Regulatory Sciences in Biomedicine
Thema 1: Introduction
1. Stakeholders in the biomedical ecosystem
Stakeholder Role Extra
Researchers Research work Scientific organisations at different levels and
(academic → clinical →translational) collaboration
Healthcare Deliver care, recruit participants, interpret
professionals tests, implement therapy.
Hospitals / CTCs Provide clinical infrastructure, trial Clinical studies require operational and legal
management and patient access. governance.
Ethics committees Independent review of ethical, scientific and Different types of EC, for the different types
/ IRBs legal acceptability. of studies.
Regulatory Assess safety, efficacy/performance and Different levels:
authorities quality. FAGG/FAMHP → EMA → WHO
Payers Decide or coordinate reimbursement. National public payers / sickness funds /
private insurance companies
Industry / CROs Develop products, run trials, manufacture, Responsible for quality systems and
submit dossiers. evidence packages.
Patients / citizens Participants, users and stakeholders in Regulation protects rights and supports trust.
priorities and acceptability.
Others: Industre & sector associations, non-profit organisations, technology transfer office, data protection
office,….
2. Why regulation exist?
• Patient/participant protection
• Scientific quality
• Transparency and trust
• Fair competition / level playing field: companies and institutions follow the same standards.
• Innovation enabling: clear rules help researchers know what evidence is needed for translation.
• International recognition
Regulation ≠ bureacracy
3. Types of regulatory instruments
Instrument Meaning
EU Regulation Directly binding in all EU Member States.
EU Directive Binding goal, but Member States
transpose into national law.
National law Country-specific legal implementation and
institutional rules.
Soft guideline Not always legally binding, but often
expected as best practice.
Institutional Local operational rules.
procedure
1
,Thema 2: Medical law and hospital organisation
Increasing juridification in healthcare
• Healthcare law has expanded due to patient rights, social/ethical debates, EU legislation, data transfer …
• Medical practice is no longer only professional ethics; it is also legal compliance.
Mandatory hospital structures in Belgium
Structure Core role
Medical director / Chief Medical a physician responsible for the smooth running of the hospital’s medical department.
Officer (CMO)
Medical Council Elected (3y) advisory body representing hospital doctors; legal discussion partner of
hospital management.
Nursing director Coordinates nursing services and collaboration with medical activities.
Ethics Committee Independent ethical review and advice.
Hospital hygiene Infection prevention and hygiene policy.
physician/committee
Pharmacy and Therapeutics Drug formulary, safe and cost-effective medication use.
Committee
Medical Equipment Committee Evaluation, selection and standardisation of medical devices/equipment.
Medical director
Organises and coordinates medical activities in the hospital, collaborating with:
• head physicians;
• medical staff;
• nursing services;
• paramedical services;
• hospital pharmacist;
• Medical Council.
Main responsibilities:
- Ensures compliance with medical laws, regulations and hospital medical rules.
- Integrates medical activity into the overall hospital activity.
- Promotes cooperation
- Promotes quality assurance (waiting time, mortality, infections, medication near-misses)
- Ensures continuity of care, admission and discharge procedures, hospital hygiene, medical files, annual
medical report, budget data for medical activities.
- Can organise a medical audit when patient safety is at risk.
- Keeps personal files on every doctor working in the hospital.-
- Ensures decentralized clinical biology testing happens under supervision of the accredited lab head.
Medical Council
• It is the legal discussion partner of the hospital administrator.
• A Medical Council is required for hospital recognition and is also an ethical obligation.
Advice about
• regulation
• number of medical staff;
• nomination of the medical director;
• nomination of heads of clinical departments;
• temporary access of other doctors to work in the hospital;
• dismissal or sanctions of medical staff;
• annual budget for medical activities;
• medical equipment needs, purchase and repair;
• agreements with third parties affecting medical activity, framework for nursing and paramedical staff;
Why is it powerful?
2
, • If the Medical Council disagrees, a mediation procedure must start.
• It has real influence over hospital decisions affecting medical care.
Access to patient files
• Access only when needed for a valid clinical role, quality/legal function or justified research procedure.
Therapeutic relationship: you need the information to diagnose or treat that patient.
• Minimum necessary access: only view what you need.
