Regulatory Sciences in Biomedicine
Lecture 1 Introduction
Stakeholders in the biomedical domain
The biomedical ecosystem = the trajectory from the lab to the
clinic.
Stakeholders in the biomedical domain:
- Healthcare professionals
- Patients
- Researchers
- Industry
- Regulatory authorities
Researchers/HCP’s and scientific organisations
International Organizations
- World Health Organization (WHO) – Guidelines and policies for global health
- International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) –
Harmonization of pharmaceutical research and registration
o ICH à organization that set the standards for safety and efficacy, so that everyone in the world uses the same
standards for safety and efficacy
- European Society of Human Genetics (ESHG) – Network for genetic research in Europe
European Scientific Networks/Societies
- European Federation of Biotechnology (EFB) – Supports biotechnological research and collaboration
- European Society for Medical Oncology (ESMO) – Network for oncology research and clinical guidelines
- European Hematology Association (EHA) – Scientific collaboration in hematology
Belgian/National Organizations
- Belgian Society of Human Genetics (BeSHG)
Ethics committees or bodies
SMEC – Social and Societal Ethics Committee: Social, behavioral, and non-medical human research
- It is not with patients, but with healthy persons
EC Research UZ/KU Leuven: Medical-scientific and clinical research involving humans
EC Care – Ethics Committee Care: Ethical aspects of patient care and use of donated human biological material
- Ethics related to care not to research
ECD – Ethical Committee for Animal Experimentation: Animal research and experimentation
- If you do tests on animals, the protocol needs to be approved by this commitee
- They’re balancing: What is the negative effect on the animal VS the advantage/benefit for the human society
EC DMM – Ethical Committee on Dual Use, Military Use & Misuse: Research with potential dual-use, military applications, or
misuse risks
- They will look whether or not what you make is still not purely military but has also dual-use namely a use that is to the
benefit of our society
1
,OBC – Education & Support Committee: Support and ethical guidance for master’s theses (often Biomedical Sciences)
PRET Team – Privacy & Ethics Team: Data protection, privacy review, and compliance support (not a formal ethics committee)
Regulatory authorities
- Regulations on different levels; it depends on the type of product that you work with
o National level (eg. E.G. FAMHP/FAGG)
o European level (eg. EMA)
o International level (eg. WHO)
European
EMA takes care of all request for central
marketing organisation
European commission: they organize the
funding programmes, they make rules at the
european level
Payers
When there is approval to come on the market with a product, the price may be very high so no one can buy it
That’s why there is a system of payers (system of reimbursement)
- This is always organized at a national level, not european level. The reason for that is that taxes are also organized at a
national level
- The amount of money that a country can spend, is different between the different countries
In Belgium, there are different types of reimbursement of payers:
- National Public Payer – INAMI / RIZIV
o Main national health insurance institution
o Sets reimbursement rules for therapies, medicines, and medical procedures
o Evaluates cost-effectiveness and clinical benefit
• They look at different criteria, how good the product works, how bad are the side effects, compared to
the price that is asked for it.
- Sickness Funds (Mutualiteiten/Mutualités)
o Administer health coverage for citizens
o Collect contributions and reimburse care according to INAMI / RIZIV regulations
o Serve as the link between patients and national reimbursement system
o They distribute the payments
- Private Insurance Companies
o Complementary or supplementary coverage
o Limited role in standard therapies
o Sometimes used for experimental or high-cost treatments
2
,Patients
- Important stakeholders
- Individual patients
- Patient organisations
o Patients can also organise themselves in groups/organisations on european or national level. They can learn
from each other, they can share experiences, do some studies, etc.
o Club of patients with the same disease, they live in different patients.
- Patient experts
o Patients experts are closely linked to patients with a disease but they can communicate it in a very clearly way,
they are more professionalized.
o They can understand what is a clinical trial, the different processes that occur at a research level.
o Patient experts are the bridge between the patients that are too sick and researchers in order to give the input.
- Citizens
o The payers reimburse medicines with the money from the citizens/society.
Industry and sector associations
How is the industry as a stakeholder in the process organized?
Industry is a broad term, it can be focussing on pharmaceutical products, on diagnostics, you
also have smaller biotech companies that can do medicine development. Another group of
industries are the CRO’s (they help the industry to guide towards the regulatory process, they
gelp to bring the medicines to the practice).
There are more but these are the main categories.
