importance of regulatory science driving biomedical ... · pdf fileimportance of regulatory...

Importance of Regulatory Science

Driving Biomedical Innovation by

Advancing Regulatory Science at

FDA

1 M-CERSI Educational Programs Conference; 04-15-13

Frank F. Weichold, M.D., Ph.D. Director

Critical Path and Regulatory Science Initiatives Office of the Chief Scientist/OC

Food and Drug Administration

[email protected]

http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/RegulatoryScience/UCM268225.pdf

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

http://www.researchamerica.org/uploads/healthdollar10.pdf



4 Biotech/Pharma

NIH

Small

Business

Academic

Physicians &

Patients

Public & Payors

Innovation is Linked to Ecosystem and Partnerships

FDA-regulated products account for 25 cents

of every consumer dollar spent in the U.S.

http://www.fda.gov/default.htm

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 FDA-regulated 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

Stratify patients by:

• Genetics

• PRO’s

• Behavior

• Disease definition

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

• 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.

Steps FDA is Taking to Drive Biomedical

Innovation:

8

Regulatory Science Publications

Innovation is a Core FDA Mission

10

THANK YOU

Advancing Regulatory Science Website http://www.fda.gov/ScienceResearch/SpecialTopics/Reg

ulatoryScience/default.htm

http://www.fda.gov/ScienceResearch/SpecialTopics/RegulatoryScience/default.htm

http://www.fda.gov/ScienceResearch/SpecialTopics/RegulatoryScience/default.htm

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

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

1Univ. Of Maryland & US FDAJoint Institute for Food safety and Applied Nutrition

Joint Institute for Food Safety and Applied Nutrition (JIFSAN)

How?How?

A collaborative effort between the University of Maryland, the U.S. Food and Drug Administration (CFSAN and CVM), and the private sector

When?When?

Established in 1996.

What?What?

A multidisciplinary research, education and outreach program – domestic and international in scope

Concepts of OperationConcepts 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


JIFSAN Programs

University of Maryland

US Food & Drug

Administration

ResearchResearch EducationEducation

OutreachOutreach

Research GrantsResearch Grants Center for Risk Communications

Research

Center for Risk Communications

Research

Internship PostdocInternship Postdoc

FoodRisk.orgFoodRisk.org ConferencesConferencesT r a i n i n gT r a i n i n g

Good Agricultural Practices

Good Agricultural Practices

Good Aquacultural Practices

Good Aquacultural Practices

Risk AnalysisRisk

Analysis


Commercially Sterile

Packaged Foods

Commercially Sterile

Packaged Foods

International Food Safety Training Laboratory


Food Inspector Training

Food Inspector Training

JIFSAN Training Portfolio

International training centerInternational training center Food Safety Risk Analysis Training Program

Food Safety Risk Analysis Training Program



Good Agricultural Practices (GAP) Good Aquacultural Practices (GAqP) Commercially Sterile Packaged Foods (CSPF) Food Inspector training (FIT)


‘

JIFSAN – The programs

0

1

2

3

4

5

6

7

8

9

2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 2010 2011 2012

FoodInspectorTraining

CommercialFoodProcessingTechnologyGoodAquaculturalPractices

GoodAgriculturalPractices

Trinidad Brazil Dominican Republic, Mexico, Puerto Rico

Mexico (2), Peru

Guatemala, Honduras, Korea

Thailand, Brazil, Mexico

Mexico, El Salvador, China.

Vietnam

Guatemala, Peru, Nicaragua

Costa Rica, Honduras.

Indonesia, Thailand

Honduras, Dominican Republic, Guatemala, Mexico, El Salvador. Bangladesh.

Morocco

Peru, Costa Rica, Mexico (2) .

Bangladesh, Malaysia, US*.

China

Mexico.

Bangladesh, China.

China.

Honduras (Central America), India (spices and botanicals). India, Vietnam.

China.

