Advanced Therapy Medicinal products Flashcards
what are the charatericts of ATMPs
high researchdriven products
innovative manufacturing processess
Heterogenous regarding their origin, type and complexity
individualized and patient-specific products
What are ATMPs?
Medcinies based on gens, tissues or cells
what are the three main types of ATMPs?
gene therapy medicines
somatic-cell therapy medcines
tissue-engineered medicines
what are combined ATMPs?
ATMPs containing one or more medical devices as an integral part of the medicine
Name the legal framwork for AT?
Regulation (EC) No 1394/2007 (CAT)
directive 2001/83/EC
Regulation (EC) No 726/2004
Commission Directive 2009/120/EC
what was the aim of the action plan on ATMPs?
streamline procedures and better adress the specific requirements of ATMP developers
procedure of advice on th evaluation of ATMPs
What is the definitions of Gene Therapy Medicinal Product (GTMP)
medician product means a biological
what are the characteristics of the GTMP (A)?
API which consist of recombinant nucleic acid used in or administered to human beings.
what is the aim of the GTMP?
used to regulate, repair, replace, adding or deleting a genetic sequence
what are the characteristics of the GTMP (b)?
its therapeutic, prophylactic or diagnostic effects relates directly to the recombinant nucleic acid sequence
give example of gene therapy targets
insulin gene in beta cells
sodium channle in lung cells
lipoprotien lipase gene in muscle cells
Definitions of somatic Cell Therapy Medicinal Product (CTMP)
it is a biological medicinla product
what are the characteristics of CTMP (a)?
consists of cells or tissues, which are substaintial manipulated to achieve certain biological characteristics, physiological functions or structural properties
what are the characteristics of CTMP (b)?
it is used to treat, prevent or diagnose a disese through the pharmacological, imumnological or metabolic action
example of CTMPS
chimeric antigen receptor (CAR)-T-Caell therapy
stem cell therapies
Definitions of Tissue Engineered Medicinal product
it is a products that consists of engineered cells or tissues
TEP is used for?
regenerating, reparing or replacing of human tissue
Difference between GTMP,CTMP and TEP
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
what is the main responsibility of the committee for Advanced Therapies?
To prepared a draft opinion on each ATMP application submitted to EMA
What are the other responsibility of CAT?
Certification of quality and non-clinical data (small and medium size company)
providing scientific recommendation, contribute of scientic advide
efficacy follow-up, pharmacovigilance,
how does the evalaution of an initial MA works in ATMP?
two assessment teams are appointed
A PRAC Rapportuer from amoungs the members and alternated of the PRAC
what is a Hospital exemption?
it is a ATMP prepared on a non-routine basic, according to specific quality standards
where is the Hosiptial exemption used?
with the same member state in a hospital under the exclusive professional, responsibility of a medical practitioner
Where does the repsonsibility lies of the HE?
with the Member state concerned
Which roll does the CAT plays in HE?
they are not involved in the hospital exexmption authorization processes
how is the procedures performed in the HE?
performed by Paul-Ehrlich Institut
does the HE need an authorisation requirement?
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
What happens when an ATMP, which was previously manufactured as a “Hospital
Exemption”, receives a central authorisation for the same indication from the EMA?
This means, the HE has to be manfutured based on a larger slace, to increase the product accessability.
What do you think about the reimbursement of a Hospital Exemption?
the reimbursement of the HE will be expainsive and quite complicated, since the manufacure of the HE is based on a small scale
what are the two Earyl development support form the EMA?
Scientific advice and protocol assistance
The ATMP classification should be used as?
first opportunity to engage with regulators, which can follow by scientific advice and ATMP certification (mall and medium enterprises)
what is the ATMP classification?
used to classified the ATMP
simple and fast procedures
legally non-binding procedures
optional and free of charg
what are the early access pathways? (EMA)
Prime for priority medicine Accelarated assessment conditional marketing authorization Authorization under exceptional circumstances compassionate use orphan drug designation
on what does the RIME bulids?
on tools such as scientific advice and accelearted assessment
what the aim if the PRIME?
to improve clinical trial designs
what are some benefits of the PRIME?
Earyl CHMP Rapporteur appointment
EMA provides scientific advice at key development milestones
confirm potential for accelerated assessment
what are the PRIME Eligibility?
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
what are the expedited programs for serious conditions in USA
Fast Track Designation
Breakthrough Therapy Designation
Accelerate aprroval
Priority Review Designation
Fast Track Designation?
nonclinical or clinical data to demonstrate potential to adress unmet medical need or drug designated as a qualified infectious diease product
Breakthrough Therapy Designation
Serious condition + preliminary clinical evidence of substantial improvement over available therapies, simila to EMA PRIME designation
Accelerated Approval
Serious condition + meaningful advantge + surrogate endpont predictive of clinical benefit or endpoint that can be measured before morbidity/mortality (similar CMA)
Priority Review Designation
Serious condition and significant safety/efficacy improvement over available treatments
Sakihake designation
medicinal products comprising human cells, genes or tissues that will be marketed are regulated generically as regenerative medical products (RMP)
Sakihake designation is similar to?
in principle to EMA’s PRIME scheme and FDAs BTS
Adaptive pathways, the aim is?
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
what are the object of the -adaptive pathways?
MP with high medical needs but difficulties in generating evidence and conducting large clinical trials
Key features of this adaptive pathways is?
Gathering evidence through real-world data and early multi-stakeholder dialogue
