What are the 6 main steps in developing a drug and the rough time course of each step?
- Discovery research- 4yrs
- Phase 1 (50 healthy volunteers used to study drugs pharamcokinetics)- 1 yr
- phase 2 (200-400 pts with disease to study its effect)- 2 yrs
- phase 3 (1000-3000 pts to evaulate efficacy in wider population and study long term safety) - 4 yrs
- regulatory review- 1 yr
- phase 4 (drug can be prescribed, 10,000 pts, effects monitored closely)
What is assesed about a compound after it is identified as potentially useful?
- potentcy and selectivity
- Pk and metabolism
- toxicity
- cost of synthesis
- chemical properties and technical issues
What us high throughput screening?
automated testing of compounds from compound banks to see if theyre effective
What is the name for when a drug is identified as effective from high throughput screening?
a hit
What is lead identification?
turning a hit into a lead by evaluating and optimising the physcial properties, selectivity, ADME, synthesis and potency
Describe phase 1 of the trial
small number of healthy volunteers. ethical and regulatory approval needed. Evaluates saftey and pharmaco- kinetics
Describe phase 2 of the trials
target pts with the disease, involve up to 400 ppl. first part is dose finding and second part measures therapeutic action and determines dose for phase 2
Describe phase 3 studies
involve many pts with disease in RCT. determines efficacy against placebo/ other treatment. required for regulation and marketing
What does the regulatory review by MRHA look at?
MHRA looks at:
phase 3 data
extended data (food interactions ect)
issues licence
What aspects of a drugs development are looked at in a clinical appraisal?
- Any selection bias?
- How was randomisation acheived?
- What treatment was the drug compared to?
- Was it double blind?
- What was the outcome measure? Was it representative?
- Could the result have been by chance?
