16th Annual NIH
SBIR/STTR Conference
Functional
Down-to-Earth
Advice
for understanding the FDA
Chris Sasiela, PhD, RAC
NHLBI Office of Translational Alliances and Coordination
Learning Objectives
 How the FDA is organized
 How drugs, biologics, and medical devices
are regulated
 Where to start
 Where to go for help
 Office of Special Medical Programs
Learning Objectives
 How the FDA is organized
 How drugs, biologics, and medical devices
are regulated
 Where to start
 Where to go for help
 Office of Special Medical Programs
FDA Organization
http://www.fda.gov/downloads/AboutFDA/CentersOffices/OrganizationCharts/UCM291886.pdf
Practical FDA for biomedical innovators
Center for Biologics
Evaluation and
Research
Office of Special
Medical Programs
Office of Medical
Products and Tobacco
Center for Drug
Evaluation and
Research
Center for Devices and
Radiological Health
Center for Tobacco
Products
Center for Biologics Evaluation and Research
http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CBER/ucm123224.htm
Practical CBER for biomedical innovators
Organized by therapeutic modality
Office of Blood
Research and Review
Office of the CBER
Director
Office of Vaccines
Research and Review
Office of Cellular,
Tissue and Gene
Therapies
De-coding Biologics
Office
Products Regulated
OBRR
Blood and blood components used for transfusion, such as red blood cells, plasma, and
platelets. Pharmaceutical products made from blood, such as clotting factors and
immunoglobulins.
Medical devices and tests used to safeguard blood, blood components, and cellular
products from HIV, hepatitis, syphilis, and other infectious agents. Reagents used to type
blood. Machines and related software used to collect blood and blood components.
OVRR
Vaccines used for the prevention of infectious diseases, such as mumps, measles, chicken
pox, diphtheria, tetanus, influenza, hepatitis, smallpox, and anthrax. Vaccines under
development to treat HIV, and to treat or prevent non-infectious conditions, including various
cancers.
Patch tests used to diagnose the causes of contact dermatitis. Extracts used to diagnose and
treat rhinitis ("hay fever"), allergic sinusitis and conjunctivitis, and bee stings.
OCTGT
Human tissues for transplantation, such as skin, tendons, ligaments, and cartilage.
Cellular products, such as human stem cells and pancreatic islets. Tissue and cellular
products have the potential to treat cancer, Parkinson's disease, hemophilia, anemia,
diabetes, and other serious conditions.
Gene therapy products that replace a person's faulty or missing genetic material. Gene
therapy research could lead to new treatments for cancer cystic fibrosis, heart disease,
hemophilia, diabetes, and infectious diseases such as AIDS.
Xenotransplantation products use live animal cells, tissues, or organs to treat human
diseases such as liver failure and diabetes, where human materials are not always available.
http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CBER/ucm133072.htm
Center for Drug Evaluation and Research
http://www.fda.gov/AboutFDA/CentersOffices/OrganizationCharts/ucm403687.htm
Practical CDER for biomedical innovators
Organized
(generally)
by disorder or
organ system
Office of Drug Evaluation I
Office of Drug Evaluation II
Office of Drug Evaluation III
Office of New
Drugs
Office of Drug Evaluation IV
Office of Antimicrobial Products
Office of Hematology and Oncology Products
An ODE by any other name…
Office of Drug Evaluation
Divisions
ODE I
http://www.fda.gov/aboutfda/centersoffi
ces/officeofmedicalproductsandtobacc
o/cder/ucm290659.htm
Cardiovascular and Renal Products
Neurology Products
Psychiatry Products
ODE II
http://www.fda.gov/aboutfda/centersoffi
ces/officeofmedicalproductsandtobacc
o/cder/ucm269644.htm
Anesthesia, Analgesia, and Addiction Products
Metabolism and Endocrinology Products
Pulmonary, Allergy, and Rheumatology Products
ODE III
http://www.fda.gov/aboutfda/centersoffi
ces/officeofmedicalproductsandtobacc
o/cder/ucm252063.htm
Gastroenterology and Inborn Errors Products
Dermatology and Dental Products
Bone, Reproductive, and Urologic Products
ODE IV
http://www.fda.gov/aboutfda/centersoffi
ces/officeofmedicalproductsandtobacc
o/cder/ucm106342.htm
Medical Imaging Products
Non-prescription Clinical Evaluation (OTC)
Non-prescription Regulation Development (OTC)
Center for Devices and Radiological Health
CDRH Management Directory by Organization
This information is current as of August 20, 2014.