GAPs: 34 trainings in 17 countriesGAPs: 34 trainings in 17 countries GAqPs: 10 trainings in 7 countriesGAqPs: 10 trainings in 7 countries


* 10 day internship in US for 9 Bangladeshi trainers (2010)

Country/market sector ownership and involvement required for sustainable development

Program aligns with country partners’ agenda and using country partners’ systems

Country partners set the agenda based on needs of the market sector

Country partner provides the resources to develop and sustain program

A success story – based on key principles

A success story – based on key principles

Planned Collaborative CentersPlanned Collaborative Centers


The Global Collaborating Center Concept

Bangladesh (GAqP) Bangladesh Shrimp and Fish Foundation

Mexico (GAP) SENASICA

India (GAP for Spices and Botanicals) CII Jubilant Bhartia Food and Agriculture

Centre of Excellence

Spice Board

Thailand (CSPF for aquaculture products)


Food safety risk analysis courses

Have trained 1300 individuals from over 32 different countries who took over 2000 course Have trained 1300 individuals from over 32 different countries who took over 2000 course

,

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


Food Safety Laboratory Capacity Training

Laboratory Training


CanadaChileChinaCosta RicaDominican RepublicGuatemalaHonduras

IndonesiaKoreaMalaysiaMexicoPhilippinesUSAVietnam

Food Safety Laboratory Capacity Training

Laboratory TrainingLaboratory 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

Participants Origins (1 year)Participants Origins (1 year)

Year 1:April: Method for Mycotoxins in FoodMay: Pesticide Residue Analysis (APEC, funded USDA)June: Cronobacter in Infant FormulaSeptember: Salmonella in ProduceOctober: E. coli (produce and meat)Oct: Microscopic and Chemical Identification of Ingredients in Dietary SupplementsNov: Pesticide Residue Analysis (+ ISO 17025)

University of MarylandMS 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)– Pre‐recorded lectures– Homework– Web conferences– Assessments

• Projects• Online presentations• Mini‐reviews

UMCP Certificate in Regulatory Science and EngineeringRequired Course:• BIOE 689 Introduction to Regulatory Affairs: Medical DevicesThree 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 [email protected]

REGULATORYSCIENCE UNIVERSITY OF SOUTHERN CALIFORNIA

http://regulatory.usc.edu

2001

•MS Regulatory Science

2005

•Certificate Program

2008

•Doctorate in Regulatory Science

2012

•MS Management of Drug Development

2013

•MS Healthcare Decision Analysis

Our program has expanded over the years

To meet the complexities of an evolving discipline

Flexibility To Choose A Program

Based on individual student’s goals and aspiration

Certificate

Food safety

Regulatory Science

Early Drug Development

Clinical Design and Management

Patient and Product Safety

Master’s

Regulatory Science

Management of Drug Development

Healthcare Decision Analysis

Doctorate Regulatory Science

MS Regulatory Science Curriculum

• Global Regulations

• Risk Frameworks

• Seminar

• Clinical Design

• Business

• Food Science

• CMC

• Ethics

• Introduction

• Drugs & Biologics

• Devices

• Foods

• Quality

• Law

• Clinical Management

• Writing

• Internship

Composed of core courses and electives

MS Management of Drug Development

Collaboration with faculty from School of Pharmacy and other academic & industry experts

• Translational Medicine

• Drug Discovery

• Preclinical

• Early Clinical

• Drug Design

• Business

• Clinical Design

• Directed Research

• Biostatistics

• Food and Drug Toxicology

• CMC

• Drugs and Biologics

• Ethics

• Intro to Regulations

• Clinical Management

MS Healthcare Decision Analysis

• Healthcare Insurance

• International

• Competitive Intelligence

• Healthcare Reform

• Drug Information

• Outcomes

• Clinical Trials and Statistics

• Project Management

• 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

Discovery Phase I Phase II Phase III NDA/BLA

Research Early Development Late Development Approval

Access

Coverage

MDD Translational Research

HDA Outcomes and Access

Regulatory Science IND to Approval

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

Weekend

8-hour days

Short-course approach

Binders and course materials

supplied

Condensed classes

Additional

Distance Modules

Virtual Office Hours

Executive approaches

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

• 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

Education Consults

Research Outreach

Designed for research and international outreach

International Center for Regulatory Science

biodesign

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

To develop leaders in biomedical technology innovation

Mission

Educate. Collaborate. Innovate.

