Intro To Drug Development

Question 1

Timeline for drug development

Answer 1

10-15 years timeline

Question 2

Drug target

Answer 2

A molecule that the drug needs to find and act upon, often it is a protein molecule eg. -enzyme

Question 3

Drivers for discovering new drugs

Answer 3

Medical need
Disease prevalence
Technical feasibility
Research and development cost
Commercial considerations

Question 4

What happens in Pre-discovery?

Answer 4

New information on disease process/psychological mechanism
Existing treatment with unexpected effects
New technologies that allow treatment to be more specific

Question 5

Sources of medicine:

Answer 5

Plants, fungus, marine life, venom etc
Synthetically made in lab

Question 6

Why pre-clinical studies?

Answer 6

To develop adequate evidence to decide that it is reasonably safe and effective to proceed with human trials of the drug

Question 7

In vitro and example

Answer 7

Outside a living organism eg. Biological essay

Question 8

In viva and example

Answer 8

Inside a living organism eg-mice

Question 9

High throughput is a process that?

Answer 9

that allows automated screening of large numbers ofchemical/biological compounds for a specific biological target

Question 10

Quality assurance (QA) programs that ensure high quality and integrity of non-clinical and clinical research conducted

Answer 10

GLP- good lab practice
GMP -good manufacturing practice
GCP - good clinical practice

Question 11

Main aim of clinical research

Answer 11

Is it safe in humans? Is it effective?

Question 12

Describe phase1 clinical trial

Answer 12

• Small number of healthy volunteers
  Researchers confirm safety and tolerability
  Researchers start with small doses and escalate according to the tolerability and safety

Question 13

Describe phase 2 clinical trials

Answer 13

-Tested on a relatively small group of the intended disease population
Get a better idea of efficacy and safety

Question 14

Phase 3 clinical trials - describe

Answer 14

• tested in a larger group of disease target population and compared against an existing treatment/placebo
  Often Last a year or more involve 7,000 patients

Question 15

Bioavailability

Answer 15

% Of drug dose reaching the systemic circulation

Question 16

Post market surveillance is for?

Answer 16

Identification and collection of information regarding drugs after their approval for use in a population

Question 17

Post market surveillance in what phase?

Answer 17

Phase 4

Question 18

Role of marketing authorisation

Answer 18

Proposed labelling
Patient information sheet

Question 19

Challenges in drug development

Answer 19

Over 90% of drugs will not make it to the market
Lengthy and complex costly process , with a high degree of uncertainty that the drug will succeed
Animal models often can’t recapitulate an entire disease
Challenges related to heterogeneity
Molecules- become more complex, poor solubility and bioavailability