I was privileged to share some of the data PPMD has collected in our Clinical Trial Expectations study at the World Muscle Society poster session. Many clinicians and industry and academic scientists came to the poster to learn about our community thoughts and preferences, and were quite interested in how they might improve the clinical trial process. Highlighting the importance of this topic to neuromuscular providers and researchers, I was invited to present this data on Saturday in the “Poster Highlights” session.


You’ve heard from me about this study at the PPMD Connect Conference and through previous blogs. Now you’ll hear from members of the advisory committee, which leads this project, about what it has been like to partner in this research and what we have learned.


Joanna Johnson, a parent and co-chair of the project advisory committee:

Being on the Advisory Committee has been enlightening and affirming for me as a parent, even more so as a parent with a son who participated in a clinical trial. Living with this disease is heartbreak and anguish, simply put, but the idea of potential treatment helps to ease the pain and hope is often times the necessary remedy to enable us to cope and give us strength.

However, as a community we need to be cautious and mindful, and clinicians and industry need to be aware of the mindset of the boys they are treating and their parents. Parents expressed a lot of confusion and contradiction in regards to hopes and expectations in relation to a clinical trial. For example, parents may say they don’t expect benefit, but also say they would not participate if they weren’t expecting benefit for their child.

We have so much information regarding research and clinical trials at our fingertips, which will inevitably affect and possibly taint our expectations for a trial. This does not even take into account that some of the information we receive comes second hand and piecemeal. As over 40% of trials fail at stage 3, we need to find ways to manage these expectations. Most importantly, clinicians and industry need to understand our needs and motivations as things move forward. 

(Raju) Kanneboyina Nagaraju, PhD, DVM, a translational researcher on our project advisory committee:

My participation in the Expectations Committee is truly an eye opening and humbling experience. As a translational scientist who is actively involved in developing experimental therapeutics for Duchenne Muscular Dystrophy, I always thought about children with Duchenne but rarely about their parents.  Research to understand their feelings and views is immensely useful to develop coping strategies for their wellbeing. Based on being on this project’s advisory committee, for the first time as a parent of two kids, the project made me to think about what my own hopes and expectations would be if one of my children was to have a condition like Duchenne. I think that my own hopes and expectations about clinical trial participation would be no different from the parents who participated in the survey. This project should help to develop anticipatory advice for parents, clinicians and the broader community regarding clinical trial hopes and expectations.

Diana Escolar, a neurologist and researcher experienced in clinical trials shared these perspectives:

This project has been important in many ways, influencing the way I design and enroll patients in clinical trials. First, it is clear that hopes and expectations may be really difficult to tease out in parents' minds. Thus, I feel it is the clinician's obligation to explore the difference with families between their optimistic hopes and what they actually expect from the trial. Though clinicians are also biased and hopeful for their patients, they can be more objective regarding the science and the possible outcomes. 

It was also clear from this data that the informed consent may not be serving its purpose. Shorter, educational consent forms might be used, with a final family test to assure they understood the risks (including the chance the trial might fail) and ins and outs of the study they are getting into.

Finally, I heard that participating in clinical trials is a burden, but families may be willing to take more risks than regulatory agencies are willing to accept. This is important information to bring forward to FDA and to influence the design of the trials. We need a balance between close monitoring, but taking risk if it means a greater success probability would be acceptable to families and boys. 

Benjamin Wilfond, MD, a bioethicist on our advisory committee:

While parents may understand that the goal of research to improve care in the future, as this study shows, parents still hold out hopes and expectations for something more. This is understandable because it is how we all cope. But this creates challenges for, and responsibilities of, researchers related to how to manage those hopes and expectations. No one wants to dash hopes, especially in this context; but this can result in a collusion that ultimately can be destructive to parents, doctors, researchers, and the community. Regardless of our hopes and expectations, research is likely to result in failures as often as wins. And when failures dash the hopes and expectations, what is left is distrust and alienation. Anything other than frank conversation will augment hope and expectations. Clinicians and sponsors cannot simply say “we are not sure if this will benefit your children.” Clinicians might say, “I wish this will benefit your child, and we are very excited by this idea. But your child is not expected to benefit. But if you join this study, part of your child’s legacy will be his contribution to helping children in the future.” This message needs to stated at every opportunity.

And finally, from advisory committee member Janice Bowie, who is an expert in community-based participator research (CBPR - the process we are using for this project) about how the process works:

Our Expectations Project is applying CBPR principles by creating a study team of a variety of stakeholders to implement the study’s objectives. Patients, families, advocacy groups, researchers, and clinicians comprise the team and meaningfully engaged in the decision-making throughout the research process. This approach has been valuable for the Duchenne study because it has given credence to both the rigor and validity of the research – making it more relevant to the lived experiences of Duchenne patients and families. It has also promoted dissemination of the findings to the beneficiaries and the clinical community, as well as encouraged the research community to see CBPR as a potential benefit.

Thanks to the parents who shared their family’s clinical trial experience, clinicians who spoke frankly about their own experiences participating on trial teams, input from individuals with Duchenne and Becker who have managed clinical trial participation, and translational researchers who shared the experiences of those “at the bench,” we have an interesting insight into our community’s perceptions and what may be done to improve the clinical trial experience. (And thanks also to the National Institutes of Neurological Disorders and Stroke, who funded this project.)


PPMD extends a huge thank-you to our project advisory committee. We’ll share more over the next few days, including information about the next phase of the study.

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