Introduction to Regulatory Sciences in
Biomedicine
Prof. Isabelle Huys | KU Leuven | February 9, 2026
Part 1 – Stakeholders in the Biomedical Domain
The Biomedical Ecosystem
• The biomedical ecosystem encompasses all actors involved in translating scientific
discoveries from the laboratory to patients and society, and none of these actors work
in isolation — they interact continuously throughout the development process.
• The five core stakeholder groups are researchers, healthcare professionals (HCPs),
industry, patients and citizens, and regulatory authorities, each with distinct but
interdependent roles.
Researchers
• Researchers are the primary drivers of biomedical innovation and can be divided into
three types depending on where they sit in the translational pipeline.
◦ Academic researchers focus on fundamental, hypothesis-driven research —
they work with cell models, animal models, and laboratory experiments to
understand mechanisms of action and identify potential targets or molecules.
◦ Clinical researchers conduct patient-oriented studies, clinical trials, and
diagnostic research, working directly with human participants to test whether
discoveries are safe and effective in practice.
◦ Translational researchers act as the bridge between these two worlds — they
work with biomarkers and human biological materials to validate findings and
prepare them for clinical application, typically in universities or specialized
institutes such as IMEC, VIB, VITO, or the EORTC.
Healthcare Professionals (HCPs)
• Healthcare professionals include doctors, nurses, pharmacists, psychologists, and
allied health staff who work in hospitals or private entities. They often carry dual roles:
as caregivers to patients and as clinical researchers in trial settings.
• HCPs are typically embedded in scientific organizations and networks that allow them
to share knowledge and align on standards — examples include the WHO at the global
level, the ICH for pharmaceutical standards, the ESMO for oncology, and the Belgian
Society of Human Genetics nationally.
Industry
• Industry is responsible for scaling, manufacturing, and bringing laboratory discoveries
to patients as viable medical products — it takes the financial and operational risk to
turn a research finding into a pill, injection, or diagnostic tool.
• Industry can be divided into two key categories with fundamentally different market
incentives:
◦ Innovators / originators develop entirely new therapies from scratch and protect
them through patents. They want to remain alone on the market for as long as
, possible to recoup their investment. In Belgium, innovators are grouped in
pharma.be; at the European level, in EFPIA for medicines and MedTech Europe
for medical technologies.
◦ Generics and biosimilars copy existing products once the innovator's patent
expires. Generics copy small chemical molecules; biosimilars copy large,
complex biological products such as insulin. These companies want to enter the
market as fast as possible, creating a natural and important tension with
innovators. They are represented by Medaxes (Belgium) and Medicines for
Europe (EU).
• Contract Research Organizations (CROs) are specialized commercial entities hired
by pharma and biotech companies to navigate the regulatory framework on their
behalf, helping to design and run trials and bring medicines to clinical practice.
Patients and Citizens
• Patients are the ultimate end-users of everything developed in the biomedical
ecosystem, and it is essential that they are involved early — not only at the end — so
that researchers and industry understand what patients actually need and want.
• Patient experts are a particularly important subgroup: they have the disease
themselves but are professionalized enough to communicate clearly with researchers,
act as a bridge between sick patients and the scientific community, and meaningfully
contribute to study design.
• Citizens matter because they fund the healthcare system through solidarity-based
taxes, which in turn finances reimbursement. Their trust in the system is therefore
essential to maintain.
• At the European level, organizations such as the European Patients Forum (EPF) and
Cancer Patients Europe advocate for patient rights and influence policy. At national
and disease-specific levels, many more patient organizations exist.
Regulatory Authorities
• Regulatory authorities ensure that research and medical products meet legal, ethical,
and safety standards before and during clinical use, operating at three levels:
◦ National level: e.g., the Federal Agency for Medicines and Health Products
(FAMHP/FAGG) in Belgium, which must approve clinical trials before they begin.
◦ European level: the European Medicines Agency (EMA) is the central authority
for marketing authorization of complex therapies; alongside it operate the
European Commission, the Heads of Medicines Agencies (HMA), the ECDC, and
others.
◦ International level: the WHO sets global health guidelines, and the ICH
harmonizes technical requirements so that the same standards of safety and
efficacy are applied worldwide.
Payers
• Payers control whether a product is actually accessible to patients by determining
reimbursement — a drug can be EMA-approved but still unaffordable if payers refuse
to reimburse it.
• Reimbursement is always organized at the national level because it is funded by
national taxes, which means wealthy countries can often reimburse therapies that
poorer countries cannot — creating real inequalities in patient access across Europe.
, • In Belgium, the key payer structures are:
◦ RIZIV/INAMI (National Institute for Health and Disability Insurance): the main
public payer that evaluates cost-effectiveness and sets reimbursement rules.