medtech vs. biopharma

medtech biopharma

Innovation

Process

mech eng

elect eng

med/surg

needs-

driven

discovery

plus need

chem eng

comput sci

biology

genetics

Disciplines

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 Biodesign Innovation Fellowships

biodesign

The biodesign process …

identify

invent

implement

1.1 Strategic

Focus

1.2

Observation &

Problem

Identification

1.3 Need

Statement

Development

2.1

Disease State

Fundamentals

2.2

Treatment

Options

2.3

Stakeholder

Analysis

2.4

Market

Analysis

2.5 Needs

Filtering

4.6 Concept

Selection

1. NEEDS FINDING

5. DEVELOPMENT STRATEGY

5.1

IP Strategy

5.3

Regulatory

Strategy

5.5 Clinical &

Marketing

Strategy

5.4

Stakeholder /

Political

Strategy

5.2

Reimbursement

Strategy

6. DEVELOPMENT PLAN

6.2

Clinical Trial

Design

6.1 R&D /

Engineering

Plan

6.4

Operating Plan

& Milestones

6.6

Funding

Sources

6.7

Business Plan

Development

6.3

Sales &

Marketing

Plan

6.5 Financial

Modeling &

Proxy

Validation

2. NEEDS SCREENING AND SPECIFICATION ID

EN

TIF

Y

INV

EN

T

IMP

LE

ME

NT

3.1

Brainstorming

4.1

Intellectual

Property

4.5 Early

Stage Design

& Prototyping

4.2

Reimbursement

4.3

Regulatory

4.4

Business

Models

3.2

Concept

Screening

4. CONCEPT SELECTION

5.6 Competitive

Advantage &

Value

Proposition

Need

Statement

Need

Specification

Multiple

Concepts

Final

Concept

& Prototype

Competitive

Advantage

Statement

Business

Plan

3. CONCEPT GENERATION

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

biodesign

needs finding

need

need

need

need

need

need

need

need

need

need

need

need

need

need

need

need

need

need

need

need

need

needs screening

and specification

need

need

need

and create a need statement

Select the top needs to pursue…

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

biodesign

need

need

need

concept generation

concept

concept

concept

concept

concept

concept

concept

concept

concept

concept selection

x

x

x

x

x

x

x

program

Business/Project Planning

• Financial Modeling

• Funds Forecasting

• Research Strategy

• Marketing Strategy

• Clinical/Regulatory Strategy

• Ethical Considerations

• Management Planning

• Business/Project Plan

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

Sch

ed

ule

Our real “product”

CERSI

http://regulatoryscience.georgetown.edu

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

Kenneth Dretchen PhD Professor and Chair, Department of Pharmacology Co-Principal Investigator, CERSI Georgetown University Medical Center



CERSI 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.




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




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 decision-

making, ▫ 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.




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.




MS-CTR Degree Requirements

• MS-CTR core courses – 23 credits

• Regulatory Science electives – 10 credits

• Capstone Research Project – 4 credits

• Total – 33 credits




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




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




Capstone Research Project

• 4 credits • Mentored research project • Individualized to each student’s career and

research goals • Based on the competencies developed in the MS-

CTR, Regulatory Science course of study • Students required to present projects during

their final (summer) term in a program-wide poster/discussion session




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, non-

credit earning)




Session Topics Objectives Date Time Invited Lecturers

1. Fundamentals of

Regulatory

Science

Describe course purpose, structure; and

requirements; introduce concepts and

history of Regulatory Science.

9/12/12 6:00 –

7:50pm

I. Shoulson

(GUMC);

C. Wilson (FDA)

2. Regulatory

Affairs and the

Law

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.

9/19/12 6:00 –

7:50pm

S. Zimmet

(GUMC);

R. Mehta (FDA)

3. Regulatory

Science and

Bioethics

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.

9/26/12 6:00 –

7:50pm

K. FitzGerald

(GUMC)

4. Biosurveillance,

Medical

Countermeasures,

and Global Health

Provide an overview of regulatory science-

informed medical countermeasures to

protect the public from threats to national

and global health and security.

10/3/12 6:00 –

7:50pm

I. Jillson (GUMC);

A. Liss (FDA)

5. Product

Manufacturing

and Quality

Examine the development and application

of new methods to manufacture and ensure

quality of medical products.

10/10/12 6:00 –

7:50pm

J. Polli (MD)

6. Clinical Research

Tools of

Regulatory

Science

Review the basic principles of clinical

trials, emphasizing scientific rationale,

organization and planning, and

methodology; introduce trial designs,

research monitoring, and statistical

analysis tools.

10/17/12 6:00 –

7:50pm

R. Temple (FDA)





1. Novel

Technologies for

Development of

Medical Products

Review ongoing efforts of regulators to

evolve regulatory review practices to

enable evaluation of innovative emerging

technologies.

10/24/12 6:00 –

7:50pm

C. Dollins (FDA);

C. Peña (FDA)

2. Toxicology and

Product Safety

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.

