Sadly, even with cancer medications, it can not be assumed that just because there is some repsonse that this corresponds to a “saved life.”

Licensing/approval should not be the only rationale for studying a drug. Perhaps reviewing agencies (or the “cancer community”) should insist that cancer treatment trials be designed in such a way that if an interim analysis shows a benefit there is some ethical and appropriate modification of the protocol allowing continuation of trial to meet the original goals and establishing whether or not there is a benefit in days of quality life added, if not cure?

Certainly, once the drug has been found to show real benefit early on, everything must be done to get it to the patients quicker. The FDA here in the US allows for the Fast Track program. However, this system also puts the impetus on pharma to continue to provide and accrue data over the longer term as part of the early approval process. Failure to comply includes the levying of fines, if I am not mistaken.

I think this is where patient groups play a key role in monitoring the long term data.

Above all, we need to remember that even though a drug saves lives, it may not be in the long run what turns out to be best for the patient. It may cause problems in the not too long term that prevent the patient from seeking other types of treatments. Specifically, if you look at some of the drugs like sutent, gleevec and others and the risk (albeit it small) for heart attacks. This suggess that patients will be in less than optimal condition to try other newer treatments.

However, with cancer you have to outweight the risks versus benefits. Missing from the equation are issues the patients need to understand with regards to quality of life prolonged and what complications, further medical treatment and financial burdens this may represent.

I can’t imagine finding answers to these issues is easy at all.

Look at the AIDS vaccine Merck attempted. When they did an interim analysis they expected to find no difference (because it was too early), but hoped to find the vaccine worked. The interim analysis showed the vaccine was worse than comparison (placebo? I don’t remember). It’s not like Merck kept going with the trial because it hoped they would turn things around and find something different at the end- why put more patients at risk. If you see statistically significant changes at an interim analysis, you can be confident those changes are real - since they needed to be so dramatic to get detected at all.

Furthermore, many drugs include open-label extension trials, specifically designed to detect additional adverse events. Once you know it works you can then start to look exclusively at safety.