The Duchenne’s treatment made by Sarepta (eteplirsen) has been in the news this week, as a troubling example of the FDA lowering its bar for approval of new medicines. The FDA expert advisory panel decided not to approve this treatment, because the evidence for any benefit is weak; but there was extensive lobbying from well-organised patients and, eventually, the FDA overturned the opinion of its own panel. There have been calls for paper retractions, and so on.

This is not the first time we’ve seen peculiar activity around the treatment. Read the rest of this entry »

There are recurring howls in my work. One of them is this: in general, if you don’t know which intervention works best, then you should randomise everyone, everywhere. This is for good reason: uncertainty costs lives, through sub-optimal treatment. Wherever randomised trials are the right approach, you should embed them in routine clinical care.

This is an argument I’ve made, with colleagues, in endless different places. New diabetes drugs are approved with woeful data, small numbers of patients in trials that only measure blood tests, rather than real-world outcomes such as heart attack, renal failure, or death: so let’s roll out new diabetes treatments in the NHS through randomised trials. We rely on observational studies to establish whether Tamiflu reduces complications of pneumonia: that’s silly, we can do trials, and we should. Statin treatment regimes in widespread use have never been compared head-to-head, using real-world outcomes such as heart attack, stroke, and death: so let’s embed randomised trials as cheaply as possible in routine clinical care (we’ve done two pilots, to document the barriers).

This week a dozen colleagues and I published yet another application of this basic, simple principle, as an editorial in the BMJ. The Cancer Drugs Fund is being marketed as a way to generate new knowledge: but in reality, the data that will be collected is weak, Read the rest of this entry »

Here’s a useful paper we’ve just published in the BMJ, documenting problems in transparency around approval processes for randomised trials. There’s a basic rule in clinical research: you’re only supposed to do a trial comparing two treatments when you really don’t know which one is best, otherwise you’d be knowingly randomising half your participants to an inferior treatment. Despite this, it’s already known that trials are sometimes conducted where one group get a substandard treatment.

We wanted to find out how ethics committees come to approve such trials. Read the rest of this entry »

Hi there, I’m doing a few events in Australia and NZ this week: in Sydney, Melbourne, Auckland (only 25 tickets left), and Brisbane. Here‘s a good fun interview I did with The Conversation that gets very nerdy, on the poor state of science, COMPare, statins, reproducibility and transparency. I’ll post a big backlog of interviews, and papers, over the next week or two. So, come, come, I’ll see you in Oz! Read the rest of this entry »