Setting Realistic Expectations for Clinical Trials
This blog post covers:
Distinguishing hope from realistic medical impacts
Weighing realistic possibilities during a clinical trial
Consulting your research team
Read the whole post below to learn more.
Week 4: Clinical Trial Education & Outreach Series
When you're part of a rare disease community, clinical trials carry a great significance. It means that you are one step closer to potentially having a specific treatment approved for your disease. For communities that are often underrepresented in research, inclusion in a clinical trial is something many patient advocacy groups strive for, and is worth celebrating when achieved. But it's important to maintain a realistic view of the potential implications of clinical trials, so that expectations can be set appropriately.
Hope vs Expectation
Hope is the belief that something could happen, while expectation is the belief something will happen. Both shape how we think about care and management, and are especially useful concepts to consider when thinking about the outcomes of a clinical trial.
It’s natural (and expected) to hope for the best case scenario; all seizures stop, someone who is non-ambulatory can walk, speech appears. However, it’s also important to understand what could or should be expected based on the therapy - What symptoms are the therapy targeting? Does the therapy target the CACNA1A gene or the Cav2.1 channel protein, or does it work on something different in the cell? The outcomes will depend on these details. This may also mean accepting that full symptom reversal may not be realistic, while maintaining the belief that meaningful improvements in quality of life are achievable.
With that distinction in mind, here are a few things to consider as you weigh whether a clinical trial is right for you or your family member:
Understand the therapy may not work
A clinical trial is, at its core, an experiment. It sets out to answer a question, and sometimes the answer can be no. The reason could be because no measurable benefit was detected, the intended effect didn’t occur within the trial's timeframe, or the trial stopped early for safety reasons. If this happens, it’s important to understand that even if a therapy doesn't pan out, your participation matters. Every result, positive or not, allows researchers and clinicians to learn how to improve treatments and design clinical trials.
Be aware of the trial phase you’re being offered
If you recall from our first post, knowing what phase a clinical trial is in is one of the most useful tools you have for managing expectations. In early-phase trials, researchers are still establishing whether a therapy is safe to give at all, rather than whether it works. Therefore, participants should not be expecting any changes in their symptoms. As a therapy advances into later phases once it has been deemed safe and efficacy is being tested, participants can focus on symptoms before and after treatment.
Know that participating in a clinical trial doesn’t guarantee you access to the treatment
As we covered in our second post, some trials are designed to have a placebo-only group, an important aspect for testing therapeutic efficacy. That means there is a chance you might not receive the therapy being tested. In most cases, you won't get to choose, or even be aware of, which group you’re in, so it’s important you are comfortable with participating in either group before enrolling in a trial that is designed this way.
Be clear on what is expected from you
Clinical trials carry a logistical weight that can be easy to underestimate in the beginning. They often require frequent visits to the trial site for repeated procedures, which can mean time away from work, from other family members, and overall changes to your daily routine. Before you commit to a study, it's worth asking yourself if you have the capacity to take it on. Consider the financial, practical, and emotional demands of participation, and weigh them against the potential benefits.
Balance urgency with the need for rigor
When you or someone you love is living with a disorder, the pace of clinical research can feel unbearably slow, and the desire for a fast answer is completely understandable. But that slowness is largely a byproduct of the rigor that is built into clinical trials to make their results trustworthy and keep participants safe. It's worth remembering this whenever you're weighing how long a trial might take.
The best way to manage expectations is to have ongoing, direct conversations with your research team, especially during the informed consent process.
Ask what the data has shown so far. What benefits are realistic? What are the risks of participation? What happens if the trial doesn't work? Make sure you genuinely understand the entire clinical trial process before you sign a consent form.
This post completes our Clinical Trial Education & Outreach Series. We hope it’s helped make this process more approachable, and that you feel ready and supported in whatever decisions about clinical trials come next.
Read the first blogpost in this series by visiting: https://www.cacna1a.org/blog/clinicaltrial1
Read the second blogpost in this series by visiting: https://www.cacna1a.org/blog/clinicaltrial2
Read the third blogpost in this series by visiting: https://www.cacna1a.org/blog/clinicaltrial3
