A trial that fails to reach its clinical endpoints isn’t necessarily a ‘failure’

Eight years ago, I walked down the corridor that connected the hospital to the clinical research wing. I arrived at what felt a bit like a movie set, but was actually the floor where clinical trial participants would receive intravenous infusions of either a placebo or the investigational drug.

It was January, and this clinical trial was investigating a novel antibiotic approach that would make it harder for the Pseudomonas aeruginosa bacteria to survive in my lungs. At the time, my health with cystic fibrosis was slowly, but surely, declining. This was the first interventional clinical trial in which I’d participated.

I have long been interested in clinical research, both as a participant and as a patient representative on protocol review committees. This trial required me to be admitted for five days for daily intravenous infusions, but I was eager to participate — and excited to lie in bed for a few days and watch Netflix, but for science.

In case you aren’t aware, as participants go through the informed consent process, they agree that they don’t expect to glean benefit from participating in the trial. I admit, though, that I hoped to get a medication that would prove to be efficacious. Despite a sedentary week in the hospital, my lung function went up 5%, which led me to suspect I received the interventional medication.

Some time later, though, I learned that the study revealed no statistically significant difference between the interventional therapy and the placebo arms. In clinical trial parlance, we’ll often say the study “failed to reach its primary endpoints.”

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But was the study a failure? I don’t think so.

Clinical trials may not reach their endpoints for various reasons, but this doesn’t constitute a failure. Failure implies that something didn’t succeed, but that depends on your definition of “success.”

Clinical research is intended to provide answers, and sometimes the answer is a resounding “no,” which is not as fun or visceral as the alternative, but it’s still incredibly important for moving the field forward. Clinical trials are designed to determine if a treatment is more effective than doing nothing. If a medication does not work better than doing nothing, it doesn’t benefit the patient or clinician to continue using it. In that way, clinical trials that don’t reach their primary endpoints are still a success.

I certainly felt let down when I learned the results, but I didn’t regret participating. I was treated with dignity and respect throughout the process, and found it to be a genuinely great experience that amplified my passion for clinical research and patient advocacy. The process filled me with a lot of hope because I know how many people are working hard to improve the lives of those with cystic fibrosis. Now, I discuss clinical research at every one of my appointments and look forward to participating in the next trial I’m eligible for.

In a perfect world, participants in clinical trials would have good, dignified experiences and every trial would achieve its aims, but trials that don’t “succeed” aren’t failures.

Note: Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Cystic Fibrosis News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to cystic fibrosis.