Con ten ts
01 Evidence based medicine
02 Study design
03 Systematic reviews
04 Mastering the basics of clinical study designs
05 Animal research – why not patients
06 Tissue engineering (TE)
07 Writing an abstract
08 Introduction regulation medical research (general and NL)
09 Informed consent and shared decision making
10 Practical: cases on informed consent
11 Introduction to ethics of PM, data and biobanking
12 Legal aspects of biobanking
13 Ethics of big data/AI in medicine
14 Legal challenges of AI in healthcare
15 Personalized trial design
16 Personalized trial outcomes
17 Innovative study designs
18 Personalized medicine in severe asthma
19 Stepped wedge design
20 Adaptive trial design
21 Delphi technique
22 Informed consent practical
, lecture 01
Evidence based m edicine
Basics of Clinical Research & Regulations
Watch movie on empirical cycle!
Research is important for development and innovation. Don’t learn the years!
Empirical research
= a methodology in research in which data is collected based on observations to gather knowledge and
test hypotheses.
An evidence based approach to ensure high quality research. Ensures research is systematic,
structured, and evidence based. Can be quantitative or qualitive.
Empirical cycle (make sure you know all 5 steps!!!!)
1. Observation -> Do an observation and generate an hypothesis from it.
2. Induction -> Conclusion form the observation. Formulate a broader hypothesis.
3. Deduction -> Process of taking a general hypothesis and making specific predictions that can be
test. What are you going to focus on?
4. Testing -> use empirical methods to test your hypothesis. What experimental setup do I need to
test the hypothesis?
5. Evaluation -> analyze results and draw conclusions. Theories might be refined, accepted, or
rejected. Can start a new cycle, where this would be the observation.
Practice: what you can expect on EXAM!!!!!!!!!!!!!!!
See advantages and disadvantages of empirical research. Be aware there are also disadvantages!
Evidence based medicine (EBM)
Medicine practiced on the basis of high-quality evidence.
Fundamentals
- Clinical experience from medical doctors
- Patient values
- Best research evidence
5 steps for EBM
1. Ask -> define clinically relevant question
2. Acquire -> search for best evidence
3. Appraise -> assess quality of evidence
4. Apply -> act on evidence
5. Assess -> to evaluate the process
Medical ethics mostly based on Tuskegee study. Study on African men with syphilis to see the effects of
the disease when untreated. They were not told they would be left untreated. Lot of men died. We need
some rules on informed consent.
Levels of evidence
Clinical practice guidelines > systematic analysis > RCT > cohort > case control > case report >
animal/laboratory.
Troubling aspects of EBM
Argue medical practice was too subjective. This view of the pyramid (?) is abandoned, but….
Criticism EBM
- P-values
- Absence of n of 1 trials -> considered best, but hard to carry out.
- Internal and external validity within systematic reviews. When they are done, usually it is in a very
homogenous population.
o Publication bias.
- Clinical expertise and patient values are sidelined.
, - Reimbursements are tied to guidelines.
- Physicians ignorance of statistics -> lack of knowledge on how to interpret the data.
PICO
Trick to make sure you formulate hypothesis/RQ in the right way. See example.
Patient/population
Intervention/interest
Comparison/control
Outcome
Time of observation
Type of question/study (teacher never uses)
Rq: What is the effect of I on O compared to C in P
Hypothesis: P O following I compared to C.
Know how to phrase them separately from each other!
01 Evidence based medicine
02 Study design
03 Systematic reviews
04 Mastering the basics of clinical study designs
05 Animal research – why not patients
06 Tissue engineering (TE)
07 Writing an abstract
08 Introduction regulation medical research (general and NL)
09 Informed consent and shared decision making
10 Practical: cases on informed consent
11 Introduction to ethics of PM, data and biobanking
12 Legal aspects of biobanking
13 Ethics of big data/AI in medicine
14 Legal challenges of AI in healthcare
15 Personalized trial design
16 Personalized trial outcomes
17 Innovative study designs
18 Personalized medicine in severe asthma
19 Stepped wedge design
20 Adaptive trial design
21 Delphi technique
22 Informed consent practical
, lecture 01
Evidence based m edicine
Basics of Clinical Research & Regulations
Watch movie on empirical cycle!
Research is important for development and innovation. Don’t learn the years!
Empirical research
= a methodology in research in which data is collected based on observations to gather knowledge and
test hypotheses.
An evidence based approach to ensure high quality research. Ensures research is systematic,
structured, and evidence based. Can be quantitative or qualitive.
Empirical cycle (make sure you know all 5 steps!!!!)
1. Observation -> Do an observation and generate an hypothesis from it.
2. Induction -> Conclusion form the observation. Formulate a broader hypothesis.
3. Deduction -> Process of taking a general hypothesis and making specific predictions that can be
test. What are you going to focus on?
4. Testing -> use empirical methods to test your hypothesis. What experimental setup do I need to
test the hypothesis?
5. Evaluation -> analyze results and draw conclusions. Theories might be refined, accepted, or
rejected. Can start a new cycle, where this would be the observation.
Practice: what you can expect on EXAM!!!!!!!!!!!!!!!
See advantages and disadvantages of empirical research. Be aware there are also disadvantages!
Evidence based medicine (EBM)
Medicine practiced on the basis of high-quality evidence.
Fundamentals
- Clinical experience from medical doctors
- Patient values
- Best research evidence
5 steps for EBM
1. Ask -> define clinically relevant question
2. Acquire -> search for best evidence
3. Appraise -> assess quality of evidence
4. Apply -> act on evidence
5. Assess -> to evaluate the process
Medical ethics mostly based on Tuskegee study. Study on African men with syphilis to see the effects of
the disease when untreated. They were not told they would be left untreated. Lot of men died. We need
some rules on informed consent.
Levels of evidence
Clinical practice guidelines > systematic analysis > RCT > cohort > case control > case report >
animal/laboratory.
Troubling aspects of EBM
Argue medical practice was too subjective. This view of the pyramid (?) is abandoned, but….
Criticism EBM
- P-values
- Absence of n of 1 trials -> considered best, but hard to carry out.
- Internal and external validity within systematic reviews. When they are done, usually it is in a very
homogenous population.
o Publication bias.
- Clinical expertise and patient values are sidelined.
, - Reimbursements are tied to guidelines.
- Physicians ignorance of statistics -> lack of knowledge on how to interpret the data.
PICO
Trick to make sure you formulate hypothesis/RQ in the right way. See example.
Patient/population
Intervention/interest
Comparison/control
Outcome
Time of observation
Type of question/study (teacher never uses)
Rq: What is the effect of I on O compared to C in P
Hypothesis: P O following I compared to C.
Know how to phrase them separately from each other!
