- “There is nothing either good or bad, but thinking makes it so.”
- William Shakespeare, Hamlet
Posted: July 30th, 2014
Shakespeare may have been thinking more of the innate nature of things when he penned this particular sentiment; but in the world of clinical trials, “attitude,” or merely what a patients thinks about a treatment can have a serious impact on how that treatment works, or doesn’t.
In a thought-provoking article in the WSJ today, author Amy Marcus explores the impact that social media is and may have on the clinical trial process. Neuralstem has had several of its patients “blogging” or otherwise interacting through social media with members of the ALS community, outlining their various victories and defeats as they battle this horrible disease. Ms. Marcus suggests that it is likely that in an upcoming efficacy trial, Neuralstem would have to conduct a blinded trial. Many of you have written or called about this, so I want to be sure the record is clear. This is not the case in the typical sense of the word.
It is typical in a late-stage trial that patients do not know if they have received the actual drug, or a placebo. This “control” on how a patient’s thoughts or hopes can effect their health is a bedrock of modern medicine. In a trial like ours, however, there will be no “sham” surgeries. We will have a control group in our efficacy trials, which is what I believe Ms. Marcus meant; but that control will be randomized patients who receive the standard of care for the disease. They will not receive any type of placebo treatment. So all the patients in our trials will know who is getting the surgery and our cells, and who is not. We will, on the other hand, make all efforts to keep the professionals who measure the progress of the patients blinded as to who is in the treatment group versus the control group. Given the ethical and safety problems with sham surgeries, and the rapid fatal nature of the disease, this seems a reasonable compromise for trial design. We will also be working to find a structure that allows the control patients the option of having the treatment after the blinded period is over.
The article goes on to talk about other possible issues that can arise from patients “talking” to each other about their treatments. There are some in the industry who believe that this communication poses a serious threat to the integrity of the trial system itself. I could not differ more strongly.
First, it is naïve to think that patients haven’t always spoken with each other, or tried to figure out if they were on a drug or a placebo. Second, these patients are fighting for their lives, and it must be remembered that they Have lives. They are not rats in a maze. Indeed, who can even imagine how intense and compacted the interplay of everyday thoughts and emotions must be when one has been given a fatal diagnosis. One of the great benefits of social media and modern communications is that it has created ever-expanding communities to be part of. Patients with fatal diseases are even more in need of community and the emotional support that comes from community than most. And they are even more in need of information on their diseases and how others are coping, and possible treatments.
There is always a balance that must be struck between pure “lab” science, and the ethics around treating humans in a trial. The balance must always tilt heavily towards humane ethical treatment of patients. The influence and prevalence of social media is only growing. This is not a genie that can be put back into the bottle; nor, in my opinion, should it be. Both the drug discovery industry and the regulatory institutions around the world are going to have to figure out ways that protect the integrity of the trial data, and accommodate patients’ needs. The two can not be in competition.