◦ Sickness funds (mutualiteiten): administer health coverage and distribute
reimbursements to patients.
◦ Private insurance companies: provide complementary coverage, particularly for
high-cost treatments such as cell-based therapies.
Local Structures at KU Leuven / UZ Leuven
• KU Leuven and UZ Leuven together form a model of how major academic medical
centers are organized, and understanding this structure is a template for how clinical
and academic centers work globally.
◦ Clinical Trial Centre (CTC): the mandatory gateway for any clinical trial involving
patients at the university or hospital — researchers must pass through the CTC to
ensure the trial is correctly designed and compliant.
◦ Biobank: governs the storage, management, and regulatory use of human
biological materials such as leftover blood or tissue. Any researcher wishing to
use such samples must obtain permission from the biobank.
◦ Research Coordination Office (DOC): helps researchers set up protocols,
identify funding at national and European levels, and connects them to the right
hospital resources.
◦ Leuven Research & Development (LRD): the university's technology transfer
and valorization office, handling patents, spin-offs, and legal/financial contracts
with industry partners.
Ethics Committees at KU Leuven
• Multiple distinct ethics committees exist, each covering a different domain of research
— knowing which committee governs which type of research is important.
◦ SMEC (Social and Societal Ethics Committee): handles surveys, interviews, and
non-medical research with healthy volunteers.
◦ EC Research UZ/KU Leuven: the main medical-scientific ethics committee for
clinical research with human participants. Prof. Minne Casteels is its chair. This
committee applies rigorous scientific and ethical scrutiny.
◦ EC Care: deals with the ethical aspects of patient care — not research — for
example, euthanasia protocols or end-of-life decisions.
◦ ECD (Animal Ethics Committee): evaluates animal experimentation protocols,
weighing the human benefit against the harm to animals.
◦ EC DMM (Dual Use, Military Use & Misuse Committee): assesses whether
civilian research could be repurposed for military or harmful defense applications
(e.g., genetically modifying a virus that could be weaponized).
◦ OBC: a support committee specifically for master's thesis students, helping them
determine which regulatory path to follow — a shortcut through the ethics
landscape.
◦ PRET Team: not a formal ethics committee, but a privacy and data protection
team that reviews GDPR compliance issues.
Stakeholder Interaction in Practice: Application to Cases
, • On the exam, you will not be asked to memorize diagrams — you will be given a
hypothetical scenario and asked to identify which stakeholders must be involved and
explain why.
• Example: a multi-country clinical trial testing vaccine efficacy involving both blood
samples and personal patient data requires coordination between all of the following:
◦ CTC: to verify the trial is correctly designed.
◦ Ethics Committee: to approve the ethical and scientific validity of the protocol.
◦ FAGG/FAMHP: to grant national regulatory approval for the clinical trial.
◦ Biobank: to authorize and manage the storage and use of blood samples.
◦ Data Protection Officer (DPO): to ensure personal patient data is handled in
accordance with GDPR.
◦ LRD/TTO: if external companies are involved, to conclude the necessary legal
and financial contracts.
• The logic to internalize: each stakeholder serves a distinct protective or enabling
function, and involving them at the right moment in the research trajectory is not
bureaucracy — it is what makes research safe, valid, and ultimately useful to patients.
The ATMP Example: Governance Across Multiple Levels
• Advanced Therapy Medicinal Products (ATMPs) — such as cell therapies and gene
therapies — illustrate how regulatory decisions happen at different levels
simultaneously, not just one.
◦ At the local level, the Ethics Committee must approve the clinical trial protocol
before any research with patients begins.
◦ At the national level, the FAGG/FAMHP must grant authorization for the clinical
trial to proceed in Belgium.
◦ At the European level, the EMA makes the final decision on marketing
authorization — whether the therapy can be sold and used across the EU —
based on the totality of clinical trial evidence.
• An important nuance: power is distributed, not held by one entity. A local Ethics
Committee can stop a study immediately with a negative opinion, before it ever
reaches the EMA. Conversely, the EMA can refuse marketing authorization even if
national and local approvals were granted. Each level has a distinct and decisive role.
Part 2 – Why Do Regulations Exist?
Historical Context: The Necessity of Regulation
• Modern regulatory systems did not emerge arbitrarily — they were built in direct
response to real disasters caused by the absence of oversight.
◦ Unethical experimentation on human subjects without consent occurred
throughout the 1930s–1970s, including the Tuskegee Syphilis Study and Nazi
experiments, which established the need for informed consent as a legal
principle.
◦ The thalidomide tragedy in the 1960s — where pregnant women took a morning-
sickness drug that caused severe limb deformities in their children — directly
drove the creation of agencies like the EMA and the FDA.
• The key historical codes (such as the Nuremberg Code and the Helsinki Declaration)
established the core principle of informed consent and Good Clinical Practice (GCP).
