Importance of Regulatory Science
Driving Biomedical Innovation by
Advancing Regulatory Science at
FDA
Frank F. Weichold, M.D., Ph.D.
Director
Critical Path and Regulatory Science Initiatives
Office of the Chief Scientist/OC
Food and Drug Administration
Frank.Weichold@fda.hhs.gov
1
M-CERSI Educational Programs Conference; 04-15-13
Industrial R & D Landscape
– Past 2010, biomedical research is in excess of $186 billion in
R&D*
– The average cost of bringing a drug to market rose to more than
$1.4 billion past 2011
– Medical Innovation sector employs nearly 1 million people **
– Biopharma industry indirectly contributed more than $300 billion
to US GDP^
– Patent cliff (89.5B revenue lost) – US accounts for more than
40% of the word’s patents in biotech
– Decrease productivity of R&D pipelines(#) and increase in
clinical trial cost
*http://www.researchamerica.org/uploads/healthdollar10.pdf
**NIH study: An Economic Engine
^PhRMA 2011
#Mckinsey quarterly
2
3
Innovation is Linked to Ecosystem and Partnerships
FDA-regulated products account for 25 cents
of every consumer dollar spent in the U.S.
NIH
Public & Payors
Academic
Physicians &
Patients
Small
Business
Biotech/Pharma
4
Why do we need Regulatory Science?
• Major investments and advances in basic sciences are
not fully translating into products to benefit patients
• Product development is increasingly costly, success
rates remain low, many uncertainties exist
• Development/evaluation tools and approaches have
neither kept pace with nor incorporated emerging
technologies
• Economic health of innovative biotech and medical
product industry at risk
5
What is Regulatory Science?
• The science of developing new tools, standards, and approaches to
assess the safety, efficacy, quality, and performance of FDAregulated products
• The critical bridge between the “too basic” scientific research
discoveries and “too applied” science of manufacturing and
marketing of regulated products
• Includes areas of preclinical and clinical research, manufacturing,
processing, and translational science in a product development and
approval process (“the cycle”)
• May improve regulatory affairs processes
Discovery
Clinical Trial
Product on Market
Patients
Redefine patient as co-researcher
Reverse engineer to drive smarter basic research and
product development
Redefine patient as co-inventor
1. Drive science
2. Drive smart product development
1. Digital engagement- app, web, social media
2. Data driven
3. Patient information- digital media
4. Continuity of care, compliance
Better performance and outcome
Stratify patients by:
• Genetics
• PRO’s
• Behavior
• Disease definition
Steps FDA is Taking to Drive Biomedical
Innovation:
• Rebuilding FDA’s small business outreach services.
(SBA)
• Building the infrastructure to drive and support
personalized and precision medicine.
• Creating an Expedited Drug Development Pathway
• Harnessing the potential of data mining and information
sharing.
• Reducing the time and cost of medical device
development, assessment and review.
• Training the next generation of innovators
• Streamlining and Reforming FDA regulations.
8
Regulatory Science Publications
Innovation is a Core FDA Mission
Advancing Regulatory Science Website
http://www.fda.gov/ScienceResearch/SpecialTopics/Reg
ulatoryScience/default.htm
THANK YOU
10
FDA Human Capital Needs
Leslie D. Wheelock, RN, MS
Director, Office of Scientific Professional Development
Office of the Chief Scientist
Office of the Commissioner
FDA
1
Outline
•
•
•
•
Federal Government and Human Capital
FDA Workforce
Human Capital Challenges
Academic Programs in Regulatory
Science
2
Federal Government and
Human Capital
• Federal workforce is central to the delivery
of services to the American public
• Human capital management is the
government’s approach to select, develop,
train and manage a high quality,
productive workforce (CHCO Act 2002)
3
FDA Workforce
• About 14,500 staff
• Average age: 46.5 years
• Length of Service
– 45%: less than 10 years of service
– 28.8%: more than 20 years of service
– 10.1%: more than 30 years of service
4
FDA Workforce
• Commissioned Corps Officers
• Civil Service
– Competitive
– Excepted Service
– Senior Executive Service
5
6
FDA Scientists (9426)
• Medical & Public Health (5271): Consumer Safety Officers,
General Health Scientist, Medical Officer, Pharmacist,
Epidemiologist, Nurse, Medical Technologist, Dentist
• Biological Sciences (1911): Microbiologists, Biologists,
Pharmacologists, Toxicologists
• Physical Sciences (1214): Chemist
• Mathematics & Statistics (424): Mathematical Statistician,
Statistician
• Engineering (405): General Engineer, Electrical Engineer,
Biomedical Engineer
• Veterinary Medicine (124): Veterinarian
• Social Sciences & Psychology (77): Social Science Analyst,
Research Psychologist, Consumer Studies Specialist
7
FDA’s Human Capital
Challenges
• Need to recruit a well–qualified
workforce force
– Related to regulatory science
• Training and development of
employees
– Related to regulatory science
– Related to rapidly changing science
8
Need to Recruit a
Well–Qualified Workforce
• Retirement : Average of 239 employees a year
are eligible
– FY 2013 – 212 scientists, 26 scientific leaders
– FY 2014 – 122 scientists, 43 scientific leaders
• Congressionally mandated hiring surges
• FDA response to Human Capital survey – 52%
indicated “My work unit is able to recruit people
with the right skills.”
9
Staff Training and
Development
• Time to train new employee: 3 years
– New scientists need the knowledge and skills
to make regulatory decisions
• Intersection of science and policy
• Scientists need to keep current
10
FDA’s Recruitment
and Training
• Hire scientists
• FDA provides regulatory science
training
– Classroom
– On-the-job mentoring
11
Academic Programs in
Regulatory Science
• Potential benefits for FDA, Industry and
Academia
– Scientists who are prepared to work in
areas related to regulatory science
– Scientists who need less time to be
oriented
– Scientists who are current in both their
scientific area and regulatory science
(tools, standards and approaches)
12
Academic Programs in
Regulatory Science
• Considerations for learning regulatory science related to
medical products:
• Product development
– Hands on experience - Industry and Academia
• Product approval
– Challenge to obtain outside FDA
– Because of limited FDA resources and time, consider
educational strategies such as case studies
– Hands on experience - Industry and Academia
» Internships that include interactions with FDA
» Industry and academia perspective of product approval
13
Thank you.
14
Partnership: Key for Success in Food Safety Education and Training
JIFSAN, University of Maryland
Univ. Of Maryland & US FDA
Joint Institute for Food safety and Applied Nutrition
1
Joint Institute for Food Safety and Applied Nutrition (JIFSAN)
When?
What?
 A multidisciplinary research, education and outreach
program – domestic and international in scope
 Established in 1996.
How?
 A collaborative effort between the University of
Maryland, the U.S. Food and Drug Administration
(CFSAN and CVM), and the private sector
Concepts of Operation
 Build programs through partnerships
 Leverage and share resources
 Create a neutral environment conducive to exchange of ideas and conducting
research
 Develop international collaborations
Univ. Of Maryland & US FDA
Joint Institute for Food safety and Applied Nutrition
2
JIFSAN Programs
Good Agricultural Practices
Good Aquacultural Practices
Commercially Sterile Packaged Foods
Risk Analysis
Food Inspector Training
Tra i n i n g
International Food Safety Training Laboratory
FoodRisk.org
Conferences
Outreach
University of Maryland
Research
Research Grants
Center for Risk Communications Research
US Food & Drug Administration
Education
Internship Postdoc
Univ. Of Maryland & US FDA
Joint Institute for Food safety and Applied Nutrition
3
JIFSAN Training Portfolio
International training center




