The problem with drug trials

Should randomised trials be the only type of evidence accepted for rolling out drug treatments? If so, then two researchers wrote in the Lancet this week that that we face...

Should randomised trials be the only type of evidence accepted for rolling out drug treatments?

If so, then two researchers wrote in the Lancet this week that that we face a problem:

The evidence we have might not be the evidence we need, and the evidence that we need may never become available.

They are writing in response to the publication of a trial of a new combination drug treatment for stomach cancer that seems to extend people’s lives by nearly 3 months.

According to these guys, there’s probably lots of effective combinations of current cheap drug treatments to treat cancer, but we’re never going to get the evidence to prove it.

The new trial must have been crazy expensive: It consisted of patients from 122 different institutions in 24 counties on 4 continents and, as the editorialists point out, it would never have been paid for had it not promised huge returns for the pharmaceutical company that funded the trial, Roche.

This raises a sticky issue. Drug companies like Roche produce treatments that save lives. But they don’t do so because they save lives. Rather, they produce the drugs because they can make the company money.

So what happens when these two motivations come apart — when drugs that could save lives don’t make drug companies money? That’s the problem that the Lancet editorialists are referring to.

If we presuppose that only randomised trials produce evidence of sufficient quality to support decisions about the allocation of scarce resources, there is a problem. There is a lot of evidence on the effects of adding expensive new drugs to conventional therapies, but little evidence for when older, less expensive interventions are combined.

And boy are these drugs expensive. In the same comment piece, the authors calculated that the cost of each year of life gained by this new treatment, is about $100,000 AUD ($85,000 USD).

The authors of the study argue that this should be rolled out as a “new standard option” in the treatment of gastric cancer — and that’s not surprising given that the manufacturer of the drug not only funded the trial, but was involved in the data analysis and editing of the report.

So how are we supposed to get evidence about cheap drugs that will help millions of people but not make money for drug companies?

I don’t know. The authors mention the model in physics where large projects that don’t have obvious practical benefits are funded by governments and research institutions. And they seem to imply that there might be a way of not relying solely on randomised trials — but they don’t say what that is.

Whatever the way forward, it seems crazy that in the mean time, we can only get evidence about drugs which, for the majority of people around the world, are prohibitively expensive when there’s almost certainly some great cheap alternatives right under our noses. Munro, A., & Niblock, P. (2010). Cancer research in the global village The Lancet DOI: 10.1016/S0140-6736(10)61022-7 Bang, Y., Van Cutsem, E., Feyereislova, A., Chung, H., Shen, L., Sawaki, A., Lordick, F., Ohtsu, A., Omuro, Y., & Satoh, T. (2010). Trastuzumab in combination with chemotherapy versus chemotherapy alone for treatment of HER2-positive advanced gastric or gastro-oesophageal junction cancer (ToGA): a phase 3, open-label, randomised controlled trial The Lancet DOI: 10.1016/S0140-6736(10)61121-X