I used to dislike surveys, polls, and questionnaires. I understood why they existed — customer or patient feedback is important — but I never really saw anything change once I took a survey.
I hadn’t considered that perhaps my feedback was unrealistic or didn’t represent the entire community. It wasn’t until I took a quality of life questionnaire before and after a clinical trial visit a few years ago that I began to understand their importance.
During my visit, I discovered questionnaires can be used as secondary or even primary endpoints for clinical trials. These are the metrics used to determine whether or not a clinical trial is “successful.” (How we define the binary of a “successful” clinical trial is a conversation for another day.)
There are, of course, instances in which a clinical trial is designated a failure based on primary and secondary endpoints, even though some people have benefitted from it. When patients go through the informed consent process before enrolling in a clinical trial, we are told that we should not expect to receive any benefits, as we may receive a placebo. Alternatively, the study medication may simply not be efficacious.
As patient-reported outcomes have become more mainstream over the last 10-20 years, discussions about how patients feel have been amplified. Validated questionnaires are legitimate scientific tools that are used to determine if hidden aspects of medications and treatments may have a bigger impact on how the patient actually feels.
Compared to traditional biometrics, such as lung function or liver enzymes, patient-reported outcomes demonstrate that experience can encompass more than numbers on a chart. In some cases, a patient may prefer a medication that is administered in the hospital once a month instead of a medication they must take three times a day, or vice versa. Quality of life questionnaires find those nuances in the clinical trial and patient experience.
Since I started working at BioNews, the publisher of this site, I have been involved in the creation of surveys to better understand patient-reported outcomes. It has been a delight to use my basic science and research background in combination with my advocacy and patient-centric experience to learn more about what patients really feel.
I believe that surveys can be used to tell the broader story of the patient experience, no matter the disease community. If done effectively, surveys can articulate what needs to be done to improve the quality of life of the entire community. There are limitations to surveys, of course: They are lagging indicators, as people are reacting to what has been done in the past.
My hope, however, is that by understanding and reacting to the patient community’s desires, we can work together to effectively anticipate what needs to be done for the next generation of patients and treatments to guarantee an improved quality of life.
It was an honor to have the opportunity to lead the design of the Cystic Fibrosis News Today survey. Our hope with this first survey is to better understand multiple aspects of the CF patient experience — demographics, symptomatology, treatment plan and side effects, interest in clinical trials, and more.
With this survey, we hope to understand where the CF community is happiest and where it wants to see the most improvement. With future surveys, we hope to learn more about the community to further impact the trajectory of cystic fibrosis treatment and continuously improve quality of life.
Follow along with my other writings at my humbly named site, www.trelarosa.com, or subscribe to my newsletter “sonder, essays” at trelarosa.substack.com.
Tré LaRosa
Tré is a 26-year-old living with cystic fibrosis in Pensacola, Florida who works as a research lead for BioNews Insights. He is an extremely passionate advocate for disabled rights and people living with chronic disease, especially cystic fibrosis. His sister, Alyssa, died at 29 due to chronic rejection of her bilateral lung transplant, pushing him further into the fight for a better world for all. “Mutations & Conversations” discusses the science and sociology that binds us all. He also loves his golden retriever Duncan very much.
