Advanced Therapy Medicinal products

Question 1

what are the charatericts of ATMPs

Answer 1

high researchdriven products
innovative manufacturing processess
Heterogenous regarding their origin, type and complexity
individualized and patient-specific products

Question 2

What are ATMPs?

Answer 2

Medcinies based on gens, tissues or cells

Question 3

what are the three main types of ATMPs?

Answer 3

gene therapy medicines
somatic-cell therapy medcines
tissue-engineered medicines

Question 4

what are combined ATMPs?

Answer 4

ATMPs containing one or more medical devices as an integral part of the medicine

Question 5

Name the legal framwork for AT?

Answer 5

Regulation (EC) No 1394/2007 (CAT)
directive 2001/83/EC
Regulation (EC) No 726/2004
Commission Directive 2009/120/EC

Question 6

what was the aim of the action plan on ATMPs?

Answer 6

streamline procedures and better adress the specific requirements of ATMP developers
procedure of advice on th evaluation of ATMPs

Question 7

What is the definitions of Gene Therapy Medicinal Product (GTMP)

Answer 7

medician product means a biological

Question 8

what are the characteristics of the GTMP (A)?

Answer 8

API which consist of recombinant nucleic acid used in or administered to human beings.

Question 9

what is the aim of the GTMP?

Answer 9

used to regulate, repair, replace, adding or deleting a genetic sequence

Question 10

what are the characteristics of the GTMP (b)?

Answer 10

its therapeutic, prophylactic or diagnostic effects relates directly to the recombinant nucleic acid sequence

Question 11

give example of gene therapy targets

Answer 11

insulin gene in beta cells
sodium channle in lung cells
lipoprotien lipase gene in muscle cells

Question 12

Definitions of somatic Cell Therapy Medicinal Product (CTMP)

Answer 12

it is a biological medicinla product

Question 13

what are the characteristics of CTMP (a)?

Answer 13

consists of cells or tissues, which are substaintial manipulated to achieve certain biological characteristics, physiological functions or structural properties

Question 14

what are the characteristics of CTMP (b)?

Answer 14

it is used to treat, prevent or diagnose a disese through the pharmacological, imumnological or metabolic action

Question 15

example of CTMPS

Answer 15

chimeric antigen receptor (CAR)-T-Caell therapy

stem cell therapies

Question 16

Definitions of Tissue Engineered Medicinal product

Answer 16

it is a products that consists of engineered cells or tissues

Question 17

TEP is used for?

Answer 17

regenerating, reparing or replacing of human tissue

Question 18

Difference between GTMP,CTMP and TEP

Answer 18

GTMP: used to diagonse effect relates directly to the recombinant nucleic acid squence
CTMP: used to treat, prevent or diagonse a disease through the pharmacological, immunological or metabolic action
TEP: view to regenerating, repairing or replacing a human tissue

Question 19

what is the main responsibility of the committee for Advanced Therapies?

Answer 19

To prepared a draft opinion on each ATMP application submitted to EMA

Question 20

What are the other responsibility of CAT?

Answer 20

Certification of quality and non-clinical data (small and medium size company)
providing scientific recommendation, contribute of scientic advide
efficacy follow-up, pharmacovigilance,

Question 21

how does the evalaution of an initial MA works in ATMP?

Answer 21

two assessment teams are appointed

A PRAC Rapportuer from amoungs the members and alternated of the PRAC

Question 22

what is a Hospital exemption?

Answer 22

it is a ATMP prepared on a non-routine basic, according to specific quality standards

Question 23

where is the Hosiptial exemption used?

Answer 23

with the same member state in a hospital under the exclusive professional, responsibility of a medical practitioner

Question 24

Where does the repsonsibility lies of the HE?

Answer 24

with the Member state concerned

Question 25

Which roll does the CAT plays in HE?

Answer 25

they are not involved in the hospital exexmption authorization processes

Question 26

how is the procedures performed in the HE?

Answer 26

performed by Paul-Ehrlich Institut

Question 27

does the HE need an authorisation requirement?

Answer 27

According to section 4b (1) of the germany medicines Act no authorisation requirement is necessary, but a manufacturing authorisation in accordance with section 13 of the AG

Question 28

What happens when an ATMP, which was previously manufactured as a “Hospital Exemption”, receives a central authorisation for the same indication from the EMA?

Answer 28

This means, the HE has to be manfutured based on a larger slace, to increase the product accessability.

Question 29

What do you think about the reimbursement of a Hospital Exemption?

Answer 29

the reimbursement of the HE will be expainsive and quite complicated, since the manufacure of the HE is based on a small scale

Question 30

what are the two Earyl development support form the EMA?

Answer 30

Scientific advice and protocol assistance

Question 31

The ATMP classification should be used as?

Answer 31

first opportunity to engage with regulators, which can follow by scientific advice and ATMP certification (mall and medium enterprises)

Question 32

what is the ATMP classification?

Answer 32

used to classified the ATMP simple and fast procedures legally non-binding procedures optional and free of charg

Question 33

what are the early access pathways? (EMA)

Answer 33

``` Prime for priority medicine Accelarated assessment conditional marketing authorization Authorization under exceptional circumstances compassionate use orphan drug designation ```

Question 34

on what does the RIME bulids?

Answer 34

on tools such as scientific advice and accelearted assessment

Question 35

what the aim if the PRIME?

Answer 35

to improve clinical trial designs

Question 36

what are some benefits of the PRIME?

Answer 36

Earyl CHMP Rapporteur appointment EMA provides scientific advice at key development milestones confirm potential for accelerated assessment

Question 37

what are the PRIME Eligibility?

Answer 37

it is address an unmet medical need preliminary data must be avaiable to show the potential to address this need and bring a major therapeutic advantage to patients

Question 38

what are the expedited programs for serious conditions in USA

Answer 38

Fast Track Designation Breakthrough Therapy Designation Accelerate aprroval Priority Review Designation

Question 39

Fast Track Designation?

Answer 39

nonclinical or clinical data to demonstrate potential to adress unmet medical need or drug designated as a qualified infectious diease product

Question 40

Breakthrough Therapy Designation

Answer 40

Serious condition + preliminary clinical evidence of substantial improvement over available therapies, simila to EMA PRIME designation

Question 41

Accelerated Approval

Answer 41

Serious condition + meaningful advantge + surrogate endpont predictive of clinical benefit or endpoint that can be measured before morbidity/mortality (similar CMA)

Question 42

Priority Review Designation

Answer 42

Serious condition and significant safety/efficacy improvement over available treatments

Question 43

Sakihake designation

Answer 43

medicinal products comprising human cells, genes or tissues that will be marketed are regulated generically as regenerative medical products (RMP)

Question 44

Sakihake designation is similar to?

Answer 44

in principle to EMA's PRIME scheme and FDAs BTS

Question 45

Adaptive pathways, the aim is?

Answer 45

aim is to balance the need for timely patient access to promising medicines with the need to collect information of safety and efficacy of the medicines

Question 46

what are the object of the -adaptive pathways?

Answer 46

MP with high medical needs but difficulties in generating evidence and conducting large clinical trials

Question 47

Key features of this adaptive pathways is?

Answer 47

Gathering evidence through real-world data and early multi-stakeholder dialogue