I want a new drug, one that won’t make me sick
One that won’t make me crash my car
Or make me feel three feet thick
I want a new drug, one that won’t hurt my head
One that won’t make my mouth too dry
Or make my eyes too red
-Huey Lewis and the News
UNMET NEED HIGHLIGHTED IN THIS POST: Critics of pharmaceutical companies’ clinical trials for drugs in development claim that such trials sometimes measure the wrong criteria for success because they’re not designed with adequate input from patients and physicians. However, these days pharma companies are listening to patients and physicians more and more.
The other day an article with the provocative title “Do Your Patients Care What Your Drug Does?” arrived in my inbox. What kind of question is that, I thought? The article’s title piqued my curiosity, though, and I had to see what it was talking about. The article argues that the outcomes measured in a clinical trial for a new drug (i.e., measures of effectiveness and side effects) before it’s submitted to the FDA often diverge from the outcomes that doctors and patients seek. And the result for the pharmaceutical company is disappointing sales.
While the types of metrics you’d want to include in a trial for a new drug may seem obvious, the outcome selection process is a lot more complicated than the layperson might imagine and mistakes are made. In a 2014 series of articles, the respected medical journal The Lancet quoted research that claimed that the most common source of waste in trials was poorly selected metrics of success.
The Other Side: Significant Strides by Pharma
However, pharma appears to be making significant progress in listening to patients. At Novo Nordisk, for instance, patients are extensively involved in all phases of trial design and execution, not to mention post-launch patient support activities. A friend who works at Novo speaks highly of the input provided by the company’s Disease Experience Expert Panels (or DEEPs for short).
In addition, we must remember that even if pharmaceutical companies are diligent about gathering rich input from patients and physicians, the FDA also has a major say in the trial endpoints chosen. Here’s an example of an FDA guidance document for cancer drug trial design.
So my conclusion at the end of the day is that the reality is far more nuanced than the one painted by that provocative article in my inbox.
