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Drug Regulatory Affairs > Advanced Therapy Medicinal products > Flashcards

Advanced Therapy Medicinal products Flashcards

1
Q

what are the charatericts of ATMPs

A

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

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2
Q

What are ATMPs?

A

Medcinies based on gens, tissues or cells

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3
Q

what are the three main types of ATMPs?

A

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

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4
Q

what are combined ATMPs?

A

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

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5
Q

Name the legal framwork for AT?

A

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

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6
Q

what was the aim of the action plan on ATMPs?

A

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

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7
Q

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

A

medician product means a biological

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8
Q

what are the characteristics of the GTMP (A)?

A

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

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9
Q

what is the aim of the GTMP?

A

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

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10
Q

what are the characteristics of the GTMP (b)?

A

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

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11
Q

give example of gene therapy targets

A

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

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12
Q

Definitions of somatic Cell Therapy Medicinal Product (CTMP)

A

it is a biological medicinla product

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13
Q

what are the characteristics of CTMP (a)?

A

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

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14
Q

what are the characteristics of CTMP (b)?

A

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

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15
Q

example of CTMPS

A

chimeric antigen receptor (CAR)-T-Caell therapy

stem cell therapies

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16
Q

Definitions of Tissue Engineered Medicinal product

A

it is a products that consists of engineered cells or tissues

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17
Q

TEP is used for?

A

regenerating, reparing or replacing of human tissue

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18
Q

Difference between GTMP,CTMP and TEP

A

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

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19
Q

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

A

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

20
Q

What are the other responsibility of CAT?

A

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

21
Q

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

A

two assessment teams are appointed

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

22
Q

what is a Hospital exemption?

A

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

23
Q

where is the Hosiptial exemption used?

A

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

24
Q

Where does the repsonsibility lies of the HE?

A

with the Member state concerned

25
Q

Which roll does the CAT plays in HE?

A

they are not involved in the hospital exexmption authorization processes

26
Q

how is the procedures performed in the HE?

A

performed by Paul-Ehrlich Institut

27
Q

does the HE need an authorisation requirement?

A

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

28
Q

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

A

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

29
Q

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

A

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

30
Q

what are the two Earyl development support form the EMA?

A

Scientific advice and protocol assistance

31
Q

The ATMP classification should be used as?

A

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

32
Q

what is the ATMP classification?

A

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

33
Q

what are the early access pathways? (EMA)

A 
Prime for priority medicine
Accelarated assessment
conditional marketing authorization
Authorization under exceptional circumstances
compassionate use
orphan drug designation
34
Q

on what does the RIME bulids?

A

on tools such as scientific advice and accelearted assessment

35
Q

what the aim if the PRIME?

A

to improve clinical trial designs

36
Q

what are some benefits of the PRIME?

A

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

37
Q

what are the PRIME Eligibility?

A

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

38
Q

what are the expedited programs for serious conditions in USA

A

Fast Track Designation
Breakthrough Therapy Designation
Accelerate aprroval
Priority Review Designation

39
Q

Fast Track Designation?

A

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

40
Q

Breakthrough Therapy Designation

A

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

41
Q

Accelerated Approval

A

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

42
Q

Priority Review Designation

A

Serious condition and significant safety/efficacy improvement over available treatments

43
Q

Sakihake designation

A

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

44
Q

Sakihake designation is similar to?

A

in principle to EMA’s PRIME scheme and FDAs BTS

45
Q

Adaptive pathways, the aim is?

A

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

46
Q

what are the object of the -adaptive pathways?

A

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

47
Q

Key features of this adaptive pathways is?

A

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

Drug Regulatory Affairs (15 decks)

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