Lecture 1: 27/9
General info
- Steven Van Cruchten & Peter Delpu4e
- 24th nov.: 2 experts come to speak
- You need some knowledge of pharmacology
- Main aim: in the end there is a seminar where we should be able to interpret a drug insert
o Drug insert (= document with all the criIcal informaIon of the drug) is import for the
physicians and they will relay on it so all the criIcal informaIon need to be in it
- It is important to connect all the topics
- Non clinical studies will become for clinical studies (it will proceed a clinical phase)
o Before you can do a study in human volunteers (phase 1) you will need a non-clinical
package. If you then want to do a study in paIents you will need to do a other non-
clinical studies.
o You will guide the clinical studies in a stepwise approach
o Target populaIon? How long will you aim to treat them -> this need to be clear in the
beginning and you can’t change that during the course of the process
- Wri4en examinaIon
Introduc0on
Phases is drug discovery and development
- Can take a very long Ime
o During covid we were spoiled
o Can be 12 years from your molecule to the drug -> costly
- Molecules is a very variable term
o Small molecular enIIes, like aspirin
o For several diseases small molecule drugs are not the right opIon -> bio
pharmaceuIcals
§ He uses the term new therapeuIcal modaliIes (covering everything that you can
imaging regarding drug development) (also gene therapy)
• Large -> difficult to reach the target => parental through injecIons
• It is also oZen difficult to find the right model to predict what you will see
in the human situaIon
o Natural products
§ He will not address this
Drug R&D
R&D spending in different industry sectors
- If you working in the biopharmaceuIcal field this is very expensive (large cost)
o Rare diseases -> you will earn less when you sell a product for a rare disease than when
you sell a drug for a common disease
§ In some cases (small target populaIon) the price needs to be high because it cost
a lot to make the drug
, R&D spending & therapeu9c areas
- Spending is increasing a lot
- Approving is going down -> needs to be compensated
- FDA 2019 small molecule drug approvals
o a lot of the drugs are against cancer, because we have many types
§ ib: inhibitor
- Japan: smaller country so the expenditure is smaller
- Full cost of a drug: over the years has it become more expensive to get a drug on the market
o More than 1 billion dollars -> explains why companies want to gain money with the drug
o If they don’t proceed with the producIon it is important they do this early = fast, fail
cheap
- Very clear that a bulk of the cost go to the clinical trials
o Phase 3 has a mulIsite environment -> mulIple faciliIes around the world (and the
locaIon depends on where you find paIents)
§ SomeImes hard to recruite paIents
o Phase 1 clinical trials: in healthy persons, oZen male students
§ No female students: companies don’t want to risk that women will be inferIle or
that you will expose the foetus (if she don’t know she is pregnant)
• The risk on issues with the male volunteer is less
• + you need to collect less non clinical data for the men
- AnIviral treatments are hot topic now
General info
- Steven Van Cruchten & Peter Delpu4e
- 24th nov.: 2 experts come to speak
- You need some knowledge of pharmacology
- Main aim: in the end there is a seminar where we should be able to interpret a drug insert
o Drug insert (= document with all the criIcal informaIon of the drug) is import for the
physicians and they will relay on it so all the criIcal informaIon need to be in it
- It is important to connect all the topics
- Non clinical studies will become for clinical studies (it will proceed a clinical phase)
o Before you can do a study in human volunteers (phase 1) you will need a non-clinical
package. If you then want to do a study in paIents you will need to do a other non-
clinical studies.
o You will guide the clinical studies in a stepwise approach
o Target populaIon? How long will you aim to treat them -> this need to be clear in the
beginning and you can’t change that during the course of the process
- Wri4en examinaIon
Introduc0on
Phases is drug discovery and development
- Can take a very long Ime
o During covid we were spoiled
o Can be 12 years from your molecule to the drug -> costly
- Molecules is a very variable term
o Small molecular enIIes, like aspirin
o For several diseases small molecule drugs are not the right opIon -> bio
pharmaceuIcals
§ He uses the term new therapeuIcal modaliIes (covering everything that you can
imaging regarding drug development) (also gene therapy)
• Large -> difficult to reach the target => parental through injecIons
• It is also oZen difficult to find the right model to predict what you will see
in the human situaIon
o Natural products
§ He will not address this
Drug R&D
R&D spending in different industry sectors
- If you working in the biopharmaceuIcal field this is very expensive (large cost)
o Rare diseases -> you will earn less when you sell a product for a rare disease than when
you sell a drug for a common disease
§ In some cases (small target populaIon) the price needs to be high because it cost
a lot to make the drug
, R&D spending & therapeu9c areas
- Spending is increasing a lot
- Approving is going down -> needs to be compensated
- FDA 2019 small molecule drug approvals
o a lot of the drugs are against cancer, because we have many types
§ ib: inhibitor
- Japan: smaller country so the expenditure is smaller
- Full cost of a drug: over the years has it become more expensive to get a drug on the market
o More than 1 billion dollars -> explains why companies want to gain money with the drug
o If they don’t proceed with the producIon it is important they do this early = fast, fail
cheap
- Very clear that a bulk of the cost go to the clinical trials
o Phase 3 has a mulIsite environment -> mulIple faciliIes around the world (and the
locaIon depends on where you find paIents)
§ SomeImes hard to recruite paIents
o Phase 1 clinical trials: in healthy persons, oZen male students
§ No female students: companies don’t want to risk that women will be inferIle or
that you will expose the foetus (if she don’t know she is pregnant)
• The risk on issues with the male volunteer is less
• + you need to collect less non clinical data for the men
- AnIviral treatments are hot topic now