Good Agricultural Practices (GAP)
Good Aquacultural Practices (GAqP)
Commercially Sterile Packaged Foods (CSPF)
Food Inspector training (FIT)
Food Safety Risk Analysis Training Program
International Food Safety Training Laboratory
‘
Univ. Of Maryland & US FDA
Joint Institute for Food safety and Applied Nutrition
4
JIFSAN – The programs
GAPs: 34 trainings in 17 countries
GAqPs: 10 trainings in 7 countries
9
Food
Inspector
Training
8
7
Commercial
Food
Processing
Technology
Good
Aquacultural
Practices
6
5
4
3
2
Good
Agricultural
Practices
1
0
2000
Trinidad
2001
Brazil
2002
2003
2004
2005
Dominican Mexico (2), Guatemala, Thailand, Republic, Peru
Honduras, Brazil, Mexico, Korea Mexico
Puerto Rico 2006
Mexico, El Salvador, China. Vietnam
2007
2008
2009
2010
2011
2012
Honduras Guatemala, Costa Rica, Honduras, Peru, Costa Mexico. (Central Peru, Honduras. Dominican Rica, Mexico America), Republic, (2) . Nicaragua
India (spices Guatemala, Mexico, El and Salvador. botanicals). Indonesia, Bangladesh. Bangladesh, Bangladesh, India, Malaysia, China. Thailand
Vietnam. US*. Morocco
China
China.
China.
* 10 day internship in US for 9 Bangladeshi trainers (2010)
Univ. Of Maryland & US FDA
Joint Institute for Food safety and Applied Nutrition
5
The Global Collaborating Center Concept A success story – based on key principles
Planned Collaborative Centers
 Bangladesh (GAqP)
 Country/market sector ownership and
involvement required for sustainable
development
 Program aligns with country partners’
agenda and using country partners’ systems

Bangladesh Shrimp and Fish Foundation
 Mexico (GAP)

SENASICA
 India (GAP for Spices and Botanicals)
 Country partners set the agenda based on
needs of the market sector

CII Jubilant Bhartia Food and Agriculture
Centre of Excellence
 Country partner provides the resources to
develop and sustain program

Spice Board
 Thailand (CSPF for aquaculture products)
Univ. Of Maryland & US FDA
Joint Institute for Food safety and Applied Nutrition
6
Food safety risk analysis courses
Have trained 1300 individuals from over 32 different countries who took over 2000 course ,
Univ. Of Maryland & US FDA
Joint Institute for Food safety and Applied Nutrition
7
Food Safety Laboratory Capacity Training
Hands-on training on standard methods for
detecting chemical and microbial contaminants
in food in a state-of-art facility being built with
the support from
Waters Corporation
Summer 2011
Univ. Of Maryland & US FDA
Joint Institute for Food safety and Applied Nutrition
8
Food Safety Laboratory Capacity Training
Laboratory Training
 Objectives

