Clinical Research… Why?

The motivation of most anything at the age of 13 is something of monetary value. In 8th grade all I saw was a dollar sign. However, what I gained by taking part in my first clinical drug study for Cystic Fibrosis (CF) is something money will never be able to buy.

My first clinical study was in 2004 for a new and improved digestive enzyme for patients with CF. This new enzyme boasted improved capability for nutritional absorption and came with hopes of being able to take less than the usual 20-30 pills per day. The health risk involved was relatively low, but ultimately my family left the decision up to me regarding whether or not I was going to participate. We met with several of the clinical study nurses in addition to my usual staff of doctors so that we could make an educated decision. After almost no hesitation on my end – again, all I saw were dollar signs – we decided to move forward with the study. Although the risk was low, the commitment to the study was high and involved a two-week hospital stay (a first for me) to monitor vitals and the drug’ effectiveness.

For those who are wondering what happened to the study pill, it was never approved by the FDA for use by the CF community. Although it was disappointing to hear, the outcome of this particular story is far from failure. I gained so much more during this study than a few extra pounds and the added convenience of taking fewer enzymes. I built a deeper relationship with my physicians and nurses, I learned more about the disease I live with, and most importantly I contributed to the continued improvement and quality of life for those living with CF.

A few years later I was asked to take part in one of the preliminary drug trials for Vertex Pharmaceuticals who were pioneering what we now know as Kalydaco and Okrambi – I think we all know how these studies turned out.

Clinical drug studies are not for everyone. Several factors such as lack of time to commit to, health factors, as well as many others may mean that now is not the right time for participation in a drug study. However, I feel it is crucial that those who are capable of participating in these types of studies at least consider doing so for the simple fact that we would not have come as far as we have without the select few who have given their time and effort into making it happen. Tremendous organizations like the CF Foundation can raise as much money as they want to, but without dedication from patients willing to contribute in the name of science those dollars are wasted.