1.2 Developing Medicines Flashcards
Name the phases of drug discovery
Idea/concept phase Feasibility Target validation Identification of potential compounds-leads Selection of candidate drug
How is HIT identification done?
High throughput screening to obtain a starting point
What stage follows on from HIT identification?
Lead identification
What processes occur in lead optimisation?
Increase potency
Optimise selectivity
Optimise physical properties and Pharmacokinetics
Ensure efficacy in disease models to give confidence for efficacy in man
What are some examples of toxicology tests?
Genetic toxicology Safety pharmacology General toxicology Reproductive toxicology Carcinogenicity
Describe phase I studies
First into man - SAD and MAD
Small number of HEALTHY volunteers
Investigates pharmacokinetics and evaluates safety
What’s the typical volunteer for phase I studies like ?
Usually healthy young men
Not taking any medication
Able to give written consent
Name some methods of volunteer recruitment for clinical trials
Newspapers
Internet
Radio
Poster displays
What principles underline consent in trials?
Use guiding ethical principles
Voluntary
Free to withdraw consent at any time
What 4 things are key to the ethics of clinical trials?
Informed consent
Efficient scientific design
Competent authority/legislation
Research ethics committee
What are the four principles of ethics?
Justice - being fair
Beneficence - doing good
Non-maleficence - not doing harm
Autonomy - allowing individual to determine what happens to him or her
Describe phase 2 studies
Target patient with disease
Involve up to 400 patients
What is a phase IIa study?
Pilot study for dose finding
What is a phase IIb study?
Measures therapeutic action and determines dose for phase III
Why is a phase II study patient unlike most other patients?
They have the disease but don’t have anything else wrong with them
Describe a phase III study
Involve patients with a disease
Large multi centre RCT - usually thousands
Last several years
Determines efficacy against standard practice/placebo
Required for regulation and marketing
What are phase IV studies?
Large scale clinical studies or pharmacovigilance for long term safety
Based on seeking new indications for product
Often outcome based
How many volunteers in phase 1 studies?
50 healthy volunteers
How many volunteers in phase 2 studies
200-400 patients with the disease (but no other conditions)
How many volunteers in phase 3 studies?
1000-3000 patients with the disease
How many patients involved in phase 4 studies?
<10,000
