Beruflich Dokumente
Kultur Dokumente
Agenda
Regulatory affairs(RA)
IND, NDA
RA
Case study
A discipline that focuses on the analysis and application of regulations in relation to the
development, government approval and marketing of healthcare products.
Science of developing new tools, standards and approaches to assess the safety, efficacy,
quality and performance of FDA-regulated products.
Agenda
Regulatory affairs(RA)
IND, NDA
RA
Case study
Diphtheria antitoxin from milk wagon horse tragedy in 1901 => 13 children died
First control over the manufacturing processes of biological products
Mandated that producers of vaccines be licensed annually
Inspections for the manufacturing facilities
All product labels were required to include the product name, expiration date, and
address and license number of the manufacture
http://www.fda.gov/AboutFDA/WhatWeDo/History/ProductRegulation/100YearsofB
iologicsRegulation/ucm070022.htm
http://www.fda.gov/AboutFDA/WhatWeDo/History/Origin/ucm054819.htm
http://www.fda.gov/AboutFDA/WhatWeDo/History/ProductRegulation/SulfanilamideDisaster/default.htm
FD&C ACT
First time manufacturers were required to prove effectiveness and safety of drug before
marketing
Required to give participants full information about the benefits and risks of drugs being
studied
Set good manufacturing practices to be followed by the drug industry
Also required that drugs introduced between 1938 and 1962 be effective - nearly 40 %
not effective
http://www.fda.gov/AboutFDA/WhatWeDo/History/ThisWeek/ucm117831.htm
Median Approval Times, New Molecular Entities (NMEs) & New Biologic
Entities (NBEs), by Fiscal Year of Receipt
Applications
requiring clinical data: $2,169,100
not requiring clinical data: $1,084,550
Supplements requiring clinical data: $1,084,550
Establishments: $554,600
Products: $104,060
Relevant Laws
Relevant Regulations
Other Guidelines
ICH: International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use
GCP: Good Clinical Practice, GMP: Good Manufacturing Practice, GLP: Good Laboratory Practice,
GCLP: Good Clnical Laboratory Practice, GVP: Good Pharmacovigilance Practice,
GCDMP: Good Clinical Data Management Practice
WMA: World Medical Association, WHO: World Health Organization
Regulations
21 CFR 11
21 CFR 50
21 CFR 54
21 CFR 56
21 CFR 312
Subpart I
21 CFR 314
Subpart C
Abbreviated application
Subpart H
Accelerated approval
21 CFR 601
Subpart E
Regulations
21 CFR 316
Orphan drugs
21 CFR 320
21 CFR 330
21 CFR 812
21 CFR 814
21 CFR 60
21 CFR 201
Labeling
21 CFR 202
CDER: IND & NDA for new drugs, orphan drugs, & OTC human drugs, ANDA
for generic drugs
CBER: BLA for biologics
CDRH: IDE & PMA for medical devices
Combination Products
Agenda
Regulatory affairs(RA)
IND, NDA
RA
Case study
In Terms of Process
Pre-clinical Studies
IND
Approval
Phase I ~ Phase III Clinical Studies
NDA
Pre-IND Meeting
: 21 CFR 312.82
1
(: , IND data )
Pre-IND(PIND) file PIND .
( FDA meeting )
, , , ( ), ,
(type A, B, C), ( )
CMC, /,
, FDA
(6-8 )
( 4 )
Pre-IND Meeting
Pre-IND meeting
Novel indication
No current Guidance Documents
Unique molecular entity, studies or indications
New sponsors or new to area of drug development
Problematic Pharm/Tox signals
New molecular entity
Avoid protocol changes
CMC,
, GCP
IND
(2, 3)
IND Dossier
Cover sheet
Table of contents
Investigational plan
Investigators brochure
Protocol
Chemistry, manufacturing, and controls information
Pharmacology and toxicology information
Previous human experiences with the investigational drug
Additional information
Relevant information
: 21 CFR 312.82
accelerate approval
2
,
: 21 CFR 312.47
3 , , ,
PK
, , ,
CMC
1 2
3
Pre-NDA Meeting
: 21 CFR 312.47
NDA dossier (, )
FDA
/
Delayed submission (original NDA 30 ) : stability data, final
audited reports( )
NDA
? ?
labeling ?
, /, , ?
NDA Dossier
Module
Module
Module
Module
Module
1
2
3
4
5
Day 0
Day 45
Filing Period
Filing meeting
Day 74 letter
Filing letter
Internal mid-cycle
review meeting
Advisory
committee
meeting
NDA approval
Day 60
Resubmission timeline
Class 1 resubmission: 2
Class 2 resubmission: 6
Launch
Fast Track
Breakthrough Therapy
Drugs to treat a serious condition and preliminary clinical evidence indicates that the
drug may demonstrate substantial improvement over available therapy on a clinically
significant endpoint(s).
Intermediate clinical endpoint
Measure of a therapeutic effect that is considered reasonably likely to predict the
clinical benefit of a drug (effect on irreversible morbidity and mortality)
EOP2 meeting
Accelerated Approval
Priority Review
Agenda
Regulatory affairs(RA)
IND, NDA
RA
Case study
LAW
34( )
Vulnerable subjects
GMP
31( )
32( )
33( )
34( )
(IND)
(KGCP)
Enforcement
Guideline
Agenda
Regulatory affairs(RA)
IND, NDA
RA
Case study
Qualification of RA Professionals
RA
, ,
Good writing skills!
Qualification of RA Professionals