REGULATORY SCIENCES IN
BIOMEDICINE
1. INTRODUCTION
THE AIM OF RESEARCHERS
Scientific validation
Testing in patients
o Can we test it directly in patients?
o Who needs to approve this?
o What about stored biological samples?
o What about patient data?
Clinical use
AI tool validation
Decision-support in clinic
Fast translation into practice
The regulatory framework:
Ethical approval by ethics committee
Use of human biological material via biobank law
Data protection & privacy (GDPR)
In vitro diagnostics regulation (IVDR & MDR)
Reimbursement & access (RIZIV or national health insurance)
Clinical validation & performance with clinical trials
Cybersecurity & software safety following ISO 27001
Liability & legal compliance
Post-market surveillance & updates by real-world performance monitoring
User training & transparency
From the very first experiment, you are already operating within a regulatory landscape
→ Regulatory science is not a final step, it runs alongside your research from the
beginning
→ If you think too late about it, the innovation might not reach patients or reach
them too late
STAKEHOLDERS
Researchers have an important position in the translation of findings from the lab to the
clinic
→ They identify molecules & their targets & try to identify the pathways in which
they interact with each other
→ They will have to work together with all the stakeholders in the field
→ Academic, clinical or translational researchers
→ Work at universities or research institutions
The industry’s job is to translate what is found in the lab in a larger scale in order to bring
it to the market
→ They can scale up the production of a molecule
1
, → The industry consists of the manufacturers
→ Can also include researchers
The regulatory authorities will look at how & whether the research is conducted according
to the ethical rules
→ Did the researchers ask permission?
→ Are they doing tests according to the correct standards?
Patient organisations & society use the treatments & medications available on the market
(= users)
→ They need to be involved in the process early on to decide what is really needed
from the industry
Health care professionals (HCPs) are involved in the care of patients
→ Will decide which medications to give to their patients a lot of the time
→ Dual roles: can be researchers at the same time
→ Work in hospitals or private entities
Universities take care of education & research
→ Research coordination office (DOC) helps researchers set up a protocol, finding
funding,…
The industry leads to valorisation (= the process of creating economic or societal value
from knowledge, research, or technology)
→ Technology transfer office helps researchers when they want to protect their
findings via patents
Hospitals & patients are protected by the clinical trial centre (CTC)
→ Research needs to pass through this office before it can be approved
The ethics committee is an independent organization
→ Not linked to a specific hospital or university
ETHICS COMMITTEES
SMEC = social & societal ethics committee
→ Social, behavioural & non-medical human research
→ Not legally recognized
EC research at UZ or KU Leuven for medical-scientific & clinical research involving
humans
EC Care for ethical aspects of patient care & use of donated human biological materials
→ Ex. A girl is being ventilated in the hospital. The doctors decide there’s nothing left
they can do, so want to stop the ventilation against her parents’ will.
ECD = ethical committee for animal experimentation
→ Explain how much pain the animals will be in
→ Explain how you will euthanise them
→ Justify why you need x amount of animals
2
,3
,EC DMM = ethical committee on dual use, military use & misuse
→ Ex. You do a clinical trial & you collaborate with some universities in China. DMM
will look at it & say you aren’t allowed to work with one of the universities because
they know they are involved in taking transplant organs from people that are in
prison.