Office of the Center Director
Office of Communication and Education
Office of Compliance
Office of Device Evaluation
Office of In Vitro Diagnostics and Radiological Health
Office of Management Operations
Office of Science and Engineering Laboratories
Office of Surveillance and Biometrics
http://www.fda.gov/aboutfda/centersoffices/officeofmedicalproductsandtobacco/cdrh/cdrhoffices/
ucm127854.htm
Practical CDRH for biomedical innovators
Organized (generally) by disorder or organ system
Anesthesiology, General Hospital,
Respiratory, Infection Control, and Dental
Ophthalmic and ENT
Neurological and Physical Medicine
Orthopedic
Surgical
Reproductive, Gastro-renal, Urological
Office of In Vitro Diagnostics
and Radiological Health
Office of Device
Evaluation
Cardiovascular
Chemistry and Toxicology
Devices
Immunology and Hematology
Devices
Molecular Genetics and
Pathology
Microbiology Devices
Radiological Health
Mammography Quality
Standards
Medical Specialties
Anesthesiology (868), General Hospital (880), Respiratory, Infection Control
(862, 864, 866), and Dental (872)
Office of Device
Evaluation
Cardiovascular (870)
Ophthalmic (886) and ENT (874)
Neurological (882) and Physical Medicine (890)
Orthopedic (888)
Surgical (878)
Reproductive (884), Gastro-renal (876), Urological (876)
Learning Objectives
 How the FDA is organized
 How drugs, biologics, and medical devices
are regulated
 Where to start
 Where to go for help
 Office of Special Medical Programs
Biologics Regulation




Regulated under the Public Health Service Act
IND for clinical development
Biologics License for marketing allowance
Co-regulate many medical devices
 specifically related to blood products
 ‘Biological Product''
 a virus, therapeutic serum, toxin, antitoxin, vaccine, blood,
blood component or derivative, allergenic product, or
analogous product, or arsphenamine or derivative of
arsphenamine (or any other trivalent organic arsenic
compound), applicable to the prevention, treatment, or
cure of a disease or condition of human beings.
Drug Regulation
 Federal Food, Drug, and Cosmetic Act
 as amended
- Orphan Drug Act (1983)
- Drug Price Competition and Patent Term
Restoration Act (1984)
- Best Pharmaceuticals for Children Act (2002)
- Pediatric Research Equity Act (2003)
- Food and Drug Administration Amendments Act
(2007)
- Food and Drug Administration Safety and
Innovation Act (2012)
Device Regulation
 FD&C Act Medical Device Amendments
- May 28, 1976
 Title 21 Code of Federal Regulations Parts 800-1299
 Classification based on risk
 Class I Low Risk (General Controls)
 Class II Medium Risk (General Controls and Special Controls)
 Class III High Risk (General Controls and Premarket Approval)
 Market allowance based on risk plus legislation
 Exempt,510(k), PMA
 Tracking
 Establishment Registration
 Medical Device Listing
 Unique Device Identification
Learning Objectives
 How the FDA is organized
 How drugs, biologics, and medical devices
are regulated
 Where to start
 Where to go for help
 Office of Special Medical Programs
www.FDA.gov
20
FOIA Electronic Reading Rooms
 CBER
 http://www.fda.gov/aboutfda/centersoffices/officeofmedicalproductsandt
obacco/cber/ucm129132.htm
 CDER
 http://www.fda.gov/drugs/guidancecomplianceregulatoryinformation/ucm
113237.htm
 CDRH
 http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsa
ndTobacco/CDRH/CDRHFOIAElectronicReadingRoom/default.htm
21
CBER Product Approval Information
 Biologics Products & Establishments
 Licensed Biological Products with Supporting
Documents
 Substantially Equivalent 510(k) Device Information
 Premarket Approvals (PMAs) and Humanitarian
Device Exemptions (HDEs)
 New Drug Applications (NDAs)
 Postmarketing Requirements and Commitments:
Introduction
 Transfer of Therapeutic Products to the Center for
Drug Evaluation and Research
Licensed Biological Products
with Supporting Documents
ALPROLIX (Biogen Idec)
Types of Supporting Information/Documents
BL 125444
Most current approval letter
Rationale for approval
In depth approval information!!
Understanding CBER Licensing Applications
Redacted documents
How do reviewers think?
