Transcript Slide 1

GOOD MORNING!
BONJOUR!
GOEDEMORGEN!
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An Introduction to certain
aspects of the US FDA
Murray M. Lumpkin, MD
Deputy Commissioner
International Programs
US Food and Drug Administration
Belgian Trade Delegation
Washington
28 June 2011
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http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm259848.htm
http://www.ema.europa.eu
/docs/en_GB/document_lib
rary/Report/2011/06/WC50
0107900.pdf
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FDA – Belgium - Europe
Long history of interactions
Confidentiality arrangements with:
Belgian Scientific Institute of Public Health
Belgian Federal Agency for Medicines and Health
Products
Quality of Medicines & HealthCare (EDQM)
Health and Consumer Protection Directorate-General
(DG SANCO)
EMA
EFSA
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Confidentiality Arrangements
Legal Framework to share:
Commercial confidential information
Pre-decisional information
Investigative – compliance information
Pharmacovigilance data, reports
NOT Trade Secret information
NO requirement to exchange anything
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FDA = Federal Drug Administration
Food and Drug Administration
Part of Department (Ministry) of Health and Human
Services
Secretary (Minister) part of President’s cabinet
(Kathleen Sibelius)
Commissioner of Food and Drugs – (Dr Margaret
Hamburg) appointed by President, confirmed by Senate,
responsible to Secretary – politicals / career
CDC, NIH, others also part of DHHS (FDA is the only
regulatory body in DHHS)
Our secretary & commissioner are not part of our
Congress (not a parliamentary system)
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FDA - Components
~12,000 employees (~7500 in Washington)
Office of the Commissioner (OC)
Office of Regulatory Affairs (ORA)
(inspectorate/enforcement)
Center for Devices and Radiologic Health (CDRH)
Center for Veterinary Medicine (CVM)
Center for Food Safety and Applied Nutrition (CFSAN)
National Center for Toxicologic Research (NCTR)
Center for Tobacco Products
Center for Biologics Evaluation and Research (CBER)
Center for Drug Evaluation and Research (CDER)
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Two medicines laws
U.S. Public Health Service Act (1902)
Most biologically derived human medicines
Biosimilars (2010)
Federal Food, Drug, and Cosmetic Act (1938)
Most chemically derived human and animal medicines
Generic Drugs (1984)
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Animal
Testing
Phase 1
Phase 2
Phase 3
Short-term
Long-term
Pre-clinical
Research
Post-authorisation
Synthesis
and
Purification
MAA Review
Discovery / Screening
NEW MEDICINE
DEVELOPMENT
IND
AP
Clinical Studies
MA
“NDA”
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IND PROCESS
(IND = Investigational New Drug)
Submit IND application before 1st clinical study in
the US (no matter what “phase” the first study is)
Regulatory vehicle under which all investigational trials are
overseen in the USA
All animal data, previous clinical data outside USA (if
any), development plan, and first protocol with special
emphasis on any safety concerns and manufacturing
process for clinical trials supplies
Make case for why it is reasonable to proceed into humans
at this point and why the plan for safety monitoring is
adequate
Include IRB (ethics committee) oversight information
(name)
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IND PROCESS
After 1st submission – must wait 30 calendar
days.
If nothing heard from FDA, may proceed on day 31.
Do not need to wait for authorising letter. In fact,
there is no “authorising letter”
Subsequent submissions are all “notifications”.
Do not need to wait to start trial, simply notify.
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IND PROCESS
FDA can place any trial (or part of a trial) on “hold”
at any time
“Hold” means trial may not proceed and, if started, no
further patients may be enrolled
If application not complete
If FDA does not believe it is reasonably safe to proceed
If FDA does not believe company is properly monitoring
for potential safety problems
If FDA believes trial design puts patients at risk for no
scientific purpose (vs not going to answer the question the
company wants to have answered)
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WHY DO WE DO THIS?
Promote and Protect Public health –
clinical trials are how we initially learn
about the safety and efficacy of new
products
Need independent oversight to help assure
that subjects are not put at unreasonable
risk and not put into trials from which we
cannot reasonably expect to learn
something scientifically relevant
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WHY DO COMPANIES
ENGAGE WITH US FDA
DURING DEVELOPMENT
Submissions are required
Essentially all engage much more than
minimally required
No direct cost for such meetings
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FDA / SPONSORS
DURING DRUG DEVELOPMENT
Interact with sponsors to maximise
efficiency and scientific robustness of their
drug development program
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VERY IMPORTANT:
PRE-IND / END OF PHASE 2
Reach Understanding of Development Goals and
Implementation
Trial designs / Where to do them
Evaluability Criteria
Define a “Win”
Characterise Product
Indications, Dosing Regimen(s)
Major Safety Parameters
Manufacturing Issues
Possible modifications with time
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SPECIAL PROTOCOLS
Carcinogenicity
Stability
P3 trials that will be primary data for an
efficacy claim
Performance goal: 45 days
Written agreements binding except when
science changes
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When can a product be authorised?
When the data show that the demonstrated
benefits outweigh the known risks for the
intended population when used as directed
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EARLY ACCESS INITIATIVES
Phase 1
Subpart E:
Phase 2
Submit MA here
Phase 3
Short-term
Long-term
IND subm
AP
Fast TrackRolling Review
Post-authorisation
Synthesis
and
Purification
Animal
Testing
Accelerated
Approval:
Clinical Studies
MAA Review
Discovery / Screening
Pre-clinical
Research
Priority Review
Treatment IND
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US - EU COMPARISON
(Nomenclature)
United States
Priority Review
European Union
Fast Track
Accelerated Approval
(Subpart H)
Conditional Approval
Subpart E
Approval under
special circumstances
Fast Track
No equivalent
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Thank You / Merci / Dank U
Questions?
Comments?
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