OBC = education & support committee
→ Support & ethical guidance for master’s theses
PRET = privacy & ethics team
→ Data protection, privacy review & compliance support
→ Looking at GDPR tools
REGULATORY AUTHORITIES
National level:
FAMHP = federal agency for medicines & health products
FAGG = federaal agentschap voor geneesmiddelen & gezondheidsproducten
European level:
EMA = European medicines agency
o Central evaluation
o Authorization of medicines
European commission
o EU legislation
o Health policy
o Organize funding programmes
HMA = heads of medicines agencies
o EU network of national authorities
EDQM = European directorate for the quality of medicines & healthcare
o Quality standards
ECDC = European centre for disease prevention & control
International level: WHO
PAYERS
National public payer like RIZIV or INAMI
→ Main national health insurance institution
→ Sets reimbursement rules for therapies, medicines & medical procedures
→ Evaluates cost-effectiveness & clinical benefit
Sickness funds (= mutualiteiten)
→ Administer health coverage for citizens
→ Collect contributions & reimburse care according to INAMI or RIZIV regulations
→ Serve as the link between patients & national reimbursement system
Private insurance companies
→ Complementary or supplementary coverage
→ Limited role in standard therapies
4
, → Sometimes used for experimental or high-cost treatments
INDUSTRY & SECTOR ASSOCIATIONS
The industry consists of big pharma, biotech SMEs, medical & diagnostic companies, &
contract research organisations (CROs)
Innovator associations bring new products on the market
→ Aim is to be the only one offering this type of product on the market for as long as
possible
→ Ex. Pharma.be, efpia, MedTech Europe
Generic & biosimilar associations copy the original products
→ Want to be on the market as soon as possible
→ Ex. Medaxes, Medicines for Europe
Non-profit organizations:
Charities & foundations
Patient organisations
Public private partnerships
WHY ARE REGULATIONS NEEDED IN BIOMEDICAL SCIENCE?
Aim of regulations:
Patient protection
Transparency
Trust
Scientific quality
There needs to be a balance between innovation and safety, and between speed and
thoroughness
Regulations as an enabling factor:
Clarity = clear rules & guidelines
o Stakeholders understand requirements & processes
Level playing field
o Ensures fair competition
o Ensures consistent standards across companies & countries
International recognition
o Facilitates global market access
o Facilitates mutual acceptance of approvals & data
Without regulation there would be uncontrolled growth & inconsistent development. This
leads to inequality & a loss of trust.
Ex. Clinical trial without regulatory framework:
Lack of participant protection
o No informed consent or safeguards
o Risk of harm or exploitation
Poor study design
o Unreliable or biased results
o Ineffective or unsafe treatments get approved
5
BIOMEDICINE
1. INTRODUCTION
THE AIM OF RESEARCHERS
Scientific validation
Testing in patients
o Can we test it directly in patients?
o Who needs to approve this?
o What about stored biological samples?
o What about patient data?
Clinical use
AI tool validation
Decision-support in clinic
Fast translation into practice
The regulatory framework:
Ethical approval by ethics committee
Use of human biological material via biobank law
Data protection & privacy (GDPR)
In vitro diagnostics regulation (IVDR & MDR)
Reimbursement & access (RIZIV or national health insurance)
Clinical validation & performance with clinical trials
Cybersecurity & software safety following ISO 27001
Liability & legal compliance
Post-market surveillance & updates by real-world performance monitoring
User training & transparency
From the very first experiment, you are already operating within a regulatory landscape
→ Regulatory science is not a final step, it runs alongside your research from the
beginning
→ If you think too late about it, the innovation might not reach patients or reach
them too late
STAKEHOLDERS
Researchers have an important position in the translation of findings from the lab to the
clinic
→ They identify molecules & their targets & try to identify the pathways in which
they interact with each other
→ They will have to work together with all the stakeholders in the field
→ Academic, clinical or translational researchers
→ Work at universities or research institutions
The industry’s job is to translate what is found in the lab in a larger scale in order to bring
it to the market
→ They can scale up the production of a molecule
1
, → The industry consists of the manufacturers
→ Can also include researchers
The regulatory authorities will look at how & whether the research is conducted according
to the ethical rules
→ Did the researchers ask permission?
→ Are they doing tests according to the correct standards?
