Samenvatting Regulatory Sciences in Biomedicine | KU Leuven |master biomedische wetenschappen| 2025/26

REGULATORY SCIENCE

1 INTRODUCTION


1.1 WHAT IS THE AIM / NEXT STEP OF THE RESEARCHERS?
Scientific validation – testing in patients – clinical use

 Can you test this directly in patients?
 Who need to approve this?
 What about stored biological samples?
 What about patient data?

Need of a regulatory framework (geheel v wetten en regels)

 ethical approval (ethics committee)  in vitro
 use of human biological material (biobank law) diagnostics
 data protection and privacy (GDPR) regulations (IVDR)
 reimbursement
and access (RIZIV)
AI tool validation – decision support in clinic – fast translation into practice

 Who is liable in case of errors?
 Transparency and bias of AI?
 Safety + effectiveness of the vaccine ?

Need of a regulatory framework:

 Ethical approval (ethics committee)  Clinical validation
 Use of human biological material (biobank law) and performance
 Data protection and privacy (GDPR)  Cybersecurity and
 In vitro diagnostics regulations (IVDR) software safety
 Reimbursement and access (RIZIV)  Liability and legal
compliance
 Post market
From the very first experiment you are already operating within regulatory
surveillance and landscape 
regulatory science is not the final step, it runs alongside your research from the
updates
beginning  User training and
transparency


1.2 STAKEHOLDERS
=all persons, organisations, institutions involved with research, develop,
regulations, approval,…

 Researchers: working in lab, identify molecules, targets, mechanisms of action
o Academic: hypothesis driven, fundamental research, mechanisms +
preclinical models
o Clinical: patient oriented, clinical studies, diagnostics + treatment
o Translational: bridge between lab + clinic, validation, preparation for
clinical application
o In universities or research institutions (IMEC, VIB)



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,  Industry: to translate what is found in lab to larger scale: manufacturing + scale
up production
 Regulatory authorities: define standards, how research is conducted according
to the ethical rules, did they ask permission,…. (control quality, safety, ethics,
compliance with regulations)
 Patients : they use these innovations: need to be involved early on, what is really
needed
o Individual patient – patient organisations – patient experts – citizens
 Health care professionals (HCP): involved in care of patients, link with the
patients



SCIENTIFIC ORGANISATIONS:
They make guidelines, standards and make international collaboration possible

International organisations:

 WHO = world health organization: guidelines + policies for global health
 ICH = international Council for Harmonisation of Technical Requirements
for Pharmaceuticals for Human Use: harmonisation of pharma regulations
internationally

European scientific networks/ societies

 EFB = European federation of biotechnology: supports biotechnol research +
collaboration
 ESMO = European Society for Medical Oncology: network for oncology
research + clinical guidelines
 EHA = European Hematology Association : scientific collaboration in
hematology

Belgian / national organizations : Belgian society of human genetics (BeSHG)



KU Leuven: 3 sub-entities

 UZ Leuven = hospital + patients : clinical trial centre (CTC) + biobank for all
bio samples
 KU Leuven = education + research : research coordination office (DOC) helps
to set up protocol, funding, direct researchers to the hospital
 LRD = Leuven research & development: valorisation = scientific knowledge
translation into economic/society value + technology transfer office (TTO)
helps researchers with patents, i.e., commercialisation, connection to industry,..



ETHIC COMITTEES OR BODIES:
SMEC = Social + Societal Ethics Social, behavioral, and non-medical human
Committee research
EC Research UZ / KU Leuven Medical-scientific and clinical research:
humans



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, EC Care – Ethics Committee Care Ethical aspects of patient care and use of
donated human biological material
ECD –Ethical Committee for Animal Animal research and experimentation
Experimentation
EC DMM –Ethical Committee on Research with potential dual-use, military
Dual Use, Military Use & Misuse applications, or misuse risks
OBC –Education & Support Support and ethical guidance for master’s
Committee theses (often Biomedical Sciences)
PRET Team – Privacy & Ethics Team Data protection, privacy review, and
compliance support (not a formal ethics
committee)

REGULATORY AUTHORITIES:
National level (FAMHP/FAGG) – European level (EMA) – international level
(WHO)

 EMA = European medicines agency: central evaluation and authorisation of
medicines
 HMA = heads of medicines agency: EU network of national authorities
 European commission: DG SANTE: EU legislation and health policy
 EDQM = European directorate for the quality of medicines and healthcare
 ECDC = European centre for disease prevention and control



PAYERS:
=always organised at national level: europe can approve drug but depending on payers
on national level, so depending on country the drug can be commercialized in that
country

National public payer (INAMI – RIZIV = rijkinstituut voor ziekte en
invaliditeitsverzekering)

 Determines which therapies and medicines will be reimbursed
 Sets reimbursement rules for therapies, medicines, and medical procedures
 Evaluates cost-effectiveness and clinical benefit
 Main national health insurance institution
 Solidary based: everyone pays small amount to the system


Sickness funds (mutualiteiten)

 Collect contributions and reimburse care according to INAMI / RIZIV
regulations
o They implement the decisions of the INAMI/RIZIV regulations
o Voeren de terugbetalingen praktisch uit voor burgers
 Serve as the link between patients and national reimbursement system


Private insurance companies:

 Complementary or supplementary coverage (aanvullende dekking voor bep
kosten)

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,  Limited role in standard therapies
 Sometimes used for experimental or high-cost treatment



INDUSTRY + SECTOR ASSOCIATIONS:
 Big pharma
 Biotech: smaller biotech companies that take a lot of risks
 CRO = contract research organization: helps industry with clinical studies,
regulations and administration  they know the regulatory framework perfectly
 Medical + diagnostic companies




Innovator associations <-> generic and biosimilar associations

 Innovator : focus on innovative medicines build from scratch : pharma.be,
efpia,..
 Generic/bio: generic= copy of small molecule <-> biosimilar = copy of biologic
product
 Generics need to wait on the innovators , innovators bring it first on market


NON PROFIT ORGANISATIONS :
Charities and foundations – patient organisations – public private partnerships  they
can support research and patients, and creeren v bewustwording



INTERACTION BETWEEN DIFFERENT STAKEHOLDERS EXAMPLE:
Multi-country study (international) on patients to study vaccine efficacy and
collect blood and data

 Researchers / clinicians: design and construct the study, interact with patients,
collect data
 Ask for ethical approval with EC
 Ask for approval of government: FAGG: looks at rules + safety
 Recruitment of pt in hospitals + clinical trial centres (CTS)
 Biobanks to store the human samples and get approval that we can work with it
 Data protection officer (DPO): human personal data: ensures that all patient
data is handled according to the privacy regulations (GDPR)
 Results can lead to product: help of Technology transfer office (TTO) +
LRD: manage intellectual property, collaborations, potential commercialization of
products
 European approval (EMA): can it go on the market (MA)
 Then reimbursement by RIZIV (national level)


Regulatory authorities on different levels: decisions in different steps of product at
different levels

 Local level: ethics committee: ask permission for protocol (before study starts)

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