10/31/12 6:00 –

7:50pm

S. Schwartz

(GUMC);

D. Jacobson-Kram

(FDA)

3. Regulatory

Science and Social

and Behavioral

Sciences

Introduce ways regulatory science will

enable health professionals and the public

to make more informed decisions about

medical and food products.

11/7/12

(Part 1)

6:00 –

6:50pm

N. Ostrove

(independent,

former FDA)

4. Regulatory

Science and Food

Safety

Introduce concepts of toxicology applied

in food science; review the assessment of

nutritional value; examine regulatory

review practices that enable a prevention-

focused food safety system.

11/7/12

(Part 2)

7:00 –

7:50pm

J. Levitt (Hogan

Lovells)

5. Regulatory

Science and

Tobacco

Products*

Develop new knowledge, skills, and

understanding of FDA’s new and broad

regulatory authorities over tobacco

products and its impact on population

health.

11/14/12 6:00 –

7:30pm*

L. Deyton (CTP-

FDA)





1. Personalized

Medicine and

Patient Outcomes

Present innovative trial design and

evaluation techniques to improve medical

product development and patient

outcomes.

11/28/12 6:00 –

7:50pm

H. Federoff

(GUMC);

C. Christopher

(Independent)

2. Bioinformatics 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.

12/5/12 6:00 –

7:50pm

S. Madhavan

(GUMC);

V. Seyfert-Margolis

(FDA)

3. Conclusions and

Review

Review course material and answer final

questions to ensure students achieved

course competencies. Students will present

their research and findings.

12/12/12 6:00 –

7:50pm

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)



http://grad.georgetown.edu/pages/apply_online.cfm


Contact

Regulatory Science at Georgetown University

Program for Regulatory Science & Medicine

Center of Excellence in Regulatory Science and Innovation

[email protected]



mailto:[email protected]

Johns Hopkins University

Center for Biotechnology Education

Thomas Colonna, PhD, JD Associate Director

Regulatory Science

[email protected]

Regulatory Science

Jamie Austin, RAC Coordinator

Biotechnology Enterprise and

Regulatory Science

[email protected]

Lynn Johnson Langer, PhD, MBA Program Director

Biotechnology Enterprise and Regulatory

Science

[email protected]

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)


• Developed with leaders from • FDA

• Industry

• Academia

• 10 Courses • 7 Required Courses

• 3 Electives

• Online or onsite


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


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 • Introduction to Regulatory

Affairs

• Food and Drug Law

• International Regulatory

Affairs

• Validation in

Biotechnology

• Intro to cGMP Compliance

• Legal Aspects of Biotech

• Managing and Leading

Biotech Professionals

• Marketing Aspects of

Biotech

• Creating a Biotech

Enterprise

• Bioscience

Communications

• Economic Dynamics of

Change in Biotechnology

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

0.0%

5.0%

10.0%

15.0%

20.0%

25.0%

30.0%

35.0%

40.0%

1999-2000 2000-2001 2001-2002 2002-2003 2003-2004 2004-2005 2005-2006 2006-2007

Biotechnology Online Enrollment 1999 - 2006

online enrollment percent of total

Biotechnology Online Enrollment

0

10

20

30

40

50

60

70

80

90

100

Taught at

Level

Expected

Learned a

Great Deal

Assignments

Effective

Encouraged

Participation

Work Load

Rigorous

Recommend

Course

Student Satisfaction Online vs. Onsite

Online (n=79)

OnSite (n=261)

Student Satisfaction, Online vs Onsite

Financial Aid

• Advanced Academic Programs

Scholarship Assistance Program

• Learn more, please visit:

http://www.jhu.edu/finaid/part_time.html

http://www.jhu.edu/finaid/part_time.html

• 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

Regulatory Science Application:

https://app.applyyourself.com/AYApplicantLogin/ApplicantConnectLogin.asp?id=jhu-aap

Contact Information

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

[email protected]

Jamie L. Austin, RAC

Biotechnology Enterprise and

Regulatory Science

[email protected]

301.448.9671

Thomas E. Colonna, PhD, JD

Associate Director Regulatory

Science

[email protected]

302.292.8342 Lynn Johnson Langer, PhD, MBA

Program Director

[email protected]

http://biotechnology.jhu.edu/graduate-education/index.html









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.

Educational background (mostly at doctoral level)

Career aspirations.

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 FDA Regulations, Methods in Toxicity Testing,

Risk/Safety Assessment, Good Regulatory Practices

Clinical Research Management 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 broader-based, 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

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