Hands on – in Lab Training

Train- the-Trainer Events

Reproducible Training
Modules (web)
 Types of participants

Foreign and State government
laboratory workers

Third-party laboratories

Industry
Year 1:
April: Method for Mycotoxins in Food
May: Pesticide Residue Analysis (APEC, funded USDA)
June: Cronobacter in Infant Formula
September: Salmonella in Produce
October: E. coli (produce and meat)
Oct: Microscopic and Chemical Identification of Ingredients in Dietary Supplements
Nov: Pesticide Residue Analysis (+ ISO 17025)
Participants Origins (1 year)
Canada
Chile
China
Costa Rica
Dominican
Republic
Guatemala
Honduras
Indonesia
Korea
Malaysia
Mexico
Philippines
USA
Vietnam
Laboratory Training
Univ. Of Maryland & US FDA
Joint Institute for Food safety and Applied Nutrition
9
University of Maryland
MS in Regulatory Science
• Part‐time, exclusively online program designed for working professionals
• Non‐thesis, but students assemble a personal portfolio in regulatory science
• Regulatory science is the science of developing new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of FDA‐regulated products
• Primarily focuses on drugs
– chemistry, manufacturing, and controls (CMC)
– clinical research
– pharmacovigilance and Phase IV research (e.g., pharmacoepidemiology)
– drug discovery
• www.pharmacy.umaryland.edu/regulatoryscience
Program Objectives
• A graduate will be able to:
– Devise and implement global strategies for drug, biologic, and device development and evaluation
– Differentiate FDA and other region requirements for drug product development and registration
– Apply principles of basic and applied pharmaceutical sciences in drug discovery and development
– Formulate critical elements of chemistry, manufacturing, & controls (CMC) to drug development
– Relate principles of clinical research design to practices in clinical trial management
– Apply critical elements of risk and utilization to post‐marketing surveillance and pharmacoepidemiology, and evaluate economic and human factors that impact drug use
Courses
•
•
•
•
•
REGS603 Drug, Biologic, and Device Regulation
REGS614 Drug Discovery
REGS631 Drug Development REGS621 Clinical Research
REGS641 Regulated Products in the Marketplace
• Five 6‐credit courses (each composed of 6 modules)
–
–
–
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Pre‐recorded lectures
Homework
Web conferences
Assessments
• Projects
• Online presentations
• Mini‐reviews
UMCP Certificate in Regulatory Science and Engineering
Required Course:
• BIOE 689 Introduction to Regulatory Affairs: Medical Devices
Three from the following list:
• BIOE 6XX Cardiovascular Engineering
• ENRE 671 Engineering Decision Making and Risk Management
• ENRE 600 Reliability Engineering
• ENME 808 / ENRE 648 Risk and Reliability in Health Care (New Course)
• ENRE 602 Reliability Analysis
• BIOE 632 Biophotonic Instrumentation and Techniques
• BIOE 645 Advanced Engineering Startup Ventures
• BIOE 653 Advanced Biomaterials
UMCP Certificate in Regulatory Science and Engineering
• Program will kickoff in Fall 2013
• Program housed in our Office of Advanced Engineering Education
• Expected to grow into an MS degree program
• Considering an opline option
Regulatory Science at USC
Eunjoo Pacifici, PharmD, PhD
International Center for Regulatory Science
University of Southern California
epacific@usc.edu
REGULATORYSCIENCE
UNIVERSITY OF SOUTHERN CALIFORNIA
http://regulatory.usc.edu
Our program has expanded over the years
2013
2012
2001
•MS
Healthcare
•MS
Decision
2008
Management Analysis
•Doctorate in of Drug
2005
Development
Regulatory
•Certificate Science
Program
•MS
Regulatory
Science
To meet the complexities of an evolving discipline
Flexibility To Choose A Program
Master’s
Regulatory Science
Certificate
Food safety
Regulatory Science
Doctorate
Regulatory
Science
Management of Drug
Development
Healthcare Decision
Analysis
Early Drug Development
Clinical Design and
Management
Patient and Product
Safety
Based on individual student’s goals and aspiration
MS Regulatory Science
Curriculum
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•
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•
•
•
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Introduction
Drugs & Biologics
Devices
Foods
Quality
Law
Clinical Management
Writing
Internship
•
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Global Regulations
Risk Frameworks
Seminar
Clinical Design
Business
Food Science
CMC
Ethics
Composed of core courses and electives
MS Management of Drug
Development
•
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•
•
•
•
•
•
Translational Medicine
Drug Discovery
Preclinical
Early Clinical
Drug Design
Business
Clinical Design
Directed Research
•
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•
•
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•
•
Biostatistics
Food and Drug Toxicology
CMC
Drugs and Biologics
Ethics
Intro to Regulations
Clinical Management
Collaboration with faculty from School of Pharmacy
and other academic & industry experts
MS Healthcare Decision Analysis
•
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Healthcare Insurance
International
Competitive Intelligence
Healthcare Reform
Drug Information
Outcomes
Clinical Trials and
Statistics
• Project Management
•
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Pricing Strategies
Partnerships and Mergers
Drug Regulation
Device Regulation
Global Regulation
Ethics
Biostatistics
Rotations
Interdisciplinary instruction by faculty from School of Pharmacy,
Shaffer Center for Health Policy & Economics, and other academic & industry experts
From Bench to Bedside
