Regulatory sciences in biomedicine – summary
INTRODUCTION – Isabelle Huys
Stakeholders
The biomedical ecosystem
Research and innovation Patient Society
->diagnosis and treatment ->health and community
- Experiments - Diagnosis - Prevention
- Discoveries - Therapy - Public health
- Preclinical research - Care - Policy
From laboratory -> to the patient -> to society
Stakeholders in the biomedical domain
Health care professionals
- They detect clinical needs and bring patients to the researchers
- Perform clinical studies
- Translate research results into practical clinical health care
- Bridge between science and patient-care
Researchers
- Central place – startpoint of innovation
- Very important position in the whole translation from findings from the lab to the clinic
- Identify new targets
- Pre-clinical research
- Proof-of-concept (to bring a therapy to the clinical development)
Patients
- They deliver important input about the quality of life, needs and feasibility and acceptance of therapy
Industry (also researchers in the industry!)
- Different types of industry
- They scale up what is found in the lab, more production is possible
- They manufacture products
1
,Regulatory authorities (ex. EMA, FDA, FAGG)
- They define the standards, which the research need to occur
- The research must conduct and accord to the rules
->they all work very closely together
Researchers
Examples of types of researchers
Academic
- Fundamental research
- Hypothesis diven
- Mechanisms and preclinical models
Clinical
- Patient-oriented research
- Clinical studies
- Diagnostics and treatment
Translational
- Bridge between lab and clinic
- Validation and proof-of-concept
- Preparation for clinical application
- They work with human material
- They translate what is in the lab and want to validate it into clinical
Universities
Research institutes (IMEC, VIB, VITO, EORTC, …)
Health care professionals – HCPs
->hospitals and private entities
- Doctors/physicians
- Nurses
- Allied health professionals
- Pharmacists
- Mental health professionals
- Public health and administrative professionals
Researcher/HCPs & scientific organisations
International organizations
- World health organization (WHO): guidelines and policies for global health
- International council for harmonization of technical requirements for pharmaceuticals for
human use (ICH) – harmonization of pharmaceutical research and registration
->ICH: everyone has to use the same: 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)
2
,At KU Leuven
- One legal entity and three operational subentities
- KUleuven functions as one legal entity, but in practice it is composed of three distinct subentities, each
with its own responsibilities, infrastructure, ad role in research, clinical trials and valorisation.
UZ LEUVEN KU LEUVEN LRD – Leuven Research and
->hospital and patients ->education and research development
->valorisation
- Clinical trial centre – CTC - Research coordination - Technology transfer
- Biobank office – DOC office (TTO)
- Several hospital - It acts as the bridge
campuses (gasthuisberg, between academic
sint raf, pellenberg) research and industry
- Responsible for patient
care and the clinical
execution of research
->ethics committee: it is an independent committee although it is closely connected to research activities
Ethics committees or bodies
There are lots of ethical committees present in the university
->important, we will be confronted with them
->we need to know the different types
Committee/body Full name Scope and focus Typical research/ activities reviewed
SMEC Social and societal Ethical review of social, - Surveys
ethics committee behavioural, and non - Interviews
medical human research - Psychology studies
- Humanities research
- Studies with healthy volunteers
- (non medical)
EC research UZ/ Medical- scientific Ethical review of medical, - Clinical trials
KU Leuven ethics committee clinical, and interventional - Medical devices
research involving humans - Drug studies
- Diagnostic studies
- Interventional procedures
EC care Ethics committee Ethical aspects of patient - Clinical care dilemmas
care care and use of donated - Use of human tissue
human biological material - Biobank-related decisions
- End-of-life care issues
ECD Ethical committee for Ethical review of animal - Animal studies
animal research - In vivo experiments
experimentation - Harm-benefit analysis for animal
use
EC DMM Ethical committee on Research with dual-use - Be weaponized
dual use, military use potential, military relevance, - Surveillance technologies
and misuse or risk of misuse - Biological agents
- Cybersecurity tools
OBC Education and Ethical support for master’s - Student research projects
support committee theses (especially - Small scale studies
biomedical sciences) - Methodological and ethical
guidance
PRET Team Privacy and ethics Data protection, GDPR - Data management plan
team compliance, privacy review - GDPR checks
