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Drug Regulatory Affairs > Seconde Exercise > Flashcards

Seconde Exercise Flashcards

1
Q

How are Gene therapy Medicinal products defined (GTMPs)?

A

they are subgroup of the advanced therapy medicinal products (ATMPs)

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2
Q

what doe GTMPs contain?

A

an active substance which contains or consists or recombinant nucleic acid (RNA or DNA)

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3
Q

what doesn’t include to the GTMPs?

A

Vaccines against infectious deiseases

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4
Q

Which regulation is sued to adressed the ATMPs?

A

(EC) no 1394/2007 and directive 2001/83/EC and (EC No 726/2004

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5
Q

Which committee deals with the ATMPs?

A

Commitee for Advanced Therapies (CAP)

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6
Q

what does the CAT does?

A

Evaluation, certification and classification of the ATMPs.

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7
Q

the certification process is mained for who?

A

Small and Medium Enterprises

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8
Q

How long is the certification process?

A

90 day

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9
Q

How long is the classification process?

A

60-day with no fee

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10
Q

which other committee is part of the assessment procedure of the ATMPs?

A

Commitee for Medicnal products for Human Use

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11
Q

Which marketing approval is used for gene modifying treatments?

A

CP

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12
Q

Which bodies are regulated the GMT in EU, USA and JP

A

USA: Center for Biologics Evaluation and Research
JP: Ministry of Health, Labour and Welfare (MHLW)
EU: EMA

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13
Q

What does the applicant has to submitted to the CBER?

A

a biologic license application (BLA)

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14
Q

What does the BLA inlcudes?

A

applicant information, product and manufacturing information, pre-clinical studies, clinical studies and labeling

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15
Q

under what does the GTP falls in JP?

A

category of regenerative medincine

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16
Q

what are the three acts of major importance to GTPs in JP?

A

The regenerative Medicine promotion Law
Act of safety of Regenrative promotion Law
Act on pharmaceutical and medical devices

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17
Q

Act on pharmaceutical and medicinal devices is?

A

separate pathway for drug approval for regenrative medicines

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18
Q

what differentiates the GTP of JP from the USA and EU regulation?

A

the GTP in Japan is allow for a conditional marketing approval after phase 2 clinical studies

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19
Q

when is the renewal of the MA for GTP in japan?

A

with 7 years, reanalyzed and granted final market approval

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20
Q

Classification of the gene therapies in EU, USA and JP

A

EU: ATMPs (GTMs, CTM, TEM)
UAs: bilogical products
Japan: Regenerative medicine

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21
Q

Arketing approval in EU, UAS and JP?

A

Centralzed marketing approval

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22
Q

Possibility of special marketing approval/authorization (EU, USA and JP?

A

EU: conditional market authorization
USa: accelerated approval
Japan: Conditional marketing approval

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23
Q

Regulations/Directives in UAS?

A

Section 357 public health service act

24
Q

CRISPR stands fo?

A

Clustered regulary interspaced short palindromic repeats

25
Q

what is the CRISPR /Cas9 system?

A

is a promising technology to insert, delect or replace DNA in an organism’s genome

26
Q

How are CRISPR/Cas9 defined?

A

gene therapy medicinla products or somatic cell therapy medicinal products (sCTMPs)

27
Q

what are mandatory for ATMPs?

A

clinical trials

28
Q

what is forbitten in the CT in ATMPs?

A

gene therapy trials resulting in modifications to the patient’s germ-line genetoc identity

29
Q

Give an example for ATMPs product?

A

ZYnteglo

30
Q

what procedures are used for suport and evaluation of MA procedures?

A

Accelerated assessment
PrIME: priority medicines
Compassionate use
emergcy approval, rolling review procedure

31
Q

Accelerated assessment?

A

used to reduced time needed for the review of a request for MA by EMA

32
Q

aim of AA?

A

early access of patient to important and innovative medicine

33
Q

how is the approach fostered AA?

A

promoting earyl dialogue between applicant and EMA, providing enhance guidance in the justification of a request of the AA,
providing more transparency in the handling of the requests

34
Q

what are the eligibility of AA?

A

unmet medical need
extent to which the MP is expected to fulfill the unmet medical need
the stength of evidence to sppourt justifying major interest from the point of viw of public health

35
Q

when does the pre-submission meeting takes place?

A

before MA-

36
Q

What happend after eligibility is granted?

A

90-30-30 day’s asessemnt procedures begins

37
Q

what is the 90-30-30 procedures?

A

1-90 first assessment by rapporteurs (CHMP) and PRAC peer-review
90 day: CHMP meeting takes place
120, CHMP gives it’s opinion

38
Q

PRIME stands for?

A

Priority Medicine

39
Q

PRIME is used for?

A

support the development of medicines that target an unmet medical need

40
Q

what is the main difference between the AA and PRIME?

A

AA application is only possible months prior the MA, while PRIME ia an optimization of the process with early interaction with the refulators together.

PRIME there is an appointment of a contact person in the EMA

41
Q

what does the AA and PRIME has in common?

A

same eligibility

products which are approved for PRIME are autiomaticall eligible for AA

42
Q

which proof does the applicant has to show for PRIME?

A

proof of concept and proof of principle

43
Q

proof of concept is for?

A

all applicant advances in clinical trial and is a proof of an exploratory trial

44
Q

proof of principle is for?

A

applicant such as SME and Academia, maily about non-clinical studies

45
Q

how long does the eligibility takes places?

A

up to 40 days , major actor are the SAWP and CHMP

46
Q

what has to be done, if the prime is granted?

A

a reafirmation of intent to request for a AA must be done

47
Q

PRIME is?

A

a scheme that facilitate the use of other early access tools auch as AA

48
Q

Compassionate use is?

A

permition of unauthorized medicines to become available to groups of patients or cohort under very specific and strict conditions

49
Q

Compassionate use is apply for

A

life-threatning, long-lasting, or seriously debilitating illnesses

50
Q

the use of compassionate use is initiated by?

A

national competent authority

51
Q

What is the implementation routes?

A

remains that of the competence of the MS

52
Q

which authorities managed the compassionate use?

A

BfArM and PEI

53
Q

eligibility for compassionate use are

A

each MS through its fedral authority define specific basic prerequisites

54
Q

the Rolling Review is?

A

use during a public health emergency to speed up the assessment

55
Q

RR?

A

a continues submission of data and immediate assesmment of the data by the EMA still the data is complete for a positive benefits risk (COVID-19)

56
Q

Emergency approval

A

use of the rolling review

57
Q

Vaccines authorides using amergency approval are given?

A

a conditional approval, basis of less comprehensive data

Drug Regulatory Affairs (15 decks)

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