• Every access is logged; patients can request the audit trail.
• Unjustified access can lead to investigation and sanctions.
• Privacy also covers seeing someone in the hospital: do not communicate who you saw.
GDPR gives data subject rights such as erasure, but Belgian medical law may require long retention (30y) of
medical records.
Thema 3: Ethical approval
1. Introduction
• Ethics Committee (EC) or Institutional Review Board (IRB) is an independent multidisciplinary body. It
analyses research involving human participants to protect ethics → based on legislation and institutional rules.
• Never self-decide that approval is unnecessary: request approval or a waiver (= vrijstelling). Retroactive
approval is normally not given.
• Ethics ~ scientific validity + protection of patient and facilitation of research
• Internation Council for Hormonisation (Quality, Safety, Efficacy, Multidisciplinary)
• Due to historical need (Nurenberg code). International → European → National
2. EC tasks
• Evaluate research on human (clinical trials), medical devices.
• Advise on human body material, biobanking, in-vitro embryos
• Evaluate compassionate use / medical need programs where relevant, theses.
• Privacy-related checks when linked to research.
3. Composition
• Expertise: physicians, 2 nurses, hospital pharmacist, methodology/statistics, ethics/philosophy, pharmacology,
psychology, General Practitioner, legal expert, patient representative (lay) and healthy volunteer representative
(2x Ph I)
• Conflict of interest must be managed
4. Principal Investigator commitments
• Qualified and familiar with the investigational product.
• Complies with GCP and other regulations; prepared for audit/monitoring → Informed consent
• Maintains delegation of responsibilities: who is authorised to do what.
• Has adequate resources: staff, facilities, recruitment capacity and time.
• Ensures staff understand protocol, product and responsibilities.
• Deliver documents: cover letter, protocol, informed consent form, CV, GCP certificate …
5. Informed consent form
• Patient-PI-medical doctor
• Purpose of the study, design, procedures, practical burden.
• Benefits - risks
• Alternative treatments
• Data/sample confidentiality and intended use.
• Financial aspects, insurance and compensation in case of injury.
• Voluntary participation and right to withdraw at any time without affecting care.
• Contact details
3
Thema 1: Introduction
1. Stakeholders in the biomedical ecosystem
Stakeholder Role Extra
Researchers Research work Scientific organisations at different levels and
(academic → clinical →translational) collaboration
Healthcare Deliver care, recruit participants, interpret
professionals tests, implement therapy.
Hospitals / CTCs Provide clinical infrastructure, trial Clinical studies require operational and legal
management and patient access. governance.
Ethics committees Independent review of ethical, scientific and Different types of EC, for the different types
/ IRBs legal acceptability. of studies.
Regulatory Assess safety, efficacy/performance and Different levels:
authorities quality. FAGG/FAMHP → EMA → WHO
Payers Decide or coordinate reimbursement. National public payers / sickness funds /
private insurance companies
Industry / CROs Develop products, run trials, manufacture, Responsible for quality systems and
submit dossiers. evidence packages.
Patients / citizens Participants, users and stakeholders in Regulation protects rights and supports trust.
priorities and acceptability.
Others: Industre & sector associations, non-profit organisations, technology transfer office, data protection
office,….
2. Why regulation exist?
• Patient/participant protection
• Scientific quality
• Transparency and trust
• Fair competition / level playing field: companies and institutions follow the same standards.
• Innovation enabling: clear rules help researchers know what evidence is needed for translation.
• International recognition
Regulation ≠ bureacracy
3. Types of regulatory instruments
Instrument Meaning
EU Regulation Directly binding in all EU Member States.
EU Directive Binding goal, but Member States
transpose into national law.
National law Country-specific legal implementation and
institutional rules.
Soft guideline Not always legally binding, but often
expected as best practice.
Institutional Local operational rules.
procedure
1
,Thema 2: Medical law and hospital organisation
Increasing juridification in healthcare
• Healthcare law has expanded due to patient rights, social/ethical debates, EU legislation, data transfer …
• Medical practice is no longer only professional ethics; it is also legal compliance.
Mandatory hospital structures in Belgium
Structure Core role
Medical director / Chief Medical a physician responsible for the smooth running of the hospital’s medical department.