How did the company present
their product?
(not all trials go into a
license application)
What was the company’s preclinical and clinical strategy?
CDER Product Approval Information
 Product Approval Information
 Drugs @ FDA
Basic Information
ND 205434
In depth approval information!!
What about the reviews?
This product was approved on July 23, 2014.
This screenshot was taken August 20, 2014.
Redaction and uploading of review information takes time!
CDRH Reading Room
510(k) Database
K140582
Product Information
Product Classification Information
510(K) Summary
Comparison to PredicateBasis of SE determination
Predicate
Identification
Device
Description
Intended Use
Sponsor
Conclusions
FDA Educational Resources
 CBER OCTGT Learn
 http://www.fda.gov/biologicsbloodvaccines/newsevents/ucm232821.htm
 CDER Learn
 http://www.fda.gov/Training/ForHealthProfessionals/default.htm
 CDRH Learn
 http://www.fda.gov/Training/CDRHLearn/default.htm
34
General Websites of Interest
 FDA Small Business Guide
 http://www.fda.gov/downloads/forindustry/smallbus
inessassistance/smallbusinessguidetofda/ucm081
030.pdf
 FDA Small Business Contacts
 http://www.fda.gov/forindustry/smallbusinessassist
ance/smallbusinessrepresentatives/
 FDA Information update request page
 http://www.fda.gov/AboutFDA/ContactFDA/StayInf
ormed/GetEmailUpdates/default.htm#top
35
Learning Objectives
 How the FDA is organized
 How drugs, biologics, and medical devices
are regulated
 Where to start
 Where to go for help
 Office of Special Medical Programs
Small Business Assistance Offices
 CDER Small Business and Industry Assistance
 CDERSmallBusiness@fda.hhs.gov
(866) 405-5367 or (301) 796-6707
 CBER Office of Communication, Outreach, and
Development
 Industry.Biologics@fda.hhs.gov
(800) 835-4709 or (301) 827-1800
 CDRH Division of Industry and Consumer
Education
 DICE@fda.hhs.gov
(800) 638-2041 or (301) 796-7100
Center Contact Information
 CBER Contacts
 http://www.fda.gov/AboutFDA/CentersOffices/Officeof
MedicalProductsandTobacco/CBER/ucm123224.htm
 CDER Contacts
 http://www.fda.gov/aboutfda/centersoffices/officeofme
dicalproductsandtobacco/cder/ucm075128.htm
 CDRH Contacts
 http://www.fda.gov/AboutFDA/CentersOffices/Officeof
MedicalProductsandTobacco/CDRH/CDRHOffices/uc
m127854.htm
38
Learning Objectives
 How the FDA is organized
 How drugs, biologics, and medical devices
are regulated
 Where to start
 Where to go for help
 Office of Special Medical Programs
Cross-Center Offices
Office of Combination Products
Office of Good Clinical Practice
Office of Special
Medical Programs
Office of Orphan Products Development
Office of Pediatric Therapeutics
Office of Combination Products
 Guidance Documents
 Early Development Considerations for Innovative
Combination Products (09/2006)
 How to Write a Request for Designation (04/2011)
 Classification of Products as Drugs and Devices
and Additional Product Classification Issues
(Draft - 06/2011)
combination@fda.hhs.gov
301-796-8930
Orphan Product Development Programs
 Designation Programs
 Orphan Drug designation
- including exclusivity
 HUD Designation
 Rare Pediatric Disease Designation
- for Priority Review Voucher Program
 Grants
 Orphan Products Grants Program
 Pediatric Device Consortia Grants Program
 Outreach Program
 Patient-related Topics
Office of Orphan Product Development
 For (non-oncology) Orphan Drug Designations:
Jeff.Fritsch@fda.hhs.gov
 For (oncology) Orphan Drug Designations, Devices, and
Rare Pediatric Disease Designations:
James.Bona@fda.hhs.gov
 For Orphan Grants:
Mary.Limon@fda.hhs.gov
 For Pediatric Device Consortia Grants:
Linda.Ulrich@fda.hhs.gov
(301) 796-8660
Office of Pediatric Therapeutics
 Scientific Activities Program
 Clinical protocol design/conduct/lessons-learned
 Ethics Program
 Necessity of proposed pediatric studies
 Safety Program
 Coordinate Pediatric Advisory Committee
 International Program
 Collaborate with other regulatory agencies
OPT@fda.hhs.gov
301-796-8659
Thank You