Regulatory Science
IND to Approval
MDD
HDA
Translational Research
Outcomes and Access
Research
Discovery
Early Development
Phase I
Late Development
Phase II
Phase III
Approval
NDA/
BLA
Coverage
Access
Graduate Certificate Programs
• Risk Management and Safety
• Food Regulation and Safety
• Clinical Design and
Management
• Early Product Development
• Regulatory Science
Allow development of specific expertise
Courses can be rolled forward into MS program
Executive approaches
Weekend
8-hour days
Condensed
classes
Short-course
approach
Additional
Distance
Modules
Binders and
course materials
supplied
Virtual Office
Hours
Hybrid/Distance Education
All courses taught in parallel by web streaming
All courses archived in multimedia format
Biweekly interactive videoconferencing session
Students at USC
Mean age: 35
n=136 (83 F/53M)
Most are working full-time
Other students are usually employed
by industry at or before graduation
Many have advanced degrees already
Professional Doctoral Studies
• Approved April 2008
• Admission into a cohort program
• Students selected by strong work
performance and achievement in MS
studies
• Opportunity for advanced placement with
suitable graduate preparation
Advanced Doctoral Courses
•
•
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Communication and Negotiation
Strategic Lifecycle Management
Global Regulation: Asia
Human Resource Management
Global Regulation: Europe
“We are preparing the future leaders for a profession
that is just beginning to exist”
Travel to study global regulatory systems
Travel to learn business travel
Doctoral Program
Thesis topics reflect current issues
Regulatory dissonance among international agencies
GCP awareness of investigator sponsors
Evaluation of the adequacy of guidance for extractable/
leachable testing
Engagement of academic investigators in the clinical trial
agreement process
Adoption of quality systems in hospital settings
FDA’s review process for clearance of imported products
through US ports
International Center for Regulatory
Science
Education
Consults
Outreach
Research
Designed for research and international outreach
Program in Biodesign
needs invent
med tech patent
license collaborate
cost effective
surgical innovation
license Stanford
FDA discovery
technology transfer
ethics & policy prototype reimbursement
specialty innovation teamwork outcomes
biodesign
Mission
To develop leaders in biomedical
technology innovation
Educate. Collaborate. Innovate.
biodesign
medtech vs. biopharma
medtech
biopharma
Disciplines
mech eng
elect eng
med/surg
chem eng
comput sci
biology
genetics
Innovation
Process
needsdriven
discovery
plus need
The Bay Area has the highest
concentration of new biotech and medtech
companies in the world
Medtech Mecca
J. Abele, C. Alferness, P. Arensdorf, P. Auerbach, D. Auth, M. Baker, J. Bakker,
L. Barish, A. Belson, M. Berman, A. Bianchi, N. Blevins, L. Bottorff, K. Bowsher,
R. Buyan, B. Byers, J. Capek, M. Carusi, T. Chou, K. Connors, B. Constantz, R.
Croce, R. Curtis, M. Cutkosky, M. Dake, A. de Bord, M. Deem, J. Delagardelle,
D. Donohoe, D. Echt, D. Feigal, R. Ferrari, F. Fischer, R. Fisher, T. Fogarty, G.
Foster, J. Garfinkle, M. Garrison, J. Geriak, G. Gershony, H. Gifford, J. Gill, J.
Gold, J. Gordon, G. Graham, J. Green, J. Grossman, E. Grube, D. Gruenfeld, L.
Guterman, S. Hamade, W. Hawkins, M. Hendricksen, J. Hickey, T. Hinohara, R.
Hirsch, E. Hlavka, D. Hoffmeister, L. Hogle, H. Holstein, M. Horsewski, S.
Hossainy, K. Im, M. Imran, F. Ingle, P. Jackson, J. Jacobs, W. Jaeger, R. Jaffe, J.
Joye, S. Jwanouskos, A. Kaganov, D. Kelley, T. Kelley, K. Kelley, F. Khosravi, G.
Kilman, T. Kohler, E. Koskinas, M. Lachman, J. Lacob, G. Lambrecht, T.
Lamson, J. Lasersohn, S. Latterman, T. Lefteroff, M. Lesh, A. Macfarlane, W.
Maloney, J. Mandato, J. Maroney, D. Mauney, A. May, M. Mayer, C. McGlynn, L.
Michael, T. Mills, N. Mourlas, D. Murphy-Chutorian, J. Nehra, C. Nelson, W.
New, S. Oesterle, J. Onopchenko, M. Paganini, J. Palmaz, O. Palmer, T. Kim
Parnell, R. Perkins, T. Petersen, D. Piacquad, H. Plain, M. Raab, G. Rao, A.
Rasdal, R. Reiss, G. Robertson, D. Roeder, E. Rogers, S. Rowinski, G. Rubin,
S. Salmon, W. Samson, J. Schox, M. Selmon, J. Shapiro, J. Shay, T. Simons, J.
Simpson, Y. So, F. St. Goar, B. Starling, B. Stern, M. Sugarman, R. Sutton, J.
Swick, R. Tabibiazar, K. Talmadge, L. Tannenbaum, R. Thomas, S. Toyloy, S.
Turnbull, J.S. Uy, B. Vale, S. Van Bladel, C. Vidal, M. Wan, K. Wasserstein, K.
Widder, A. Will, J. Wilson, S. Wolf, T. Wollaeger, P. Young, R. Zider…& others
Real-World Mentors
Biodesign Innovation Fellowships
The biodesign process …
identify
invent
implement
biodesign
IDENTIFY
1. NEEDS FINDING
1.1 Strategic
Focus
2. NEEDS SCREENING AND SPECIFICATION
1.2
Observation &
Problem
Identification
INVENT
3. CONCEPT GENERATION
3.1
Brainstorming
1.3 Need
Statement
Development
2.1
Disease State
Fundamentals
2.3
Stakeholder
Analysis
2.5 Needs
Filtering
Need
Statement
2.2
Treatment
Options
2.4
Market
Analysis
Need
Specification
4. CONCEPT SELECTION
3.2
Concept
Screening
4.1
Intellectual
Property
4.3
Regulatory
Multiple
Concepts
4.2
Reimbursement
4.4
Business
Models
IMPLEMENT
5.3
Regulatory
Strategy
5.2
Reimbursement
Strategy
5.4
Stakeholder /
Political
Strategy
5.5 Clinical &
Marketing
Strategy
4.6 Concept
Selection
Final
Concept
& Prototype
6. DEVELOPMENT PLAN
5. DEVELOPMENT STRATEGY
5.1
IP Strategy
4.5 Early
Stage Design
& Prototyping
5.6 Competitive
Advantage &
Value
Proposition
Competitive
Advantage
Statement
6.1 R&D /
Engineering
Plan
6.4
Operating Plan
& Milestones
6.2
Clinical Trial
Design
6.5 Financial
Modeling &
Proxy
Validation
