The Duchenne community has waited 20 years to be where we are. We are reminded daily of how far we have come, how the landscape has changed, how there are close to 20 clinical trials to choose from. While these changes within are landscape bring us to a hopeful time in Duchenne, the journey to this point has not been painless for the families involved.
As we have watched clinical trials in Duchenne expand, we have witnessed the impact of participating in these trials on children and families. Trials with complex protocols that may extend for lengthy periods of time exert significant pressures that families feel, and exert on each other. Examples of children and parents rebelling against the pressure, trying their very best to cope or, more often, not coping at all, creep onto social media and into our hearts.
Last fall, PPMD had a discussion with Dr. Robert “Skip” Nelson (Deputy Director and Senior Pediatric Ethicist in the Office of Pediatric Therapeutics (OPT) in the Office of the Commissioner) that illuminated concerns that PPMD, industry, and the FDA all shared – concerns of the burdens being placed on participants and families involved in pediatric research. It became clear that it was time to examine and address the needs of our community more closely.
Photo from today's "Unique Burdens of Pediatric Clinical Trials" meeting
Today and tomorrow, PPMD will convene the "Unique Burdens of Pediatric Clinical Trials" meeting. This meeting will include presentations of the perspectives of parents/patients, industry, and primary investigators, on four main areas that have been identified as areas of burden in clinical trial participation.
These four areas include:
A published summary of this meeting will be available to the community.
We appreciate the FDA’s participation in this meeting and their openness to discussion. The burden of trial participation for children appears to be a priority for the agency. In fact, later this month we expect the FDA to announce that they will be holding a Pediatric Advisory Committee Meeting soon. This meeting will take a closer look at trial protocols, in particular the use of ports.
In all areas of life we have heroes – firemen who rush into burning buildings to pull out stranded victims, athletes that dig a little deeper to find that shred of spirit needed to push them across the finish line, workers who sacrifice whatever is needed to help just one more person.
In Duchenne, we don't have to look farther than our clinical trial sites to find our heroes. Every day, trial participants dig deep into their spirit to bear the burdens and make the sacrifices needed to make sure that new therapies are safe, that they work, that they make the tumultuous journey from bench to bedside – that these new therapies are available to every patient that wants and needs them.
PPMD heralds these families, and recognizes the tremendous burdens that they endure. We are hopeful that, together, we can help make being a hero a little less painful.