Biomedicine
Prof. Isabelle Huys | KU Leuven | February 9, 2026
Part 1 – Stakeholders in the Biomedical Domain
The Biomedical Ecosystem
• The biomedical ecosystem encompasses all actors involved in translating scientific
discoveries from the laboratory to patients and society, and none of these actors work
in isolation — they interact continuously throughout the development process.
• The five core stakeholder groups are researchers, healthcare professionals (HCPs),
industry, patients and citizens, and regulatory authorities, each with distinct but
interdependent roles.
Researchers
• Researchers are the primary drivers of biomedical innovation and can be divided into
three types depending on where they sit in the translational pipeline.
◦ Academic researchers focus on fundamental, hypothesis-driven research —
they work with cell models, animal models, and laboratory experiments to
understand mechanisms of action and identify potential targets or molecules.
◦ Clinical researchers conduct patient-oriented studies, clinical trials, and
diagnostic research, working directly with human participants to test whether
discoveries are safe and effective in practice.
◦ Translational researchers act as the bridge between these two worlds — they
work with biomarkers and human biological materials to validate findings and
prepare them for clinical application, typically in universities or specialized
institutes such as IMEC, VIB, VITO, or the EORTC.
Healthcare Professionals (HCPs)
• Healthcare professionals include doctors, nurses, pharmacists, psychologists, and
allied health staff who work in hospitals or private entities. They often carry dual roles:
as caregivers to patients and as clinical researchers in trial settings.
• HCPs are typically embedded in scientific organizations and networks that allow them
to share knowledge and align on standards — examples include the WHO at the global
level, the ICH for pharmaceutical standards, the ESMO for oncology, and the Belgian
Society of Human Genetics nationally.
Industry
• Industry is responsible for scaling, manufacturing, and bringing laboratory discoveries
to patients as viable medical products — it takes the financial and operational risk to
turn a research finding into a pill, injection, or diagnostic tool.
• Industry can be divided into two key categories with fundamentally different market
incentives:
◦ Innovators / originators develop entirely new therapies from scratch and protect
them through patents. They want to remain alone on the market for as long as
, possible to recoup their investment. In Belgium, innovators are grouped in
pharma.be; at the European level, in EFPIA for medicines and MedTech Europe
for medical technologies.
◦ Generics and biosimilars copy existing products once the innovator's patent
expires. Generics copy small chemical molecules; biosimilars copy large,
complex biological products such as insulin. These companies want to enter the
market as fast as possible, creating a natural and important tension with
innovators. They are represented by Medaxes (Belgium) and Medicines for
Europe (EU).
• Contract Research Organizations (CROs) are specialized commercial entities hired
by pharma and biotech companies to navigate the regulatory framework on their
behalf, helping to design and run trials and bring medicines to clinical practice.
Patients and Citizens
• Patients are the ultimate end-users of everything developed in the biomedical
ecosystem, and it is essential that they are involved early — not only at the end — so
that researchers and industry understand what patients actually need and want.
• Patient experts are a particularly important subgroup: they have the disease
themselves but are professionalized enough to communicate clearly with researchers,
act as a bridge between sick patients and the scientific community, and meaningfully
contribute to study design.
• Citizens matter because they fund the healthcare system through solidarity-based
taxes, which in turn finances reimbursement. Their trust in the system is therefore
essential to maintain.
• At the European level, organizations such as the European Patients Forum (EPF) and
Cancer Patients Europe advocate for patient rights and influence policy. At national
and disease-specific levels, many more patient organizations exist.
Regulatory Authorities
• Regulatory authorities ensure that research and medical products meet legal, ethical,
and safety standards before and during clinical use, operating at three levels:
◦ National level: e.g., the Federal Agency for Medicines and Health Products
(FAMHP/FAGG) in Belgium, which must approve clinical trials before they begin.
◦ European level: the European Medicines Agency (EMA) is the central authority
for marketing authorization of complex therapies; alongside it operate the
European Commission, the Heads of Medicines Agencies (HMA), the ECDC, and
others.
◦ International level: the WHO sets global health guidelines, and the ICH
harmonizes technical requirements so that the same standards of safety and
efficacy are applied worldwide.
Payers
• Payers control whether a product is actually accessible to patients by determining
reimbursement — a drug can be EMA-approved but still unaffordable if payers refuse
to reimburse it.
• Reimbursement is always organized at the national level because it is funded by
national taxes, which means wealthy countries can often reimburse therapies that
poorer countries cannot — creating real inequalities in patient access across Europe.
, • In Belgium, the key payer structures are:
◦ RIZIV/INAMI (National Institute for Health and Disability Insurance): the main
public payer that evaluates cost-effectiveness and sets reimbursement rules.
◦ Sickness funds (mutualiteiten): administer health coverage and distribute
reimbursements to patients.
