make resource allocation decisions and to develop individual strategic investment plans for the research activities” of each of the institutes that have a common format. The plans would also be required to “identify strategic focus areas” in which the resources of each institute and center “can best contribute to the goal of expanding the knowledge on human health.”

The bill further specifies the content of the plans including identifying funding priority for NIH overall: “In developing and maintaining a strategic investment plan, the NIH director would be required to ensure that at least 55 percent of the funds that are used by [NIH] to support extramural research for any fiscal year are used to support basic biomedical extramural research.”

The strategic focus areas are required to “include up to 10 topics, to be known as Mission Priority Focus Areas, which best serve the goals of preventing or eliminating the burden of a disease or condition and scientifically merit an enhanced and focused research engagement campaign over the next 5 years.” The measure directs the NIH director to “ensure that rare and pediatric diseases and conditions remain a priority.”

The agency would be given a maximum of 270 days after enactment of the legislation to complete the initial strategic investment plans and make them accessible on the NIH’s website. In addition, the NIH director, in consultation with the directors of the institutes and centers, would be directed to “conduct metrics reviews for each strategic focus areas.” The plan would be reviewed every five years.

Biomedical Research Working Group to Reduce Administrative Burden on Researchers

The measure would establish a “Biomedical Research Working Group,” with the NIH director serving as chair. The working group’s duties would include:

(1)    reviewing “literature and reports” on administrative burdens of researchers funded” by NIH, improving replicability of NIH-funded research;

(2)   providing recommendations to the NIH director to reduce administrative burdens with respect to the extent to which (and how) the grant proposal submission and progress report requirements of the NIH should be restructured, streamlined, and simplified;

(3)   evaluating and providing recommendations on the extent to which it is required by Congress to provide any statutory authority to implement any recommendations proposed; and

(4)  preparing a plan, including timeframes, for implementing recommendations proposed for which congressional action is not required.

The proposed legislation stipulates that the membership of the working group include federal members (NIH director, NIH Division of Program Coordination, Planning, and Strategic Initiatives director, NIH Extramural Programs director, NIH Intramural Program director) and seven non-federal members representing physicians, health practitioners, academies, scientists, and entrepreneurs whose work, research specialization, or professional expertise includes a significant focus on basic and clinical research funded by NIH. It further requires the HHS Secretary in consultation with the NIH director to appoint three of the members, the Speaker and Minority Leader of the House of Representatives each to appoint one individual, and the Majority and Minority Leaders of the Senate to each appoint one individual.

The Working Group would be required to submit a report to Congress no later than one year after the date of the enactment, followed by periodic reports by the NIH director on implementation of measures to reduce administrative burdens no later than six months after the submission of the Working Group’s report. The NIH director would be required to submit a report to Congress on the extent to which the director has implemented the measures.

NIH Travel

The discussion draft contains a placeholder for a section to address NIH travel policies.

Increasing NIH Accountability

The 21st Century Cures Act would address the appointment of the 27 institute and center directors. It states that the director of the National Cancer Institute (NCI) would remain a presidential appointment. The NIH director will continue to appoint the other directors to whom they will directly report. The measure stipulates that the IC directors would be appointed to a term of four years and gives the NIH director the authority to remove directors prior to the expiration of their terms. The directors, however, may be reappointed, and there is no limit on the number of terms a director may serve. Current directors’ terms would commence with the enactment of the Act.

Review of Certain Awards by Directors

One of the most glaring changes to the current grant process is that the measure states: “Before an award is made by a national research institute or national center for a grant for a research program or project…other than an award constituting a renewal of such a grant, the director of such national institute or center shall personally review and approve the award.” The directors are to take into consideration whether the “goals of the research program or project are a national priority and have public support; whether other agencies are funding programs or projects to accomplish the same goals; and whether the monetary investment is worth the potential scientific discovery.”

A GAO study would be required not later 270 days after the date of enactment of this Act on the extent to which biomedical research conducted or supported by Federal agencies is duplicative, and submit a report to the Congress on the results of such study, including recommendations on how to prevent such duplication. The GAO is to complete a study on the “extent to which there is waste, fraud, and lack of consistency” with the NIH mission in the conduct and support of research. The GAO would also be required to provide a report that includes an analysis of how amounts reserved under such section have been used and the impact of that funding on the each of the areas that received funding.

Funding for the Common Fund

The measure includes placeholders authorizing additional funding for the Common Fund and the BRAIN Initiative.

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Posted in Issue 3 (February 24), Update, Volume 34 (2015)

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