L1

Question 1

Why do BTFs exist?

Answer 1

transaction costs in absence of a firm would be too high

Question 2

What is efficacy?

Answer 2

How well the drug works in controlled conditions in a clinical trail

Question 3

What is effectiveness?

Answer 3

How well the drug works in the real world on patients

Question 4

What is a key characteristic of modern drugs?

Answer 4

Precision eg immunotherapy

Question 5

What 5 things go into strategy?

Answer 5

time
risk
pricing
convergence
precision

Question 6

What are 4 qualities of the BP?

Answer 6

safe
efficacy
quality
price

Question 7

What happens if the precise drug enters a market quicker than normal?

Answer 7

more likely for S/Es to occur

Question 8

What is convergence?

Answer 8

need diagnostic equipment to identify genetic material in individuals for the precise drug to be effective

Question 9

What is the Centocoor case?

Answer 9

15/04/1992 - FDA does not approve nebacumab developed by CENTOCOOR ( founded 1979) - not to commercialize mAbs

Question 10

What did CENTOCOOR develop?

Answer 10

Developed 7 mAbs including HA-1A from stanford against gram -ve bacteria

Question 11

Define raising?

Answer 11

Receiving money from investors

Question 12

What is IND?

Answer 12

investigation into new drug in US

Question 13

What is CTA?

Answer 13

clinical trail application in EU

Question 14

How did CENTOXIN get on in its clinical trail?

Answer 14

reduced mortality in pts by 47% who went into septic shock & GNS by 39%

Question 15

What is NDA?

Answer 15

new drug application to FDA

Question 16

What is MAA?

Answer 16

marketing authorisation application to EMA

Question 17

What is SAC?

Answer 17

scientific advisory committe of FDA

Question 18

What was CENTOCOORs downfall?

Answer 18

First european sales were below expectations - NE journal of med - not able to reproduce
Nov 1991 - showed leathality in dogs by NIH trails & inability to treat septic shock