◦ Private insurance companies: provide complementary coverage, particularly for
high-cost treatments such as cell-based therapies.
Local Structures at KU Leuven / UZ Leuven
• KU Leuven and UZ Leuven together form a model of how major academic medical
centers are organized, and understanding this structure is a template for how clinical
and academic centers work globally.
◦ Clinical Trial Centre (CTC): the mandatory gateway for any clinical trial involving
patients at the university or hospital — researchers must pass through the CTC to
ensure the trial is correctly designed and compliant.
◦ Biobank: governs the storage, management, and regulatory use of human
biological materials such as leftover blood or tissue. Any researcher wishing to
use such samples must obtain permission from the biobank.
◦ Research Coordination Office (DOC): helps researchers set up protocols,
identify funding at national and European levels, and connects them to the right
hospital resources.
◦ Leuven Research & Development (LRD): the university's technology transfer
and valorization office, handling patents, spin-offs, and legal/financial contracts
with industry partners.
Ethics Committees at KU Leuven
• Multiple distinct ethics committees exist, each covering a different domain of research
— knowing which committee governs which type of research is important.
◦ SMEC (Social and Societal Ethics Committee): handles surveys, interviews, and
non-medical research with healthy volunteers.
◦ EC Research UZ/KU Leuven: the main medical-scientific ethics committee for
clinical research with human participants. Prof. Minne Casteels is its chair. This
committee applies rigorous scientific and ethical scrutiny.
◦ EC Care: deals with the ethical aspects of patient care — not research — for
example, euthanasia protocols or end-of-life decisions.
◦ ECD (Animal Ethics Committee): evaluates animal experimentation protocols,
weighing the human benefit against the harm to animals.
◦ EC DMM (Dual Use, Military Use & Misuse Committee): assesses whether
civilian research could be repurposed for military or harmful defense applications
(e.g., genetically modifying a virus that could be weaponized).
◦ OBC: a support committee specifically for master's thesis students, helping them
determine which regulatory path to follow — a shortcut through the ethics
landscape.
◦ PRET Team: not a formal ethics committee, but a privacy and data protection
team that reviews GDPR compliance issues.
Stakeholder Interaction in Practice: Application to Cases
, • On the exam, you will not be asked to memorize diagrams — you will be given a
hypothetical scenario and asked to identify which stakeholders must be involved and
explain why.
• Example: a multi-country clinical trial testing vaccine efficacy involving both blood
samples and personal patient data requires coordination between all of the following:
◦ CTC: to verify the trial is correctly designed.
◦ Ethics Committee: to approve the ethical and scientific validity of the protocol.
◦ FAGG/FAMHP: to grant national regulatory approval for the clinical trial.
◦ Biobank: to authorize and manage the storage and use of blood samples.
◦ Data Protection Officer (DPO): to ensure personal patient data is handled in
accordance with GDPR.
◦ LRD/TTO: if external companies are involved, to conclude the necessary legal
and financial contracts.
• The logic to internalize: each stakeholder serves a distinct protective or enabling
function, and involving them at the right moment in the research trajectory is not
bureaucracy — it is what makes research safe, valid, and ultimately useful to patients.
The ATMP Example: Governance Across Multiple Levels
• Advanced Therapy Medicinal Products (ATMPs) — such as cell therapies and gene
therapies — illustrate how regulatory decisions happen at different levels
simultaneously, not just one.
◦ At the local level, the Ethics Committee must approve the clinical trial protocol
before any research with patients begins.
◦ At the national level, the FAGG/FAMHP must grant authorization for the clinical
trial to proceed in Belgium.
◦ At the European level, the EMA makes the final decision on marketing
authorization — whether the therapy can be sold and used across the EU —
based on the totality of clinical trial evidence.
• An important nuance: power is distributed, not held by one entity. A local Ethics
Committee can stop a study immediately with a negative opinion, before it ever
reaches the EMA. Conversely, the EMA can refuse marketing authorization even if
national and local approvals were granted. Each level has a distinct and decisive role.
Part 2 – Why Do Regulations Exist?
Historical Context: The Necessity of Regulation
• Modern regulatory systems did not emerge arbitrarily — they were built in direct
response to real disasters caused by the absence of oversight.
◦ Unethical experimentation on human subjects without consent occurred
throughout the 1930s–1970s, including the Tuskegee Syphilis Study and Nazi
experiments, which established the need for informed consent as a legal
principle.
◦ The thalidomide tragedy in the 1960s — where pregnant women took a morning-
sickness drug that caused severe limb deformities in their children — directly
drove the creation of agencies like the EMA and the FDA.
• The key historical codes (such as the Nuremberg Code and the Helsinki Declaration)
established the core principle of informed consent and Good Clinical Practice (GCP).
