by Craig Klugman, Ph.D.

For patients with a serious illness, accessing cutting edge drugs has just taken a new turn. In the past, a patient with cancer would undergo conventional treatments. If that failed, then he or she would be considered for a clinical trial of a new drug that may (or may not) have the potential to affect the disease. Entry into trials is strict with controls for potential subjects’ age, sex, type and stage of cancer, treatments already tried, and more. And trials are limited in size. Depending on the type of trial the researchers are looking for safety, efficacy, side effects, or dosing. Keeping the numbers of subjects low ensures that fewer people will be harmed if the drug is found to be dangerous. This is one reason why we test new medications.

At least, that’s how it worked until now. In 2014, the way to get access to experimental drugs is to shame the company into giving them to you. In March 2014, supporters of 7-year-old Josh Hardy used social media to pressure Chimerix, a biotech company in North Carolina, to permit compassionate use of a new medication. The company’s drug brincidofovir is an antiviral that Josh’s family thought might fight the infection Josh was suffering after a bone marrow transplant. Almost 20,000 people signed an online petition and celebrities weighed in on the appeal to the company. After all that pressure, the Chimerix worked with the FDA to create a new Phase 3 (confirmation of safety and efficacy among a large number of people who have the disease) clinical trial and made Josh their first subject.

Meanwhile in California, a man is using social media to get access to experimental antibody drugs to help his wife, Mikaela Knapp, whose kidney cancer did not respond to current treatments. They used a change.org petition (with 200,00 signatures), a blog, Facebook, Twitter and more. Two companies who make these new drugs have already refused to make their drugs available but a third, Merck, is in discussions. Most of these drugs are in phase 1 trials, meaning they are being reviewed for safety among healthy volunteers to determine safety.

Is this the new face of illness? He or she with the largest media push gets the drug? In an age when new treatments can cost $168,000 (new hepatitis C drug), should we be distributing medicines based on who can shame a company the most? Or who can appeal to public opinion the most? Josh is a cute 7-year-old white boy. The Knapps have loaded their social media campaigns with pictures and videos of their smiling faces, looking like a cute (white) couple embarking on an adventurous life. It’s the story that they are trying to sell.

These efforts are understandable. If one is sick and has tried all that medicine has to offer, it becomes attractive to look for new possibilities and many of those hopes are in drugs in clinical trials. The families want to do everything possible to help their loved one. This hope may be misplaced and may be misunderstood.

A drug under clinical trial is not a treatment, it is research. And the goal of research is to test the efficacy and safety of the drug on subjects, not to cure any patient. And since the person in question does not meet the standards for the research protocol, the research is slowed down meaning it will be longer until more people can get an approved drug that is shown to work (or the company starts a new research trial for the one person which has the same affect of extending the timeline). By going around the research enterprise, these well-intentioned, suffering families are undermining the process for assuring that safe, effective medications make their way to people in need.

In large part, these social media campaigns are leveraging numbers. If enough people sign on, the company either feels pressured into giving the drug or shamed into it. After all, who wants to be known as the company that let a 7-year-old boy die? With a petition of 200,00 signatures, what company wants to risk having a boycott against its consumer products division. Saying no is risking a public relations nightmare.

So those with a good social network, with know how of social media, with money to pay for a campaign, with a heart-tugging story and pretty pictures can use all of that advantage to get the drugs. It’s no longer who can afford the drug or who qualifies for the trial, but rather who gets the most “likes.”

If the compassionate use program has problems, then we should fix it. But these attempts to “jump the line” and get preferential treatment are unjust. This is similar to campaigns on billboards and the internet to secure an organ for a loved one through advertised direct donation, thus circumventing the system. While this can make things better for the individual, it does make things worse for the people who followed the rules and were part of the system. It also certainly makes things worse for the person who lacks the means to take these steps.

Perhaps clinical trial participation should be a game show where potential subjects have to share their story to a panel of celebrity judges and using online voting, the American people decide who gets the drug, the organ, or the hospital bed. Of course this is ludicrous and while I can empathize with patients and families who feel they have no hope but to grasp at straws, for the population as a whole, these appeals to emotion through social media do more harm than good.

The other option is simply to open up the floodgates, do away with structured testing. If there’s a drug and you want to try it, then go ahead. If it turns out to be toxic, or destroys your liver, well you are on your own. The reality is that we need to test drugs for safety and efficacy. To do that we need a system that balances benefits and burdens and we have that. Can it be improved, sure. Let’s look at changing the policy before letting pretty pictures and a sad story be the arbiter of medical care.

The good news is that Josh is doing well since he received the experimental treatment and has been moved out of the ICU. The bad news is that no one knows what the safety and side effects issues might be for him and for the delays in FDA approval that would have ultimately saved many other lives. But this is America and we regularly sacrifice the good of the many for the one, if that one is cute and has a good story.