REGULATORY AFFAIRS
ADI Acceptable daily intake
NOAEL & LOAEL & safety factor
ADR Adverse drug reactions
Response to a medicinal product which is noxious and unintended
aGMP Appropriate GMP
AI Artificial intelligence
AMP Auxiliary medicinal product
A licensed medicine used in an interaction study as CYP3A4 substrate, not
expected to be prescribed with the IMP
non-AMP: food products, eg alcohol, used in an interaction study
ANDA Abbreviated new drug application
Data approved of generic by FDA
API Active pharmaceutical ingredient
Submit information to authorities:
Ph.Eur => CEP (MAH)
ASMF (EDMF) open & closed
All in CTD
aQbD Quality by design
ENHANCED METHOD traditional (OFAT)
CQA (ATP)
DOE
Spaces: NOR/MOR, design space (PAR, MODR), EoF, total space =
experimental domain + unexplored space, multiple factors at a time
Control strategy (PAT, SST, parametric release), product-process-analytical
understanding, regulatory flexibility
ASMF Active substance master file
CTD module 3: 3.2.S
OPEN: send directly to authorities, just enough to continue development (no
confidential information, known by the applicant)
CLOSED (much more in detail)
ATIMP Advanced therapeutic investigational medicinal products
ATMP Advanced therapeutic medicinal products
1394/2007
-gene therapy MP
-somatic cell therapy MP
-Tissue engineered MP
APPLICATION:
Registered: MAA
Clinical trials: CAT, ATIMP
Hospital exemption: non-routine, quality standards, use within same MS,
use in hospital, excl responsibility medical practitioner, individual medical
prescription for individual patient
ATP Analytical target profile
BI Budget impact => affordability
Level 1: aantal boxes * prijs per box
Level 2: level 1 – other expenses
Level 3: cost on the HCB
BMWP Biosimilar medicinal product working party (temporary WP)
BTC Blood tissue and cell (legislation)
,CA Competent authorities
CAM Complementary & alternative medicines
CAM Chicken chorioallantoic membrane
CAP Common agriculture policy
Capability Meeting of the specification limits
Cp index=tolerance/6s
Tolerance= USL-LSL
Zusl = (USL-u)/s
Zlsl = (u-LSL)/s
Cpk= Zmin/3
Cpk & Cp > 1.67 (min 1.33)
CAT Committee for advanced therapies
Application & evaluation
Voor aanvraag ATMP statuut
CBMP Cell based medicinal product => SCTP & TEP
Cosmetic Op de markt door qualified persoon
Cosmetic regulation
ANNEX I
Ensure compliance with regulation
Safety
Compounds!!!
Cosmetic claims
Annex I
-legal compliance
-truthfulness
-evidence support
-Honesty
-Fairness
-informed decision making
CDG Commissie terugbetaling geneesmiddelen
CEA Cost-effectiveness acceptability
CEO Chief executive officer
CEP Certificate of suitability
Issues by the EDQM
Wilt zeggen: quality is in compliance met Ph. Eur
CEP Cost-effectiveness plane
CESP Common European Submission Portal: system providing mechanism for exchange
of information between applicants and national regulatory agencies
cGMP Current GMP
CHMP Committee for medicinal products for human use
CP!!
CJEU Court of justice of the EU
Overrules nationals
CMC Chemistry manufacturing controls: technical
CMDh Coordination group of mutual recognition and decentralised procedure for human
medicinal products
CMS Concerned member state (DCP)
COMP Committee for orphan medicinal products
COP27 Agreement goal of limiting temperature rise to 1.5 degrees Celsius by the end of
this century
Concrete actions
, Accountability & transparency
Workstreams of mitigation, adaption, finance
Council of EU Government ministers
Presidency changes every 6 months
Topic-specific
CP Centralized procedure
1 marketing authorisation in EU
1 name
1 common product information
Mandatory for:
ATMP
Orphan (<5/10 000)
Certain therapeutic classes: aids, cancer, diabetes, viral diseases,
neurodegenerative
Bio-technological
TIMETABLE
*D-14: validation (logicstics)
*D0: start CP
*until day 120: assessment phase 1, LoQ
*D120: clock stop
*D121: RtQ
*D120-210: assement phase 2
*D180: LOI & clock stop
*D210: CHMP opinion (majority) => linguistic process
*D277: single European market authorisation => UITGEREIKT DOOR EC
CPNP Cosmetic products notification portal
CQA Critical quality attributes
CRM Commission reimbursement medicines
CRM Customer relationship management
CTA Clinical trial application
CTD Common technical dossier
Format bepaald door ICH (in EU, Japan, US)
Set of specifications for the application dossier for the registration of medicine
NOT about the content, where + how presentation
M1: general information (not really part of the CTD)
1.0 Cover letter
1.1 TOC
1.2 Application: legal basis (full/abridged)
8(3) full application
10.1 generic, 10.3 hybrid, 10.4 biosimilar
10a well established, 10b new fixed combination, 10c informed
consent
16a traditional herbals
1.3 Product information
SmPC
1.4 Three experts
1.5 Specific requirements different applications (conditional/exceptional)
1.6 Environmental risk assessment
WWTP, RQ, COP27, EUROPEAN GREEN DEAL
1.7 Orphan market exclusivity