It’s important to know and understand the associations because there is sometimes attraction between both (the
originators/innovators and generics or biosimilars)
- Innovator associations
o Pharma.be, MedTech Europe, efpia, …
- Generic and biosimilar associations
o Medaxes, medicines for europe, …
Not for profit organisations
There are some organisations that are in between
- Charities & foundations
- Patient organisations
- Public private partnerships - IHI (= Innovative Health Initiative)
Interaction between the different stakeholders – example
Possible exam question: story and what types of organisations may be involved
- List them and explain why you think these organisation can have a place in the whole trajectory
3
, Regulatory authorities – ATMP example
One of the most complex types of medicines are advanced therapy
medicinal products (ATMP)
- Cell therapy, gene therapy, …
Everything that is assessed in the context of ethics, occurs at the national
level (the local level)
If an ATMP is brought on the market, you need approval from the national level (fagg) in order to conclude the clinical trial
So locally researchers need to ask permission to the ethics committee to do the research, but in order to do the clinical trial you
need to have permission at the national level from the medicines agencie at the nationla level (example fagg)
What is decided at the European level?
The product that is positively advised by the ethics committee locally which have received positive advice from the national
authorities to conduct the clinical trial and if the clinical trial ends up positive, showing that the therapy works and is safe. Then,
it’s at the European level (European medicines agency) to decide about the marketing authorisation of this complicated therapy.
Main message:
For a certain product in the biomedical area, decisions on whether to go further occur at different levels: locally, national and
European.
Different steps in the development of products are decided at different levels, and it depends on what type of approval that you
need.
Why regulations in biomedical science?
In the past there were a lot of clinical trials that occured chaotically (uncontrolled, not regulated, consequences for patient safety)
à unethical approaches (WW2) without consent
Aim of regulations
- Patient protection
- Scientific quality
- Transparency
- Trust
ð We need to balance this strive for innovation and strive to speed up new treatment to the market, we have to balance it
with safety, to do everything that the safety of the patient is guaranteed and that the science is done thoroughly à if
you go too fast, you can miss important steps
Regulation as an enabling factor
1. Clarity
Clear rules and guidelines help all stakeholders understand requirements and processes
2. Level Playing Field
Ensures fair competition and consistent standards across companies and countries
3. International Recognition
Facilitates global market access and mutual acceptance of approvals and data
What would happen without regulation?
- Uncontrolled growth, inconsistent development
- Inequality
- Loss of trust
4
Lecture 1 Introduction
Stakeholders in the biomedical domain
The biomedical ecosystem = the trajectory from the lab to the
clinic.
Stakeholders in the biomedical domain:
- Healthcare professionals
- Patients
- Researchers
- Industry
- Regulatory authorities
Researchers/HCP’s and scientific organisations
International Organizations
- World Health Organization (WHO) – Guidelines and policies for global health
- International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) –
Harmonization of pharmaceutical research and registration
o ICH à organization that set the standards for safety and efficacy, so that everyone in the world uses the same
standards for safety and efficacy
- European Society of Human Genetics (ESHG) – Network for genetic research in Europe
European Scientific Networks/Societies
- European Federation of Biotechnology (EFB) – Supports biotechnological research and collaboration
- European Society for Medical Oncology (ESMO) – Network for oncology research and clinical guidelines
- European Hematology Association (EHA) – Scientific collaboration in hematology
Belgian/National Organizations
- Belgian Society of Human Genetics (BeSHG)
Ethics committees or bodies
SMEC – Social and Societal Ethics Committee: Social, behavioral, and non-medical human research
- It is not with patients, but with healthy persons
EC Research UZ/KU Leuven: Medical-scientific and clinical research involving humans
EC Care – Ethics Committee Care: Ethical aspects of patient care and use of donated human biological material
- Ethics related to care not to research
ECD – Ethical Committee for Animal Experimentation: Animal research and experimentation
- If you do tests on animals, the protocol needs to be approved by this commitee
- They’re balancing: What is the negative effect on the animal VS the advantage/benefit for the human society
EC DMM – Ethical Committee on Dual Use, Military Use & Misuse: Research with potential dual-use, military applications, or
misuse risks
- They will look whether or not what you make is still not purely military but has also dual-use namely a use that is to the
benefit of our society
1
,OBC – Education & Support Committee: Support and ethical guidance for master’s theses (often Biomedical Sciences)
PRET Team – Privacy & Ethics Team: Data protection, privacy review, and compliance support (not a formal ethics committee)
Regulatory authorities
- Regulations on different levels; it depends on the type of product that you work with
o National level (eg. E.G. FAMHP/FAGG)
o European level (eg. EMA)
o International level (eg. WHO)
European
EMA takes care of all request for central
marketing organisation
European commission: they organize the
funding programmes, they make rules at the
european level
Payers
When there is approval to come on the market with a product, the price may be very high so no one can buy it
That’s why there is a system of payers (system of reimbursement)
- This is always organized at a national level, not european level. The reason for that is that taxes are also organized at a
national level
- The amount of money that a country can spend, is different between the different countries
In Belgium, there are different types of reimbursement of payers:
- National Public Payer – INAMI / RIZIV
o Main national health insurance institution
o Sets reimbursement rules for therapies, medicines, and medical procedures
o Evaluates cost-effectiveness and clinical benefit
• They look at different criteria, how good the product works, how bad are the side effects, compared to
the price that is asked for it.