Officer (CMO)
Medical Council Elected (3y) advisory body representing hospital doctors; legal discussion partner of
hospital management.
Nursing director Coordinates nursing services and collaboration with medical activities.
Ethics Committee Independent ethical review and advice.
Hospital hygiene Infection prevention and hygiene policy.
physician/committee
Pharmacy and Therapeutics Drug formulary, safe and cost-effective medication use.
Committee
Medical Equipment Committee Evaluation, selection and standardisation of medical devices/equipment.
Medical director
Organises and coordinates medical activities in the hospital, collaborating with:
• head physicians;
• medical staff;
• nursing services;
• paramedical services;
• hospital pharmacist;
• Medical Council.
Main responsibilities:
- Ensures compliance with medical laws, regulations and hospital medical rules.
- Integrates medical activity into the overall hospital activity.
- Promotes cooperation
- Promotes quality assurance (waiting time, mortality, infections, medication near-misses)
- Ensures continuity of care, admission and discharge procedures, hospital hygiene, medical files, annual
medical report, budget data for medical activities.
- Can organise a medical audit when patient safety is at risk.
- Keeps personal files on every doctor working in the hospital.-
- Ensures decentralized clinical biology testing happens under supervision of the accredited lab head.
Medical Council
• It is the legal discussion partner of the hospital administrator.
• A Medical Council is required for hospital recognition and is also an ethical obligation.
Advice about
• regulation
• number of medical staff;
• nomination of the medical director;
• nomination of heads of clinical departments;
• temporary access of other doctors to work in the hospital;
• dismissal or sanctions of medical staff;
• annual budget for medical activities;
• medical equipment needs, purchase and repair;
• agreements with third parties affecting medical activity, framework for nursing and paramedical staff;
Why is it powerful?
2
, • If the Medical Council disagrees, a mediation procedure must start.
• It has real influence over hospital decisions affecting medical care.
Access to patient files
• Access only when needed for a valid clinical role, quality/legal function or justified research procedure.
Therapeutic relationship: you need the information to diagnose or treat that patient.
• Minimum necessary access: only view what you need.
• Every access is logged; patients can request the audit trail.
• Unjustified access can lead to investigation and sanctions.
• Privacy also covers seeing someone in the hospital: do not communicate who you saw.
GDPR gives data subject rights such as erasure, but Belgian medical law may require long retention (30y) of
medical records.
Thema 3: Ethical approval
1. Introduction
• Ethics Committee (EC) or Institutional Review Board (IRB) is an independent multidisciplinary body. It
analyses research involving human participants to protect ethics → based on legislation and institutional rules.
• Never self-decide that approval is unnecessary: request approval or a waiver (= vrijstelling). Retroactive
approval is normally not given.
• Ethics ~ scientific validity + protection of patient and facilitation of research
• Internation Council for Hormonisation (Quality, Safety, Efficacy, Multidisciplinary)
• Due to historical need (Nurenberg code). International → European → National
2. EC tasks
• Evaluate research on human (clinical trials), medical devices.
• Advise on human body material, biobanking, in-vitro embryos
• Evaluate compassionate use / medical need programs where relevant, theses.
• Privacy-related checks when linked to research.
3. Composition
• Expertise: physicians, 2 nurses, hospital pharmacist, methodology/statistics, ethics/philosophy, pharmacology,
psychology, General Practitioner, legal expert, patient representative (lay) and healthy volunteer representative
(2x Ph I)
• Conflict of interest must be managed
4. Principal Investigator commitments
• Qualified and familiar with the investigational product.
• Complies with GCP and other regulations; prepared for audit/monitoring → Informed consent
• Maintains delegation of responsibilities: who is authorised to do what.
• Has adequate resources: staff, facilities, recruitment capacity and time.
• Ensures staff understand protocol, product and responsibilities.
• Deliver documents: cover letter, protocol, informed consent form, CV, GCP certificate …
5. Informed consent form
• Patient-PI-medical doctor
• Purpose of the study, design, procedures, practical burden.
• Benefits - risks
• Alternative treatments
• Data/sample confidentiality and intended use.
• Financial aspects, insurance and compensation in case of injury.
• Voluntary participation and right to withdraw at any time without affecting care.
• Contact details
3
