clinical drug development Flashcards

Question

non inferiority trial

Answer 1

phase 3 and you show that the new treatment is at least as effective as. It should not be worse, it might even be better but you don't check that. One sided testing then and easier to rpove

Answer 2

phase 3 and you show that the new treatment is at least as effective as. It should not be worse, it might even be better but you don't check that. One sided testing then and easier to rpove

Answer 3

investigate the effectiveness of drug in real life and the safety too in the context of pharmacovigilance. I trie to get the best real possible complete picture of benefit and risk of the drug

Answer 4

a pseudo clinical trial designed by the company that is put in place to promote the use of a product that was recently approved. They try too appear like they want to answer a scientific question but it is really more of a marketing strategy to get the physician to prescribe the drug

Answer 5

objective and measurable endpoint such as death or survival or decreased morbidity

Answer 6

they are parameters more something that have to be measured such as a biomarker. Something related to a clinical endpoint with a sort of predictive parameter that can replace the clinical endpoints when you try to show your drug is better than the existing one, ou take surrogate endpoint to how it. Advantage too cause it is quicker to get these information than morbidity

Answer 7

describe how patient feel function survive such as quality of life survival morbidity mortality etc. depending upon what you think the most important are for this drug, you can refer to certain endpoints as being primary or secondary

Answer 8

standard of care

Answer 9

randomized controlled trial it is prospective and analytical and experimental studies it is the most convincing one advantages is that they can be controlled for the unknown and unmeasurable cofounder, very powerful in terms of statistics. however you a some limitation such as the number of participant, the exposure and the follow up there are some inclusion nd exclusion criteria (limit external validity) artificial expensive ethical etc so don't forget these

Answer 10

Interactive voice response system

Answer 11

negative expectation resulting in undesirable effect

Answer 12

difference of effect between the real compound and placebo

Answer 13

EXPLORATory clinical trial application part of the early development before/part of phase 1 limited non-clinical toxicology so very limited human exposure no therapeutic intend no intention to loof at MTD MRSDA divided by 50 allow earlier differentiation of promising compounds expose human earlier reduced cost of closed medicine fewer studies and animals microdosing 500 ug less you can use it to look at the biodistribution and receptor occupancy using PET PK grounds carbon 14 plasma concentration tissue distribution and R occupancy

Answer 14

one disease but we look for different possible therapy (breast cancer or covid)

Answer 15

here it is one therapy that is tested in different disease (cancer too). Eg tissue/tumor agnostic treatment so they ignore the type of tissue where the tumor is occurring and just focus on the type of mutation.

Answer 16

umbrella and kucket approach together. you think about all the different combination possible and therefore taste more combination more efficiently and quicker

Answer 17

proof of principle

Answer 18

developmental and reproductive toxicity

Answer 19

is randomization of people in two groups that are given another treatment for the whole duration of the trial the advantages are that this can be long lasting, easy to perform and anlyse however, you introduce a large variability because the two groups are technically not identical so you really need a big number of participant to get over this.

Answer 20

you divide in two groups and give them a certain treatment. After x amount of time you stop the treatment, allow for a washout time and exchange the treatment or no treatment between the group. This reduces the number of participants and variability, as every subject has its own control. However, there are different problem such as the period effect of disease which is when the disease itself is not sufficiently stable and does not allow to perform a cross over design some subject could die during the first part, so you need people that will be ok over the two parts of the treatment you also have the carry over effect of R - it is when participant in the first group that get the drug get better or cured you cannot really switch them to the other treatment the washout period can also be very comp^licated (for example not done with AB) as people need to get back to the baseline very complicated analyse

Answer 21

exploratory investigational new drug

Answer 22

are a last alternative approach to increase the efficiency of drug development at the beginning, it was really one treatment one disease one protocol. The new approach tries to maximise the number of questions answered in a shorter time they design overarching protocol to answer multiples question at the same time such as for precision medicine, in antibiotic research or oncology it can be really complicated and expensive to make a new protocol every time for each different mutation responsible for a certain type of breast cancer so we try to bring everything in one protocol it includes the umbrella/platform trial, the basket. bucket trial and adaptive design the advantages are that it uses a common screening platform there is 1 overlooking organisation, it uses a network of experiences clinical centres, you can use the same controls group however it does cost a lot of time and money, it requires a complex organisation and the start up time can be long

Answer 23

it is about 1 signle case/observation that is unusual and rare. You can then challenge dechallenge rechallenge to observe the relationship between exposure and consequence advantages is that is it cheap and hypothesis generating, as well as easy however it give little evidence on causalityn you can't test these hypothesis pbservational and descriptive

Answer 24

when you are pooling diffferent cases reports with a comparable history - you try to get a description of a characteristic and outcome among a group: its great as it can estimate the number of incidences (rule of three) and hypothesis generating. However you again have little evidence on causality, you have no control population to compare with and no hypothesis testing it is again observational and descriptive

Answer 25

observational and analytical you compare cases with control and you look at the differences in preceding exposure - so retrospective you know who has the disease and who not and you look at how is the exposure different you then have the risk in odds ratio adv are that you can investigate rare and lates response, can check for multiple exposure at the tiam and is easy quick cheap however it is sensitive to bias in terms of selection, information, recall and you also can get some confounding effect

Answer 26

are observational and analytical. However, this one involves a cohort that will be followed over time from exposure to a consequence. it can be prospective, follow up over time it can be retrospective and ambidirectional too. adv is that multiple responses can be investigated like smoking can give COPD/Lung cancer, you can check for rare exposure and you get information on incidence selection bias is less likely neg is that you need a very large group, it is also sensitive to bias on outcome data and it is very long experiment

Answer 27

it is simple to use and has historical evidence based however the approach is typically done on small molecule such as NCE ignore step 5 and too simple allomertric calling

Answer 28

minimal effective dose

Answer 29

also allows to predict PD does the binding predict a good response

clinical drug development Flashcards

(53 cards)