Some thoughts about clinical trials

There’s an interesting article from the New York Times yesterday about whether clinical trials work – not just melanoma trials, but trials of new treatments for all kinds of conditions. My reading of many posts on the Melanoma Resource Foundation’s Patient Information Page (MPIP) over the last year has led me to contemplate this subject. Here’s what I think about it.

The article was prompted  by findings released and discussed at the American Society of Clinical Oncology meeting last month. In a nutshell, it said that researchers consider failed trials a success if they further knowledge about cancer mechanisms and how certain treatments affect them – whether the patient responds to the treatment or not. Some people wonder how a trial can be a success when so many patients don’t respond.

For the community of patients, loved ones, and caregivers, at least those affected by melanoma, clinical trials of promising new therapies provide more “tricks in the bag,” more steps along the way to “beating the beast,” as one MPIP frequenter puts it. For too many, there is no such thing – it’s just a way to buy a few more months or years, hoping that a REAL cure will be found before our time is up. We’ll hope to be among the more than 38% of patients for whom anti-PD1 trials (experimental therapies that turn off the “programmed death” gene that makes some melanoma cells so deadly) create a durable response (in this case, more than 11 months so far). And if not, perhaps researchers will learn about some other mechanism while we are fighting or biding our time to see whether our disease advances, or until some other hopeful therapy enters trials that we could sign up for. More hope for us, more knowledge for the researchers … more life to live in the meantime.

Drug companies, according to the article, consider clinical trials a success if they lead to FDA approval of the drug so that people will have to pay to find out whether they work. Something is wrong with this system …

We want the trials to succeed because it offers hope, even if only 38% respond. Once the therapy is approved, it will cost us (or Medicare, or our insurer) handsomely. It doesn't deter us from hoping for success.

I don’t have alternatives to suggest. I’ll leave that to the policy folks to figure out.

  • Read more about anti-PD1 research and melanoma here.