(not a formal ethics - Data-sharing agreements
committee) - Privacy risk assessments
3
, Regulatory authorities
National level European level International level
FAMHP/FAGG European medicines agency EMA WHO
- Central evaluation and authorization of medicines
- If companies want to bring a product on the market
in Europe, they need to pass the EMA
European commission
-> DG SANTE
- EU legislation and health policy
- They organize the funding programs
- The big funding that can go to research
- They make the rules at the European level
Heads of medicines agencies
->HMA
- EU network of national authorities
- National medicines agencies can talk to each other,
how it is in the other countries so they can learn from
each other
European directorate for the quality of medicines and
healthcare EDQM
- Quality standards for medicines and healthcare
European centre for disease prevention and control ECDC
- Infectious disease prevention and control
Payers
->sometimes a product Is on the market, but is to expensive, that is why there is a system of payers
needed
It is a system that is organized on the national levels
- Because taxes are also decided on national levels
- Inequalities in patient access, because some countries cant afford
In Belgium there are different types of payers:
1. National public Payer – INAMI/RIZIV
- Main national health insurance institution
- Sets reimbursement rules for therapies, medicines, and medical procedures
- Evaluates cost-effectiveness and clinical benefit
2. Sickness Funds (mutualiteiten) – the bridge
- Administer health coverage for citizens
- Collect contributions and reimburse care according to INAMI/ RIZIV regulations
- Serve as the link between patients and national reimbursement system
3. Private insurance companies
- Complementary or supplementary coverage
- Limited role in standard therapies
- Sometimes used for experimental or high-cost treatments
Patient and citizens
- Individual patient
→ They have experience with illness and receiving care
→ They can share their personal experiences with disease, treatment and quality of life
→ There experience is essential for understanding real patient needs
4
INTRODUCTION – Isabelle Huys
Stakeholders
The biomedical ecosystem
Research and innovation Patient Society
->diagnosis and treatment ->health and community
- Experiments - Diagnosis - Prevention
- Discoveries - Therapy - Public health
- Preclinical research - Care - Policy
From laboratory -> to the patient -> to society
Stakeholders in the biomedical domain
Health care professionals
- They detect clinical needs and bring patients to the researchers
- Perform clinical studies
- Translate research results into practical clinical health care
- Bridge between science and patient-care
Researchers
- Central place – startpoint of innovation
- Very important position in the whole translation from findings from the lab to the clinic
- Identify new targets
- Pre-clinical research
- Proof-of-concept (to bring a therapy to the clinical development)
Patients
- They deliver important input about the quality of life, needs and feasibility and acceptance of therapy
Industry (also researchers in the industry!)
- Different types of industry
- They scale up what is found in the lab, more production is possible
- They manufacture products
1
,Regulatory authorities (ex. EMA, FDA, FAGG)
- They define the standards, which the research need to occur
- The research must conduct and accord to the rules
->they all work very closely together
Researchers
Examples of types of researchers
Academic
- Fundamental research
- Hypothesis diven
- Mechanisms and preclinical models
Clinical
- Patient-oriented research
- Clinical studies
- Diagnostics and treatment
Translational
- Bridge between lab and clinic
- Validation and proof-of-concept
- Preparation for clinical application
- They work with human material
- They translate what is in the lab and want to validate it into clinical
Universities
Research institutes (IMEC, VIB, VITO, EORTC, …)
Health care professionals – HCPs
->hospitals and private entities
- Doctors/physicians
- Nurses
- Allied health professionals
- Pharmacists
- Mental health professionals
- Public health and administrative professionals
Researcher/HCPs & scientific organisations
International organizations
- World health organization (WHO): guidelines and policies for global health
- International council for harmonization of technical requirements for pharmaceuticals for
human use (ICH) – harmonization of pharmaceutical research and registration
->ICH: everyone has to use the same: 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)
2
,At KU Leuven
- One legal entity and three operational subentities
- KUleuven functions as one legal entity, but in practice it is composed of three distinct subentities, each
with its own responsibilities, infrastructure, ad role in research, clinical trials and valorisation.
