Biospecimen-Based Research and the 21st Century Cures Act

Posted by Abdul Ally on Feb 1, 2017 10:25:00 AM

21st_Century_Cures_Act_Image1.png“God Willing, This Bill Will Save Lives.” So said Vice President Joe Biden, in sharing his thoughts on the potential ramifications of the passing of the 21st Century Cures Act (H.R.6), made possible by the bipartisan leadership of Republicans and Democrats in the Senate and the House on December 13th 2016.

According to Vice President Biden; “This Bill will help us combat the heroin and prescription opioid epidemic ripping apart families and communities. It invests in programs to improve mental health treatment and suicide prevention. It will provide resources for President Obama’s BRAIN and Precision Medicine initiatives, so that our world class researchers can figure out how to better prevent, treat and eventually cure Alzheimer’s, epilepsy and traumatic brain injuries. And this is the part that’s personal for me and for millions of Americans: the 21st Century Cures Act invests $1.8 billion to help us end cancer as we know it. For over a year, I’ve been leading our Cancer Moonshot 2020 to fundamentally change the culture of our fight against cancer and inject a sense of urgency into it. This Bill goes a long way to help us – investing in promising new therapies, enhancing prevention and detection efforts in every community regardless of zip code, and bringing us closer to the day when there are vaccines for all kinds of cancer, just as we have them for measles or mumps.”

Some Key Features of this Historic Bill
A vital benefit to the passage of the 21st Century Cures Act is the injection of funding into the NIH and clinical research.  Under H.R. 6, the NIH will receive at least an additional $1.86 billion per year for the next five years ($9.3 billion total) and the FDA will receive an additional $550 million. H.R. 6 also reauthorizes the NIH for the first time since 2007.  In addition to annual budget increases of $1.86 billion per year, the Bill promises an additional $2 billion per year up to 5 years to create an NIH innovation fund (with oversight from a nine-member advisory board appointed by the Director of NIH) and also makes additional monetary investments which continue to fund The BRAIN and Precision Medicine Initiatives over the next 10 years to tackle neurological disorders including Alzheimer’s, touching nearly everyone involved in the new patient-focused drug development pipeline (initial discovery through biospecimen-based research and into clinical trials). Also included are reforms to the regulation of medical devices, specifically targeting those perceived to be low-risk (including software) to be deregulated –typical examples include electronic health record software and exercise trackers.

Key parts of the Act have received a great deal of attention and discussion, especially those sections that addressed funding and altered FDA approval criteria. However, those conducting basic research will be primarily affected by the parts of this legislation that did not generate headlines.  For instance, Section 2039 allows the Secretary of HHS to direct government contractors to collect reimbursement for the use of biological samples and other research products on behalf of the federal agency providing the resources.

The Sections listed below highlight some of the items included in the Act that could have an impact on research involving biospecimens, and particularly federally funded research.

Title I, Subtitle B, Section 1021, National Institutes of Health (NIH) RESEARCH STRATEGIC PLAN.  In this section, the Director of NIH is mandated to develop and maintain a five year strategic plan to leverage the best scientific opportunities through the identification of strategic focus areas (referred to as ‘Mission Priority Focus Areas’) and the establishment of objectives, identifying research opportunities, with a priority given to advancing research into pediatric and rare diseases and conditions (further specified in subtitle E, section 1081). Another significant priority includes a requirement that the Director of NIH maintain the biomedical workforce of the future by including the participation of scientists who are traditionally underrepresented in the scientific workforce. The initial strategic plan is due no later than 270 days after enactment of this subsection, and shall be made publicly available on the website of the NIH at such time.

Title I, subtitle C, Section 1042, Report.  In this Report section, the Director of NIH is mandated to report on efforts by the NIH to attract, retain, and develop emerging scientists, including underrepresented individuals in the sciences, such as women and other minorities.

Title II:  Discovery, Section 2014, Data Sharing.  This allows the Director of NIH to require grant recipients to share the data generated from NIH-funded research.  The data must be shared in a manner that is consistent with Federal laws and regulations for the protection of human research participants, proprietary data, and national security interests.

Title II:  Discovery, Section 2037, National Center for Advancing Translational Sciences (NCATS).  Allows the NCATS to support clinical trials through the end of phase IIB (previously IIA), and also allows NCATS to support clinical trial activities into phase III for treatment of a rare disease or condition, under specific circumstances.

Title II:  Discovery, Section 2039, Enhancing the Rigor and Reproducibility of Scientific Research.  This requires the Secretary of HHS, acting through the Director of NIH, to convene a working group to develop recommendations for a formal policy to enhance the rigor and reproducibility of NIH-funded scientific research, including pre-clinical study design and data sharing.

Title II:  Discovery, Section 2043, Reimbursement for Research Substances and Living Organisms.  Where research products are made available through contractors, allows the Secretary of HHS to direct such contractors to collect payments on behalf of the Secretary for the costs incurred to make available such research products. The amounts collected are to be credited to the appropriations accounts that incurred the cost to make the research product involved available.

Title III:  Advancing New Drug Therapies, Section 3011, Qualification of Drug Development Tools. Establishes a review pathway at FDA for biomarkers and other drug development tools that can be used to help shorten drug development time and reduce the failure rate in drug development.

Title III:  Advancing New Drug Therapies, Section 3036, Standards for Regenerative Medicine and Advanced Therapies.  Parts (a) and (b) require the FDA to consult with stakeholders and the National Institute of Standards and Technology to establish standards to support the development of advanced therapies. Part (c) defines “regenerative medicine and advanced therapies” to refer to the meaning given the term in Section 506(g) of the Federal Food, Drug, and Cosmetic Act (cell therapy, gene therapy, gene-modified cell therapy, therapeutic tissue engineering products, human cell and tissue products, and combination products using any such therapies or products).

While the entire legislation is 996 pages, available at, pages 265 through 454 focus on the FDA, Medicare and Medicaid, and pages 454 through the end (Division B) address reforms intended to help families in mental health crisis.

Interested in some additional reading? Check out our eBook Protecting Your Protocol: Operating a Flawless Clinical Agent Repository. It discusses the process of how clinical agents are received, filling orders for clinical pharmacies, and ultimately how to minimize risk, all while adhering to FDA guidelines.


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