December 2022: A Patient’s Perspective Participating in Clinical Trials

December 2022: A Patient’s Perspective Participating in Clinical Trials

Anne Lacey We are pleased to present the final article in a three-part series on clinical trials sponsored by Vertex Pharmaceuticals Incorporated. This article was written by Anne Lacey, a person living with type 1 diabetes from New York, NY.

Click to read the first article, What Are Clinical Trials and Why Are They Important?
Click to read the second article, Innovating in Clinical Trials.

As someone whose life has been significantly improved by clinical trials, I am eager to share my experiences with others who may want to consider participating themselves. I was diagnosed at age six with type 1 diabetes (T1D) which increased my risk for strokes, heart attacks, amputations from gangrene, blindness, and death, just to mention a few examples. When I was diagnosed, doctors said it would not be possible to have children, but I was fortunate to beat the odds of having two children by the time I reached my thirties. However, I was dealing with complications when my children were young, and my son had also just been diagnosed with diabetes.

Growing up with T1D, I chose to participate in clinical trials, as I understood that nothing would change for those of us with the disease unless the researchers were able to study and test new approaches and therapies. Over my lifetime, I have participated in approximately 20 clinical trials and never regretted my decisions to participate to help researchers find potential treatments and hopefully cures. There are always risks and benefits to weigh when considering participation in clinical trials. Asking the physicians who cared for me about research underway with my disease, I would listen for trials that were focused on issues I was experiencing, then ask their opinions on my potential participation. With this information, I went and did my homework (all very accessible online now) about the pros and cons, how it might benefit me and others, then I would meet with the clinical trial coordinators to ask many questions. I’ve always asked to speak with others who have gone before me in the trial (or similar trials elsewhere) to get their input.

The most beneficial clinical trial for me was also the most clinically involved and complex of all those in which I’ve participated. The main reasons I decided to participate are:

  1. The potential that the trial might be successful and, if so, allow for potential treatments and someday a cure to be developed so others (like my son!) could benefit vs what happens if the clinical trial might not have enough patients participating, which means that trial closes and the questions being investigated would not be available for anyone.
  2. The potential benefit of entering the trial which might allow me to live longer and be there for my children vs the existential risks of continuing along the road I was on with the disease as it was.
  3. The potential that a trial may alter the course of my disease and what medications I would need to support it.
  4. The potential that the trial might be successful and help my disease.

This analysis made my decision crystal clear - of course, I would participate for all of the above reasons! However, there are potential risks associated with participating in clinical trials that individuals should consider. The treatment may cause side effects, the treatment may not work, you may not be in the treatment group, and/or the trial could be an inconvenience to you.

For those considering trial participation, consult with your healthcare provider. I can also share a few tips that helped me:

  • The trials I chose to participate in were best suited for my specific clinical situation; I recommend others consider their own clinical needs when assessing whether to participate in a trial.
  • Participating in clinical trials means you will be getting potential help in caring for your disease during the course of the trial, even if the trial is not successful.
  • It can be disappointing to participate in trials where the outcome is not successful, but your participation allows potential therapies and cures for diseases like T1D to be investigated and informs other researchers in that field to try a different approach. Being part of a “trial” means that potential treatments are being “tried out” and there are no guarantees the trial will work. However, I encourage you to keep in mind that your decision to participate in a clinical trial has the potential to benefit hundreds, thousands, or maybe even millions of individuals living with serious or life-threatening diseases like T1D. Each one of us has the ability to make a huge impact.

© 2022 Vertex Pharmaceuticals Incorporated l VXR-US-02-2200264 (v1.0) l 09/2022