Patient organisations & society use the treatments & medications available on the market
(= users)
→ They need to be involved in the process early on to decide what is really needed
from the industry
Health care professionals (HCPs) are involved in the care of patients
→ Will decide which medications to give to their patients a lot of the time
→ Dual roles: can be researchers at the same time
→ Work in hospitals or private entities
Universities take care of education & research
→ Research coordination office (DOC) helps researchers set up a protocol, finding
funding,…
The industry leads to valorisation (= the process of creating economic or societal value
from knowledge, research, or technology)
→ Technology transfer office helps researchers when they want to protect their
findings via patents
Hospitals & patients are protected by the clinical trial centre (CTC)
→ Research needs to pass through this office before it can be approved
The ethics committee is an independent organization
→ Not linked to a specific hospital or university
ETHICS COMMITTEES
SMEC = social & societal ethics committee
→ Social, behavioural & non-medical human research
→ Not legally recognized
EC research at UZ or KU Leuven for medical-scientific & clinical research involving
humans
EC Care for ethical aspects of patient care & use of donated human biological materials
→ Ex. A girl is being ventilated in the hospital. The doctors decide there’s nothing left
they can do, so want to stop the ventilation against her parents’ will.
ECD = ethical committee for animal experimentation
→ Explain how much pain the animals will be in
→ Explain how you will euthanise them
→ Justify why you need x amount of animals
2
,3
,EC DMM = ethical committee on dual use, military use & misuse
→ Ex. You do a clinical trial & you collaborate with some universities in China. DMM
will look at it & say you aren’t allowed to work with one of the universities because
they know they are involved in taking transplant organs from people that are in
prison.
OBC = education & support committee
→ Support & ethical guidance for master’s theses
PRET = privacy & ethics team
→ Data protection, privacy review & compliance support
→ Looking at GDPR tools
REGULATORY AUTHORITIES
National level:
FAMHP = federal agency for medicines & health products
FAGG = federaal agentschap voor geneesmiddelen & gezondheidsproducten
European level:
EMA = European medicines agency
o Central evaluation
o Authorization of medicines
European commission
o EU legislation
o Health policy
o Organize funding programmes
HMA = heads of medicines agencies
o EU network of national authorities
EDQM = European directorate for the quality of medicines & healthcare
o Quality standards
ECDC = European centre for disease prevention & control
International level: WHO
PAYERS
National public payer like RIZIV or INAMI
→ Main national health insurance institution
→ Sets reimbursement rules for therapies, medicines & medical procedures
→ Evaluates cost-effectiveness & clinical benefit
Sickness funds (= mutualiteiten)
→ Administer health coverage for citizens
→ Collect contributions & reimburse care according to INAMI or RIZIV regulations
→ Serve as the link between patients & national reimbursement system
Private insurance companies
→ Complementary or supplementary coverage
→ Limited role in standard therapies
4
, → Sometimes used for experimental or high-cost treatments
INDUSTRY & SECTOR ASSOCIATIONS
The industry consists of big pharma, biotech SMEs, medical & diagnostic companies, &
contract research organisations (CROs)
Innovator associations bring new products on the market
→ Aim is to be the only one offering this type of product on the market for as long as
possible
→ Ex. Pharma.be, efpia, MedTech Europe
Generic & biosimilar associations copy the original products
→ Want to be on the market as soon as possible
→ Ex. Medaxes, Medicines for Europe
Non-profit organizations:
Charities & foundations
Patient organisations
Public private partnerships
WHY ARE REGULATIONS NEEDED IN BIOMEDICAL SCIENCE?
Aim of regulations:
Patient protection
Transparency
Trust
Scientific quality
There needs to be a balance between innovation and safety, and between speed and
thoroughness
Regulations as an enabling factor:
Clarity = clear rules & guidelines
o Stakeholders understand requirements & processes
Level playing field
o Ensures fair competition
o Ensures consistent standards across companies & countries
International recognition
o Facilitates global market access
o Facilitates mutual acceptance of approvals & data
Without regulation there would be uncontrolled growth & inconsistent development. This
leads to inequality & a loss of trust.
Ex. Clinical trial without regulatory framework:
Lack of participant protection
o No informed consent or safeguards
o Risk of harm or exploitation
Poor study design
o Unreliable or biased results
o Ineffective or unsafe treatments get approved
5