6.3
Sales &
Marketing
Plan
6.6
Funding
Sources
6.7
Business Plan
Development
Business
Plan
Start With “Boot Camp”
• Intensive introduction
to clinical field
• Lectures by clinical
faculty
• Engineering & business
overview
• Team building
Clinical Immersion
• Team “lives” in hospital and
clinics
• Observe with fresh eyes, ask
“naïve” questions
• Develop list of >200 needs
needs finding
needs screening
and specification
need
need
need
need
need
need
need
need
need
need
need
need
need
need
need
need
need
need
need
need
need
need
need
need
biodesign
Select the top needs to pursue…
and create a need statement
Need Specification
•
•
•
•
•
Verify & Validate
Quantify the Potential Benefit
Develop Criteria for Screening
Rank the Needs
Create A Written Need
Specification
NOW it’s time to invent…
Concept Development
•
•
•
•
•
Brainstorm
Prototype
In-Vivo / In-Vitro Modeling
Screen Based On Criteria
Create A Concept
Specification
concept
generation
concept
selection
x
concept
need
need
concept
concept
x
concept
x
x
concept
program
concept
concept
need
x
x
concept
concept
x
biodesign
Business/Project Planning
•
•
•
•
•
•
•
•
Financial Modeling
Funds Forecasting
Research Strategy
Marketing Strategy
Clinical/Regulatory Strategy
Ethical Considerations
Management Planning
Business/Project Plan
Schedule
August 1
welcome
Aug
innovation boot camp
engineering, business
intro to med technologies
initial clinical immersion
Sept
specialty immersion
Oct
needs screen & specification
Nov, Dec
brainstorming, concept development
Jan
prototyping, concept selection
Feb
development strategy
March
externship
April-June
project planning, launch
Our real “product”
Georgetown University:
Regulatory Science Graduate
Education Program
Ira Shoulson MD
Professor of Neurology, Pharmacology, and Human Science
Director, Program for Regulatory Science and Medicine (PRSM)
Principal Investigator, CERSI
Georgetown University Medical Center
CERSI
Kenneth Dretchen PhD
Professor and Chair, Department of Pharmacology
Co-Principal Investigator, CERSI
Georgetown University Medical Center
http://regulatoryscience.georgetown.edu
Sponsored by the U.S. Food and Drug Administration (U 01 FD 4319)
Please note: The views presented do not
necessarily reflect those of the FDA.
CERSI
http://regulatoryscience.georgetown.edu
Overview: Regulatory Science
Graduate Education at Georgetown
• Unique track in Regulatory Science builds on core curriculum of the
Master of Science in Clinical and Translational Research (MS-CTR)
of the Georgetown-Howard Universities Center for Clinical and
Translational Science (GHUCCTS; an NIH-supported CTSA)
• Flexible format and design to accommodate working professionals
▫ Part- or full-time enrollment status
▫ Online, with some in-person classes
• Relevant for regulators, policy makers, or public health researchers
• Faculty from Georgetown and affiliated institutions, including FDA
• Introduction to Regulatory Science first offered in Fall 2012
CERSI
http://regulatoryscience.georgetown.edu
Core Competencies
• GHUCCTS Master of Science in Clinical and Translational Research
(MS-CTR) provides a foundation in key skills such as:
▫
▫
▫
▫
Applications of bioethics to research,
Systematic clinical trial design and administration,
Data collection and analysis, and
Methods of behavioral and social sciences.
• The Regulatory Science concentration is appropriate for trainees
who are interested in developing:
▫ Broad understanding of regulatory science applications to public health,
▫ Enhanced analytic skills and methods for evidence-based decisionmaking,
▫ An understanding of the role of novel technologies, tools, and
approaches to assessing safety and quality of medical, food, or tobacco
products, and
▫ Behavioral and social science skills in communicating risk and benefit.
CERSI
http://regulatoryscience.georgetown.edu
Teaching, Learning, and Real-World
Application
• This area of concentration will include didactic
instruction, student-led and mentored exercises, and
discussion in carefully structured courses to provide this
knowledge base.
• Candidates will hold or currently pursue medical,
master, or terminal degrees in related disciplines,
ensuring that courses will be taught with a high level of
rigor.
• Successful degree candidates will apply scientific
knowledge and analytical capabilities and realize career
opportunities in academia, government, or industry.
CERSI
http://regulatoryscience.georgetown.edu
MS-CTR Degree Requirements
• MS-CTR core courses – 23 credits
• Regulatory Science electives – 10 credits
• Capstone Research Project – 4 credits
• Total – 33 credits
CERSI
http://regulatoryscience.georgetown.edu
MS-CTR Core Curriculum
•
•
•
•
•
•
•
•
•
Research Ethics with Human Subjects (3)
Introduction to Biostatistics in Clinical Research (3)
Study and Clinical Trial Design (2)
Project Development in Clinical Research (1)
Epidemiologic Methods (3)
Clinical Research Administration (3)
Social and Behavioral Aspects of Public Health (3)
Core Clinical Research Seminars (1)
Capstone Research Project (4)
Total Core Credits: 23
CERSI
http://regulatoryscience.georgetown.edu
MS-CTR Regulatory Science Electives
• Introduction to Regulatory Science (2)
• Analytic Approaches for Regulatory Science (3)
• Approaches for Assessing Safety, Quality, and
Benefit (3)
• Communicating Risk and Benefit for Public
Health (2)
Total Electives Credits: 10
CERSI
http://regulatoryscience.georgetown.edu
Capstone Research Project
• 4 credits
• Mentored research project
• Individualized to each student’s career and
research goals
• Based on the competencies developed in the MSCTR, Regulatory Science course of study