- Sickness Funds (Mutualiteiten/Mutualités)
o Administer health coverage for citizens
o Collect contributions and reimburse care according to INAMI / RIZIV regulations
o Serve as the link between patients and national reimbursement system
o They distribute the payments
- Private Insurance Companies
o Complementary or supplementary coverage
o Limited role in standard therapies
o Sometimes used for experimental or high-cost treatments
2
,Patients
- Important stakeholders
- Individual patients
- Patient organisations
o Patients can also organise themselves in groups/organisations on european or national level. They can learn
from each other, they can share experiences, do some studies, etc.
o Club of patients with the same disease, they live in different patients.
- Patient experts
o Patients experts are closely linked to patients with a disease but they can communicate it in a very clearly way,
they are more professionalized.
o They can understand what is a clinical trial, the different processes that occur at a research level.
o Patient experts are the bridge between the patients that are too sick and researchers in order to give the input.
- Citizens
o The payers reimburse medicines with the money from the citizens/society.
Industry and sector associations
How is the industry as a stakeholder in the process organized?
Industry is a broad term, it can be focussing on pharmaceutical products, on diagnostics, you
also have smaller biotech companies that can do medicine development. Another group of
industries are the CRO’s (they help the industry to guide towards the regulatory process, they
gelp to bring the medicines to the practice).
There are more but these are the main categories.
It’s important to know and understand the associations because there is sometimes attraction between both (the
originators/innovators and generics or biosimilars)
- Innovator associations
o Pharma.be, MedTech Europe, efpia, …
- Generic and biosimilar associations
o Medaxes, medicines for europe, …
Not for profit organisations
There are some organisations that are in between
- Charities & foundations
- Patient organisations
- Public private partnerships - IHI (= Innovative Health Initiative)
Interaction between the different stakeholders – example
Possible exam question: story and what types of organisations may be involved
- List them and explain why you think these organisation can have a place in the whole trajectory
3
, Regulatory authorities – ATMP example
One of the most complex types of medicines are advanced therapy
medicinal products (ATMP)
- Cell therapy, gene therapy, …
Everything that is assessed in the context of ethics, occurs at the national
level (the local level)
If an ATMP is brought on the market, you need approval from the national level (fagg) in order to conclude the clinical trial
So locally researchers need to ask permission to the ethics committee to do the research, but in order to do the clinical trial you
need to have permission at the national level from the medicines agencie at the nationla level (example fagg)
What is decided at the European level?
The product that is positively advised by the ethics committee locally which have received positive advice from the national
authorities to conduct the clinical trial and if the clinical trial ends up positive, showing that the therapy works and is safe. Then,
it’s at the European level (European medicines agency) to decide about the marketing authorisation of this complicated therapy.
Main message:
For a certain product in the biomedical area, decisions on whether to go further occur at different levels: locally, national and
European.
Different steps in the development of products are decided at different levels, and it depends on what type of approval that you
need.
Why regulations in biomedical science?
In the past there were a lot of clinical trials that occured chaotically (uncontrolled, not regulated, consequences for patient safety)
à unethical approaches (WW2) without consent
Aim of regulations
- Patient protection
- Scientific quality
- Transparency
- Trust
ð We need to balance this strive for innovation and strive to speed up new treatment to the market, we have to balance it
with safety, to do everything that the safety of the patient is guaranteed and that the science is done thoroughly à if
you go too fast, you can miss important steps
Regulation as an enabling factor
1. Clarity
Clear rules and guidelines help all stakeholders understand requirements and processes
2. Level Playing Field
Ensures fair competition and consistent standards across companies and countries
3. International Recognition
Facilitates global market access and mutual acceptance of approvals and data
What would happen without regulation?
- Uncontrolled growth, inconsistent development
- Inequality
- Loss of trust
4
