Clinical Trials (Q4)

Timeline of drug development

1. Drug discovery

2. Preclinical development

3. Clinical development

4. Regulatory approval

Aspects of drug discovery

Target selection

Lead-finding

Load optimisation

Pharmacological profiling

Duration of drug discovery

2 to 5 years

With approximately 100 projects

Ends when a drug candidate is found

Aspects of preclinical development

Pharmacokinetics

Short-term toxicology

Formulation

Synthesis scale-up

Very expensive

Duration of preclinical development

2-5 years

With 20 compounds

Ends with development compound decided

Overview of clinical development

5.7 years

involves humans following animal testing

four stages (phase I-IV)

Safety testing

Acute toxicity

Sub-acute toxicity

Chronic toxicity

Reproductive toxicity

Carcinogenicity

Mutagenicity

Goals of phase I clinical development

Identify proper dosing and side effects in healthy volunteers.

Aspects of phase I clinical development

20-80 healthy volunteers

Investigates safety, tolerability (adverse effects?), pharmacokinetics (ADME), pharmacodynamics (fulfils purpose?)

Goals of phase II clinical development

Provides preliminary information on how well the treatment works in a clinical setting.

Aspects of phase II clinical development

100-300 patients

Testing of dosage and efficacy

Long term toxicology studies

Given to terminally ill patients (cancer drugs)

Goals of phase III clinical development

Compare the new drug with existing treatments

Must show some benefit compared to older formulations

Aspects of phase III clinical development

1000-10,000 patients (up to 30,000)

Cost-effective

Randomised multicentre double-blind trials

Confirms data from phase II in large group

Goals of phase IV clinical development

Continuing evaluation once on the market

New indications can be pursued

Detection of long term or rare side effects