• Students required to present projects during
their final (summer) term in a program-wide
poster/discussion session
CERSI
http://regulatoryscience.georgetown.edu
Introduction to Regulatory Science
• Survey course:
▫
▫
▫
▫
▫
Examines the issues that arise in the field of regulatory science
Introduce students to fundamental principles of regulatory science
Distinguishes between regulatory science and regulatory affairs
Provides an overview of the innovative clinical research tools
Focuses on FDA’s eight priority areas for advancing regulatory science
• Expert lecturers from GU, FDA, and other academic partners lecture
and guide discussion of key issues.
• Open to students from Georgetown, FDA, other academic centers,
and other government / nonprofit / research organizations.
• Students may opt to participate as guests (non-degree seeking, noncredit earning)
CERSI
http://regulatoryscience.georgetown.edu
Session Topics
1. Fundamentals of
Regulatory
Science
2. Regulatory
Affairs and the
Law
3.
Regulatory
Science and
Bioethics
4.
Biosurveillance,
Medical
Countermeasures,
and Global Health
Product
Manufacturing
and Quality
Clinical Research
Tools of
Regulatory
Science
5.
6.
CERSI
Objectives
Describe course purpose, structure; and
requirements; introduce concepts and
history of Regulatory Science.
Examine the legal issues that arise in the
field of regulatory science; introduce key
food and drug legislation and related legal
issues; explain life cycle of scientific and
clinical research.
Describe reasoning skills required to
explain and justify decisions regarding
ethical questions and dilemmas; review the
nine key training areas identified by NIH
for the responsible conduct of research.
Provide an overview of regulatory scienceinformed medical countermeasures to
protect the public from threats to national
and global health and security.
Examine the development and application
of new methods to manufacture and ensure
quality of medical products.
Review the basic principles of clinical
trials, emphasizing scientific rationale,
organization and planning, and
methodology; introduce trial designs,
research monitoring, and statistical
analysis tools.
Date
9/12/12
Time
6:00 –
7:50pm
9/19/12
6:00 –
7:50pm
9/26/12
6:00 –
7:50pm
K. FitzGerald
(GUMC)
10/3/12
6:00 –
7:50pm
I. Jillson (GUMC);
A. Liss (FDA)
10/10/12
6:00 –
7:50pm
J. Polli (MD)
10/17/12
6:00 –
7:50pm
R. Temple (FDA)
http://regulatoryscience.georgetown.edu
Invited Lecturers
I. Shoulson
(GUMC);
C. Wilson (FDA)
S. Zimmet
(GUMC);
R. Mehta (FDA)
Session Topics
1. Novel
Technologies for
Development of
Medical Products
2. Toxicology and
Product Safety
3.
4.
5.
Regulatory
Science and Social
and Behavioral
Sciences
Regulatory
Science and Food
Safety
Regulatory
Science and
Tobacco
Products*
CERSI
Objectives
Review ongoing efforts of regulators to
evolve regulatory review practices to
enable evaluation of innovative emerging
technologies.
Introduce modern toxicology tools and
methods to analyze pre-clinical and
clinical data that informs regulators about
the safety of medical products, including
pharmaceuticals, biologics, and devices.
Introduce ways regulatory science will
enable health professionals and the public
to make more informed decisions about
medical and food products.
Introduce concepts of toxicology applied
in food science; review the assessment of
nutritional value; examine regulatory
review practices that enable a preventionfocused food safety system.
Develop new knowledge, skills, and
understanding of FDA’s new and broad
regulatory authorities over tobacco
products and its impact on population
health.
Date
10/24/12
Time
6:00 –
7:50pm
Invited Lecturers
C. Dollins (FDA);
C. Peña (FDA)
10/31/12
6:00 –
7:50pm
S. Schwartz
(GUMC);
D. Jacobson-Kram
(FDA)
11/7/12
(Part 1)
6:00 –
6:50pm
N. Ostrove
(independent,
former FDA)
11/7/12
(Part 2)
7:00 –
7:50pm
J. Levitt (Hogan
Lovells)
11/14/12
6:00 –
7:30pm*
L. Deyton (CTPFDA)
http://regulatoryscience.georgetown.edu
Session Topics
1. Personalized
Medicine and
Patient Outcomes
2.
Bioinformatics
3.
Conclusions and
Review
CERSI
Objectives
Present innovative trial design and
evaluation techniques to improve medical
product development and patient
outcomes.
Introduce the application of information
sciences to biology; review basics of
molecular biology and genetics, and tools
used to analyze protein sequences;
describe how diverse data can improve
outcomes.
Review course material and answer final
questions to ensure students achieved
course competencies. Students will present
their research and findings.
Date
11/28/12
Time
6:00 –
7:50pm
12/5/12
6:00 –
7:50pm
12/12/12
6:00 –
7:50pm
http://regulatoryscience.georgetown.edu
Invited Lecturers
H. Federoff
(GUMC);
C. Christopher
(Independent)
S. Madhavan
(GUMC);
V. Seyfert-Margolis
(FDA)
K. Dretchen
(GUMC);
R. Filice (FDA)
Admissions Requirements
Application deadline: Fall 2013 – May 15, 2013
1.
Application form –
http://grad.georgetown.edu/pages/apply_online.cfm
2. Non-refundable application fee – $80
3. Statement of purpose – A personal statement describing
past exposure to CTR or regulatory science, reasons of
interest in the program, and career goals
4. Resume/CV
5. Three written recommendations
6. GRE – not required
7. TOEFL/IELTS, if applicable
8. Official transcripts (all prior institutions)
CERSI
http://regulatoryscience.georgetown.edu
Contact
Regulatory Science at Georgetown University
Program for Regulatory Science & Medicine
Center of Excellence in Regulatory Science and Innovation
regulatoryscience@georgetown.edu
CERSI
http://regulatoryscience.georgetown.edu
Johns Hopkins University
