Though surrogate endpoints are argued to have the advantage of allowing quicker drug development and patient access, it is questionable whether studies based on surrogate measures of efficacy provide optimal, or even meaningful, information for patients and clinicians. Moreover, our analysis raises the possibility that regulatory and current research practices have created a situation in which critical information about the outcomes that matter most to patients might never be generated once oncology drugs are approved for widespread use.

That’s not a good situation. I’m sure that there will be room to argue about some of the figures in this paper – and there’s plenty of room to argue about whether the situation here in the US, with the FDA, is any better. (There are plenty of arguments waiting when such data become available, too, believe me). But in general, I have to say that I agree with the conclusions that regulatory agencies should be asking for more survival data – if not at approval, then in required postmarketing surveillance. For those cheering this on, though, be aware that nothing comes for free: if you want more solid data on real-world survival, then you’re going to be paying for that, in both money and time. Tightening up the survival requirements will inevitably slow things down, and if you’re in the “Regulations are strangling drug innovation” camp, you’re not going to care for the idea much to start with.

But you pays your money and you takes your choice: faster, cheaper, and less proven – or slower, more expensive, and more proven. The only way I can see to split the difference is as mentioned above: if you’re going to approve on surrogate endpoints, then make it mandatory to collect data on what happens afterwards, and set an appropriate date to review it, with approval to be rescinded if things don’t work out. Sounds good – but remember what happened when the FDA did that with Avastin’s breast cancer indication? Nothing is simple, not with so many lives and so much money on the line.