Big pharma is failing.

The current model for big pharma is to use very large trials to deal with a secular increase in the placebo response rate. These commercial organqanizations partner with clinical groups to recruit patients. There is significant money paid for recruiting patients, and more for keeping patients in the system to completion.

It may be that the modern drugs do not work. Or, more likely, the patients recurited in these studies are different from those in the earlier studies: the older studies, with more severe patients, show better differentiation between placebo and treatment.

In phase II drug trials, the agent is administered to patients with a particular disease or condition. These studies usually include hundreds of patients and are essential for establishing the agent’s effect size, dose range, and general safety and tolerability profile. Phase III trials are much larger and can involve 300 to 3,000 participants. These studies can confirm the phase II findings and provide additional safety information. Recently, these trials have been global and have included as many as 200 different research sites. An important observation from a number of recently completed phase III trials is that the active drug resulted in similar symptom reduction as in the phase II trials. However, the placebo response was substantially greater, and the active drug did not separate from placebo. Another characteristic of these studies is that they often have aggressive timelines. Sites and contract research organizations are reimbursed based on the number of subjects who are randomized, and there is often pressure to meet deadlines.

The conceptual ideas around these trials needs to be reconsidered. They now cost too much. The article suggests some ways forward. It may be better to limit those who are entered in the trial to the more severe: if medications are to be used later and talking therapy earlier (which has a certain face validity) then smaller and sicker patient populations are needed.

It may be that meta analysis of earlier trials will help.

But the big pharma trial is now failing.

One thought on “Big pharma is failing.

  1. Other issue is that the medications are getting so fine-scale in approach that there might not be a test population large enough. Related to that, you also have the issue that most medications don’t really work on “edge cases”, which is why the the placebo effect is so high.

    This also goes in with something I’ve observed in social sciences. Most population situations actually have a 30/40/30 split. 30% “something works”; 30% “it does nothing!”; 40% depending on how you phrase things. This is the reason why political polling can be all over the place. Very finely scaled questions or processes can shift a huge chunk of a response population.

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