Center for Biotechnology Education
Regulatory Science
Thomas Colonna, PhD, JD
Lynn Johnson Langer, PhD, MBA
Jamie Austin, RAC
Associate Director
Regulatory Science
Program Director
Biotechnology Enterprise and Regulatory
Science
Coordinator
Biotechnology Enterprise and
Regulatory Science
tcolonn1@johnshopkins.edu
ljlanger@jhu.edu
jausti12@jhu.edu
Why Johns Hopkins Biotechnology?
•
Worldwide Leader in Biotechnology Education
•
Online Courses accessible Worldwide
•
Fully online
• MS in Regulatory Science
•
MS in Biotechnology - Concentration in Regulatory Affairs
Expert Faculty from Government
and Private Industry
• JHU Medical
• MedImmune
(Astra-Zeneca)
• Merck
• SAIC
• 20/20 Gene Systems
• NCBI
• NIH
• Human Genome
Sciences
• J Craig Venter Institute
• National Cancer Institute
• USAMRIID
• FDA
Student Professions
•
•
•
•
•
•
•
•
Regulatory Professionals
QA/QC Specialists
Lawyers
Engineers
Scientists
Business Professionals
Venture Capitalists
Pharmacists
Campus Locations
• Montgomery County Campus, Rockville,
MD
• Online Programs, Worldwide
Master of Science in
Regulatory Science
MS in Regulatory Science
Admission Requirements
•
•
•
Undergrad degree in science/engineering
3.0+ GPA
Prerequisite courses:
• One semester Biochemistry & Cell Biology
OR
• Bioscience for Regulatory Affairs
• TOEFL (100 on Internet test)
MS in Regulatory Science
• Developed with leaders from
• FDA
• Industry
• Academia
• 10 Courses
• 7 Required Courses
• 3 Electives
• Online or onsite
MS in Regulatory Science
Required Courses
• Biological Processes
• Introduction to Regulatory Affairs
• Translational Biotechnology: From Intellectual
Property to Licensing
• Introduction to cGMP Compliance
• Clinical Development of Drugs and Biologics
• Food and Drug Law
• Practicum
MS in Regulatory Science
Elective Course Examples
•
•
•
•
•
•
•
•
•
Clinical Trial Design
QA/QC for Pharmaceuticals and Biotech
International Regulatory Affairs
Managing and Leading Biotech Professionals
Marketing in a Regulated Environment
Medical Device Regulation
Biomedical Software Regulation
Introduction to Food Safety
Validation in Biotechnology
Research Opportunities
• Independent Studies in Regulatory Affairs
• Must first complete at least 5 courses
• Identify study topic & mentor
• Project proposal & final written document
• Bioscience Regulatory Affairs Thesis
• Must first complete 9 courses
• Two semesters of thesis project
• First semester is Independent Study
General MS Biotechnology
Admission Requirements
•
•
•
•
•
•
Undergraduate degree in
science/engineering
3.0+ GPA
2 semesters of college chemistry
2 semesters of organic chemistry
2 semesters of biology
TOEFL (100 on Internet test)
MS in Biotechnology
Program Requirements
• Four Core Courses:
–
–
–
–
Biochemistry
Molecular Biology
Cellular Biology I
Cellular Biology II
• Six Electives
MS in Biotechnology
Concentrations
• Biotechnology Enterprise
• Regulatory Affairs
•
•
•
•
Biodefense*
Bioinformatics
Molecular Targets and Drug Discovery Technologies*
General
*Not Offered Online
Thesis Optional (11 courses)
Biotechnology
Elective Course Examples
• Managing and Leading
Biotech Professionals
• Marketing Aspects of
Biotech
• Creating a Biotech
Enterprise
• Bioscience
Communications
• Economic Dynamics of
Change in Biotechnology
• Introduction to Regulatory
Affairs
• Food and Drug Law
• International Regulatory
Affairs
• Validation in
Biotechnology
• Intro to cGMP Compliance
• Legal Aspects of Biotech
Online Education
•
•
•
•
Highly Interactive
Asynchronous
Rigorous
Same curriculum & faculty
• Onsite & online
• No residency requirement
• Ten years of online experience
Technology in Online Courses
• Adobe Connect
• Streaming Video
• Voice-over PowerPoint
Biotechnology Online Enrollment
Biotechnology Online Enrollment 1999 - 2006
40.0%
35.0%
30.0%
25.0%
20.0%
15.0%
10.0%
5.0%
0.0%
1999-2000
2000-2001
2001-2002
2002-2003
2003-2004
online enrollment percent of total
2004-2005
2005-2006
2006-2007
Student Satisfaction Online
vs. Onsite vs Onsite
Student Satisfaction,
Online
100
90
80
70
60
50
Online (n=79)
OnSite (n=261)
40
30
20
10
0
Taught at
Level
Expected
Learned a
Great Deal
Assignments Encouraged
Effective
Participation
Work Load
Rigorous
Recommend
Course
Financial Aid
• Advanced Academic Programs
Scholarship Assistance Program
• Learn more, please visit:
http://www.jhu.edu/finaid/part_time.html
Regulatory Science Application:
• AAP Apply Yourself online application
• $75.00 application fee
• Resumé or curriculum vitae
• Official undergraduate transcript from all
universities attended
• 500-word statement of purpose
• International students:
• TOEFL
• WES evaluation
Contact Information
Jamie L. Austin, RAC
Biotechnology Enterprise and
Regulatory Science
jausti12@jhu.edu
301.448.9671
Thomas E. Colonna, PhD, JD
Associate Director Regulatory
Science
tcolonn1@jhu.edu
302.292.8342
Lynn Johnson Langer, PhD, MBA
Program Director
ljlanger@jhu.edu
For current and comprehensive admissions information, full course
listings and descriptions, and other program information, please visit
biotechnology.jhu.edu.
•
Admissions Inquiries
202.452.1940
aapadmissions@jhu.edu
The University of Arkansas for Medical
Sciences Program in Regulatory Sciences
Jay Gandy, Ph.D.
Department of Environmental & Occupational Health
University of Arkansas for Medical Sciences (UAMS)
Degree Programs in Regulatory Sciences Conference
University of Maryland College Park
April 15, 2013
Why a Regulatory Sciences
Training Program at UAMS?
Historical NCTR/UAMS
collaborations in education and
research.
 Existing graduate programs in
pharmacology & toxicology
 Proximity to FDA research center

