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| "success vs failure vs non-approval" and "benefits outweighing the risk".
What does that mean? What does the "risk" part mean? | |
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Whenever we start developing a new drug, there's a risk (indeed, a 9/10 chance, from the start) that it won't be effective or safe enough to be used, so the research spend up to that point is lost. That's the degree of risk, put simply.
The potential patient group for this drug, had it been successful, was HUGE - "
our estimate is that dalcetrapib could have prevented 100 thousand deaths, strokes or other serious CV events every year." " Overall, more than 200 million patients worldwide could have benefited from this medicine." - so the risks were weighed up against the potential benefits and found to be worthwhile taking.
"success vs failure vs non-approval" refers to the (large) added costs and potential reputation damage if we'd decided to continue developing the drug despite the negative interim results. It is possible that the trials may have gone on to show efficacy, but the chances are much lower, and if we'd tried to push it through for registration and been rejected (i.e. not approved) then it would be very damaging for the company, as well as expensive.
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| And "phase III"? Is that when the "risk" is measured? | |
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No. Risk is continually measured. Phase three is when we start large-scale trials on real patients, with a view to using the results to show that the medicine is safe and effective. It (approximately) marks the transition from Research to Development, and involves significant cost and resources.
The whole 'risk' thing is being highlighted as it's a sign of the application of a policy of 'smart risk-taking' over the last few years. We must be prepared to take risks, but recognising them and being prepared to reassess based on changing information allows us to mitigate those risks as much as possible.