ADI Acceptable daily intake
NOAEL & LOAEL & safety factor
ADR Adverse drug reactions
Response to a medicinal product which is noxious and unintended
aGMP Appropriate GMP
AI Artificial intelligence
AMP Auxiliary medicinal product
A licensed medicine used in an interaction study as CYP3A4 substrate, not
expected to be prescribed with the IMP
non-AMP: food products, eg alcohol, used in an interaction study
ANDA Abbreviated new drug application
Data approved of generic by FDA
API Active pharmaceutical ingredient
Submit information to authorities:
Ph.Eur => CEP (MAH)
ASMF (EDMF) open & closed
All in CTD
aQbD Quality by design
ENHANCED METHOD traditional (OFAT)
CQA (ATP)
DOE
Spaces: NOR/MOR, design space (PAR, MODR), EoF, total space =
experimental domain + unexplored space, multiple factors at a time
Control strategy (PAT, SST, parametric release), product-process-analytical
understanding, regulatory flexibility
ASMF Active substance master file
CTD module 3: 3.2.S
OPEN: send directly to authorities, just enough to continue development (no
confidential information, known by the applicant)
CLOSED (much more in detail)
ATIMP Advanced therapeutic investigational medicinal products
ATMP Advanced therapeutic medicinal products
1394/2007
-gene therapy MP
-somatic cell therapy MP
-Tissue engineered MP
APPLICATION:
Registered: MAA
Clinical trials: CAT, ATIMP
Hospital exemption: non-routine, quality standards, use within same MS,
use in hospital, excl responsibility medical practitioner, individual medical
prescription for individual patient
ATP Analytical target profile
BI Budget impact => affordability
Level 1: aantal boxes * prijs per box
Level 2: level 1 – other expenses
Level 3: cost on the HCB
BMWP Biosimilar medicinal product working party (temporary WP)
BTC Blood tissue and cell (legislation)
,CA Competent authorities
CAM Complementary & alternative medicines
CAM Chicken chorioallantoic membrane
CAP Common agriculture policy
Capability Meeting of the specification limits
Cp index=tolerance/6s
Tolerance= USL-LSL
Zusl = (USL-u)/s
Zlsl = (u-LSL)/s
Cpk= Zmin/3
Cpk & Cp > 1.67 (min 1.33)
CAT Committee for advanced therapies
Application & evaluation
Voor aanvraag ATMP statuut
CBMP Cell based medicinal product => SCTP & TEP
Cosmetic Op de markt door qualified persoon
Cosmetic regulation
ANNEX I
Ensure compliance with regulation
Safety
Compounds!!!
Cosmetic claims
Annex I
-legal compliance
-truthfulness
-evidence support
-Honesty
-Fairness
-informed decision making
CDG Commissie terugbetaling geneesmiddelen
CEA Cost-effectiveness acceptability
CEO Chief executive officer
CEP Certificate of suitability
Issues by the EDQM
Wilt zeggen: quality is in compliance met Ph. Eur
CEP Cost-effectiveness plane
CESP Common European Submission Portal: system providing mechanism for exchange
of information between applicants and national regulatory agencies
cGMP Current GMP
CHMP Committee for medicinal products for human use
CP!!
CJEU Court of justice of the EU
Overrules nationals
CMC Chemistry manufacturing controls: technical
CMDh Coordination group of mutual recognition and decentralised procedure for human
medicinal products
CMS Concerned member state (DCP)
COMP Committee for orphan medicinal products
COP27 Agreement goal of limiting temperature rise to 1.5 degrees Celsius by the end of
this century
Concrete actions
, Accountability & transparency
Workstreams of mitigation, adaption, finance
Council of EU Government ministers
Presidency changes every 6 months
Topic-specific
CP Centralized procedure
1 marketing authorisation in EU
1 name
1 common product information
Mandatory for:
ATMP
Orphan (<5/10 000)
Certain therapeutic classes: aids, cancer, diabetes, viral diseases,
neurodegenerative
Bio-technological
TIMETABLE
*D-14: validation (logicstics)
*D0: start CP
*until day 120: assessment phase 1, LoQ
*D120: clock stop
*D121: RtQ
*D120-210: assement phase 2
*D180: LOI & clock stop
*D210: CHMP opinion (majority) => linguistic process
*D277: single European market authorisation => UITGEREIKT DOOR EC
CPNP Cosmetic products notification portal
CQA Critical quality attributes
CRM Commission reimbursement medicines
CRM Customer relationship management
CTA Clinical trial application
CTD Common technical dossier
Format bepaald door ICH (in EU, Japan, US)
Set of specifications for the application dossier for the registration of medicine
NOT about the content, where + how presentation
M1: general information (not really part of the CTD)
1.0 Cover letter
1.1 TOC
1.2 Application: legal basis (full/abridged)
8(3) full application
10.1 generic, 10.3 hybrid, 10.4 biosimilar
10a well established, 10b new fixed combination, 10c informed
consent
16a traditional herbals
1.3 Product information
SmPC
1.4 Three experts
1.5 Specific requirements different applications (conditional/exceptional)
1.6 Environmental risk assessment
WWTP, RQ, COP27, EUROPEAN GREEN DEAL
1.7 Orphan market exclusivity
