Cell, gene- and tissue therapy – Samenvatting
From information transfer to: looking for reliable information, validate, analyse, synthetise, transfer
and problem solving with information
Exam questions:
- Imagine this situation, what would you do to solve it? Where does this fit that is applying?
- Which of these products are ATMPs? Why and why not? Which class do they belong to?
Explain?
- Compare and contrast biotechnological medicines with ATMPs
- Indicate the most important thrusts of regulation 1394/2007/EU. What are major changes in
this respect compared to Directive 2001/83/EC?
- Explain the mechanism of action of imlygic
- What criteria are assessed when deciding whether to bring an ATMP to the market? Which
body or bodies are involved in this decision?
- Discuss the role of ATMPs in the treatment of melanoma. Contrast with classical therapies
and biotechnological drugs. Which ethical, regulatory and economic aspects are specifically
related to ATMPs?
- Bonus: Which therapy do you think is the most promising? Motivate your answer
1
, 1. Introduction to advanced therapies, a new class of medicines
EMA
- Centralized registration procedure (Regulation) => Marketing everywhere in the EEA (European
Economic Area = EU countries + Norway and liechtenstein)
- Important drugs: HIV, cancer, diabetes, neurodegenerative disorders, autoimmune and
other immune dysfunctions, viral diseases
- Ophan drugs (rare diseases and in some cases also serious diseases
2 Most important committees within the 7 committees of EMA:
- Biotechnological drugs (CHMP; committee for human medicinal products)
- ATMP (CAT; committee for advanced therapies)
ATMP
= Medicinal products based on manufacturing processes, focussed on various gene transfer-
produced bio-molecules and/or biologically advanced therapeutic modified cells as active substances
or part of active substances.
- Directive 2001/83/EC (National procedure)
- Modules 1 to 5 apply
= Medicinal products for humane use: a gene therapy medicinal product, a somatic cell therapy
medicinal product and a tissue engineered product/tissue manipulated product
- Regulation 1394/2007/EC (Central procedure)
- A stronger definition than the directive
- A tissue engineered product (TEP) = a product that contains or consists of engineered cells
or tissues and is presented as having properties for or is used in or administrered to human
beings with a view to regenerating, repairing or replacing a human tissue.
- A combined advanced therapy medicinal product = an advanced therapy medicinal product
that fulfils the following conditions: it must incorporate, as an integral part of the product, 1
or more medical devices within the meaning of article 1(2)(a) of Directive 93/42/EEC
(National procedure) or 1 or more active implantable medical devices. Its cellular or tissue
part must contain viable cells or tissues or its cellular or tissue part containing non-viable
cells or tissues must be liable to act upon the human body with action that can be
considered as primary to that of the devices referred to.
2
, => GTMP, CTMP and TEP
- Gene therapy product are ATMPs but NOT when it is aimed at vaccines for infectious
diseases. DNA that is manipulated is an ATMP, but when it is only isolated, it is NOT an
ATMP. Cells have to be transduced.
- Tissue preparation = tissue taken and used for transplantation (NOT an ATMP)
- Tissue manipulation product = tissue taken, in culture and add growth factors and
something changes in the cells (ATMP)
European law
3
, ATMP legislation and scope
EMA:
+ EC (ethical committee) + RIZIV + FAGG (for re-funding)
- For the CAT is a regulation 726/2004 applied, unless the regulation is differently founded
(<-> CHMP: human medicinal products -> directive or national procedure)
- De head of the bureau makes sure to coordinate between the CAT and other comittees of
the bureau, such as the CHMP and the COMP, the working groups of the comittees and
scientific advisory groups.
- Working groups of the committees = group that only consist of experts in the field,
coming from the national competent authorities
- Scientific advisory groups = patients and healthcare staff who provide advice on
questions about the correct use of drugs in practice
Function of CAT in ATMP registration
4
From information transfer to: looking for reliable information, validate, analyse, synthetise, transfer
and problem solving with information
Exam questions:
- Imagine this situation, what would you do to solve it? Where does this fit that is applying?
- Which of these products are ATMPs? Why and why not? Which class do they belong to?
Explain?
- Compare and contrast biotechnological medicines with ATMPs
- Indicate the most important thrusts of regulation 1394/2007/EU. What are major changes in
this respect compared to Directive 2001/83/EC?
- Explain the mechanism of action of imlygic
- What criteria are assessed when deciding whether to bring an ATMP to the market? Which
body or bodies are involved in this decision?
- Discuss the role of ATMPs in the treatment of melanoma. Contrast with classical therapies
and biotechnological drugs. Which ethical, regulatory and economic aspects are specifically
related to ATMPs?
- Bonus: Which therapy do you think is the most promising? Motivate your answer
1
, 1. Introduction to advanced therapies, a new class of medicines
EMA
- Centralized registration procedure (Regulation) => Marketing everywhere in the EEA (European
Economic Area = EU countries + Norway and liechtenstein)
- Important drugs: HIV, cancer, diabetes, neurodegenerative disorders, autoimmune and
other immune dysfunctions, viral diseases
- Ophan drugs (rare diseases and in some cases also serious diseases
2 Most important committees within the 7 committees of EMA:
- Biotechnological drugs (CHMP; committee for human medicinal products)
- ATMP (CAT; committee for advanced therapies)
ATMP
= Medicinal products based on manufacturing processes, focussed on various gene transfer-
produced bio-molecules and/or biologically advanced therapeutic modified cells as active substances
or part of active substances.
- Directive 2001/83/EC (National procedure)
- Modules 1 to 5 apply
= Medicinal products for humane use: a gene therapy medicinal product, a somatic cell therapy
medicinal product and a tissue engineered product/tissue manipulated product
- Regulation 1394/2007/EC (Central procedure)
- A stronger definition than the directive
- A tissue engineered product (TEP) = a product that contains or consists of engineered cells
or tissues and is presented as having properties for or is used in or administrered to human
beings with a view to regenerating, repairing or replacing a human tissue.
- A combined advanced therapy medicinal product = an advanced therapy medicinal product
that fulfils the following conditions: it must incorporate, as an integral part of the product, 1
or more medical devices within the meaning of article 1(2)(a) of Directive 93/42/EEC
(National procedure) or 1 or more active implantable medical devices. Its cellular or tissue
part must contain viable cells or tissues or its cellular or tissue part containing non-viable
cells or tissues must be liable to act upon the human body with action that can be
considered as primary to that of the devices referred to.
2
, => GTMP, CTMP and TEP
- Gene therapy product are ATMPs but NOT when it is aimed at vaccines for infectious
diseases. DNA that is manipulated is an ATMP, but when it is only isolated, it is NOT an
ATMP. Cells have to be transduced.
- Tissue preparation = tissue taken and used for transplantation (NOT an ATMP)
- Tissue manipulation product = tissue taken, in culture and add growth factors and
something changes in the cells (ATMP)
European law
3
, ATMP legislation and scope
EMA:
+ EC (ethical committee) + RIZIV + FAGG (for re-funding)
- For the CAT is a regulation 726/2004 applied, unless the regulation is differently founded
(<-> CHMP: human medicinal products -> directive or national procedure)
- De head of the bureau makes sure to coordinate between the CAT and other comittees of
the bureau, such as the CHMP and the COMP, the working groups of the comittees and
scientific advisory groups.
- Working groups of the committees = group that only consist of experts in the field,
coming from the national competent authorities
- Scientific advisory groups = patients and healthcare staff who provide advice on
questions about the correct use of drugs in practice
Function of CAT in ATMP registration
4