UZ LEUVEN KU LEUVEN LRD – Leuven Research and
->hospital and patients ->education and research development
->valorisation
- Clinical trial centre – CTC - Research coordination - Technology transfer
- Biobank office – DOC office (TTO)
- Several hospital - It acts as the bridge
campuses (gasthuisberg, between academic
sint raf, pellenberg) research and industry
- Responsible for patient
care and the clinical
execution of research
->ethics committee: it is an independent committee although it is closely connected to research activities
Ethics committees or bodies
There are lots of ethical committees present in the university
->important, we will be confronted with them
->we need to know the different types
Committee/body Full name Scope and focus Typical research/ activities reviewed
SMEC Social and societal Ethical review of social, - Surveys
ethics committee behavioural, and non - Interviews
medical human research - Psychology studies
- Humanities research
- Studies with healthy volunteers
- (non medical)
EC research UZ/ Medical- scientific Ethical review of medical, - Clinical trials
KU Leuven ethics committee clinical, and interventional - Medical devices
research involving humans - Drug studies
- Diagnostic studies
- Interventional procedures
EC care Ethics committee Ethical aspects of patient - Clinical care dilemmas
care care and use of donated - Use of human tissue
human biological material - Biobank-related decisions
- End-of-life care issues
ECD Ethical committee for Ethical review of animal - Animal studies
animal research - In vivo experiments
experimentation - Harm-benefit analysis for animal
use
EC DMM Ethical committee on Research with dual-use - Be weaponized
dual use, military use potential, military relevance, - Surveillance technologies
and misuse or risk of misuse - Biological agents
- Cybersecurity tools
OBC Education and Ethical support for master’s - Student research projects
support committee theses (especially - Small scale studies
biomedical sciences) - Methodological and ethical
guidance
PRET Team Privacy and ethics Data protection, GDPR - Data management plan
team compliance, privacy review - GDPR checks
(not a formal ethics - Data-sharing agreements
committee) - Privacy risk assessments
3
, Regulatory authorities
National level European level International level
FAMHP/FAGG European medicines agency EMA WHO
- Central evaluation and authorization of medicines
- If companies want to bring a product on the market
in Europe, they need to pass the EMA
European commission
-> DG SANTE
- EU legislation and health policy
- They organize the funding programs
- The big funding that can go to research
- They make the rules at the European level
Heads of medicines agencies
->HMA
- EU network of national authorities
- National medicines agencies can talk to each other,
how it is in the other countries so they can learn from
each other
European directorate for the quality of medicines and
healthcare EDQM
- Quality standards for medicines and healthcare
European centre for disease prevention and control ECDC
- Infectious disease prevention and control
Payers
->sometimes a product Is on the market, but is to expensive, that is why there is a system of payers
needed
It is a system that is organized on the national levels
- Because taxes are also decided on national levels
- Inequalities in patient access, because some countries cant afford
In Belgium there are different types of payers:
1. National public Payer – INAMI/RIZIV
- Main national health insurance institution
- Sets reimbursement rules for therapies, medicines, and medical procedures
- Evaluates cost-effectiveness and clinical benefit
2. Sickness Funds (mutualiteiten) – the bridge
- Administer health coverage for citizens
- Collect contributions and reimburse care according to INAMI/ RIZIV regulations
- Serve as the link between patients and national reimbursement system
3. Private insurance companies
- Complementary or supplementary coverage
- Limited role in standard therapies
- Sometimes used for experimental or high-cost treatments
Patient and citizens
- Individual patient
→ They have experience with illness and receiving care
→ They can share their personal experiences with disease, treatment and quality of life
→ There experience is essential for understanding real patient needs
4