Conceptual Genesis of Regulatory
Sciences program at UAMS
Proposed 2006
 Concept document prepared and
circulated 2008
 FDA/State of Arkansas MOU 2011.
 One component of MOU is the
formation of a Regulatory Sciences
educational and research program
at UAMS.

Targeted Potential Students




Post-doctoral Fellows at NCTR
Post-doctoral Fellows at UAMS
Graduate students in Pharmacology
and Toxicology at UAMS
Other Graduate students at UAMS
(e.g., biochemistry, physiology,
etc.)
Profile of UAMS Regulatory
Sciences Students

Type of Student interested in training
in Regulatory Sciences at our
institution.


Career aspirations.


Educational background (mostly at
doctoral level)
Government and Industry
Motivations for additional training in
Regulatory Sciences

Employment enhancement
Why add Regulatory Sciences Training
to traditional graduate education?

Although well trained in basic or
clinical sciences, most newly minted
doctoral students or post-doc who
join regulatory agencies or regulated
industries have not developed the
critical skills needed to apply their
science in the context of regulatory
decision-making.
Goals of UAMS Regulatory
Sciences Program



Provide students with insight into the
complexities of the laws, regulations,
policies, risk assessments, risk-benefit
analyses and risk management
processes.
Working knowledge of regulatory
science that can foster leadership in
regulatory science for industry,
government and academia.
Provide a more competitive background
for regulatory science-based careers.
Curriculum Design – Specific
Learning Objectives


Develop a broad understanding of the
regulatory framework of the U.S. Food
and Drug Administration, including the
administrative structure and legislative
mandate of the agency.
Become familiar with the various
regulations that govern development of
new drug, medical devices, cosmetics,
and food ingredients and how they are
tested and approved for commerce.
Curriculum Design – Learning
Objectives (continued)



Develop the critical skills needed to assess
and apply basic science for regulatory
decision-making.
Develop a understanding of the types of
scientific data required for product safety
assessment and product approval.
Learn risk assessment/product safety
assessment methods and skills that
contribute to how scientific data from
animal testing are used to predict human
risk.
Curriculum Design – Learning
Objectives (continued)



Master risk assessment/product safety
assessment methods and skills applicable to the
collection of and utilization of human data that
can be used to estimate and predict population
risk.
Understand the regulations that govern clinical
drug trials and how clinical trials are designed
and managed for new drug approval.
Become knowledgeable of data quality
requirements for scientific studies submitted in
support of regulatory decisions.
 GLPs, GCPs, GMPs
Certificate in Regulatory Sciences
Initial Curriculum – 4 courses (12 hrs)
Principles of Food and Drug
Regulations
 Methods in Product Safety
Assessment/Risk Assessment
 Clinical Trials Design and
Management
 Good Regulatory Practices

Initiated Fall 2012
22 Students in first two courses
 Students from nine countries
(India, China, Mexico, Saudi
Arabia, Pakistan, Canada,
Ukraine, US)

Future Program Plans




Distance Learning offering of the
Certificate in Regulatory Sciences.
Certificate in Regulatory Sciences –
Multiple Tracks.
Master of Science in Regulatory
Sciences.
Masters of Public Health (MPH) with
emphasis in Regulatory Sciences.
Program Plans – Multi-track Certificate
Courses

Food Safety and Pre-clinical drug
development track


Clinical Research Management


FDA Regulations, Methods in Toxicity Testing,
Risk/Safety Assessment, Good Regulatory Practices
FDA Regulations, Clinical Trials Design &
Management, Good Regulatory Practices, Statistical
Methods for Clinical Trials
Pharmaceutical Quality Compliance

FDA Regulations, Good Regulatory Practice, Quality
Management Audits & Inspections, Chemical Basis
of Pharmaceuticals Manufacturing
Two new faculty positions
created in Regulatory Science

First position filled September
2012


Incorporation of epigenetic
mechanisms in product safety
assessment
Recruitment for second position
underway.
The End
Questions?
What does industry want from
regulatory science training?
Robert J. Meyer, MD
Virginia Center from Translational
and Regulatory Sciences
Recently ex-Merck, VP of GRA
Regulatory/Development Hiring
• Merck sought individuals for regulatory hiring with an
advance degree who understood medical science and drug
development first and foremost
– This is not a rare commodity in science-based organizations, but
getting such talent interested in the challenges of regulatory
science/affairs can be a challenge
• A second “desirable” (but not a sine qua non) is some
regulatory knowledge/experience
– MRL has an extensive series of courses that teach the basics of
global regulations (US, EU, Japan….)
– There are many external programs that teach the fundamentals
of regulation and regulatory processes
– But, much of the knowledge of how regulators think and act
comes from direct experience/activities – another challenge
Regulatory/Development Hiring
• What struck me in moving from FDA to industry is
how little even very seasoned and successful
regulatory personnel in industry know about the
details of how FDA acts and thinks
– Part of this is due to FDA and the formality of most
interactions
– Part of this is a lack of effective interchange
– This would be mitigated by better informal discussions
between industry and FDA on matters of the
intersection of science and regulation (potential role
of academia in fostering these discussions)
Regulatory/Development Hiring
• Merck also has participated in some limited
PharmD internships and rotational programs
internally to provide regulatory experiences
• What is not sufficiently available is a broaderbased, experiential training:
– Case-based education of how science, regulations/law
and policy intersect
– How to most effectively work with regulators to
advance regulatory science
– A curriculum that imparts the “fun” of solving for
regulatory complexities in a global environment to
affect an efficient, high POS development strategy
Broader Regulatory Science
Workforce Needs
• Innovative industry very much wants a regulatory
workforce that is conversant, if not expert, in evolving
science
• Good regulatory scientists also understand the nexus of
cutting edge science, regulatory history/framework
and policy
• Great regulators would look to provide regulation and
regulatory structures that are value added and efficient
• In short, regulated industry benefits from a highly
competent regulatory workforce that then is deployed
in sensible ways to do work that matters to public
health