Over the past few years I have talked to a lot of parents, individuals with Duchenne, and clinicians about clinical trials. This probably won’t be a surprise to you: I often hear how desperation and a feeling of running out of time affect how people think and feel about clinical trials.      

Many bioethicists are worried about situations like this. They wonder whether people who are feeling desperate make informed decisions and really understand clinical trials. They talk about therapeutic misconception, which means that people think a clinical trial is designed to benefit them/their child directly, rather than test or compare treatment methods in a study group. Do we, as a community, tend to think of clinical trials as a hoop to jump through to get our hands on that maybe-wonder-drug to “treat” our children? Could be.  Does that mean that parents don’t make informed choices about participating in clinical trials? I don’t think so.

Here’s the thing about bioethicists (no offense to any out there): sometimes they miss the heart of things; the emotion; the real driving force. In addition to desperation, the common thing I hear from families and clinicians is the importance of hope. We need to have hope for a better future for our own children, and for the next generation of people with Duchenne.  

We have to balance our expectations and hopes in a clinical trial. We have to appreciate the difference between bringing our child to the doctor for an approved treatment specifically for his symptoms, versus seeing that same doctor in the context of a clinical trial. It sounds so easy, but we know it’s not. At PPMD, we are working on a grant from the National Institutes of Neurological Disorders and Stroke (NINDS) to understand expectations and hopes in Duchenne and Becker clinical trials. Where do the communities’ expectations and hopes come from? When do high expectations or hopes help people? When do they cause harm? The overall goal is to improve the wellbeing of families who are thinking about or participating in research.

We have started by interviewing parents, individuals with Duchenne or Becker age 12 or older, researchers, clinicians, and industry sponsors. Later, we’ll do a survey to get information from a much larger group of parents. 

If you would like to learn more about this PPMD project, visit our study page. We’re still looking for individuals to be interviewed, so let me know if you’d like to share your insights on these topics!

Holly Peay, Sr. Director, Education & Outreach
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