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NIST’S ROLE IN SUPPORTING THE BIOTECHNOLOGY INDUSTRY Laurie E. Locascio, Ph.D.

Director, Material Measurement Laboratory

2014 Biomanufacturing Technology Summit

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NATIONAL INSTITUTE OF STANDARDS & TECHNOLOGY (NIST)

Non-regulatory agencyestablished in 1901 in theUS Department of Commerce.

Mission: to promote US innovation and industrial competitiveness by advancing measurement science, standards & technology

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“Industry’s National Laboratory”partnering/serving industry to help maintain US leadership in science and technology products

NIST- WHO WE ARE

The National Metrology Institutetop of the US standards pyramid working toward global measurement harmonization and traceability to the SI

Department of Commercedeveloping standards to support international trade and commerce

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• ~ 3,000 employees• ~ 2,800 associates and facilities

users• ~ 1,300 field staff in partner

organizations • Two main campuses- Gaithersburg,

MD and Boulder, CO• FY13 Budget: 763M Congressional

Appropriations

NIST: BASIC STATISTICS

Gaithersburg, MD62 buildings; 578 acres

Boulder, CO26 buildings; 208 acres

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STRATEGIC PARTNERSHIPS

8 active NIST strategic partnerships across the U.S.• JILA – Atomic and molecular physics,

Boulder, CO• JQI – Quantum science, College

Park, MD• NCCoE – Cybersecurity, Rockville,

MD• CHiMaD – Advanced Materials,

Chicago, IL• And 4 related to biological

measurement science

Inst. for Bioscience and Biotech. Research (IBBR)University of Maryland

Hollings Marine Laboratory (HML)

Charleston, SC

Brookhaven National Labs (BNL)

Upton, NY

Advances in Biological/ Medical Measurement

Science (ABMS)Stanford Univ, CA

NIST grows its capabilities by building and leveraging through strategic partnerships

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FACILITATING COMMERCE THROUGH MEASUREMENT SCIENCE & TECHNOLOGY

• Deep research expertise underpins technological innovation – e.g., new materials, advanced clinical diagnostics and therapies, advanced communications, etc.

• Non-regulatory status enables important role as a convener that facilitates collaboration between industry and government

• 4 Nobel Prizes since 1997• MacArthur Fellowship winner in 2003• Kyoto Prize winner in 2011• National Medal of Science winners in 1998 and 2008• ~ 60 National Academy Members (10 current)• ~120 National Society Fellows

A world-leading measurement science program

Bill Phillips1997 Nobel Prize

in Physics

Eric Cornell2001 Nobel Prize

in Physics

John Hall2005 Nobel Prize

in Physics

Dan Shechtman2011 Nobel Prize

in Chemistry

John Cahn2011 Kyoto

Prize

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National reference laboratory for measurements in the

chemical, biological, and material sciences MML serves a broad range of industry sectors ranging from transportation to biotechnology and provides research, measurement services, and measurement quality assurance tools for addressing problems of national importance such as:

• assessment of climate change• renewable energy• the nation’s aging infrastructure• environmental quality• food safety and nutrition

MML is also responsible for coordinating the NIST-wide Standard Reference Materials and Standard Reference Data programs

• forensics and homeland security• health care measurements• manufacturing (ranging from

advanced materials to photovoltaicsto biologic drugs)

MATERIAL MEASUREMENT LABORATORY

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MML ROLE IN THE BIOSCIENCESDevelop, provide, and advance the necessary measurement infrastructure -o methods, o reference materials, reference data, o technologies, and models

To enable accurate, comparable, and reliable measurements of o biological molecules (nucleic acids, proteins,

metabolites)o systems (viruses, bacteria, cells, tissues)o materials (therapeutics, drug delivery platforms,

scaffolds)o devices (pacemakers, stents)

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IBBR AND NIST

• History: Longstanding partnership of >25y and NIST’s first coordinated effort in the biosciences

• Mission: To conduct groundbreaking biomolecular and measurement science research to generate innovative technologies and solutions

• NIST supports this mission as a key agency in the President’s innovation agenda and as the Nation’s metrology laboratory

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NIST-FDA COOPERATION “FOR SUPPORTING INNOVATION IN THE REGULATORY ENVIRONMENT”

Credit: Image ©Nantel/courtesy Shutterstock

• NIST and FDA identified 3 areas for collaboration to benefit both agencies supporting innovation in the regulatory environment

• NIST role in its collaborations with FDA is development and • dissemination of measurement methods and standards to support and

facilitate regulatory reform• NIST is often looked to as a neutral convener and plays a critical role in

bringing together industry, other agencies, and other stakeholders

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Phase IIIEfficacysafety

ApprovalPre-clinicaltesting

Phase ISafety

MarketingPhase IIEfficacysafety

ApprovalMeasurements

Reliable analytical measurement underpins development and regulatory approval of originator and biosimilar drugs

FDA cites analytical characterization as being particularly important in demonstrating biosimilarity

From the Advanced Manufacturing Partnership 2.0 report, May 2014: “Unlike many other industries for which the fundamental sciences is highly advanced, biomanufacturing of biologics exploits what remains only a partial understanding of protein science, cell biology, bioprocess control, and biomedicine.” A technical gap: high fidelity analytical techniques

BIOTECHNOLOGY PRODUCTS: THE IMPORTANCE OF ANALYTICAL CHARACTERIZATION

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SUPPORTING BIOPHARMA INDUSTRY

FDA

NISTIndustry

Regulation, Guidance,Research

Fundamental Measurement Science, Standards, Data

Current, Future Products & Analytical

Characterization

• The complexity of biopharmaceuticals is barrier to reliable analytical characterization and therefore a barrier to acceptance

• Powerful, yet complex, analytical technologies for assessing product quality are emerging to fill gaps

• A scientifically sound metrology infrastructure will enable high quality data interpretation, provide measurement assurance, and inform regulatory decisions

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NIST PROGRAM IN BIOMANUFACTURINGMeasurement science, tools, standards to support development, manufacturing, regulatory approval of biologic drugs

Congressional Subcommittee Hearing -Need for Measurement Standards to Facilitate R&D of Biologic Drugs, 2009Dr. Anthony Mire-Sluis (Amgen)Dr. Patrick VJJ Vink (Mylan)Dr. Steven Kozlowski (FDA) Dr. Willie May (NIST)

NIST program developed with Input from critical stakeholders:

“With the development of new analytical methods comes the need for new standards to evaluate them.”Steve Kozlowski, FDA

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FDA and biotech industry require better measurements to improve biologic drug quality and safety and to lower costs• No measurement basis to assess

“sameness” of biologic drugs – impedes generic development

• Lack of measurements and control of biomanufacturing processes leads to batch variability – increased production costs

Aggregation of biologic drugs is linked to immunogenicity, a major

safety issue in patients

NIST targeting:1. Improved measurement/standards for protein stability,

aggregation, particles2.Structural and biochemical measurements to determine

“sameness” of biologic drugs3.Measurements to enable improved understanding and

control of production cells

NIST PROGRAM IN BIOMANUFACTURING

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9:30 am Steven Kozlowski, Ph.D.Director, Office of Biotechnology ProductsFood and Drug Administration

10:15 am Break

10:30 am Michael J. Tarlov, Ph.D.Division Chief, Biomolecular MeasurementThe National Institute of Standards and Technology

11:15 am William E. Bentley, Ph.D.Robert E. Fischell Distinguished Professor and Chair, Department of BioengineeringUniversity of Maryland, College Park

12:00 pm Panel discussion with morning session presentersLed by John Marino, Ph.D.

Morning Session - Bioanalytical Characterization of Biosimilars