Lecture 3

Question 1

what is clinical research?

Answer 1

-medical research that involves people like us it involves human participants and helps translate basic research into new treatments to gain insight on how disease and health progress

Question 2

volunteer participation in investigations with the objective of……

Answer 2

discovering better ways to treat, diagnose and understand human disease

Question 3

clinical trials are sponsored by what?

Answer 3

-by various organizations like physicians, foundations, medical institution and pharmaceutical companies

Question 4

what is randomization?

Answer 4

process by which treatments are assigned to participants by chance to avoid any bias aka double blind

Question 5

what do clinical trials compare

Answer 5

they compare new therapy with existing therapy to determine if the new is as better or better than the existing treatment

Question 6

true or false in all studies there is a placebo

Answer 6

false it is in some

Question 7

why is there unique eligibility criterias in each study?

Answer 7

to identify appropriate participants and keep then safe and to ensure researchers xan find the new information that they need

Question 8

all trials have guidelines about who can participate called the…..

Answer 8

inclusion/exclusion criteria

Question 9

what are criteria based on…

Answer 9

age, gender. stage of disease. current medications, previous treatment and other medical conditions

Question 10

what are the main infos on the 4 phases?

Answer 10

-is it safe
-does it do what it is supposed to do
-how does it compare
-what happens in the long term

Question 11

Phase 1: is it safe? purpose and number of peeps

Answer 11

-find out whether a medical approach is safe and can be tolerated: we assess side affects

Question 12

phase 2: does it do what it is supposed to do: purpose and number of peeps

Answer 12

-start testing whether a medical approach works. Continue monitoring for side effects, determine opportune dose and collect data
-between 100-300 peeps

Question 13

phase 3: how does it compare? purpose and peeps

Answer 13

-confirm whether a medical approach works: continue monitoring side effects
-usually 1000 or more

Question 14

phase 4 what happens in the long term: purpose and number of peeps

Answer 14

-when a medical approach goes to a market, continue gathering info for long term effects
-number of peeps: thousands

Question 15

why participate to a study

Answer 15

-gain access to new research treatments before widely available
-receive regular and careful medical attention from a research team
-help other by contrinuting to medical research
-play an active role in your own health care

Question 16

what are adaptive trial designs

Answer 16

-basically you can put phase 1-2 together and 3-4
-allos trial flexibility by using accumulated results

Question 17

true or false: adaptive trial designs are more efficient, informative and ethical

Answer 17

true
they use less participants and better use of time and money

Question 18

what is a part of the clinical research uni

Answer 18

-neurologists
-raters: questionnaires
-nurses: blood draws and vitals
-imaging center aka mri

Question 19

what is nusinersen used for

Answer 19

treatment of spinal muscular atrophy

Question 20

what is tofersen used for

Answer 20

as for a rare form of ALS: usually hits older people

Question 21

what is the gene for ALS

Answer 21

sod1 and it is genetic

Question 22

why was valor not a slay for tofersen

Answer 22

-thet thought it would be a huge slay
-They got fucked and failed to meet primary disease
-Indicators of the disease were getting better tho
-People that were normal progressors are regaining normal function
-Mistake was on the clinical designs
-They got recovery from the disease

Question 23

how does insurance works after the drug is approved by health canada

Answer 23

After drug approved by canada our province has to decide if it is gonna be available for us and through insurances and if its in hospitals